Clinical Trials Reporting and Publication

Clinical Trials Reporting and Publication

Order Code RL32832
Clinical Trials Reporting and Publication
Updated July 12, 2007
Erin D. Williams
Specialist in Bioethical Policy
Domestic Social Policy Division

Clinical Trials Reporting and Publication
Summary
The central issue before Congress with respect to clinical trials reporting and
publication is how to balance the potential beneficial public health effects of
requiring that clinical trials data be made public with the burdens that such
requirements may place on companies and their innovation. Clinical trials, which
are conducted regularly to test the effects of new pharmaceuticals and medical
devices, cost a significant amount of money, and by their nature may present some
risk to the people who participate in them. Manufacturers as well as medical journal
editors have been reluctant to publish clinical trial data indicating that products in
development are harmful or ineffective. The availability of such information might
save a duplication of effort and studies that harm or fail to help patients.
While current federal regulations require the publication of some clinical trials
data, and some private entities have taken steps to encourage publication, there is no
requirement that the public have access to all standardized clinical trials data — be
it notice of trial launch or research results through a centralized system such as a
registry. Food and Drug Administration (FDA) regulations require sponsors of trials
that test the effectiveness of new drugs for serious or life-threatening conditions to
register with the Department of Health and Human Services (HHS) at
[http://clinicaltrials.gov/], although not all such trials are listed there. Clinical trial
data from National Institutes of Health (NIH)-funded research may be made public
through a Freedom of Information Act request only if the findings were used by the
federal government in developing an agency action that has the force and effect of
law. The International Committee of Medical Journal Editors (ICMJE) requires, for
publication of clinical trial results, that a sponsor have posted its trial in a public
registry before enrolling patients. A voluntary registry of recent controlled trials
results was created in October 2004 by the Pharmaceutical Research and
Manufacturers of America (PhRMA).
Proposals for public access to all or most clinical trial data raise a variety of
issues. These relate to the goals of providing public access, the appropriateness of
the information and its presentation for the audience, the timing of a trial’s inclusion,
whether reporting should be mandatory, potential conflicts of interest, and whether
medical device trials should be included.
Nine relevant bills have been introduced during the 110th Congress, two of
which also reauthorize key Food and Drug Administration programs. These bills are
the Food and Drug Administration Revitalization Act (S. 1082), which the Senate
passed on May 9, 2007, and the Food and Drug Administration Amendments Act of
2007 (H.R. 2900), which the House passed on July 11, 2007. Both bills would
require the registration of clinical trials, some of which must currently be registered
at [http://clinicaltrials.gov]. H.R. 2900 would also require the subsequent posting of
clinical trial results. Differences between the two bills are expected to be addressed
in conference. Seven other bills also contain relevant provisions: S. 467, S. 484/H.R.
1561, S. 468/H.R. 788, and S. 830/H.R. 1494.
This report will be updated on a regular basis.

Contents
Introduction: Current Federal Regulations . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1
Clinical trials are the gold standard . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2
Federal regulations require the publication of certain clinical
trial information and encourage the disclosure of some results . . . 3
Non-Federal Activities . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5
World Health Organization (WHO) promotes trial registry
standards, portal, and registration of all clinical trials . . . . . . . . . . 5
The International Committee of Medical Journal Editors (ICMJE)
Clinical Trial Publication Policy requires registration . . . . . . . . . . 6
American Medical Association (AMA) recommends a
comprehensive clinical trials registry . . . . . . . . . . . . . . . . . . . . . . . 7
The Association of American Medical Colleges (AAMC)
develops principles for clinical trials reporting . . . . . . . . . . . . . . . 7
The Institute of Medicine (IOM) supports mandatory trial registration
and results reporting . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 7
The pharmaceutical industry favors limited, voluntary clinical
trial registration and reporting . . . . . . . . . . . . . . . . . . . . . . . . . . . . 8
Legislation . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 9
FDA User Fee Reauthorization Legislation (S. 1082 and H.R. 2900) . . . . . 9
Other Clinical Trials Bills . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 9
Registry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10
Results Database . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11
Issues . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11
Goals . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11
Appropriateness/Presentation . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11
Timing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12
Voluntary or Mandatory/Penalties . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12
Conflicts of Interest . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12
Devices . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12
Appendix A. World Health Organization, International Clinical Trials
Registry Platform, Registration Data Set (Version 1.0) . . . . . . . . . . . . . . . . 35
List of Tables
Table 1. Comparison of Proposals for Clinical Trials Reporting and
Publication in the 110th Congress . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14

Clinical Trials Reporting and Publication
Introduction: Current Federal Regulations
In 2004, Congress and others raised questions about the safety and effectiveness
of several FDA-approved biomedical products on the market. These included certain
antidepressants, Merck’s pain relief drug, Vioxx, Boston Scientific’s cardiac stents,
and other drugs and medical devices. Discussion about ways to help ensure safety
and effectiveness of biomedical products focused primarily on two questions:
whether data from all clinical trials should be made publicly available, and whether
FDA’s processes for product approval and post-market surveillance and study are
adequate. This report focuses on the first of these questions.1
The central issue before Congress with respect to clinical trials reporting and
publication is how to balance the potential beneficial public health effects of
requiring that clinical trials data be made public with the burdens that such
requirements may place on companies and their innovation. On one hand, companies
may lose a competitive advantage if their competitors are alerted to their clinical
trials activities and failures. On the other hand, the public may be harmed if a
particular type of clinical trial is repeated — particularly if an earlier trial
demonstrated that a product was ineffective or harmful. In addition, if clinical trial
data are to be made public, the timing and contents of the disclosure may prove to be
pivotal, both with respect to competitive innovation and public safety.
Clinical trials reporting can mean public access to results after a trial’s
conclusion, to a proposed plan before a trial is begun, or both. There is no
centralized system for either type of reporting; thus, different trials may have the
same title, one trial may be reported in several places under different titles, and many
trials are never reported. Researchers have traditionally reported pre- and post-
market trial results in peer-reviewed medical journals, which have historically tended
to favor publication of clinical trials demonstrating successful intervention; the
results of negative or inconclusive trials often go unpublished.2 Other venues for the
dissemination of research results are industry, government, or university press
releases and presentations at medical conferences. Researchers — who may be
affiliated with a product’s manufacturer, a university, the government, or an
association established to find better treatments for a particular disease — may have
various motives for publishing or not publishing results. Some observers have
1 For further information about whether FDA’s processes for product approval and
post-market surveillance and study are adequate, see CRS Report RL32797, Drug Safety and
Effectiveness: Issues and Action Options After FDA Approval, by Susan Thaul.
2 “Pressure Mounts for Clinical Trial Registry,” Medicine & Health, vol. 58, no. 24 (June
21, 2004), pp. 2-3.

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expressed concern that a lack of transparency, particularly for negative data, could
adversely affect medical decision-making.3
The lack of transparency may be amplified in part by sponsors’ contractual
requirements of their researchers. This concern was raised by two May 2005 medical
journal articles, suggesting that contractual “gag” clauses might prohibit clinical trial
investigators from examining data independently or submitting a manuscript for
publication without first obtaining the consent of trial sponsors. According to one
of the articles, sponsors with a financial interest in the outcomes of clinical research
could thus suppress negative results and interfere with the publication of unfavorable
data on safety.4 The other article, which described results from a survey of medical
school research administrators responsible for negotiating clinical trial agreements
with industry sponsors, reported that industry provides approximately 70% of
funding for clinical drug trials in the United States.5 The survey results suggested
that 85% of the administrators’ offices would not approve provisions that gave
industry sponsors the authority to revise manuscripts or to decide whether results
should be published. Administrators’ responses varied regarding whether contracts
could contain provisions allowing sponsors to insert their own statistical analyses in
manuscripts, draft manuscripts, or prohibit investigators from sharing data with their
parties after the trial’s conclusion.
In order to fully understand the debate surrounding clinical trials reporting and
publication, a basic understanding of clinical trials themselves and of the current
federal requirements — both of which are presented below — is essential. The slate
of issues that frequently arise during discussions of clinical trials reporting and
publication, all of which are addressed below in the “Issues” section of this report,
include questions related to the goals of publication, the materials’ appropriateness
and presentation, the timing the disclosures, whether disclosure should be voluntary
or mandatory with penalties, overcoming potential conflicts of interest, and whether
medical devices should be included in reporting requirements.
Clinical trials are the gold standard. Clinical trials, which are the gold
standard for assessing drug and device safety and effectiveness both before and after
they are marketed in the United States, are scientific studies that systematically test
interventions on human beings. They may include behavioral studies or other
biomedical investigations, such as those that test drugs and medical devices. As
described by FDA, clinical trials are generally conducted in four phases following
successful animal testing.6 Phase I trials study a new drug or device in a small group
3 Robert Steinbrook, “Public Registration of Clinical Trials,” JAMA, vol. 351, no. 4 (July
22, 2004), p. 315.
4 Robert Steinbrook, “Gag Clauses in Clinical-Trial Agreements,” New England Journal of
Medicine, vol. 352, no. 21 (May 26, 2005), p. 2160.
5 Michelle Mello, et al., “Academic Medical Centers’ Standards for Clinical-Trial
Agreements with Industry,” New England Journal of Medicine, vol. 352, no. 21 (May 26,
2005), p. 2202.
6 For further information on the role of federal agencies in evaluating biomedical products,
(continued...)

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of people (20-80) to evaluate its safety, determine a dosage range for drugs, and
identify gross side effects. Phase II trials study the product in a larger group of
people (100-300) to see whether it is effective for a specific purpose and to further
evaluate its safety. Phase III trials investigate the product in a large group of people
(1,000-3,000), to confirm the product’s effectiveness, monitor side effects, and
collect information that will allow the drug, treatment or device to be used safely.
Phase IV trials are usually large-scale studies, conducted after the FDA approves a
product for marketing in order to demonstrate effectiveness in a broader clinical
context and to watch for rare side effects that may not be identified until significant
numbers of people have used the product.
Federal regulations require the publication of certain clinical trial
information and encourage the disclosure of some results. The federal
government has historically regulated certain aspects of some clinical trials by
attaching conditions to those conducted with federal research funds, and/or by
creating requirements that must be met before a drug or device can be marketed in
the United States. Most federal funding occurs through the Department of Health
and Human Services’ (HHS) National Institutes of Health (NIH). According to
NIH’s regulations issued pursuant to a provision in the Omnibus Consolidated and
Emergency Supplemental Appropriations Act, 1999 (P.L. 105-277), research data
relating to published research findings produced under an award that were used by
the federal government in developing an agency action that has the force and effect
of law — a limited number of research results if any — must be released if a
Freedom of Information Act request is made.7
Beginning in May 2005, the NIH has requested that investigators with
manuscripts that are accepted for publication, and that are the result of research
supported in whole or in part with direct costs from NIH, submit them voluntarily to
the National Library of Medicine’s (NLM’s) PubMed Central.8 (The NLM, which
is located on the NIH campus in Bethesda, Maryland, is the world’s largest medical
library.) This effort would enables free access to results published elsewhere and
would not facilitate access to previously undisclosed results. The NIH announcement
was preceded by a July 2004 House committee recommendation that NIH provide
free public access to the complete text of articles and supplemental materials
generated by NIH-funded research.9
6 (...continued)
see CRS Report RS21962, From Bench to Bedside: The Role of Health and Human Services
(HHS) Agencies in the Evaluation of New Medical Products, by Michele Schoonmaker.
7 Uniform Administrative Requirements for Grants and Agreements With Institutions of
Higher Education, Hospitals, and Other Non-Profit Organizations; Final Rule (Office of
Budget Management, Circular A 110), Federal Register, Vol. 65, No. 52, Page 14406
(March 16, 2000), at [http://grants2.nih.gov/grants/policy/a110_fed_reg_20000316.pdf].
8 National Institutes of Health, “Policy on Enhancing Public Access to Archived
Publications Resulting from NIH-Funded Research,” NOT-OD-05-022, February 2, 2005,
at [http://grants2.nih.gov/grants/guide/notice-files/NOT-OD-05-022.html].
9 U.S. Congress, House Committee on Appropriations, Departments of Labor, Health and
Human Services, and Education and Related Agencies Appropriations Bill, 2005, report to
(continued...)

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Both pre-market approval and post-market monitoring of medical drugs and
devices marketed in the U.S. are the responsibility of HHS’s FDA. Each FDA center
that reviews and approves biomedical products for human use — the Center for Drug
Evaluation and Research, the Center for Devices and Radiological Health, and the
Center for Biologics Evaluation and Research — posts summaries of safety and
effectiveness data from clinical trials that support approved applications for new
products, or new uses of approved products; FDA does not otherwise post clinical
trials data.
The FDA Modernization Act of 1997 (FDAMA, P.L. 105-115, Section 113)
required the Secretary of HHS to establish a clinical trials registry, intending the
availability of information to increase the access of individuals to cutting-edge
medical care available only through research protocols. Sponsors of trials testing the
effectiveness of life-threatening disease or condition treatments (drugs, but not
devices) that are being conducted to obtain FDA approval for marketing,10 under an
expanded use protocol11 of an investigational new drug application to FDA, or on
Group C12 cancer drugs are required to register. In addition, any trial (drug, device,
or other) that has been approved by a human subject review board (or equivalent) and
conforms to the regulations of the appropriate national or international health
authority may also be included.
In response to FDAMA, the NLM established a clinical trials registry and made
it available to the public in 2000 [http://clinicaltrials.gov]. It was later reported that
an FDA analysis found that in 2002 only 48% of trials of cancer drugs had been
registered, and a preliminary review indicated the listing rate for drugs for some other
serious diseases is in the single digits. Some companies had reportedly listed no
studies; some trials were listed without identifying the sponsoring company or the
drug being tested.13 In March 2002, FDA issued a guidance document, instructing
9 (...continued)
accompany H.R. 5006, 108th Cong., 2nd sess., H.Rept. 108-636 (Washington, GPO, 2004).
10 Pursuant to 21 U.S.C. § 355(i).
11 An expanded use protocol is one that allows for widespread patient access to an
investigational new drug not yet approved for marketing, when the drug has shown promise
for treating a serious or life-threatening condition, there is no comparable or satisfactory
alternative therapy, and the sponsor is actively pursuing permission to market the drug (21
U.S.C. § 360bbb(c)).
12 Group C “was established by agreement between FDA and the National Cancer Institute
(NCI). The Group C program is a means for the distribution of investigational agents to
oncologists for the treatment of cancer under protocols outside the controlled clinical trial.
Group C drugs are generally Phase 3 study drugs that have shown evidence of relative and
reproducible efficacy in a specific tumor type. They can generally be administered by
properly trained physicians without the need for specialized supportive care facilities.
Group C drugs are distributed only by the National Institutes of Health under NCI
protocols.” Information Sheets: Guidance for Institutional Review Boards and Clinical
Investigators,1998 Update, Drugs and Biologics, FDA, at [http://www.fda.gov/oc/ohrt/irbs/
drugsbiologics.html].
13 Shankar Vedantam, “Drugmakers Prefer Silence on Test Data,” Washington Post, July 6,
(continued...)

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industry how and when to participate in the registry [http://www.fda.gov/cder/
guidance/4856fnl.htm].
A 2005 survey conducted by FDA’s Office of Special Health Issues indicated
that 67% of companies required to register their trials had done so.14 The 2005
survey results were not comparable to those of 2002 due to methodological
differences. It was reported that FDA did not plan to continue to monitor whether
companies registered beyond 2006.15
In a July 2004 announcement unrelated to [http://clinicaltrials.gov/], the FDA
announced that clinical trial sponsors could use a standard format, the Study Data
Tabulation Model (SDTM) developed by the nonprofit organization Clinical Data
Interchange Standards Consortium (CDISC), to submit clinical trials data to the
agency [http://www.cdisc.org/index.html]. While the data would not necessarily be
made public, according to the FDA, providing a consistent framework and format for
clinical trial information is expected to enhance data integration opportunities and
thereby reduce data management barriers for sharing the latest clinical trial data.16
Non-Federal Activities
A number of national and international groups recommended that clinical trial
reporting be centralized, standardized, and/or include both positive and negative
results, and have taken steps toward that goal.
World Health Organization (WHO) promotes trial registry standards,
portal, and registration of all clinical trials. In May 2006, the WHO, the
United Nations specialized agency for health which supports and funds much of the
international research on marginalized populations, began urging research institutions
and companies to register all medical studies that test treatments on human beings,
including the earliest studies, whether they involve patients or healthy volunteers.17
This dovetails with another WHO initiative: the International Clinical Trials
Registry Platform (ICTRP), which aims to standardize the way information on
medical studies is made available to the public. As a part of the ICTRP, WHO has
13 (...continued)
2004, p. A1.
14 “FDAMA Section 113: Status Report on Implementation,” FDA Office of Special Health
Issues, August 2005, at [http://www.fda.gov/oashi/clinicaltrials/section113/113report/
default.htm].
15 “FDA to Stop Tracking Industry Compliance With Clinical Trial Registry,” Inside
Washington Publishers, September 26, 2006.
16 “FDA Announces Standard Format That Drug Sponsors Can Use to Submit Human Drug
Clinical Trial Data,” FDA News, July 21, 2004, at [http://www.fda.gov/bbs/topics/
news/2004/NEW01095.html].
17 “The World Health Organization announces new standards for registration of all human
medical research,” World Health Organization website, May 19, 2006, [http://www.who.
int/mediacentre/news/releases/2006/pr25/en/index.html].

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recommended that 20 key details — such as title, funding source, research ethics
review, and outcome measures — be disclosed at the time studies are begun, that a
Universal Trial Reference Number be assigned to each trial, and that minimum
standards for the reporting of trial results be defined. (See Appendix A for a
complete list of key details.) As the ICTRP progresses, WHO plans to launch a
one-stop Search Portal for searching compatible registries worldwide.18
Some organizations have voiced opposition to the WHO efforts. The
Pharmaceutical Research and Manufacturers of America (PhRMA) has reportedly
opposed publicizing information early in the clinical trial, arguing that disclosing
early research data does little to help doctors and patients, and may impede
innovation by alerting competitors to companies’ activities.19 For similar reasons, the
Advanced Medical Device Medical Technology Association (AdvaMed) has
reportedly attempted unsuccessfully to allow device firms to delay disclosure of some
required data elements.20 AdvaMed argued that the issue was more pronounced for
device than drug manufacturers because device development process is iterative,
involving improvements over a period of time.
Since April 2004, all clinical trials approved by the WHO ethics review board
have been required to be registered at their outset and assigned a unique identification
number.21 A London-based group of biomedical publishing companies agreed to
maintain a no-charge, online register of these numbered trials at
[http://www.controlled-trials.com] to identify and track them throughout their life
cycle. The system was designed to avoid the problem of publication bias by posting
information on trial starts and their results.
The International Committee of Medical Journal Editors (ICMJE)
Clinical Trial Publication Policy requires registration. The ICMJE consists
of the editors of 12 major journals, including the New England Journal of Medicine,
The Lancet, and the Journal of the American Medical Association. In order for a
sponsor to have its clinical trial results published in one of the ICMJE journals, the
ICMJE requires it to have posted its trial in a public registry before enrolling
patients.22 The policy applies to any trial that started recruiting human subjects on
or after July 1, 2005. The ICMJE did not advocate any particular registry, but cited
18 “International Clinical Trials Registry Platform” World Health Organization website, May
19, 2006, [http://www.who.int/ictrp/en/].
19 “PhRMA Opposes UN Plan for Trial Data Disclosure,” Inside Washington Publishers,
May 30, 2006.
20 “AdvaMed, WHO at Odds Over Global Trial Registry Standards,” Inside Washington
Publishers, July 19, 2006.
21 Gerd Antes, “Registering clinical trials is necessary for ethical, scientific and economic
reasons,” Bulletin of the World Health Organization, May 2004, vol. 82, no. 5, at
[http://www.who.int/bulletin/volumes/82/5/en/321.pdf].
22 Catherine De Angelis et al., “Clinical Trial Registration: A Statement from the
International Committee of Medical Journal Editors,” New England Journal of Medicine,
vol. 351, no. 12 (September 16, 2004), p. 1250, at [http://content.nejm.org/cgi/content/full/
351/12/1250].

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[http://clinicaltrials.gov/] as the only database currently meeting its requirements. In
June 2005, the ICMJE specified the minimum set of data elements necessary for a
trial to be considered fully registered, adopting the WHO list of 20 items.23
American Medical Association (AMA) recommends a
comprehensive clinical trials registry. In an effort at dovetailing with the
ICMJE requirements, in December 2004, the AMA House of Delegates committed
the organization to take all appropriate action to protect the rights of physician
researchers to present, publish, and disseminate data from clinical trials.24 In June
2004, the AMA recommended that HHS create a comprehensive, centralized clinical
trials registry. The AMA further called on all institutional review boards to make
registration in this database a condition of their approval of the bioethical aspects of
clinical trials.25 Noting the AMA’s position, Senators Tim Johnson and Christopher
Dodd called for a national clinical drug trial registry in a July 8, 2004 letter to the
heads of NIH and FDA.26
The Association of American Medical Colleges (AAMC) develops
principles for clinical trials reporting. In January 2006, the AAMC Executive
Committee approved a set of principles designed to promote standards for analyzing
and reporting the results of sponsored clinical research.27 The principles include,
among other things, that researchers have an ethical obligation to make their results
public, that contracts with sponsors should require a good-faith effort to publish
results, and that trials should be fully registered according to ICMJE standards within
21 days of their outset either in [http://clinicaltrials.gov/] or elsewhere.
The Institute of Medicine (IOM) supports mandatory trial
registration and results reporting. The IOM, a National Academies institute,
conducted a workshop on developing a national clinical trials registry.28 Workshop
participants presented a range of views on the need for registries, registry content,
23 Catherine DeAngelis et al., “Is This Clinical Trial Fully Registered?: A Statement from
the International Committee of Medical Journal Editors,” New England Journal of
Medicine, vol. 352, no. 23 (June 9, 2005), p. 2436, and [http://www.icmje.org/
clin_trialup.htm].
24 American Medical Association, “610. Physicians and Clinical Trials,” December 2004
Resolutions, at [http://www.ama-assn.org/meetings/public/interim04/resolutions.pdf].
25 Joseph M. Heyman, “AMA Encouraged by Early Signs of Industry Support for National
Clinical Trials Registry,” American Medical Association, press release, June 18, 2004, at
[http://www.ama-assn.org/ama/pub/category/print/13909.html].
26 “Senators Call for National Registry of Clinical Drug Trials,” Senator Tim Johnson, press
release, July 8, 2004, at [http://johnson.senate.gov/~johnson/releases/200407/2004708B20.
html].
27 Susan Ehringhaus and David Korn, “Principles for protecting Integrity in the Conduct and
Reporting of Clinical Trials,” Association ofAmerican Medical Colleges, January 6, 2007,
at [http://www.aamc.org/research/clinicaltrialsreporting/clinicaltrialsreporting.pdf].
28 Committee on Clinical Trials, Institute of Medicine of the National Academies,
Developing a National Registry of Pharmacologic and Biologic Trials (Washington, DC:
The National Academies Press, 2006), at [http://books.nap.edu/catalog/11561.html#toc].

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implementation issues, and next steps. A separate draft publication published by
IOM in 2006 recommended that Congress require industry drug sponsors to register
phase 2-4 clinical trials at [http://clinicaltrials.gov/], and that initial postings be
supplemented by a summary of safety and efficacy results.29
The pharmaceutical industry favors limited, voluntary clinical trial
registration and reporting. The pharmaceutical industry’s reaction to clinical
trials reporting has been mixed, although as litigation and FDA and congressional
interest have increased, some individual manufacturers and groups have volunteered
to make some of their clinical trials data public. How the industry defines the types
of trials to include (e.g., hypothesis-testing or late-phase only) could affect a
registry’s utility. Initially skeptical, PhRMA introduced its own clinical trials
database in October 2004 at [http://www.clinicalstudyresults.org]. Companies that
market drugs in the United States can voluntarily post the positive and negative
results of controlled trials (mainly Phase III and IV studies) completed after October
2002 on the PhRMA database. As of April 16, 2007, 60 companies had posted
results for 343 drugs. According to FDA, more than 10,000 drugs are approved for
marketing in the United States. In January 2005, PhRMA additionally called for its
members to voluntarily post all hypothesis-testing clinical trials on NLM’s registry,
clinicaltrials.gov.
In January 2005, an international pharmaceutical federation of which PhRMA
is a member, the International Federation of Pharmaceutical Manufacturers and
Associations (IFPMA), announced that its members would voluntarily disclose
summary results of all industry-sponsored clinical trials.30 Trial results would be
published in a standard, non-promotional summary that would include a description
of trial design and methodology, results of primary and secondary outcome measures
described in the protocol, and safety results. In October 2005, IFPMA announced
that it had launched a search portal of clinical trial registries and databases
worldwide.31
Legislation
A number of bills related to clinical trials reporting and publication have been
introduced in the 110th Congress.
29 Committee on the Assessment of the US Drug Safety System, Institute of Medicine of the
National Academies, The Future of Drug Safety: Promoting and Protecting the Health of
the Public, Advance Copy, Tuesday September 26, 2006, (Washington, DC: National
Academies Press, 2006), at [http://books.nap.edu/books/0309103045/html].
30 The announcement was made jointly with PhRMA, the European Federation of
Pharmaceutical Industries and Associations (EFPIA), and the Japanese Pharmaceutical
Manufacturers Association (JPMA). International Federation of Pharmaceutical
Manufacturers and Associations (IFPMA), “Global Industry Position On Disclosure of
Information About Clinical Trials,” IFPMA Press Release, January 6, 2005, at
[http://www.ifpma.org/News/NewsReleaseDetail.aspx?nID=2205].
31 IFPMA, “IFPMA Improves Biomedical Data Transparency with Launch of First
Worldwide Clinical Trials Portal,” IFPMA Press Release, September 21, 2005, at
[http://www.ifpma.org/clinicaltrials.html].

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FDA User Fee Reauthorization Legislation
(S. 1082 and H.R. 2900)
Two major pieces of legislation, which would reauthorize FDA drug and
medical device user fee authorities, contain clinical trials databank titles or subtitles.
Each bill has been passed by its respective chamber of Congress, leaving differences
between the bills to be addressed in conference. The Food and Drug Administration
Reauthorization Act (S. 1082), introduced by Senator Kennedy, passed the Senate on
May 9, 2007. The bill is composed of titles on the topics of reauthorizing the
Prescription Drug User Fee Act and the Medical Device User Fee Act, promoting
drug safety, encouraging the development of pediatric medical drugs and devices,
addressing drug importation, promoting food safety, and enabling domestic pet turtle
market access. One subtitle of the drug safety provisions (Title II, Subtitle C) would
create a clinical trial registry and could lead to the creation of a results database
following rulemaking by the HHS Secretary.
The Food and Drug Administration Amendments Act of 2007 (H.R. 2900),
introduced by Representative Pallone on June 28, 2007, passed the House on July 11,
2007. H.R. 2900 is similar to S. 1082, but does not contain provisions related to drug
importation, food safety, or domestic pet turtle market access. Its title on clinical
trials databanks (Title VIII) would require both the registration of clinical trials and
the posting of their results. One controversial provision that was dropped from H.R.
2900 (and was never present in S. 1082) specified that Act would not have had any
legal effect on — and thus would have allowed — causes of action for damages
under state law.
Other Clinical Trials Bills
One bill introduced in the 110th Congress is solely focused on clinical trials
registration and reporting: S. 467, the Fair Access to Clinical Trials (FACT) Act,
introduced by Senator Dodd. Similar legislation was introduced in the 109th
Congress by Senator Dodd (S. 470) and Representative Waxman (H.R. 3196), and
in the 108th Congress by Senator Dodd (S. 2933) and Representative Markey (H.R.
5252).
Several other bills focused on promoting drug and device safety at the FDA
contain clinical trials databank provisions as well. The Enhancing Drug Safety and
Innovation Act of 2007 (S. 484/H.R. 1561), introduced by Senator Enzi and
Representative Waxman, is composed of titles designed to address the following
topics at FDA: risk evaluation and mitigation strategies, the Reagan-Udall Institute
for Applied Biomedical Research, clinical trials, and conflicts of interest. The
clinical trials title of each bill contains provisions that would create a clinical trial
registry and results database. Although many provisions of S. 484 and H.R. 1561 are
identical, those related to clinical trials reporting and publication are different.
Two other pieces of legislation with provisions related to clinical trials reporting
and publication are the identical companion bills S. 468 and H.R. 788, the Food and
Drug Administration Safety Act of 2007, introduced by Senator Grassley and
Representative Tierney. This measure would establish a Center for Postmarket

CRS-10
Evaluation and Research for Drugs and Biologics at FDA. It would enable the
Center Director to require certain pre- and postmarket studies, and would require the
HHS Secretary to make information about those studies available to the public.
The remaining two bills with provisions related to clinical trials reporting and
publication are the pediatric Medical Device Safety and Improvement Act of 2007
(S. 830 / H.R. 1484) introduced by Senator Dodd and Representative Markey. The
bills would expand tracking of FDA pediatric device approvals, modify and tighten
the humanitarian device exemption (which waives user fees associated with the
FDA’s review of medical device applications), require the NIH Director to designate
a point of contact to assist those seeking funding for pediatric device development,
create demonstration grants for improving pediatric device availability, amend
regulations governing the office of pediatric therapeutics and the pediatric advisory
committee, and enable the Secretary to order certain postmarket studies as a
condition of approval of pediatric medical devices. The bills would also require the
HHS Secretary, acting through the FDA Commissioner, to establish a database of
clinical trials on pediatric devices. The database would include trials conducted in
conjunction with the aforementioned postmarket studies, or with FDA premarket
device approval, clearance, or qualification as for the humanitarian device exemption.
Details of five proposals for clinical trials reporting and publication contained
in S. 1082, H.R. 2900, S. 467, S. 484 and H.R. 1561 are discussed in the text that
follows, and compared with current law in Table 1. Due to the narrow scope of the
proposal for clinical trial publication contained in S. 830/H.R. 1484, it is not
incorporated into the text or table. For similar reasons, S. 468/H.R. 788 is not
incorporated into the table. For purposes of this report, the repository of clinical trial
information submitted at the outset of the trial is referred to as a registry, and the
repository of the trial conclusions is referred to as a results database.
Registry. Current law: Only trials that meet all three of the following criteria
must be included in the registry, clinicaltrials.gov: (1) The trial is testing a drug; (2)
The trial is being conducted to obtain FDA approval for marketing, is conducted
pursuant to an expanded use protocol of investigational new drug application to
FDA, or is conducted on a Group C cancer drug; and (3) The trial tests treatments
of serious or life-threatening conditions. Other trials that have been approved by a
human subject review board (or equivalent) and conform to the regulations of the
appropriate national or international health authority may also be included.
Each of the legislative proposals would expand the scope of the current law,
which requires only the registration of certain drug trials, to include trials related to
biologics as well. All but S. 484 would also require the inclusion of medical device
trials. S. 467 would also allow for the results of other types of trials to be voluntarily
submitted. All but S. 467 would also expand the registry to include trials beyond
those for the treatment of life-threatening diseases or conditions.
Results Database. Current law: there is no requirement that the results of
clinical trials be made publically available, except those included as a portion of
what FDA publishes upon its approval of an application.

CRS-11
Most of the bills (all but S. 1082) would require public disclosure of study
results. S. 1082 would require the NIH Director to issue a report and HHS Secretary
to create a rule based on that report regarding the best way to make clinical study
results available to the public.
Issues
Issues surrounding the possibility of clinical trials reporting and publication
have focused on a range of topics. Those topics are discussed below, with an
accompanying analysis of the clinical trials reporting and publication provisions
contained in S. 1082, H.R. 2900, S. 467, S. 484, H.R. 1561, and S. 468/H.R. 788.
Goals. Proponents of public access to clinical trials data cite the need to
provide information to members of the general public, health care workers, and
researchers, both to help inform treatment decisions and to help eliminate abuses.
Industry advocates have also cited the potential benefits of public awareness of the
resources necessary to get a drug approved, and the elimination of duplicated failed
efforts. PhRMA cites making clinical trial results for U.S.-marketed pharmaceuticals
more transparent, and providing information to practicing physicians and their
patients. Each of the legislative proposals aims to make information available and
understandable to members of the public.
Appropriateness/Presentation. Some have questioned whether
registration and publication of clinical trials and their results are the best mechanism
for ensuring patient safety, both because the language may be too technical for lay
audiences, and because numerous trials may need to be viewed together in order to
draw meaningful conclusions — an analysis that would be difficult for many doctors
as well. (A single clinical trial may generate thousands of pages of documentation.)
These questions have led some to focus on how information might be presented in
an audience-appropriate way. PhRMA’s registry contains a link to drug labels, a
bibliography, and a summary of results in a format developed by industry
consensus.32 All of the bills would contain information accessible to both the general
public and professionals. Three bills, H.R. 2900, S. 484 and H.R. 1561, have the
additional specific requirement that the results database contain both a technical and
a nontechnical summary report, which might meet the differing requirements of
professionals and lay persons.
Timing. Some have argued that only clinical study results are important to
judging effectiveness, so publication of a trial’s inception is not necessary. Others
have argued that some registration at inception is necessary to avoid abuse, and is
helpful for connecting potential subjects with various trials. FDAMA requires that
notice of a qualifying trial be submitted to [http://clinicaltrials.gov/] no later than 21
days after the trial is open for enrollment. PhRMA’s database only accepts results
from completed trials. S. 1082 and S. 467 would generally require registration within
21 days that a trial is opened for enrollment. H.R. 2900, S. 484, and H.R. 1561
would require enrollment within 14 days after the first patient is enrolled, except for
32 Structure and Content of Clinical Study Reports; Guideline Approved by the International
Conference on Harmonization, July 1996, at [http://www.fda.gov/cder/guidance/iche3.pdf].

CRS-12
medical device clinical trials. H.R. 2900 would not allow the public release of the
information until the device is approved or cleared by FDA. S. 467/H.R. 788 would
require that information about the study be posted not less frequently than every 90
days.
For clinical trial results, H.R. 2900, S. 467, and H.R. 1561 would require them
to be submitted within one year of the earlier of the trial’s actual or estimated
completion date. S. 484 would require results submissions not later than one year
after the last patient has his or her last medical visit, and S. 467 / H.R. 788 would
require results to be submitted upon completion of the study. All the bills except for
S. 467 / H.R. 788 would allow for extensions for results submission in certain
circumstances, such as when publication in a peer-reviewed journal is pending. S.
467 / H.R. 788 may also allow for such extensions by nature of the fact that the
Director of the act-created Center for Postmarket Evaluation and Research for Drugs
and Biologics would determine the studies completion date, and might therefore be
capable of delaying the date if presented with good cause. S. 1082 does not create
a results databank and, therefore, does not specify when results would have to be
released.
Voluntary or Mandatory/Penalties. Concerns about the potential
regulatory burden on smaller drug and device manufacturers, as well as about the
potential for intellectual property problems, have led some to call for voluntary
registration and publication. The desire to protect public safety and to reduce abuse
has led others to back mandatory reporting. PhRMA’s registry is voluntary. The
reporting proposed in all of the bills would be mandatory (with limited exceptions
for trials not conducted on drugs, devices, or biological products and those completed
before the bill’s enactment) and would carry penalties for noncompliance.
Conflicts of Interest. Some commentators have focused on the need for
public disclosure of financial and other arrangements between researchers and
sponsors in order to demonstrate potential conflicts of interest that may affect clinical
trial design, interpretation of data, and presentation of results. The PhRMA database
does not include information about funding relationships, though products there are
identifiable by company, which may also be the trial funding source. All of the bills
would require the disclosure of funding source(s), among other things.
Devices. Some have questioned whether information about clinical trials
related to medical devices should be included in the registry. The medical device
advocacy group, Avamed, points out that FDA regulation of devices is different from
its regulation of drugs. Devices are often approved based on analytical comparisons
to existing products rather than on the conduct of new clinical trials. Devices as
compared to drugs often tend to present a lower risk to patients, tend to be
manufactured by smaller companies, tend to have a short market life due to frequent,
incremental refinements rather than major breakthroughs, and tend to require more
financial incentives to test. PhRMA’s database contains only information related to
drug trials; those proposed in all of the bills except S. 468 / H.R. 788 would include
trials related to medical devices. S. 467 / H.R. 788 would require the HHS Secretary,
in consultation with the FDA Commissioner, the Director of the Center for
Postmarket Evaluation and Research for Drugs and Biologics, and the Director of the
Center for Devices and Radiological Health, to submit to Congress a report that

CRS-13
identifies gaps in the current process of postmarket surveillance of devices approved
under the Federal Food, Drug, and Cosmetic Act, includes recommendations on ways
to improve gaps in postmarket surveillance of devices, and identifies the changes in
authority needed to make those improvements.

CRS-14
Table 1. Comparison of Proposals for Clinical Trials Reporting and Publication in the 110th Congress
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
Title
Data bank of
Food and Drug
Food and Drug
FACT Act
Food and Drug
Enhancing Drug
information on
Administration
Administration
Administration
Safety and
clinical trials for
Revitalization Act
Amendments Act
Revitalization Act Innovation Act of
drugs for serious or
of 2007
2007
life-threatening
diseases and
conditions
Sponsor
Senator Kennedy
Representative
Senator Dodd
Senator Enzi
Representative
Pallone
Waxman
Law Amended
(Existing law) PHSA PHSA (42 U.S.C.
PHSA Title IV (42
PHSA (42 U.S.C.
Subsection (i) of
Subsection (i) of
(42 U.S.C. § 282 (j))
282), as amended;
U.S.C. 281, et seq.) 282), as amended
section 402 of
section 402 of
and Section 492
by Public Law
PHSA (42 USC
PHSA (42 USC
A(a) of the PHSA
109-482; and
282 as amended
282), as amended
Section 492 A(a)
by PL 109-482).
by PL 109-482.
of the PHSA
Registry and/or
Registry only
Registry expanded, Both.
Both
Both
Both
Results
(clinicaltrials.gov);
and includes links
Database
with sponsor consent, to certain results.
Required
registry may also
Results database to
include information
be created by HHS
about the results of
Secretary
registered trials,
rulemaking

CRS-15
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
including potential
following
toxicities or adverse
recommendations
effects
to be made in NIH
Director’s report
about best,
validated method of
making trial results
publically
available.
Product Trial
REGISTRY:
REGISTRY:
BOTH:
BOTH:
BOTH:
BOTH:
Types Included
Drugs
Drugs, devices,
Drugs, devices,
Drugs, biologics,
Drugs, biologics,
Drugs, devices,
biologics
and biologics.
devices.
eventually
biologics
Information about
possibly devices
other trials may be
voluntarily
submitted.
Public Access
REGISTRY:
REGISTRY:
BOTH:
BOTH:
BOTH:
BOTH:
Yes, via information
Yes, via Internet.
Yes, via Internet.
Yes, via
Yes, via Internet.
Yes, via Internet.
systems, which are to Internet posting and FOIA request
information
Internet posting
FOIA request
include toll-free
FOIA request
disclosures not
systems, which are and FOIA request
disclosures not
telephone
disclosures limited
available for results to include toll-free disclosures limited available for
communications
to terms of the act. for which the
telephone
to terms of the act. results for which
Secretary
principal
communications.
Secretary
the principal
promulgates
investigator is
Provisions related
promulgates
investigator is
regulations that
seeking
to disclosure of
regulations that
seeking

CRS-16
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
notice of posting be publication.
FDA reviews
notice of posting
publication.
part of informed
Old versions of
supersede FOIA.
be part of
consent.
updated postings
informed consent.
remain available,
with trackable
changes the public
can see.
Location of
REGISTRY:
REGISTRY:
BOTH: NLM and
BOTH:
BOTH:
BOTH:
Databases
NLM at NIH is
NLM at NIH
NIH
Not specified, but
NIH.
NIH.
current location
REGISTRY:
bill amends the
REGISTRY:
REGISTRY:
Either supplants or
portion of the
Either supplants or Either supplants or
builds on
USC related to the builds on
builds on
clinicaltrials.gov,
current registry,
clinicaltrials.gov,
clinicaltrials.gov,
whichever is more
which is located at whichever is more whichever is more
efficient.
NLM at NIH.
efficient.
efficient.
Links Between
REGISTRY:
REGISTRY:
BOTH:
Not specified,
REGISTRY:
BOTH:
Registry,
Not specified; except Entries link to
Corresponding
except that the
Entries link to
Corresponding
Results
that the activities of
certain existing
registry and results
Secretary shall
results entries.
registry and
Database
the data bank are to
results.
database entries
assign each
results database
be integrated and
link to one another. clinical trial a
entries link to one
coordinated with
unique identifier
another.
related activities of
to be included in
other agencies of the
the registry and in
DHHS, and, to the
the database.
extent practicable,

CRS-17
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
coordinated with
other data banks
containing similar
information.
Who Submits
REGISTRY:
REGISTRY:
BOTH:
BOTH:
BOTH:
BOTH:
Information
Sponsor
Responsible party
Responsible party
Responsible party
Responsible party
Responsible party
(RP): sponsor; if no (RP): primary
(RP): if such
(RP): sponsor, or
(RP): primary
sponsor exists-
sponsor as defined
clinical trial is the
principal
sponsor as defined
grantee, contractor
by WHO, or
subject of an
investigator if
by WHO, or
or awardee of
principal
investigational
designated by
principal
federal funding; if
investigator (PI) if
new drug
sponsor
investigator (PI) if
designated by
designated by
application or an
designated by
sponsor, grantee,
sponsor and if PI is application for an
sponsor and if PI
contractor or
responsible for
investigational
is responsible for
awardee - principal conducting the
device exemption
conducting the
investigator.
trial, has access to
— the sponsor; if
trial, has access to
and control over
not — the person
and control over
data, has the right
that provides the
data, has the right
to publish trial
largest share of
to publish trial
results, and has the
monetary support,
results, and has
responsibility to
but if that person
the responsibility
meet the RP
is federal or state
to meet the RP
responsibilities.
agency — the
responsibilities.
principal
investigator; if the
main funder is a
nonprofit — the

CRS-18
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
nonprofit alone or
jointly with the
principal
investigator; if a
request is made to
the Secretary that
another person be
the RP, and that
person provides
monetary support
for the trial is
responsible for the
conduct of the trial
and will be
responsible for
submitting
required trial
information —
that person.

CRS-19
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
Who Receives
REGISTRY:
REGISTRY:
BOTH:
BOTH:
BOTH:
BOTH:
Information
HHS Secretary,
Director of NIH
Director of NIH
HHS Secretary,
Director of NIH
Director of NIH
acting through the
acting through the
NIH Director
NIH Director
Timing of
REGISTRY:
REGISTRY:
REGISTRY:
REGISTRY:
REGISTRY:
REGISTRY:
Submission
Not later than 21
-Initially: not later -Initially: not later
-Initially: not
-Initially: not
-Initially: not
days after the
than 21 days after
than 14 days after
later than 21 days
later than 14 days
later than 14 days
approval of the
the first patient is
first patient is
after the trial is
after first patient
after first patient
protocol
enrolled.
enrolled
opened for
is enrolled
is enrolled
-Change in
-Updates: not less
enrollment.
-Change in
-Updates: not less
enrollment status: than once every 6
RESULTS:
Enrollment
than once every 6
not later than 30
months
-Initially: implied Status: not later
months
days after change.
-Change in
same date as for
than 30 days after
-Change in
-Completion of
Enrollment
registry. (To the
change
Enrollment
trial: not later than Status: not later
extent practicable, -Final
Status: not later
30 days after the
than 30 days after
the Secretary
Submission: Not
than 30 days after
last patient enrolled change
ensures that where later than 30 days
change
in the clinical trial
-Notice of trial
the same
after last enrolled
-Notice of trial
has completed his
completion: Not
information is
patient has last
completion: Not
or her last medical
later than 30 days
required for the
medical visit
later than 30 days
visit, whether the
after final
registry and the
RESULTS:
after final
clinical trial
collection of data
database (such as
-Generally: Not
collection of data
conducted
from subjects for
initial information later than 1 year
from subjects for
according to the
primary and
required for the
after last enrolled
primary and
prespecified
secondary
database), a
patient has last
secondary
protocol or plan
outcomes
process exists to
medical visit
outcomes
was terminated
RESULTS:
allow the RP to
(extensions
RESULTS:
(extensions
-Generally: Not
make only one
possible).
-Generally: Not
possible).
later than 1 year
submission.
-Changes in
later than 1 year

CRS-20
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
after earlier of
-Results: not later regulatory status: after earlier of
estimated or actual
than 1 year than
within 30 days
estimated or actual
completion date
the earlier of the
after change
completion date
(extensions
trials’ estimated or
(extensions
possible)
actual completion
possible)
-Updates: every 6
date (extensions
-Updates: every 6
months for 10 years possible).
months for 10
from when initial
BOTH:
years from when
posting was
-Changes:
initial posting was
required
within 30 days of
required
-Changes in
the date on which
-Changes in
regulatory status:
the RP or principal
regulatory status:
within 30 days after investigator
within 30 days
change
became aware of
after change
the change
Timing of
REGISTRY:
REGISTRY:
REGISTRY:
BOTH:
REGISTRY:
REGISTRY:
Posting
Not specified
-Trials of drugs
-Not specified
In making
-Not specified
-Not specified
and biological
(NIH Director
information about
(NIH Director
(NIH Director
products: within
ensures the registry clinical trials
ensures the
ensures the
30 days of
information is
publicly available, registry
registry
submission
made publically
the Secretary shall information is
information is
-Trials of devices:
available via
make information
made publically
made publically
within 30 days of
Internet) except
available as soon
available via
available via
clearance under
that NIH Director
as practicable after Internet)
Internet)
section 510(k) of
may not make
receiving the data, RESULTS:
RESULTS:
the FFDCA or
registry
and shall seek to
(delays of up to 2
(delays of up to 2
approval under
information about
be as timely and
years possible if
years possible if
sections 515 or
device trials public
transparent as
seeking
seeking
520(m) of the
until the device is
possible.
publication)
publication)

CRS-21
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
FFDCA
approved or cleared (Postponement
-Pre-approval
-Pre-approval
-Links to trial
by FDA.
and extensions for studies: not later
studies: not later
results (from FDA RESULTS: (delays publication are
than 30 days after
than 30 days after
and NIH
of up to 2 years
possible).
approval or
approval or
information) that
possible if seeking
issuance of not
issuance of not
form the basis of
publication)
approvable letter
approvable letter
an efficacy claim
-Pre-approval
-Post-approval
-Summaries of
or are conducted
studies: not later
studies generally: medical, clinical
after the drug or
than 30 days after
not later than 30
pharmacology
biologic is
approval or
days after
reviews of pre-
approved or the
issuance of not
submission
approval and
device is cleared
approvable letter
-Post-approval
new use studies:
or approved: not
-Summaries of
studies of new
within 90 days of
earlier than 30 days medical, clinical
uses in which the applicable date
after the date of
pharmacology
manufacturer is a -Post-approval
approval or
reviews of pre-
trial sponsor and studies generally:
clearance, not later
approval and new
certifies it is
within 30 days of
than 30 days after
use studies: within
seeking or will
submission
the produce
90 days of
seek approval
-Post-approval
becomes publically applicable date
within 1 year: not studies of new
available.
-Post-approval
later than 30 days
uses in which
studies generally:
after approval,
manufacturer is a
within 30 days of
issuance of not
trial sponsor and
submission
approvable letter,
certifies it is
-Post-approval
or application
seeking or will
studies of new
withdrawal, or 2
seek approval
uses in which
years after
within 1 year: not
manufacturer is a
certification.
later than 30 days
trial sponsor and
after approval,
certifies it is
issuance of not

CRS-22
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
seeking or will
approvable letter,
seek approval
or application
within 1 year: not
withdrawal; or 2
later than 30 days
years after
after approval,
certification.
issuance of not
approvable letter,
or application
withdrawal; or 2
years after
certification.
Searchable By
REGISTRY:
REGISTRY:
REGISTRY:
BOTH:
REGISTRY:
REGISTRY:
Not specified (But
-Indication, using
-Trial enrollment
Not specified (But -Enrollment status -Trial enrollment
for a list of required
Medical Subject
status
for a list of
-Approval status
status
data elements, see
Headers
-Trial sponsor
required data
RESULTS:
-Trial sponsor
that entry below.)
-Source of support
RESULTS:
elements, see that
-Each financial
RESULTS:
-Study phase
-Status of FDA
entry below.)
sponsor
-Status of FDA
-Treatment
application
-Clinical trial
application
-Recruitment status -Trial phase
phase
-Trial phase
-Age group
-Product name
-Safety issue
-Product name
(including pediatric -Each financial
-Drug name
-Each financial
subpopulations)
sponsor
BOTH:
sponsor
-Study location
BOTH:
-Indication, using
BOTH:
-National Clinical
-Indication, using
Medical Subject
-Indication, using
Trial number or
Medical Subject
Headers
Medical Subject
other identification Headers
-Sponsor
Headers
number
-Safety issue being
-Safety issue
studied
being studied
-Trial sponsor

CRS-23
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
Trials Included
REGISTRY:
REGISTRY:
BOTH:
REGISTRY:
REGISTRY:
BOTH:
-Investigational new -Device trials:
-Drug, device,
- Non-phase I
-Premarket:
-Drug, device,
drug trials: trials
prospective study
biologic clinical
clinical trials of
Trials to verify
biologic clinical
(whether federally or
of health outcomes
trials: trials testing drugs, devices,
efficacy and
trials: Trials
privately funded) of
comparing an
a products’ safety
biologics: trials
establish doses
testing a products’
experimental
intervention against or effectiveness if
testing a treatment -Confirmatory:
safety or
treatments for serious a control in human
conducted in the
for a
All
effectiveness if
or life- threatening
subjects intended to U.S. or if the
life-threatening
RESULTS:
conducted in the
diseases and
support an
product has FDA
disease or
-Premarket:
U.S. or if the
conditions under
application under
approval or is the
condition, that are Trials to verify
product has FDA
regulations
section 520 (m) [re
subject of an
federally funded,
efficacy and
approval or is the
promulgated pursuant humanitarian
application for
used in requesting establish doses if
subject of an
to section 21 USC
devices] or 515 [re
FDA approval.
FDA approval,
recommended by a application for
355(i) [re
premarket approval
and/or conducted
required GAO
FDA approval.
investigational new
of devices] or a
in the United
study and required
drugs].
report under section
States.
by the HHS
-Treatment use of
510(k) [re device
RESULTS:
Secretary through
investigational new
clearance] of the
- Non-phase I
rulemaking;
drugs:
FFDCA; pediatric
Drug, device, or
fast track product
information
postmarket
biologic clinical
trials if used as the
pertaining to
surveillance as
trials, and those
basis for efficacy.
experimental
required under
required by the
-Confirmatory:
treatments for serious section 522 of the
HHS Secretary in Premarket
or life-threatening
FFDCA (as
the interest of
confirmatory trials
diseases and
amended by the
public health: if
BOTH:
conditions that may
bill).
federally funded,
-Postmarket: all.
be available -
-Drug and biologic
used in requesting -Pediatric
(i) under a treatment
trials: a controlled
FDA approval,
Pharmacokinetic:
investigational new
clinical
and/or
all
drug application that
investigation of a
conducted in the
has been submitted to product subject to
United States.

CRS-24
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
the Secretary under
section 505 [re
BOTH:
21 USC 360bbb(c);
drug approval] or
clinical trial
or
351 [re approval of
means a research
(ii) as a Group C
biological
study in human
cancer drug (as
products] of the
volunteers to
defined by the
FFDCA.
answer specific
National Cancer
-Other trials:
health questions,
Institute).
voluntary
including
submissions may
treatment,
be made.
prevention,
diagnostic,
screening, and
quality-of-life
trials
Exceptions
REGISTRY:
REGISTRY:
BOTH:
BOTH:
BOTH:
BOTH:
(trials not
Information relating
-Device trials:
-Pharmacokinetic
-Phase I clinical
-Exploratory
-Pharmacokinetic
included)
to an investigation if
limited studies to
and toxicity
trials conducted
trials solely to
and toxicity
the sponsor has
gather essential
studies: a clinical
solely to test the
assess safety,
studies: a clinical
provided a detailed
information used to trial to determine
safety of an
evaluate
trial to determine
certification to the
refine the device or the safety of a use
unapproved drug
pharmacokinetics, the safety of a use
Secretary that
design a pivotal
of a drug that is
or unlicensed
or verify efficacy
of a drug that is
disclosure would
trial and that is not
designed solely to
biological product, -Observational
designed solely to
substantially interfere intended to
detect major
pilot or feasibility
studies
detect major
with the timely
determine safety
toxicities in the
studies conducted
toxicities in the
enrollment of
and effectiveness of drug or to
to confirm the
drug or to
subjects in the
a device.
investigate
design and
investigate
investigation, unless
-Drug and
pharmacokinetics,
operating
pharmacokinetics,
the Secretary, after
Biologic Trials:
unless the clinical
specifications of
unless the clinical
the receipt of the
Phase I trials.
trial is designed to
an unapproved or
trial is designed to
certification, provides
investigate
not yet cleared
investigate

CRS-25
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
the sponsor with a
pharmacokinetics
medical device
pharmacokinetics
detailed written
in a special
may be included
in a special
determination that
population or
with RP consent.
population or
such disclosure
populations; and
-Clinical trials of
populations; and
would not
-Feasibility
other health-
-Feasibility
substantially interfere
studies: a small
related
studies: a small
with such enrollment.
clinical trial to
interventions may
clinical trial to
determine the
be included with
determine the
feasibility of a
consent of RP.
feasibility of a
device, or a clinical
device, or a
trial to test
clinical trial to test
prototype devices
prototype devices
where the primary
where the primary
focus is feasibility.
focus is feasibility.
Registry Data
-Purpose of each
-WHO elements
-WHO elements
-Trial title
-Sponsor
-WHO elements
Elements
experimental drug
(See Appendix A.) (See Appendix A.) -Unique identifier
-Trial purpose
(See Appendix
-Eligibility criteria
-City, state, zip
-City, state, zip
-Trial description
-Patient
A.)
-Location of trial
code of study
code, toll free
-Trial phase
population
-City, state, zip
sites
-Toll free number
phone number of
-Trial type
description
code of study
-Point of contact for
for study
study
-Trial purpose
-General
-Estimated
enrollment
-Whether there is
-Estimated
-Primary,
description of
completion date
-Description of
expanded access
completion date
secondary
results, trial design -RP identity and
whether and how the
for unapproved
-RP identity and
outcome measures changes, and
contact
manufacturer or
drugs and biologics contact information -Date outcome
reasons for
information
sponsor will respond
under FFDCA
-Whether there is
measures will be
changes
-Whether there is
to requests for
section 561 [re
expanded access
assessed
-WHO elements
expanded access
protocol exception,
emergency
for unapproved
-Dates and details
(See Appendix
for unapproved
with appropriate
situations, patient
drugs and biologics of revisions to
A.)
drugs and
safeguards, for
access to
under FFDCA
outcomes
-City, state, zip
biologics under
single- patient and
treatments for
section 561 [re
-Eligibility and
code of study
FFDCA section

CRS-26
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
expanded protocol
serious diseases,
emergency
exclusion criteria
-Whether
561 [re emergency
use of the new drug,
treatment uses]
situations, patient
-Whether and how compassionate use situations, patient
particularly in
-Other data
access to
requests for
is available
access to
children
elements as
treatments for
single-patient and
-Elements
treatments for
-With sponsor
appropriate
serious diseases,
expanded protocol specified by
serious diseases,
consent, may include
-Links to results
treatment uses]
use (particularly in Secretary
treatment uses]
information about the from certain FDA
-Restrictions on
children) will be
-Restrictions on
results of included
submissions, NIH
non-employees’
addressed
non-employees’
trials, including
information
discussion or
-Trial and
discussion or
potential toxicities or (Medline cites and
publication of
enrollment status
publication of
adverse effects
NLM database of
results
at individual sites
results
product labels), and -Elements specified -Estimated
-Elements
previously existing
by Secretary
completion date
specified by
databank entries
-Trial location
Secretary
-RP identity and
contact
information
-Sponsor
-Funding source
-Experimental
treatments for
serious or life-
threatening
conditions
available under a
treatment
investigational
new drug
application or as a
Group C cancer
drug.

CRS-27
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
Results Data
None.
None.
-Registry data
-Title
-Indication studied -Indication studied
Elements
elements, plus:
-Unique identifier
-Safety issue
-Safety issue
TECHNICAL
-Product tested
-Status of FDA
-Status of FDA
SUMMARY:
-Trial description
application
application
-Each sponsor
in lay language
-Trial phase
-Trial phase
-Scientific point of
-Trial phase, type
TECHNICAL
TECHNICAL
contact
-Trial purpose
REPORT:
REPORT:
-Description of
-Demographic
-Each sponsor
-Each sponsor
patient population
data
-Scientific point of -Scientific point of
-Summary data
-Estimated
contact
contact
describing
completion date
-Description of
-Description of
achievement of
-Study sponsor
patient population patient population
primary and
and funding
-Summary of
-Summary of
secondary
source
aggregate data
aggregate data
endpoints,
-Primary,
assessing primary
assessing primary
assessment of
secondary
and secondary
and secondary
secondary
outcome measures endpoints, safety
endpoints, safety
endpoints, safety
-Date outcome
information
information
information
measures assessed -Information about -Information about
-Information about
-Dates, details of
subjects quit trial
subjects quit trial
subjects who quit
outcome revisions -Restrictions on
-Restrictions on
trial
-Actual
non-employees’
non-employees’
-Restrictions on
completion date,
discussion or
discussion or
non-employees’
reason for
publication of
publication of
discussion or
difference from
results.
results.
publication of
estimate
-Link to peer
-Link to peer
results
-If terminated,
reviewed
reviewed
-Link to peer
reason for
publications
publications
reviewed
termination
-completion date
-completion date
publications
-Results summary
-FDA adverse
-FDA adverse
-completion date
with trial design,
regulatory action
regulatory action

CRS-28
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
-FDA adverse
methodology,
NONTECHNICA NONTECHNI-
regulatory action
outcome
L REPORT:
CAL REPORT:
NONTECHNI-
measures,
-Point of contact
-Point of contact
CAL SUMMARY: summary data
-General
-General
-Point of contact
tables, statistical
description of
description of
-General
significance of
results, trial design results, trial design
description of
results
changes, and
changes, and
results, trial design
-Safety data,
reasons for
reasons for
changes, and
including adverse
changes
changes
reasons for changes event information
BOTH
BOTH
BOTH
-Peer-reviewed
REPORTS:
REPORTS:
REPORTS:
publications
-Trial purpose
-Trial purpose
-Trial purpose
-Description of
-Trial sponsor
-Trial sponsor
-Trial sponsor
results review
-General
-General
-General
process, protocol
description of
description of
description of
-Status of FDA
results, trial design results, trial design
results, trial design
application or
changes, and
changes, and
changes, and
reason trial not
reasons for
reasons for
reasons for changes submitted to FDA
changes
changes
[NIH Director to
include links to
Medline citations,
NLM database
product labels,
prior databank
entries.]
Enforcement
REGISTRY:
REGISTRY:
BOTH:
BOTH:
BOTH:
BOTH:
and Corrections None specified.
-RP ensures
-RP ensures
-Sponsors of FDA -RP ensures
-RP ensures
General mechanisms
submissions not
submissions not
new drug
submissions not
submissions not
for enforcing
false or misleading. false or misleading. applications
false or
false or

CRS-29
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
compliance with
-No federal agency
-No federal agency
submit to
misleading.
misleading.
FDA requirements
may release
may release
Secretary
-No federal
-No federal
may be applicable,
research grant
research grant
certification of
agency may
agency may
but have not been
funds to
funds to
compliance with
release grant funds release research
applied by FDA.
noncompliant RPs.
noncompliant RPs.
FFDCA. If not,
to noncompliant
grant funds to
-For applicable
-Secretary consults
after hearing,
RPs.
noncompliant RPs.
trials funded by
with other federal
Secretary imposes -FDA
-For applicable
FDA, NIH, AHRQ, agencies to
$10,000/day civil
Commissioner
trials funded by
or VA, progress
determine whether
monetary penalty
verifies required
FDA, NIH,
report forms
studies funded by
until certification
submissions were
AHRQ, or VA,
include
them and
submitted. If
made when
progress report
certification of
conducted under 45 information is
considering
forms include
compliance.
CFR 46 [re federal
inaccurate and
applications for
certification of
Agency heads
protections for
sponsor knew or
investigational
compliance.
verify compliance
human subjects] are should have
drug exemptions,
Agency heads
before releasing
applicable clinical
known, after
new drug
verify compliance
grant funds to RPs. trials, and to
notice and
approvals,
before releasing
Secretary consults
develop procedures hearing, Secretary biologics licences. grant funds to
with other federal
to ensure results
orders sponsor to
After notice to RP, RPs. Secretary
agencies to
submission.
pay civil monetary opportunity to
consults with
determine whether
-NIH Director
penalty of
correct, Secretary
other federal
studies funded by
checks registry to
$100,000 to
refuses to file
agencies to
them and
ensure
$2,000,000 for any application.
determine whether
conducted under 45 corresponding
30-day period.
-Secretary checks
studies funded by
CFR 46 [re federal
results are filed.
-To be eligible for registry to ensure
them and
protections for
After notice to RP,
a federal grant,
corresponding
conducted under
human subjects]
opportunity to
contract, or
results are filed.
45 CFR 46 [re
merit similar
correct, Director
cooperative
After notice to RP, federal protections
procedures.
reports
agreement,
opportunity to
for human
-Applications or
noncompliance to
principal
correct, Secretary
subjects] merit
submissions under
federal agencies
investigator
reports
similar

CRS-30
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
FFDCA sections
and Office of
certifies Act
noncompliance to
procedures.
505, 515, 520(m),
Human Research
compliance.
federal agencies
-NIH Director
351, or 510(k) [re
Protections, posts
Noncompliance,
and Office of
checks registry to
new drugs,
notice of
after notice, leads
Human Research
ensure
biologics and
noncompliance in
to ineligibility,
Protections, posts
corresponding
devices], must have registry and
posting of
notice of
results are filed.
certifications of
database.
noncompliance
noncompliance in
After notice to RP,
compliance.
-FDA
notice in database. registry and
opportunity to
-Secretary may
Commissioner to
database.
correct, Director
impose FFDCA
verify submissions
-In trial with
-Secretary ensures reports
penalties for
are made for trials
nonfederal
content is not false noncompliance to
noncompliance.
in applications
support, Act
or misleading and
federal agencies
under FFDCA
noncompliance
non-promotional
and Office of
sections 505, 505(i) leads to notice,
by checking a
Human Research
515, 520(g), 351, or opportunity to
representative
Protections, posts
510(k) [re new or
correct, hearing,
sample. After
notice of
exempt drugs,
$10,000/day
notice to RP,
noncompliance in
biologics and
penalty until
opportunity to
registry and
devices]. After 30
compliant.
correct, Secretary
database.
days after notice,
may impose
-Applications or
failure to correct
FFDCA penalties
submissions under
leads to Secretary’s
FFDCA sections
refusal to file,
505, 515, 351, or
approve, or clear
510(k) [re new
application.
drugs, biologics
-Secretary to
and devices], must
review documents
have certifications
to ensure they are
of compliance.
non-promotional,
-Secretary may
not false or
impose FFDCA
misleading. 30
penalties for

CRS-31
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
days after notice of
noncompliance,
noncompliance,
including civil
penalties may
monetary penalties
apply.
($10,000/day for
-Secretary may
first violation,
impose FFDCA
$20,000/day for
penalties for
each subsequent
noncompliance,
violation) created
including civil
by Act.
monetary penalties
created by the Act
(not more than
$10,000/day, and
not more than
$15,000/ for all
violations of an
individual or
nonprofit
adjudicated in a
single proceeding).
Required
None.
RESULTS:
BOTH: Not later
BOTH:
RESULTS:
None required by
Studies or
The NIH Director
than 1 year after
Not later than 1
Not earlier than 2
clinical trials title.
Reports
conducts a study to enactment,
year after
years after results
determine the best,
Comptroller
enactment,
database
validated methods
General submits a
Secretary submits
established,
of making trial
report to Congress
to appropriate
Comptroller
results public after
on a study to
committees of
General initiates a
the approval of a
determine whether
Congress a report
GAO study of
drug that is the
information in the
on the status of the inclusion of
subject of an
registry and
implementation of certain premarket

CRS-32
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
applicable drug
database is
Act requirements
trials: burden to
trial. Director
considered
including number
sponsors and
submits findings to promotional and to
and types of trials
agencies, benefit
the HHS Secretary
evaluate the
submitted.
to patients and
within 18 months
implementation of
REGISTRY:
health providers,
of initiating the
the database.
Secretary
recommendations.
study.
contracts with
Makes report to
IOM to conduct a
HELP, Energy and
study of the extent Commerce.
to which data
submitted to the
registry and
database have
impacted the
public health. Not
later than 6
months after the
contract date, IOM
submits study to
Secretary
Authorized
REGISTRY:
BOTH:
BOTH:
BOTH:
BOTH:
BOTH:
Appropriations
Such sums as may be $10,000,000 each
$10,000,000 each
Such sums as may $10,000,000 each
$10,000,000 each
necessary; Fees
FY
FY
be necessary
FY
FY
collected under
section 21 USC 379h
[re FDA prescription
drug user fees] may
not be used for the
registry.

CRS-33
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
Preemption
None.
BOTH:
BOTH:
None.
BOTH:
BOTH:
Yes. No state or
Yes. No state or
Yes. No state or
Yes. No state or
political
political
political
political
subdivision of a
subdivision of a
subdivision of a
subdivision of a
state may require or state may require or
state may require
state may require
effect registration
effect registration
or effect
or effect
of trials or results.
of trials or results.
registration of
registration of
trials or results.
trials or results.
Safe Harbor
None.
BOTH:
BOTH:
None.
BOTH:
BOTH:
Somewhat.
Somewhat.
Somewhat.
Somewhat.
Compliant
Compliant
Compliant
Compliant
submissions shall
submissions shall
submissions shall
submissions shall
not be considered
not be considered
not be considered
not be considered
(1) by Secretary as
(1) by Secretary as
(1) by Secretary as (1) by Secretary as
evidence of a new
evidence of a new
evidence of a new evidence of a new
intended use
intended use
intended use
intended use
different from
different from
different from
different from
labeling, or (2) as
labeling, or (2) as
labeling, or (2) as
labeling, or (2) as
FFDCA labeling,
FFDCA labeling,
FFDCA labeling,
FFDCA labeling,
adulteration, or
adulteration, or
adulteration, or
adulteration, or
misbranding.
misbranding.
misbranding.
misbranding.
Effective Dates
REGISTRY:
REGISTRY:
BOTH:
Not specified.
BOTH:
BOTH:
Currently operational -Generally:
-Databases
Databases to be
-Databases
October 1, 2007
established not
established not
established not
-Regulations
later than 1 year
later than 1 year
later than 1 year
become effective
after enactment.
after enactment.
after enactment.
90 days after
issuance of HHS
Secretary’s
issuance of final

CRS-34
Current Law
S. 1082
H.R. 2900
S. 467
S. 484
H.R. 1561
rule. (Final rule
issued pursuant to
Act to be issued not
later than 18
months after Act’s
enactment, and
after notice and
comment.)
-Funding
restrictions take
effect 210 days
after regulations’
effective date.

CRS-35
Appendix A. World Health Organization,
International Clinical Trials Registry Platform,
Registration Data Set (Version 1.0)
ITEM
DEFINITION / EXPLANATION
Primary
Name of Primary Register, and the unique ID number assigned by the Primary
Register and
Register to this trial.
Trial ID #
Date of
Date when trial was officially registered in the Primary Register YYYY/MM/DD.
Registration in
Primary
Register
Secondary ID#s
Other identifying numbers and issuing authorities besides the Primary Register, if
any. Include the sponsor name and sponsor-issued trial number (e.g., protocol
number) if available. Also include other trial registers that have issued an ID
number to this trial. There is no limit on the number of Secondary ID numbers that
can be provided.
Source(s) of
Major source(s) of monetary or material support for the trial (e.g., funding agency,
Monetary or
foundation, company).
Material
Support
Primary
The individual, organization, group or other legal person taking responsibility for
Sponsor
securing the arrangements to initiate and/or manage a study (including arrangements
to ensure that the study design meets appropriate standards and to ensure
appropriate conduct and reporting). In commercial trials, the primary sponsor is
normally the main applicant for regulatory authorization to begin the study. It may
or may not be the main funder.
Secondary
Additional individuals, organizations or other legal persons, if any, that have agreed
Sponsor(s)
with the primary sponsor to take on responsibilities of sponsorship.
A secondary sponsor may have agreed
-to take on all the responsibilities of sponsorship jointly with the primary sponsor;
or
-to form a group with the primary sponsor in which the responsibilities of
sponsorship are allocated among the members of the group; or
-to act as the sponsor’s legal representative in relation to some or all of the trial
sites; or
-to take responsibility for the accuracy of trial registration information submitted.
Contact for
Email address, telephone number, or postal address of the contact who will respond
Public Queries
to general queries, including information about current recruitment status
Contact for
Email address, telephone number, or postal address, and affiliation of the person to
Scientific
contact for scientific queries about the trial (e.g., principal investigator, medical
Queries
director employed by the sponsor). For a multi-center study, enter the contact
information for the lead Principal Investigator or overall scientific director.
Public Title
Email address, telephone number, or postal address, and affiliation of the person to
contact for scientific queries about the trial (e.g., principal investigator, medical
director employed by the sponsor). For a multi-center study, enter the contact
information for the lead Principal Investigator or overall scientific director.

CRS-36
Scientific Title
Scientific title of the study as it appears in the protocol submitted for funding and
ethical review. Include trial acronym if available.
Countries of
The countries from which participants will be, are intended to be, or have been
Recruitment
recruited.
Health
Primary health condition(s) or problem(s) studied (e.g., depression, breast cancer,
Condition(s) or
medication error). If the study is conducted on healthy human volunteers belonging
Problem(s)
to the target population of the intervention (e.g., preventative or screening
Studied
interventions), enter the particular health condition(s) or problem(s) being
prevented. If the study is conducted using healthy human volunteers not belonging
to the target population (e.g., a preliminary safety study), an appropriate keyword
will be defined for users to select.
Intervention(s)
Enter the specific name of the intervention(s) and the comparator/control(s) being
studied. Use the International Non-Proprietary Name if possible (not brand/trade
names). For an unregistered drug, the generic name, chemical name, or company
serial number is acceptable. If the intervention consists of several separate
treatments, list them all in one line separated by commas (e.g., “low-fat diet,
exercise”).
The control intervention(s) is/are the interventions against which the study
intervention is evaluated (e.g., placebo, no treatment, active control). If an active
control is used, be sure to enter in the name(s) of that intervention, or enter
“placebo” or “no treatment” as applicable.
For each intervention, describe other intervention details as applicable (dose,
duration, mode of administration, etc).
Key Inclusion
Inclusion and exclusion criteria for participant selection, including age and sex.
and Exclusion
Criteria
Study Type
A single arm study is one in which all participants are given the same intervention.
Trials in which participants are assigned to receive one of two or more interventions
are NOT single arm studies. Crossover trials are NOT single arm studies.
A trial is “randomized” if participants are assigned to intervention groups using a
method based on chance (e.g., random number table, random computer-generated
sequence, minimization, adaptive randomization).
Date of First
Anticipated or actual date of enrollment of the first participant (YYYY/MM).
Enrollment
Target Sample
Number of participants that this trial plans to enroll.
Size
Recruitment
Recruitment status of this trial.
Status
-Pending: participants are not yet being recruited or enrolled at any site
-Active: participants are currently being recruited and enrolled
-Temporary halt: there is a temporary halt in recruitment and enrollment
-Closed: participants are no longer being recruited or enrolled
Primary
Outcomes are events, variables, or experiences that are measured because it is
Outcome(s)
believed that they may be influenced by the intervention. The Primary Outcome
should be the outcome used in sample size calculations, or the main outcome(s)
used to determine the effects of the int[ervention(s).
Enter the names of all primary outcomes in the trial as well as the pre-specified
timepoint(s) of primary interest. Be as specific as possible with the metric used
(e.g., “% with Beck Depression Score > 10” rather than just “depression”).
Examples:
Outcome Name: all-cause mortality, Timepoints: 5 years; or Outcome Name: Mean
Beck Depression Score, Timepoint: 18 weeks

CRS-37
Secondary
Secondary outcomes are events, variables, or experiences that are of secondary
Outcome(s)
interest or that are measured at timepoints of secondary interest. A secondary
outcome may involve the same event, variable, or experience as the primary
outcome, but measured at timepoints other than those of primary interest (e.g.,
Primary outcome: all-cause mortality at 5 years; Secondary outcome: all-cause
mortality at 1 year, 3 years), or may involve a different event, variable, or
experience altogether (e.g., Primary outcome: all-cause mortality at 5 years;
Secondary outcome: hospitalization rate at 5 years).
Enter the name and timepoint(s) for all secondary outcomes of clinical and/or
scientific importance. Be as specific as possible with the metric used (e.g., “% with
Beck Depression Score > 10” rather than just “depression”). Examples: Outcome
Name: all-cause mortality, Timepoints: 6 months, 1 year; or Outcome Name: Mean
glycosylated hemoglobin A1C, Timepoints: 4 and 8 weeks
Source: WHO, ICTRP, “Registration Data Set (version 1.0),” (March 16, 2007), at [http://www.who.
int/ictrp/data_set/en/], visited Apr. 16, 2007.