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Proposed Rule Calls for Clinical Trial Results to be Reported, Even When Product Isn't Approved

Posted 19 November 2014 | By Alexander Gaffney, RAC

Proposed Rule Calls for Clinical Trial Results to be Reported, Even When Product Isn't Approved

The US Department of Health and Human Services (DHHS) today unveiled new proposed requirements for its online clinical trials register,, that will require pharmaceutical and medical device companies to submit more information regarding patient enrollment, the progress of trials and the results of those trials--even when a product is never approved in the US.


Under section 801 of the Public Health Service Act (PHSA), as amended by the Food and Drug Administration Amendments Act (FDAAA) of 2007, DHHS was directed to establish a clinical trial registry data bank. That data bank,, is run by the National Institutes of Health (NIH)'s National Library of Medicine (NLM).

The website serves as a unified hub for the oversight of clinical trials, and includes information regarding the sites where a trial is conducted, the general design of a study and information about its sponsor.

As DHHS explains in its new Federal Register notice, "Under section 402(j) of the PHSA, those responsible for specified clinical trials of FDA-regulated products have been required to submit registration information to since 26 December 2007, summary results information for clinical trials of approved products since 27 September 2008, and adverse events information since 27 September  2009."

New Proposed Rule

Under a new proposal unveiled on 19 November 2014, numerous changes would be made to the way in which clinical trials results are recorded and reported.

For example, DHHS's proposal calls for one—and only one—entity to be responsible for submitting information about an "applicable clinical trial." Unless otherwise determined, that entity will always be the sponsor of the clinical trial, DHHS said.

Another change: Trials will have to be registered within 21 days after enrolling the first patient, and include information on how to enroll in the study, the location of the study, contact information for the study and data elements meant to "improve" the public's understanding of the trial.

For the first time, the information provided to will also need to include information on "how to obtain expanded access to investigational drugs used in applicable clinical trials," but only if those products are available through an expanded access (e.g. "compassionate use") program.

Focus on Results

The proposed changes also focus heavily on the submission of results. The rule "proposes to extend the requirement for results submission to applicable clinical trials of drugs, biological products, and devices that are not approved, licensed, or cleared by FDA," DHHS explains in its Federal Register notice.

"This proposed rule would require the submission of tables of data summarizing demographics and baseline characteristics of the enrolled participants and primary and secondary outcomes, including results of any scientifically appropriate statistical tests."

Those results would need to be submitted "not later than one year after the completion date of the clinical trial," DHHS noted. "No patient-specific data are required to be submitted by this proposed rule or by the law this proposed rule is intended to implement," it noted.

The number of adverse events suffered by patients would also need to be included in the results reported to the register—an unusual addition considering FDA already accepts and analyzes much of that information. However, the adverse event information could be particularly useful for further research on products which never obtained approval, for which little regulatory data is generally available.

Adverse event information would need to be summarized in one submitted table of information, while another table would summarize all events which took place at a frequency greater than 5% in any arm of the trial "regardless of whether such adverse events were anticipated or unanticipated," DHHS wrote.


Clinical Trials Registration and Results Submission

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