The National Institutes of Health (NIH) has warned clinical trial sponsors – including not only drug and device companies, but also universities and hospitals with investigator-initiated trials – to submit significant additional data to “as soon as possible” or face potential penalties under the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act (PHS Act).

This follows the NIH’s policy change and updates to the FAQs, both in the wake of the government’s decision not to appeal a federal court’s ruling in Seife et al. v. HHS et al., 18-cv-11462 (NRB) (S.D.N.Y. Feb. 24, 2020).

Background on Reporting Requirements

One of Congress’ goals when it enacted the Food and Drug Administration Amendments Act (FDAAA) in 2007 was to ensure that clinical trial sponsors did not “cherry-pick” the publicly available results of their clinical trials by withholding unfavorable results. Section 801 of the FDAAA, therefore, requires that “Responsible Parties” – clinical trial sponsors or sponsor-investigators – report “Basic Results” for certain “Applicable Clinical Trials” (ACTs) on

On September 21, 2016, the Department of Health and Human Services (HHS) finalized 42 CFR Part 11 (the Final Rule), which details the FDAAA’s results reporting requirements and largely mirrors the language in the FDAAA. In the preamble to the Final Rule, however, HHS articulated an interpretation of the Basic Results reporting requirement that exempted any ACT completed before January 18, 2017 (the Final Rule effective date), as long as the FDA had not, prior to study completion, granted approval or clearance of the investigational product. This interpretation amounted to a Basic Results reporting reprieve for a large number of ACTs initiated after September 27, 2007 (or that were ongoing as of December 26, 2007) and completed by January 17, 2017. Reporting Requirement Policy Change Details

The Seife court rejected HHS’ interpretation, ruling that the FDAAA requires Responsible Parties to report Basic Results for ACTs even if FDA grants marketing authorization after the trial is completed. In light of the government’s decision not to appeal the Seife ruling, the NIH has fully adopted the holding and now requires Basic Results of ACTs to be submitted as soon as possible for all ACTs completed before January 18, 2017, if the ACT studies a product that is approved, licensed, or cleared by FDA at any time. An FAQ on further states that “FDA and NIH may take action against responsible parties if they do not submit required results information for [clinical trials] affected by the Federal Court’s decision.” Section 801 of the FDAAA authorizes significant civil monetary penalties for non-compliance, and non-compliance could also jeopardize existing or future grant funding from NIH and FDA under the PHS Act.

Next Steps for Submitting ACT Results

Responsible Parties should determine which, if any, of their clinical trials are affected by this change and prepare and submit the Basic Results of those trials to Basic Results include demographic characteristics of the ACT’s patient sample, primary and secondary outcomes, a point of contact for information regarding the ACT, and agreements that may limit what a principal investigator may say about an ACT after its completion.

If you have any questions about this new policy change or need assistance submitting previous ACT results, please contact the author.