The US District Court for the Southern District of New York issued a potentially significant opinion with respect to ClinicalTrials.gov results posting on February 24. If upheld, clinical study sponsors and investigators may need to post certain study results for 10 years’ worth of clinical trials (2007–2017), which the US Department of Health and Human Services (HHS) had previously excluded from the requirement.
By way of background, the 2007 FDA Amendments Act (FDAAA) required that HHS include certain study results, referred to as basic results, for approved drugs and biologics on ClinicalTrials.gov. Clinical study sponsors and investigators were required to submit these study results within one year of the estimated or actual study completion date, whichever was earlier, or such other period that may be provided by regulation. However, if a study investigated a product that was not yet FDA approved, the FDAAA provided for the delayed posting of study results.
In 2016, HHS promulgated ClinicalTrials.gov regulations, which were effective January 18, 2017. Under the final rule, if a study’s primary completion date predated the rule’s effective date, basic study results would only be required to be posted if the applicable drug or biologic was approved at the time of the study’s completion.
Specifically, and as quoted in the court’s decision, the preamble to the final rule stated:
For purposes of this final rule, the marketing status of a product will be determined based on its marketing status on the primary completion date [of the study]. Thus, if a drug product . . . is approved . . . for any use as of the primary completion date, we will consider that [study] to be a trial of an approved . . . product. Similarly, if a drug product . . . is unapproved . . . for any use as of the primary completion date, regardless of whether it is later approved . . . we will consider that [study] to be a trial of an unapproved . . . product.
As a result, and as described by the court, if a study was “completed after the enactment of FDAAA . . . but before the [f]inal [r]ule’s effective date . . . [sponsors and investigators] need not submit [basic results] if the [study] studied a product that was approved after the [study’s] completion date.”
In its opinion, the court found that the “FDAAA unambiguously requires [sponsors and investigators of approved products] to submit, and ClinicalTrials.gov to include,” basic results for studies completed after the enactment of the FDAAA but before the effective date of the ClinicalTrials.gov final rule, even if, at the time of the study’s completion, the applicable product was not yet approved.
The court further rejected the government’s argument that such a ruling requires sponsors to retroactively post 10 years of study results that were not previously required. The court stated that sponsors and investigators “knew since the FDAAA’s enactment in 2007 that the statute required them to submit [basic results] for each [applicable study] of a product that is approved. It was only when HHS promulgated the [f]inal [r]ule nearly ten years after FDAAA’s enactment and almost six years after the statutory deadline for doing so, and included in its preamble an interpretation of it that was contrary to the text of the FDAAA, that HHS definitively told responsible parties that they were not required to submit [basic results] for pre–[final rule]” studies of unapproved products.
The exact impact of the court’s ruling is yet to be seen. Technically, as a result of the ruling, sponsors and investigators that did not post basic study results in reliance on HHS’s prior interpretation could be deemed to be out of compliance with the statutory posting requirements and subject to enforcement action, including monetary penalties. However, exactly how the government will respond to this holding is currently unclear. Some possible options would be for HHS to require the retroactive posting of study results within a certain timeframe, to exercise an official or unofficial policy of enforcement discretion, or to appeal the court’s decision.
While industry waits to see what HHS will do, it would be prudent for sponsors and investigators to review their clinical studies to see whether any fall into the category of studies of unapproved products completed after the FDAAA but prior to the ClinicalTrials.gov final rule, which may require retroactive results posting. Sponsors and investigators should also consider beginning to compile the information required for results posting so that if (or when) HHS requires the posting of these additional results, the applicable information is readily accessible.