Last week, the U.S. Department of Health and Human Services (“HHS”) announced that FDA intends to update its regulations governing clinical studies of new drugs. More specifically, FDA intends to update Parts 312 and 16 of Title 21 of the Code of Federal Regulations (the “Code” or “CFR”). In its announcement, HHS stated that the revisions will be focused on defining and clarifying “the roles and responsibilities of the various persons engaged in the initiation, conduct, and oversight of clinical investigations subject to [investigational new drug] requirements.” The announcement also notes that the changes will “better protect the rights, safety and welfare of subjects and help ensure the integrity of clinical trial data.”
I don’t think anyone disagrees that these are important goals and that FDA should be commended for recognizing their importance and taking steps to achieve them. However, these are not the only goals FDA should be striving to achieve as it revamps these regulations. As noted in the January 29, 2015 Innovation for Healthier Americans report issued by Senators Alexander and Burr, clinical trials are becoming longer and the number of procedures subjects are required to undergo continues to increase. This not only makes trials more expensive, but also makes it harder to enroll and retain subjects. Addressing the rising costs and enrollment difficulties is one of the drivers behind the 21st Century Cures Act which is working its way through the House and the companion legislation working its way through the Senate. Although addressing these goals has bipartisan support and FDA legislation traditionally has been less susceptible to the partisan gridlock in Congress, there is no guarantee that Congress will be able to address these issues legislatively. Therefore, it would be advisable for FDA also to focus on streamlining the regulatory process and establishing a framework for the use of surrogate endpoints and adaptive clinical trial designs. This can serve two purposes. First, it hedges against the possibility that a legislative fix will not materialize. Second, it reduces the potential that FDA will need to revisit these regulations again if a legislative fix does materialize.
In addition to expanding the goals FDA is pursuing with these changes, FDA should also expand the scope of the regulations that it will update to achieve these goals. According to the announcement, FDA only intends to update Parts 312 and 16. However, it is unclear how FDA can achieve these goals if it limits the scope of changes to these parts of the Code. Part 312 primarily focuses on the division of responsibilities as between the sponsor and the investigator. However, there are other parties and entities responsible for the oversight of clinical investigations, including institutional review boards (IRBs) and the institutions where the studies are conducted. Specifically, IRBs are responsible for ensuring the protection of rights, safety, and welfare of clinical trial subjects. Although Part 312 requires the Sponsor and the Investigator to ensure IRB approval and continuing oversight, Part 312 does not specifically address the roles and responsibilities of an IRB or the research institutions. Instead, these are addressed in Part 56. Therefore, it is unclear how FDA will be able to effectively clarify the “roles and responsibilities of the various persons engaged in the initiation, conduct, and oversight of clinical investigations” and “better protect the rights, safety and welfare of subjects” without also updating Part 56.
I look forward to seeing the changes FDA proposes to make to Part 312. I just hope that these changes enhance the protection of study subjects and ensure the integrity of the data generated by clinical trials in a manner that also makes conducting and participating in clinical trials less burdensome than it is currently.
 21 CFR Part 16 governs regulatory hearings to which investigators are entitled to in the event they are disqualified pursuant to 21 CFR § 312.70 or to which sponsors are entitled in the event FDA terminates an IND.