SAN DIEGO—As sponsors prepare for the pending clinical trial diversity requirements from the US Food and Drug Administration, they should not anticipate receiving any waivers, Tamy Kim, director for regulatory affairs and policy at the agency’s Oncology Center of Excellence, said.
Speaking to a standing-room-only crowd at the DIA Global Annual Meeting on 17 June, Kim said, “FDA can waive the requirement to submit a diversity action plan. We anticipate that this will be very rare. However, it can happen.”
Key Takeaways
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Once the diversity action plan requirement goes into effect, FDA will allow waivers, but only during public health emergencies or if sponsors show that studies can’t be conducted with one.
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The much-anticipated guidance outlining what should be in a diversity action plan is nearly six months overdue.
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Sponsors that don’t follow a diversity action plan can expect to face an advisory committee or a post-marketing commitment.
To get a waiver, FDA must determine that prevalence or incidence of the disease “makes it impractical to carry out a study with the diversity action plan” or there must be a public health emergency that would impede the plan, she said.
“If a diversity action plan waiver is requested ... the FDA must grant or deny the waiver within 60 days of receipt,” Kim said.
Kim was essentially repeating the criteria outline in the Food and Drug Omnibus Reform Act, which create the diversity action plan requirement. So far, the statute remains the only official information about how sponsors should create their “goals for clinical study enrollment, disaggregated by age group, sex, race, geographic location, socioeconomic status, and ethnicity” for Phase III studies.
FDA was supposed to issue a guidance on the “format and content” of the plans within one year after the law was signed (29 December 2022) so the document is almost six months overdue, though it is now under White House review and could be released shortly. The agency is mandated to finalize the guidance nine months after the comment period closes, and the requirements go into effect six months after the final guidance is issued.
Assuming a standard 60-day comment period and that the draft will be released sooner rather than “FDA soon,” sponsors likely have less than two years before they are required to submit diversity action plans or request waivers.
FDA’s Compliance Conundrum
If a sponsor doesn’t submit, or doesn’t follow, a diversity action plan, there is no particular enforcement specified in the legislation. FDA could, of course, simply not approve an application that was produced without a plan. But when asked about potential consequences, Kim did not go that far.
“It’s kind of a complicated issue, because the guidance is not out yet,” she said. “However, we do have experience when sponsors, either commercial or research, are not hitting their enrollment goals. It kind of depends on how far off they are.”
If a sponsor is “not too far off, this is not that bad. You’re putting in the effort and only like 2% off. Okay, like way to go. Here’s a pat on the back, keep on doing what you’re doing. Some of them are in very small populations like a pediatric cancer population. So to a certain extent you connect to the situation and see if that makes sense.”
However, “if it’s something that’s drastically wrong, and there’s examples of this … if you’re way off it could be discussed at an advisory committee. Not only that, … you may have to address it in a [post marketing commitment], particularly with a safety issue.”
Kim also reassured sponsors that “global data could be used to support a diversity action plan. However, you do have to watch out for certain things like availability of certain therapies outside of the US … as well as an environmental extrinsic factors that may affect the etiology of disease.”
Senior FDA leadership has also taken pains to say that the diversity requirements will not be onerous, with Commissioner Robert Califf noting recently that the agency would not expect population-specific quotas as part of the action plans. (Also see "US FDA Commissioner Robert Califf Says No Formulas For Trial Diversity" - Pink Sheet, 12 June, 2024.)
Some in industry, however, have said that a firmer hand by the agency could actually be preferable in order to encourage use of the plans, given sponsor worries about the time and expense they might add to development plans. (Also see "‘Do It Or We’re Not Going To Approve Your Drug:’ Industry Reps Ask FDA For Trial Diversity Sticks" - Pink Sheet, 14 May, 2024.)