FDA draft guidance may turn diversity in clinical trials from nice-to-have into a must-have

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Supporting diversity in clinical trials and actually achieving it are two different issues. There is much agreement in pharma that including a broad swath of participants in clinical trials leads to better drugs and outcomes, but most studies in the US are still largely recruited from the same homogenous group. A draft guidance from the FDA aims to change that.

The agency is building upon past guidance providing a roadmap for sponsors to develop a race and diversity plan, with the goal of enrolling more people from underrepresented racial and ethnic groups in clinical trials.

Aman Khera, Global Head of Regulatory Strategy at Worldwide Clinical Trials

Permission granted by Worldwide Clinical Trials

“This new guidance has upped the ante by making diversity considerations a ‘must-have’ versus a ‘nice-to-have’ in clinical trials,” says Aman Khera, the global head of regulatory strategy at Worldwide Clinical Trials. “It brings accountability into the drug development process while showing how seriously the agency will respond.”

According to the draft guidance, sponsor companies are now responsible for a tangible deliverable to the FDA, Khera says.

“They will be expected not only to understand potential equity barriers but to explain what they’re doing to overcome them for their product in development,” she explains.

The FDA may refine the draft guidance before it’s finalized, and the public comment period ends on June 13. But the industry shouldn’t wait to start preparing, Khera says.

“I’m sure that we’ll see some quick lessons even as early as this year,” she says. “Therefore, sponsor companies close to filing may even want to go into pre-NDA, pre-BLA or other meetings with the FDA and discuss that perhaps the trial populations might not hit as diverse a patient population as desired — and be open to negotiation on next steps.”

In particular, Khera says the FDA will be looking for proof in the form of data and metrics.

“There will be no way around it; We’ll have to demonstrate actionable targets that ensure a clinical trial population truly represents the patient population,” Khera says.

A long-standing problem

If there was ever any question that clinical trials had a diversity problem, COVID-19 vaccine trials erased all doubt, says Fabio Gratton, chief experience officer at THREAD, a technology and consulting service provider enabling electronic clinical outcome assessments and decentized clinical trials (DCTs ).

“You would recruit a 30,000-person vaccine trial, and within a month, you almost had it recruited, and 98% of the people were white, between the ages of 25 and 40,” he says. “Where was everybody else?”

The lack of diversity in clinical trials is due to a host of factors.

“It’s not like people are going out of their way to exclude [certain groups]. I think it’s just that they’re starting with the low-hanging fruit,” Gratton says.

It takes a lot more effort to search out a diverse group of participants than it does to dip the bucket into the usual well. Barriers to a broader inclusion range from a lack of awareness about clinical trials, to inconvenient trial locations or other logistical burdens, he says.

Another issue, and one that the FDA covers in its draft guidance, is the challenge of trust. Past abuses, such as the Tuskegee Syphilis Study, have also made some groups understandably leery of research participation. But promoting diversity is crucial to ensuring that medications are safe and effective, Gratton says, because people of different races and ethnicities react to drugs differently.

“It’s not a one-size-fits-all,” Gratton says.

Building a diversity plan

To make increased diversity a reality, sponsors will need concrete plans to bring new participants into the fold — no easy task.

“It’s already difficult to recruit for clinical trials. It’s already difficult to bring medications to market. We already know all the stats about how many trials failed to recruit, and how many trials are delayed by years just because you can’t find the right patient. So, there is certainly going to be some apprehension,” Gratton says.

Regardless of the challenges involved, this issue is not going away, Khera says. Companies can lay the groundwork for compliance by focusing on several critical areas:

Engage regulatory professionals: Because this is an FDA initiative, regulatory leaders need to drive the diversity plan. They should be involved and engage with the FDA early and often, Khera says. It’s also crucial that they collaborate closely with their operations teams, she adds.

Prioritizing partnerships: Companies need to build ties with patient advocacy groups, institutional review boards, contract research organizations and other partners to advance the diversity plan, Khera says. They should also get to know their patient population. “I’m a big advocate for listening. You’ve got to start with the people who are impacted,” Gratton says. “It’s not just the advocacy groups or the patient communities — it’s everybody and anybody who is affected by the disease.”

Take a flexible approach: If clinical trials aren’t meeting diversity targets, organizations need to be prepared to revise their approach. This requires an understanding of where the gaps are and how to close them, Khera says. Companies should think of their diversity plan as a living document.

Focus on the big picture: “Organizations should carefully look at the diversity plan at a holistic level,” Khera says. “Understand that although cost is involved, the plan is essential to prevent the organization from getting stuck on issues that could hinder approvals. Ultimately, we may learn if there is a variation of a drug in safety and effectiveness for certain groups sooner rather than later.”

Reduce friction: Removing barriers to trial participation is necessary to improve enrollment and retention. “I think decentralized trials are absolutely a critical step to ensuring that there is more access and participation by the underserved,” Gratton says.

The use of technology and new recruitment approaches can also help make the process easier. Gratton created CureClick, a proprietary crowdsourcing platform designed to help accelerate awareness and participation in clinical research, which was recently acquired by THREAD. This crowdsourcing approach can help streamline the patient education process, introducing people to research opportunities that they may not have otherwise been aware of, he says.

Ultimately, the FDA draft guidance and its final iteration will require a shift in mindset and an investment in change. But if companies are successful in increasing diversity, it will ultimately benefit everyone.

“Just because it’s difficult doesn’t mean it’s not something we should do. Because at the end of the day, prescribing the wrong medication to someone is not really acceptable,” Gratton says.

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