FDA Mulling Guidance on Who Can Enroll in Cancer Studies

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By Jeannie Baumann

Cancer clinical trial sponsors need to enroll a broader scope of subjects, including HIV patients and children, and the FDA is working on guidance to make that happen, the agency’s oncology chief told Bloomberg BNA.

“We would like clinical trials to more accurately reflect the patient population that will actually be using the drug, rather than a very artificial construct of patients that are hand picked to go on the clinical trial,” Richard Pazdur, director of the Food and Drug Administration’s Oncology Center of Excellence, said in an April 20 interview with Bloomberg BNA.

Clinical trials often have strict requirements on how to define the study population to protect patients from undue harm and collect useful safety and efficacy data. But Pazdur said clinical trial designers frequently just copy the eligibility criteria from one protocol to another “without really thinking through” if they apply to the study at hand.

The FDA wants sponsors to design study eligibility criteria to include the types of patients who will be using the drug once it’s approved, Pazdur said. That population also should be reflected in the product label, he added.

NEJM Perspective

Pazdur co-authored a perspective in the April 20 issue of the New England Journal of Medicine on eligibility criteria. That perspective, Reevaluating Eligibility Criteria — Balancing Patient Protection and Participation in Oncology Trials , is a first step in a much larger effort to engage in dialogue.

“In oncology trials, specific populations are often categorically excluded without a clear reason,” the paper said. Patients with HIV, for example, are commonly excluded from trials because of concerns about immunodeficiency and drug interactions. Pazdur said, “A more rational approach would allow for inclusion of patients with HIV whose CD4+ [T-cell, or white blood cell] counts exceed a predetermined level or who have no opportunistic infections,” an indication their immune systems aren’t highly compromised.

Jeff Allen, president and chief executive officer of Friends of Cancer Research, agreed. “With the progress that has been made in that disease area, there are people that may be HIV positive but are leading fairly normal lives, and it’s being managed as a chronic condition,” Allen told Bloomberg BNA April 20. “Often times, they’re developing cancer because they’re living longer. So this is an important subset of the population that could be included in clinical research with greater frequency.”

He noted that Friends, the FDA and the American Society of Clinical Oncology held a workshop last year on HIV status and eligibility criteria for clinical trials.

Friends supports the FDA’s effort to revisit evaluation criteria, Allen said. “Their analysis shows that there are certainly opportunities to do that,” he said. “And I think it’s really great that they introduce in their article too some initial thoughts on different methodologies.”

Those methodologies could be used to broaden eligibility criteria and ensure data can still accurately show the effect of the drug, Allen said.

Papers, Draft Guidance

Julia Ann Beaver, a co-author of the NEJM perspective and an associate director in the FDA’s oncology division, said the agency is involved with many stakeholders like ASCO and Friends that are also looking at the eligibility issue and whose working groups will be publishing papers on the subject soon.

Pazdur said the ASCO working groups will publish papers on the practice of excluding patients with HIV, brain metastases due to concerns about shortened life expectancy or increased seizure risk, and minors.

He said the practice of excluding minors delays pediatric studies. And, the perspective noted, “Separate adult and pediatric cancer centers, distinct cooperative research groups, and oncologists specializing in different populations do not often conduct unified adult–pediatric clinical trials or drug-development programs.”

After the working groups publish their papers, Pazdur said, “hopefully we will evaluate that information and start writing a guidance on it.” The FDA is in the very preliminary stages of guidance development, but there’s no timeline for completing it, he added.

To contact the reporter on this story: Jeannie Baumann in Washington at jbaumann@bna.com

To contact the editor responsible for this story: Randy Kubetin at RKubetin@bna.com

For More Information

The New England Journal of Medicine perspective is at http://www.nejm.org/doi/full/10.1056/NEJMp1615879.

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