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FDA selects eight new patient groups for input

Monday, July 20, 2015

The FDA released a list of eight new conditions that it plans to assess during the next two years as part of its Patient-Focused Drug Development (PFDD) program. The program seeks to inform regulatory decision-making by soliciting patients’ perspectives on their diseases, treatment options and preferred endpoints for experimental therapies.

Through the PFDD initiative, part of FDA’s commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), the agency has undertaken difficult but important work that supports the wider patient-centricity movement. The FDA has committed to holding at least 20 public meetings on specific disease areas through September 2017.

The eight new disease areas, which will each be the subject of a separate public meeting, include autism, alopecia areata, hereditary angioedema, non-tuberculous mycobacterial infections, patients who have received an organ transplant, psoriasis, neuropathic pain associated with peripheral neuropathy and sarcopenia.

“There has been an increasing recognition of the critical role that patients can play in informing the understanding of the therapeutic context of benefit-risk assessment and informing the clinical development programs for new drugs,” said Theresa Mullin, Ph.D., director of the FDA’s Office of Strategic Programs, who leads the Center for Drug Evaluation and Research (CDER) PFDD initiative. “The FDA has significantly increased its focus on engaging patients to gain their perspective and insights as have other parties including patient advocacy organizations, industry sponsors and re­searchers.”

Mullin said the new disease areas were chosen after consideration of almost 2,700 public comments received in response to a Federal Register notice of disease areas for possible inclusion in the PFDD program. In­dividual patients submitted the majority of the comments, but patient advocacy groups, caregivers, healthcare providers, profes­sional societies, scientific and academic experts and pharmaceutical companies also contributed recommendations.

To meet the PFDD selection criteria, the disease areas need to be chronic or affect the patients’ daily activities and have few or no treatment options available. The FDA also chose disease areas that have a severe impact on subpopulations, such as children or the elderly, and those that have aspects not formally captured in clinical trials.

A schedule of the meetings for the ad­ditional disease areas and Voice of the Patient reports that summarize the participants’ experiences and perspectives will be posted on the FDA’s website.

The newly selected group of diseases and conditions will bring the FDA through the end of the current authorization period for PDUFA, which is set to expire in 2017. Mullin said the FDA will continue discussions with patients and other stakeholders in prepara­tion for PDUFA VI. In the meantime, recog­nizing that there are many more disease areas that could be addressed during the next two years, the FDA recently posted information encouraging public stakehold­ers to plan their own externally led patient-focused meeting.

Kay Holcombe, senior vice president of sci­ence policy at the Biotechnology Industry Organization (BIO), said that patients have different perceptions of the benefits and risks of medicines based on their personal experi­ence with their diseases and it is critical that the patients’ voices be heard.

Holcombe added that it’s unclear how feedback from the PFDD meetings will be in­corporated into revising policy or review of individual product development programs. She said that much of the information from PFDD meetings is anecdotal and not scal­able to the thousands of diseases afflicting patients and the ever-changing healthcare environment.

“We strongly support establishing a framework for incorporating patient views into the development processes in a more structured, scientifically valid and transpar­ent way, with respect to both patient input for benefit-risk assessments and use of pa­tient experience data in regulatory decision-making,” she said. “Just as we take a scientific approach to drug development, we must take a scientific approach when gathering and evaluating patient input.”

Mullin added that PDUFA VI might include consideration of ways to advance the science of patient engagement. The development of new tools and approaches to incorporate the patients’ perspective will be an important undertaking, she said, that will require good scientific methods, patient engagement and, in many cases, may require tailoring tools and endpoints to the particular disease and what matters most to people living with the disease.


Email comments to Karyn Korieth at karyn.korieth@centerwatch.com.

This article was reprinted from Volume 19, Issue 28, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »

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