jueves, 6 de octubre de 2016

Our 20th Patient-Focused Drug Development meeting: Enhancing the patient’s voice in FDA’s approach to drug review and development | FDA Voice

Our 20th Patient-Focused Drug Development meeting: Enhancing the patient’s voice in FDA’s approach to drug review and development | FDA Voice







Our 20th Patient-Focused Drug Development meeting: Enhancing the patient’s voice in FDA’s approach to drug review and development

By: Theresa M. Mullin, Ph.D.
Since the launch of the Patient Focused Drug Development program as part of the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), we have worked intensively to explore ways to enhance the patient’s voice in drug development. Recently we reached a particularly gratifying milestone in this important work — our 20th Patient-Focused Drug Development (PFDD) public meeting.
Theresa MullinThe PDUFA program provides much needed funding from the pharmaceutical industry to support FDA’s premarket review activities and the agency’s work to encourage drug development. Under PDUFA V, FDA committed to obtain patients’ views in at least 20 disease areas over the course of the program’s five year period, which ends in September, 2017. That means conducting a public meeting for each disease area to obtain patient perspectives on the impact of the condition on daily life and current treatment approaches. Our 20th PFDD meeting, with patients who have received organ transplants, took place on September 27thWith that meeting completed, we fulfilled our commitment — one year ahead of schedule.
The PFDD meetings have given us the opportunity to strengthen our understanding of the targeted disease areas and hear directly from patients, their families, and caregivers about the symptoms that matter most to them; the impact of the disease on daily life, and their experiences with currently available treatments. Having this kind of input is extremely valuable for us because hearing what patients care about can help us determine how best to facilitate drug development for a particular disease area. Hearing the patients’ perspectives also helps us understand how patients view the benefits, risks, and burdens of treatments for their condition.
While FDA plays a critical role in drug development, we are only one of the players in the process; other stakeholders, including healthcare providers and industry sponsors, who have attended the PFDD meetings to hear from patients, are also gaining valuable information. The  PFDD meetings have also helped  identify areas of unmet need within the patient population (e.g., the psoriasis meeting highlighted the need for treatments for the pediatric population living with psoriasis) and helped raise awareness and focus engagement within the patient community itself (e.g., in preparation for the narcolepsy meeting patient groups collaborated to form a coalition called Unite Narcolepsy). We’ve chronicled this and more in our Voice of the Patient reports, which provide a detailed account of the valuable input we’ve heard at each meeting.
The Voice of the Patient reports are intended to be useful to both our FDA colleagues conducting reviews and the broader community. These reports summarize what FDA heard through patient speaker panels, audience participation, the webcast, and submissions to the public docket. Each report faithfully captures this information as a valuable resource for the FDA review divisions and is distributed internally to the relevant review divisions for reference when advising sponsors on their drug development programs and when assessing products under review in that disease area.
As drug development advances in the 21st Century, sponsors are using increasingly sophisticated and vital forms of technology to generate the medicines of the future. But there is a critical part of drug development — gaining ever increasing importance in the process — that has little to do with advanced technology. Instead, it has to do with listening to patients and their personal experiences living with their disease and its treatment, and determining the best ways to reliably capture this perspective so that it can be better integrated into decision making.
We believe that the long-term impact of PFDD will be better, more informed FDA decisions and oversight both during drug development and during our review of a marketing application. We are extremely grateful to all of the hundreds of patients and their loved ones who have so generously and, in some cases, courageously, participated in our meetings and have shared their personal stories, experiences, and perspectives.
We may have met the letter of our PDUFA commitment, but we are not finished. Patient-Focused Drug Development is a priority for FDA. Beyond the 20 meetings we have already held, we plan to hold four more PFDD meetings by the end of FY2017. Additionally, we recognize that there are many more disease areas to address. To help expand the benefits of FDA’s PFDD initiative, FDA welcomes similar patient-focused meetings organized by the patient groups themselves. For this parallel effort to FDA’s PFDD initiative, interested patient groups can submit a letter of intent. More information is outlined on FDA’s website.
One of the most valuable things we can do as regulators at FDA is simply to listen. I’m reminded of that each time we hold a PFDD public meeting. FDA will continue to listen — and learn — and we look forward to continuing to gain the additional insights that only patients, their families, and caregivers can provide.
Theresa M. Mullin, Ph.D., is Director of FDA’s Office of Strategic Programs in the Center for Drug Evaluation and Research