In recent years the U.S. Food and Drug Administration (FDA) has endeavored to place patients at the heart of its decision making. Here we explore the primary engagement opportunities with the Agency for advocacy organizations.
In the past, the development and approval of new drugs and technologies has relied on expert opinion rather than asking patients and families themselves what they consider most important. But this is changing as we enter an era of patient-centered medicine. Within the FDA, patients and caregivers are becoming more actively involved in decisions about their healthcare options and treatment regimens.
One of the centerpieces of FDA’s move towards strengthened patient engagement has been the three-year Patient-Focused Drug Development initiative*. This was established as part of the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) and put into effect through a series of 20 meetings covering a host of different disease areas from lung cancer and hemophilia, to alpha-1 antitrypsin deficiency.
Part of an initiative to gather patient perspectives, the aim of these meetings was to provide the Agency with the opportunity to hear directly from patients and caregivers about their experiences, the impact of living with their condition and treatment approaches. Following each meeting, FDA published a ‘Voice of the Patient’ summarizing the patient testimony as part of an effort to communicate what improvements patients would most like to see in their daily lives to industry and regulators.
As Dr. Francis Kalush, Center for Drug Evaluation and Research (CDER) within FDA, told Change Together: “Over the past three years we have significantly increased our focus on engaging patients to recognize the critical role they play in informing our understanding of living with a particular condition, the therapies they receive and how we might find ways to develop new treatments for these diseases.”
This concept also feeds into FDA’s Patient Representative Program. Here, patients and primary caregivers with direct experience in a particular disease participate in scientific advisory committees to bring their perspective to discussions about new and already approved medicines and devices, as well as provide early input into the product development and review process. Similarly, the first-ever Patient Engagement Advisory Committee was established in September 2015 to advise the FDA Commissioner on a range of topics including clinical trial design, unmet clinical needs, patient-reported outcomes and integrating the patient perspective into the regulatory process.
Other related activities at the Agency include a biweekly newsletter for patients, caregivers, patient advocates and other healthcare professionals providing information on new product approvals, safety warnings and regulatory guidances, as well as meetings and workshops.
In August, for example, the FDA sponsored a workshop for the public, patients, patient advocacy groups and industry to discuss whether the current regulatory paradigm for antidiabetic drug therapies addresses the needs of patients with diabetes and to identify additional outcomes, beyond hemoglobin A1c (HbA1c), that are of direct relevance and importance to those living with the disease. The Agency also continues to work with the Clinical Trials Transformation Initiative (CTTI), a public-private partnership focused on developing and driving adoption of practices that will increase the quality and efficiency of clinical trials, to create a forum for exchanging information, ideas and experiences on issues of interest to patients and patient advocates – and how this feeds into the agency’s patient engagement experience.
Earlier this year, CDER sponsored a public workshop aimed at developing a roadmap for effectively engaging with the agency on behalf of patients. The roadmap is expected to include tips on how to set up and make the most of meetings at FDA; how to prepare an effective presentation for FDA staff; and how patients can interact and provide input to the Agency.
Workshop attendees also heard from the ‘pros’ on how best to engage with the Agency, with the Alliance for Aging Research advising other advocacy organizations to educate themselves on the clinical trial process, learn what information is useful for regulatory purposes, reconcile their goals with those of the research community and regulated industry – and above all realize that successful engagement does not always equal drug approval!
“For several years, the FDA has been working to focus on the needs and goals of the patient as the Agency makes decisions about drug therapies for the advancement and protection of public health,” notes John J. Whyte, Director of Professional Affairs and Stakeholder Engagement at CDER in a blog for FDA Voice. “These efforts can only be as effective as our ability to connect with the patients and their representatives we seek to engage.”
Now, all eyes are on how the FDA builds on these efforts through the patient-focused drug development provisions in PDUFA VI. As outlined in the goals letter agreed upon by FDA and the biopharmaceutical Industry, with input from many stakeholders, part of these commitments will see a deeper dive into the science involved in incorporating the patient voice into the drug development process and regulatory decision making. This includes developing a clearer path for early agreement on endpoints and outcomes measurement, and employing patient-focused experts within each review division to ensure progress and consistency of approach.
For more information on the FDA’s patient engagement programs, please click here*.
To find out more about Professional Affairs and Stakeholder Engagement at the FDA, please click here*.
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