This fall, the U.S. Food and Drug Administration (FDA) will host its first ever Patient-Focused Drug Development meeting in the field of organ transplantation, as part of a new initiative to gather patient perspectives on their individual condition and the impact on their daily life, as well as current and new treatment approaches.
Patients and patient representatives will be asked a variety of questions, including which symptoms have the greatest impact day to day, general health concerns post-transplant, and what they’d look for in an ideal treatment to manage their transplanted organ. They will also be asked for thoughts about their current treatment regimen – which may include immunosuppressants, antibiotics, antivirals and over-the-counter medicines – and in particular how well the most significant symptoms they experience post-transplant are managed.
Paul Conway, president of the American Association of Kidney Patients commented, “The efforts of the FDA to substantively reach out to patients, seek out their opinions and listen to their perspectives and experiences is both sincere and highly valuable. As kidney patients and patient advocates, and especially transplant patients, we each have an obligation to fellow patients and future patients to be engaged in the process and share our insights with policy-makers responsible for monitoring and improving care quality. We encourage patients to visit the FDA website to learn more on how to participate, and we applaud the FDA for allowing transplant patients the opportunity to help shape the standards under which we live and not simply learn about them after the fact.”
Organ transplantation is the focus of one of a series of 20 Patient-Focused Drug Development meetings taking place over a five-year period, each centered on a specific disease area. This initiative was developed from performance commitments made under the 2012 revision of the Prescription Drug User Fee Act (PDUFA) with the aim of creating a clearer picture of disease severity and greater understanding of the variety of treatment approaches within a specific therapy area. The FDA says this is a critical component of its regulatory decision-making and communication.
As a spokeswoman for the FDA explained, “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. 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.”