Enhancing the Patient Perspective and Experience in Drug Development and Review


FDA Commissioner, Scott Gottlieb, MD released a statement this morning, stressing the FDA’s intention to incorporate the patient experience into the regulatory authority’s benefit-risk assessments.

Two people with the same disease might have very different goals, according to U.S. Food and Drug Administration (FDA) Commissioner, Scott Gottlieb, MD.

In an effort to address these realities, Gottlieb released a statement this morning, stressing the FDA’s intention to incorporate the patient experience into the regulatory authority’s benefit-risk assessments. Firsthand knowledge of a serious illness from somebody living with it every day communicated in scientific terminology that patients can appreciate and understand, is a vital part of successful drug development. It will undoubtedly lead to the approval of safe and effective products that can deliver significant clinical benefits in diseases and disease states.

“Tools for capturing the patient experience may be quantitative or qualitative, but they are transforming nearly every aspect of medical product development,” Gottlieb said in his statement. “Patients are teaching us about the benefits that matter most to them and the risks that they are most concerned about. Patients are, rightly so, becoming the driving force of the medical research enterprise.”

This most recent statement is one of many similar messages sent by Gottlieb he was elected into office in early 2017. More than anyone who has come before him, he has ensured that the FDA has made significant strides in prioritizing patient engagement as part of the process for clinical development. In early March, he announced that the FDA would host the first joint Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting with a pair of rare disease-focused organizations.

“Having patient voice involved in the conversation is especially needed right now,” said Paul Melmeyer, Director of Federal Policy for the National Organization for Rare Disorders (NORD) at the New Jersey Rare Disease Day Observation.

“While we have made a substantial advancement in progress pertaining to the number of treatments that are being developed for those with rare diseases, we still have 95% of rare disease patients without any approved treatments. There’s still such a great amount of need within the rare disease patient community. Patients and their loved ones are uniquely capable of describing that situation.”

In the statement, Gottlieb announced that the FDA conducted patient-focused drug development meetings in over 20 disease areas.

With the meetings, the regulatory authority concluded that patient input can: inform the clinical context and provide insights to frame the assessment of benefits and risk; and provide a direct source of evidence regarding the benefits and risks if methodologically-sound data collection tools could be developed and used within clinical studies of an investigational therapy.

“The FDA is now developing guidance to enable more widespread development of such patient experience data to inform regulatory decision-making,” said Gottlieb in the statement.

Other efforts that could more thoroughly integrate patients’ experiences and perspectives include: hosting patient-focused drug development meetings to advance a more systematic way of gathering patients’ perspectives on their conditions and available treatments; encouraging patient stakeholders and others to conduct their own externally-led patient-focused drug development meetings; providing patients, caregivers, advocates and others with more channels to provide meaningful input into drug development and regulatory decision-making and to more easily access information provided by others; and launching pilot programs — and advancing policies, in collaboration with the medical community – that help foster the design of clinical trials that place less burden on patients.

“We know that, in the battle against disease, engaged and informed patients are our best allies and our greatest resource,” Gottlieb concluded.

For more from the FDA, follow Rare Disease Report on Facebook and Twitter.

Related Videos
Signs and Symptoms of Connective Tissue Disease
How Gene and Cell Therapy Is Developing in Dermatology
Joyce Teng, MD, PhD, discusses how therapeutic advances in fields like epidermolysis bullosa should progress treatment discourse in other rare dermatoses.
The Prospect of Pz-cel in RDEB Treatment, with Peter Marinkovich, MD
Comparing New Therapies for Dystrophic Epidermolysis Bullosa
Reviewing 2023 with FDA Commissioner Robert M. Califf, MD
Dunia Hatabah, MD | Image Credit: HCPLive
Ricky Safer: What Clinicians Need to Know About PSC
Ryan T. Fischer, MD: Long-Term Odevixibat Benefit for Alagille Syndrome
Saeed Mohammad, MD: IBAT Inhibitors for Cholestatic Disease
© 2024 MJH Life Sciences

All rights reserved.