Janet Woodcock, M.D.—Director, Center for Drug Evaluation and Research (CDER)

People with chronic diseases are really experts in that disease. They tell the FDA which factors matter when they choose a course of treatment, and that might be different than what doctors think.

Under the PDUFA-5 Patient-Focused Drug Development Initiative, FDA committed to conducting a series of meetings focused on specific diseases. In the meetings, we hear the patient perspective of the impact of their condition on their daily life, on the treatments, and on how to choose a treatment. We also hear from patients in webcasts by phone or through the public docket, and these meetings are a critical aspect of FDA’s decision making and give us the context in which a regulatory decision is made.

CDER has held so many patient-focused drug development meetings. After each meeting, we develop and publish a document called “Voice of the Patient” that’s a summary report capturing the patient input. This report allows federal agencies, academic researchers, drug developers, patient stakeholders, consortia, and patients to use the information.

Patients’ perspectives are just critically important to the FDA, and they are so meaningful in determining how drugs should be developed. For example, during the autism meeting, patients identified communication difficulties as one of the top significant health effects for individuals with autism. They told us that some of the symptoms currently targeted by drug developers as endpoints, such as stimming, are not as important to the community.

Patients also want their experience described with the words that they use to best describe how it feels. For example, at the chronic fatigue syndrome and myalgic encephalomyelitis meeting, participants said that the clinically used term “malaise” was inaccurate, and they believe it should be termed as “crash” or “collapse,” what they use.

Drug companies, sometimes working with researchers or patient groups, conduct trials and then submit applications for new drugs to the FDA, and they can incorporate patient perspectives into their drug development program. This can help, for example, in designing the proper clinical trials both that measure the right endpoints but also are convenient and allow patients to participate.

They can also help identify areas of unmet need—for example, a symptom that hasn’t been relieved by current therapy—and they can also help raise awareness within the patient community of trials and thus help with recruitment.

Incorporating the voice of the patient is a very high priority for CDER.

We need to collect patient input so it becomes data that can further inform regulatory decision making, and we need methods to do that, and we’re going to have to provide then guidance to patient communities, researchers, and drug developers on pragmatic and methodologically sound strategies.

So, I cannot stress enough how important having the patient and the patient’s voice at the table is during drug development and in evaluating the safety and effectiveness of new medicines.