FDA’s Latest Efforts in Patient-focused Drug Development … Now in Full Swing!

By: Richard M. Klein

Patients are very much at the center of what FDA does, and the agency has long sought input from patients and caregivers regarding their treatment options and needs. For many years, FDA has included the patient perspective at advisory committee meetings and during the review of new medical products in selected meetings with FDA and product developers.

More recently, FDA has encouraged and fostered the use of patient-reported outcome measures in clinical trials, such as impact on quality of life or pain control, to support labeling claims in medical product development. We are excited about our current efforts to expand the role of the patient perspective: the agency’s Patient-Focused Drug Development initiative. Part of FDA’s commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), this initiative will gather patients’ perspectives on the impact of a condition on daily life and the available therapies to treat that condition.  

Understanding a condition’s severity and its treatment options is a part of the benefit-risk assessments that help FDA make decisions about the medical products it regulates. As part of this commitment, FDA will hold at least 20 public meetings over the next 5 years, each focused on a different disease area. The information discussed in these meetings will be summarized for consideration by FDA review divisions when making regulatory decisions in that therapeutic area. 

Our first meeting, with chronic fatigue syndrome (CFS) and myalgic encephalomyelitis (ME) patients, was held on April 25. CFS/ME are debilitating diseases for which there are currently no FDA-approved treatments. At the meeting, we heard directly from patients, their caretakers, and patient advocates about the symptoms that matter most to them, the impact their disease has on their daily lives, and their experiences with treatments they have tried.  A complete video of the meeting and a transcript of the proceedings are posted on our website. We are also preparing a summary report about this meeting to be posted on the Web. We established a public docket that will remain open through August 2, 2013, so the public can provide additional written comments. 

Our second meeting, being held today, is focusing on HIV-AIDS. Although more than 30 drugs have been approved to treat patients with this disease, much remains to be learned from patients about how well the drugs they are currently taking for HIV-AIDS are working, what symptoms the drugs are not fully addressing, and how the therapies are affecting them. The meeting will also provide an opportunity for patients to express their thoughts about the evolving science of “HIV cure research” and their decisions about participating in the research. We are very interested in hearing from patients how FDA can communicate better about how that research might affect them. 

Our next meeting, scheduled for June 28, will focus on lung cancer. Exact dates for other future meetings have not yet been set, but we’ve already selected 13 additional disease areas (for a total of 16 for fiscal years 2013-2015) and listed them on our website. The remaining meeting topics will be selected by the end of fiscal year 2015. Each of the meetings will be webcast for those who cannot attend. The webcast recording and transcript of each meeting will be available on FDA’s website. We will open a public docket for each meeting so people can submit written or electronic comments.  

We want patients to be involved. It is important to FDA to hear from patients, caregivers, and patient advocates. We welcome everyone’s comments and will try to keep people as informed as possible throughout the process through our website. 

The patient perspective is an important part of the drug development process. Through social media, the Internet, and advocacy groups, patients have become more informed about how drugs and devices are developed. FDA is committed to using this information to help foster the development and availability of safe and effective drugs that meet the needs of the American public. 

Richard M. Klein is Director of the Patient Liaison Program in FDA’s Office of Health and Constituent Affairs.