Publication Examines Patient-focused Drug Development Efforts

Paper from Eleanor Perfetto, PhD, reviews current PFDD activities and offers suggestions for incorporating patient input into drug development.

By Laura Bogart
February 27, 2015

Eleanor Perfetto, PhD, MS, professor in the Department of Pharmaceutical Health Services Research (PHSR) at the University of Maryland School of Pharmacy, has published a landmark paper on the importance of patient-focused drug development (PFDD) in the journal Medical Care. PFDD is a new initiative from the Food and Drug Administration (FDA) intended to align with one of the core goals of the Affordable Care Act – a focus on patient-centered approaches to health care design and delivery.

The patient-centered approach to health care leverages patients as partners in research and health care decision making. Perfetto’s paper examines current PFDD activities and offers suggestions for incorporating effective patient input in drug development. “Before the PFDD initiative, the FDA typically engaged patients in the very late stages of drug development, and even then on a very limited basis,” explains Perfetto. “Now, there’s more of an understanding that, in order to get a full understanding of the disease or condition we’re trying to treat, we need to hear the voices of people who live with these diseases and conditions.”

To analyze the FDA’s current PFDD initiative and its implications for patients and other key stakeholders such as payers and clinicians, as well as provide insights for improvement, Perfetto and her team conducted an exhaustive search of regulation, FDA guidance materials and presentations, and peer-reviewed literature relevant to PFDD. From there, they reflected upon the PFDD initiatives’ early successes in engaging particular patient communities, and its potential for using those communities to inform the pharmaceutical industry about which aspects and elements of treatments for particular diseases are most important to patients.

In the paper, titled “Patient-Focused Drug Development: A New Director for Collaboration,” the authors advocate for formalized collaborations between patient groups and researchers in order to develop a more codified conceptual framework for PFDD. Patients who are involved from at the start of a clinical trial to evaluate a new treatment can make suggestions that have a huge impact on the success of that research. For instance, patients may recommend making the language used in research questions more streamlined and “user-friendly,” which will facilitate more honest, accurate answers from responders.

Co-authors on the paper are Laurie Burke, RPh, MPH, former director of Study Endpoints and Labeling Development for the FDA; Elisabeth Oehrlein, a graduate student in PHSR, and Robert S. Epstein, MD, MS, former president of Medco Health Solutions, Inc.

Though Perfetto and her co-authors are hopeful about all that the PFDD initiative can achieve, they write that, “There is a need for a call to action to define what patient engagement means and to articulate the range of appropriate methodologies for engagement in PFDD.” They emphasize the importance of developing systematic methods of incorporating patients into the process and of ensuring that patients have plenty of opportunities to share substantive feedback.

To further define the concept of PDFF, Perfetto has organized a conference on the topic through the Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI) that will be held on March 9 at the Hyatt Regency Hotel in Bethesda, Md. The conference will provide a forum for all PFDD stakeholders to gather for an open dialogue. Patients, caregivers, and patient advocacy groups, as well as regulators from the FDA; researchers from academia, industry, and other agencies; payers; and other stakeholders will come together to discuss the following topics:

To learn more or to register for the conference, visit

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