Patient-Focused Drug Development (PFDD) is a new initiative from the Food and Drug Administration (FDA) intended to bring patient perspectives into an earlier stage of product development. The goal is that patients will be able to provide context for benefit-risk assessments and input to review divisions, and also aid in the development of new assessment tools, study endpoints, and risk communications. This paper provides a summary on what is known to date about FDA’s PFDD initiative and describes implications for patients, researchers, payers, and the biopharmaceutical industry. It also provides a roadmap for stakeholders to consider in defining their role in and in shaping PFDD’s direction, and for expanding PFDD principles to conditions beyond the current 20 under FDA consideration.
A search was conducted of the peer-reviewed and gray literature using PubMed and Google. This included laws, FDA guidance documents, the peer-reviewed literature, and FDA presentations for content relevant to the search term “patient-focused drug development.”
Currently, FDA activities within PFDD are limited to gaining patient insights through 20 disease-specific meetings. However, many stakeholders see the initiative much more generally as representing a broad shift toward patient centeredness in biopharmaceutical product development.
Depending upon the trajectory taken and whether or not all PFDD aims are eventually addressed, the initiative has the potential to change product development in fundamental ways. Further research should explore how patient input on disease manifestation and treatment options is best ascertained from patients and documented before initiating and during drug development.
*Department of Pharmaceutical Health Services Research, University of Maryland School of Pharmacy, Baltimore, MD
†LORA Group LLC, Royal Oak, MD
‡Epstein Health LLC, Woodcliff Lake, NJ
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E.M.P. previously worked for Pfizer and consults with various pharmaceutical companies. L.B. previously worked at the FDA and consults with a wide variety of biopharmaceutical companies. E.M.O. completed paid internships at GlaxoSmithKline and Bristol-Myers Squibb. R.E. previously worked at Medco Health Solutions, consults for a variety of companies, and sits on the Board of Directors for Illumina, Aveo, and Proteus Digital.
Reprints: Elisabeth M. Oehrlein, BA, Department of Pharmaceutical Health Services Research, University of Maryland School of Pharmacy, 220 Arch Street, 12th Floor, Baltimore, MD 21201. E-mail: firstname.lastname@example.org.