title: Patient-Focused Drug Development: A New Direction for Collaboration.
abstract: BACKGROUND
  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.
  
  
  METHODS
  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."
  
  
  RESULTS
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: *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.
authors.forename: Eleanor M
authors.initials: EM
authors.lastname: Perfetto
authors.forename: Laurie
authors.initials: L
authors.lastname: Burke
authors.forename: Elisabeth M
authors.initials: EM
authors.lastname: Oehrlein
authors.forename: Robert S
authors.initials: RS
authors.lastname: Epstein
mesh_terms: D003299:Cooperative Behavior; D055808:Drug Discovery / Q000458:organization & administration*; D004345:Drug Industry / Q000458:organization & administration; D006801:Humans; D007343:Insurance Carriers; D010358:Patient Participation / Q000379:methods*; D018802:Patient-Centered Care / Q000458:organization & administration*; D017712:Peer Review, Research; D018570:Risk Assessment; D014481:United States; D014486:United States Food and Drug Administration / Q000458:organization & administration*