PMID- 25494232
OWN - NLM
STAT- MEDLINE
DCOM- 20151117
LR  - 20220317
IS  - 1537-1948 (Electronic)
IS  - 0025-7079 (Linking)
VI  - 53
IP  - 1
DP  - 2015 Jan
TI  - Patient-Focused Drug Development: A New Direction for Collaboration.
PG  - 9-17
LID - 10.1097/MLR.0000000000000273 [doi]
AB  - CONTEXT: 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." FINDINGS: 
      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.
FAU - Perfetto, Eleanor M
AU  - Perfetto EM
AD  - *Department of Pharmaceutical Health Services Research, University of Maryland 
      School of Pharmacy, Baltimore, MD daggerLORA Group LLC, Royal Oak, MD double daggerEpstein Health 
      LLC, Woodcliff Lake, NJ.
FAU - Burke, Laurie
AU  - Burke L
FAU - Oehrlein, Elisabeth M
AU  - Oehrlein EM
FAU - Epstein, Robert S
AU  - Epstein RS
LA  - eng
PT  - Journal Article
PL  - United States
TA  - Med Care
JT  - Medical care
JID - 0230027
SB  - IM
MH  - *Cooperative Behavior
MH  - Drug Discovery/*organization & administration
MH  - Drug Industry/organization & administration
MH  - Humans
MH  - Insurance Carriers
MH  - Patient Participation/*methods
MH  - Patient-Centered Care/*organization & administration
MH  - Peer Review, Research
MH  - Risk Assessment
MH  - United States
MH  - United States Food and Drug Administration/*organization & administration
EDAT- 2014/12/11 06:00
MHDA- 2015/11/18 06:00
CRDT- 2014/12/11 06:00
PHST- 2014/12/11 06:00 [entrez]
PHST- 2014/12/11 06:00 [pubmed]
PHST- 2015/11/18 06:00 [medline]
AID - 00005650-201501000-00003 [pii]
AID - 10.1097/MLR.0000000000000273 [doi]
PST - ppublish
SO  - Med Care. 2015 Jan;53(1):9-17. doi: 10.1097/MLR.0000000000000273.