PMID- 32098551
OWN - NLM
STAT- MEDLINE
DCOM- 20210107
LR  - 20210107
IS  - 1744-8379 (Electronic)
IS  - 1473-7167 (Linking)
VI  - 20
IP  - 1
DP  - 2020 Feb
TI  - The evolution of patient-focused drug development and Duchenne muscular 
      dystrophy.
PG  - 57-68
LID - 10.1080/14737167.2020.1734454 [doi]
AB  - Introduction: There is a groundswell of interest from patient, industry, and 
      regulatory groups to rigorously and transparently integrate patient-voice into 
      regulatory decision-making. Patient-focused drug development (PFDD) is an 
      approach established by the US Food and Drug Administration to systematically 
      incorporate patient experiences into drug development and evaluation. It has 
      created a demand for scientific advancement to measure and integrate 
      patient-voice into decision-making.Areas covered: This narrative review describes 
      the evolving nature of advocacy-regulatory relations preceding PFDD, 
      characterizes current PFDD and other patient-engagement activities, and explores 
      future opportunities for patient participation along the drug development 
      pipeline. We present Duchenne muscular dystrophy as a case study to illustrate 
      how PFDD is being operationalized by patient groups and regulators using both 
      verbal and written data sources.Expert opinion: PFDD represents the most 
      widespread approach yet to integrate the patient voice as a source of evidence to 
      inform regulatory decision-making. Regulatory approvals are just one frontier in 
      drug development. On the horizon remain uncertainties in how patient experience 
      can inform post-marketing surveillance, pricing, reimbursement, and health 
      technology assessment. Patient-input may be particularly crucial to demonstrate 
      the value of expensive first-generation rare disease treatments that confer 
      meaningful benefits but do not meet traditional thresholds for 
      cost-effectiveness.
FAU - Crossnohere, Norah L
AU  - Crossnohere NL
AUID- ORCID: 0000-0002-2811-1330
AD  - Department of Health, Behavior and Society, Johns Hopkins School of Public 
      Health, Baltimore, MD, USA.
AD  - Department of Biomedical Informatics, The Ohio State University Wexner Medical 
      Center, Columbus, OH, USA.
FAU - Fischer, Ryan
AU  - Fischer R
AD  - Parent Project Muscular Dystrophy, Hackensack, NJ, USA.
FAU - Crossley, Emily
AU  - Crossley E
AD  - Duchenne UK, London, UK.
FAU - Vroom, Elizabeth
AU  - Vroom E
AD  - Duchenne Parent Project Muscular Dystrophys, Amsterdam, NL, Netherlands.
FAU - Bridges, John Fp
AU  - Bridges JF
AD  - Department of Health, Behavior and Society, Johns Hopkins School of Public 
      Health, Baltimore, MD, USA.
AD  - Department of Biomedical Informatics, The Ohio State University Wexner Medical 
      Center, Columbus, OH, USA.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20200306
PL  - England
TA  - Expert Rev Pharmacoecon Outcomes Res
JT  - Expert review of pharmacoeconomics & outcomes research
JID - 101132257
SB  - IM
MH  - Decision Making
MH  - Drug Approval
MH  - Drug Development/*methods
MH  - Humans
MH  - Muscular Dystrophy, Duchenne/*drug therapy
MH  - Patient Participation/*methods
MH  - Technology Assessment, Biomedical/methods
MH  - United States
MH  - United States Food and Drug Administration
OTO - NOTNLM
OT  - Public policy
OT  - decision making
OT  - patient advocacy
OT  - patient preference
OT  - patient-centered
OT  - review
EDAT- 2020/02/27 06:00
MHDA- 2021/01/08 06:00
CRDT- 2020/02/27 06:00
PHST- 2020/02/27 06:00 [pubmed]
PHST- 2021/01/08 06:00 [medline]
PHST- 2020/02/27 06:00 [entrez]
AID - 10.1080/14737167.2020.1734454 [doi]
PST - ppublish
SO  - Expert Rev Pharmacoecon Outcomes Res. 2020 Feb;20(1):57-68. doi: 
      10.1080/14737167.2020.1734454. Epub 2020 Mar 6.