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Review
. 2020 Feb;20(1):57-68.
doi: 10.1080/14737167.2020.1734454. Epub 2020 Mar 6.

The evolution of patient-focused drug development and Duchenne muscular dystrophy

Norah L Crossnohere  1   2 , Ryan Fischer  3 , Emily Crossley  4 , Elizabeth Vroom  5 , John Fp Bridges  1   2
Affiliations
Review

The evolution of patient-focused drug development and Duchenne muscular dystrophy

Norah L Crossnohere et al. Expert Rev Pharmacoecon Outcomes Res. 2020 Feb.

Abstract

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.

Keywords: Public policy; decision making; patient advocacy; patient preference; patient-centered; review.

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