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

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-centered
• patient advocacy
• patient preference
• review

Article Highlights

• Regulatory patient-focused drug development initiatives seek to identify and develop appropriate and rigorous methods of measuring patient voice and integrating it as a form of evidence into approval decisions.

• Highly professional patient groups, such as Parent Project Muscular Dystrophy, are pioneers in advocating for the use of patient preference and experience information to inform regulatory decision-making.

• Integrating patient experience information into post-market surveillance, pricing, and reimbursement are emerging priorities for rare disease groups and regulatory agencies internationally.

Declaration of Interest

NL Crossnohere and JFP Bridges have received funding from Parent Project Muscular Dystrophy. R Fischer is an employee of Parent Project Muscular Dystrophy. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer Disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Data Availability

All data are presented in the manuscript.

Additional information

Funding

This work was supported by the Parent Project Muscular Dystrophy. Parent Project Muscular dystrophy has received support from Pfizer for benefit-risk research.