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

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. PMID: 32098551 [PubMed - as supplied by publisher]
Source: Expert Review of Pharmacoeconomics and Outcomes Research - Category: Health Management Tags: Expert Rev Pharmacoecon Outcomes Res Source Type: research