Niche Markets and Evidence Assessment in Transition: A Critical Review of Proposed Drug Reforms

• Published in: Medical law review Vol. 22; no. 2; pp. 200 - 220
• Main Authors: Gibson, Shannon G., Lemmens, Trudo
• Format: Journal Article
• Language: English
• Published: England Oxford University Press 01.06.2014
• Subjects: Advertising as Topic; Cost effectiveness; Drug Approval - legislation & jurisprudence; Drug development; Drug Discovery; Drug Industry; Drugs; Europe; Government Regulation; Great Britain. National Health Service; Health Care Reform - economics; Health Care Reform - legislation & jurisprudence; Humans; Law and legislation; Legislation, Drug; Orphan Drug Production; Pharmaceutical industry; Pharmacogenetics; Prescription Drugs - economics; Reimbursement Mechanisms - legislation & jurisprudence; UNITED STATES; GREAT BRITAIN; Europe
• ISSN: 0967-0742; 1464-3790
• DOI: 10.1093/medlaw/fwu005

In response to rising demands and treatment costs, and the need to achieve better value for money in the face of tight fiscal constraints, both the National Health Service and the public drug reimbursement system are undergoing important reforms. Concurrently, the pharmaceutical sector itself is also alleged to be experiencing significant changes, perhaps most notably, a decline of the blockbuster model of drug development and a growing focus on niche market products. As pharmaceutical development strategies evolve and the resulting drug products become more complex, regulatory and policy responses must be able to evolve along with them. We explore how in numerous jurisdictions, including the UK, proposals for ‘adaptive licensing’ on the regulatory side and ‘performance-based risk sharing agreements’ on the funding side are shifting the focus of drug regulation and reimbursement towards more incremental access to new therapies and more post-market evidence generation. However, serious questions remain about how such reforms can be successfully implemented and whether they can balance demands for earlier access to promising new therapies with the need for robust evidence on safety, efficacy, and cost-effectiveness.