Access to orphan drugs in western Europe: can more systematic policymaking really help to avoid different decisions about the same drug?

Abstract

When the Scottish government recently launched a £21 million fund to provide patients with rare diseases access to treatment, Alex Neill, the Scottish Health Secretary, stated that ‘it was only right that Scottish patients with rare conditions had access to innovative medicines which were clinically justified, and that they were not disadvantaged due to the very high cost of these treatments’. This statement raises questions about the criteria that play a role in reimbursement decisions on orphan drugs. This editorial examines the criteria that were used in the decisions about the reimbursement of an orphan drug for Pompe disease and explores methods to improve the transparency and consistency of reimbursement decisions for orphan drugs in general.

Keywords:

• access
• multi-criteria decision analysis
• orphan drugs
• Pompe disease
• reimbursement

Financial & competing interests disclosure.

This study was financially supported by the Netherlands Organization for Health Research and Development (ZonMw; grant no. 152002045). 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.

No writing assistance was utilized in the production of this manuscript.