PNS14 VALUE ASSESSMENT OF ORPHAN DRUGS

Orphan drugs assessment in the centralised procedure.

On the basis of the author's experience as member of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and in order to facilitate the access of new orphan drugs to the patients, some suggestions were given. Among these the following should be taken into account by the regulatory bodies: 1) conditional approval or approval under exceptional circumst...

Principles for consistent value assessment and sustainable funding of orphan drugs in Europe

The European Orphan Medicinal Products (OMP) Regulation has successfully encouraged research to develop treatments for rare diseases resulting in the authorisation of new OMPs in Europe. While decisions on OMP designation and marketing authorisation are made at the European Union level, reimbursement decisions are made at the national level. OMP value and affordability are high priority issues ...

Scientific and Social Value Judgments for Orphan Drugs in Health Technology Assessment.

OBJECTIVES We explore how broader aspects of a treatment's value and the impact of the condition on patients not captured by routine health technology assessment (HTA) methods using clinical and economic evidence, defined as "other considerations," may influence HTA processes in different settings. METHODS Countries included were England, Scotland, Sweden, and France. Data sources were the pu...

Orphan drugs revisited.

Commentary Orphan drugs revisited

C. MCCABE, A. TSUCHIYA, K. CLAXTON and J. RAFTERY From the Section of Health Economics and Decision Science, School of Health and Related Research, University of Sheffield, Department of Economics, University of Sheffield, Sheffield, Department of Economics and Related Studies, University of York, York, and Wessex Institute for Health Research and Development, University of Southampton, Southam...