SCIENTIFIC AND SOCIAL VALUE JUDGMENTS FOR ORPHAN DRUGS IN HEALTH TECHNOLOGY ASSESSMENT

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...

Health technology assessment: oncology drugs with orphan designation as an example

Background Since (limited) health care resources have to be invested efficiently, HTA/ health technology assessment is applied ever more often in many health care systems for ‘rational decision-making’. Around 40% to 50% of all drugs with orphan designation are high-priced oncology drugs. Within EUnetHTA (an EU-supported project) a work package deals with the pre-coverage exchange of early asse...

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 ...

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...

Value-Based Health Technology Assessment and Health Informatics