TO WHAT EXTENT DO DISEASE AND TREATMENT CHARACTERISTICS INFLUENCE HTA-BASED RECOMMENDATIONS FOR A SAMPLE OF ORPHAN DRUGS IN THREE COUNTRIES, AND COULD THESE INDICATE WHETHER ORPHAN DRUGS HAVE A “SPECIAL STATUS”?

Author(s)

Nicod E
London School of Economics and Political Science, London, UK

Routine HTA methods may not adequately capture all the important considerations of a treatment’s value and the impact of the condition on the patient given that evidence is often incomplete. This study aims to explore the influence of broader considerations of scientific and social value judgments on reimbursement decisions for a sample of orphan drugs. OBJECTIVES To identify and compare the extent to which these broader considerations not captured by the incremental cost-effectiveness ratio (ICER) influenced HTA decision-making process in three countries; and, on this basis, explore whether orphan drugs have a “special status”. METHODS Countries included were England, Scotland and Sweden. Ten drug-indication pairs with EMA orphan designation and all appraised by NICE were selected. Publicly available HTA reports were coded using thematic analysis to systematically identify and compare these broader considerations across countries using an existing analytical framework.  RESULTS

Conference/Value in Health Info

2014-11, ISPOR Europe 2014, Amsterdam, The Netherlands

Value in Health, Vol. 17, No. 7 (November 2014)

Code

PSY106

Topic

Health Policy & Regulatory, Health Technology Assessment

Topic Subcategory

Decision & Deliberative Processes, Pricing Policy & Schemes, Reimbursement & Access Policy

Disease

Rare and Orphan Diseases

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