Orphan Medicinal Products: Challenges in Cost-Effectiveness

Questions have been raised by stakeholders, such as manufacturers and patient organizations, about the appropriateness of the cost-effectiveness criterion in national coverage assessment procedures (reimbursement procedures) in cases where the clinical data at time of launch show limitations. These disputes have been exacerbated by the lack of transparency, in some countries, in the relationship between cost-effectiveness and health authorities’ decisions for covering medicinal products, and, the lack of a clear formalized structure or framework, which justifies the criteria, arguments and final coverage decision by health authorities.

Consequently the following questions are addressed in this report: 

- "Which clinical and economic issues complicate cost-effectiveness evaluation of orphan diseases and orphan medicinal products?”

- “What is the role of cost-effectiveness in coverage decisions for orphan medicinal products and how to deal with the exceptional outcomes of cost-effectiveness evaluation?”

- “Should arguments (criteria) other than cost-effectiveness be given the main emphasis in coverage assessment procedures of orphan medicinal products, when the clinical data (effectiveness) of a medicinal product at time of launch show limitations?” 

In this report we provide guidance on coverage decisions for (orphan) medicinal products that show limitations of the clinical data at the time of launch. A Simple Transparent Algorithmic Multidisciplinary Procedure (STAMP) for the purpose of optimising the input for the standard set of decision criteria (effectiveness, cost-effectiveness and budget impact).