PHP127 Reimbursement of Orphan Drugs: What is the Difference?

Abstract

Orphan drugs are subject to regulatory and reimbursement regulations that differ with respect to application process and necessary documentation. An orphan drug status granted by the European Commission gives marketing exclusivity in the EU for 10 years after approval. Reimbursement hurdles are also supposedly lower for orphan drugs in Europe than usually. Definition and assessment process of orphan drugs for reimbursement were reviewed and analyzed. Differences to other drugs are outlined and reimbursement decisions presented. The German law on health care reform (AMNOG) implemented in 2011 requires that with market access newly approved products demonstrate their innovation through a reimbursement dossier to avoid reference group pricing. For orphan drugs, manufacturers must also submit a dossier but the additional medical benefit is regarded as having been proofed by the market authorization itself. Thus proof of additional benefit does not need to be presented but information on relevant patient groups and on the extent of this additional benefit. However, if annual sales of an orphan drug within the statutory health insurance exceed 50 million EUR, a full assessment is made. For pirfenidone, the first orphan drug assessed under the new law, IQWiG (Institute for Quality and Efficiency in Health Care) declined an additional therapeutic benefit but the G-BA (Joint Federal Committee) did not follow this conclusion in accordance to the law. In Italy pirfenidone was grouped into the lowest reimbursement class. Unlike Germany, Italy has special funds set aside for orphan drugs, France has an early access program, and many countries are struggling with how to create a reimbursement process that reflects the different regulatory provisions for orphans. Although orphan drugs are often regarded as unquestioned reimburseable, differences in respective processes and assessments exists. Manufacturers are requested to build Market Access arguments carefully and expect challenges in orphan drug indications as well.