PSY108 - Similarities and Differences In Orphan Drug Reimbursement In 16 European Countries

Abstract

Since the introduction of the European rare diseases policy in 2000, 82 drugs have received orphan status. As the orphan drug market is rapidly expanding, this number is expected to increase. The research objective was to identify how European countries manage the increase in available orphan drugs and whether there are similarities or differences in orphan drug reimbursement policies across Europe. We assessed orphan drug policies in 16 European countries, grouped into four archetypes: health insurance markets (Austria, Belgium, France, Germany, The Netherlands, Switzerland); devolved markets (Italy, Spain); health economic markets (Ireland, Portugal, UK); Nordic country markets (Denmark, Norway, Sweden). Policies were assessed considering five key factors: key stakeholders; treatment setting; evidence requirements; reimbursement processes; source of funding. In all country archetypes, except Nordic markets, the reimbursement assessment of orphan drugs used for in- and out-patient care is the same. All archetypes, except health insurance markets, follow the same processes for orphan and non-orphan drugs. Although in Scotland and England, drugs classified as ultra-orphan go through specialised processes. In devolved markets, economic and clinical data are considered; in health insurance markets, economic data are only considered under certain circumstances in some countries. Nordic countries have fewer evidence requirements than other archetypes. In both health economic and health insurance markets, orphan drug funding varies with treatment setting. In contrast, there are strong differences in the reimbursement of orphan drugs in Nordic and devolved markets. Relevant stakeholders in reimbursement assessment are national HTA (health technology assessment) agencies or regional authorities. In all archetypes, except health insurance markets, regional authorities are the relevant funding stakeholders. There are several similarities and differences in orphan drug reimbursement across European countries. While some countries do not differentiate between orphan and non-orphan drugs in decision-making or funding, others have implemented specific policies.