Access Mechanisms for Orphan Drugs: A Comparative Study of Selected European Countries

This study compares pricing and reimbursement (P&R) policy for 43 orphan medicinal products (OMPs) across seven EU Member States: France, Germany, Italy, The Netherlands, Spain, Sweden and the UK. Examined in particular are the standards of evidence and the criteria for P&R decisions, the availability of access before full licensing approval, and requirements for studies after the drug is available on the market. Requirements and practice vary substantially across the seven countries. For example, when access decisions are based primarily on clinical evidence, countries are flexible in their criteria and their approach, accepting lower risk-benefit ratios for OMPs than for other new medicines. When a formal health technology assessment (HTA) process is required, however, evidence requirements are the same as for non-OMP drugs (except in The Netherlands). Countries also vary with respect to pre-market access and requirements for post-launch studies. Although OMPs account for a small portion of total drug spending, continuingly scarce health care resources may create demands for cost effectiveness evidence even for OMPs. This could create serious challenges. The authors recommend greater cooperation across countries to develop a more robust evidence base for OMPs, with much of this research done after the drug appears on the market. Cooperation across stakeholders is essential; interactions involving the EMA, HTA bodies and sponsors early in the development process can help avoid delays by identifying potential evidence issues then and developing ways to address them.