The European Commission’s (EC) Orphan Medicinal Products Regulation intended to incentivise the research, development and marketing of new treatments for rare and chronically disabling or life-threatening diseases. Marketing authorisation granted to orphan medicinal products (OMPs) is however only the first step; patients have access to medicines once reimbursement or health technology assessment (HTA) decisions are implemented by national health systems.
Our analysis found that since the implementation of the OMPs Regulation in 2000, 143 OMPs obtained a marketing authorisation in the EU. These OMPs are most widely accessible in Germany and France. In the other countries between 30% and 60% of OMPs are reimbursed. In England, less than 50% of OMPs are routinely funded by the NHS, with one-third of these recommended by NICE. The remaining products are directly procured and made available to patients by NHS England via commissioning policies or through the Cancer Drugs Fund. In Germany reimbursement is automatically granted to all medicines which receive a marketing authorisation, immediately after it. For the other countries, the shortest time from authorisation to a reimbursement decision is observed in France and Italy which takes on average 19 months.