orphan drugs

ISPOR 2022: Changing landscape of Orphan Drug Reimbursement – Evidence from EU4 and England



This study compares the rates of positive reimbursement decisions in EU-4 and England for orphan drugs approved by the European Commission (EC) in 2015 and 2020 to determine whether patient access to Orphan Drugs (ODs) is improving.


ODs approved by the EMA in 2015 and 2020 were identified. Their reimbursement status in EU-4 and England was recorded as of June 2017 and June 2022 respectively.

Time to Reimbursement (TTR) was calculated for reimbursed ODs.


12 ODs approved by EMA in 2015 and 22 ODs approved in 2020 were analysed.

The percentage of positive recommendations (out of those which entered the HTA process) remained the highest in Germany (92%→100%), increased in Italy (50→73%) and England (20%→41%), and decreased in Spain (45%→39%). A decrease was also noted in France (64%→33%), however, thanks to the early access program (ATU), patients could access respectively 81% and 94% of assessed ODs.

Germany remained the country with the shortest median TTR (1.1→1.3 months). For the remaining countries, an overall improvement can be seen in median TTR in Italy (13.6→10.1 months) and France (14→11.1 months), while median TTR in Spain (12.6→18 months) and England (8.7→12.2 months) increased.


Germany continuously provides the most robust patient access to ODs, owing to the strategy of not limiting access during the price negotiation. Wide access to ODs is maintained in France despite an increased percentage of ODs still in the price negotiation due to the Early Access programme.  The largest improvement can be seen in Italy which corresponds to the legislative changes in the pricing negotiation process and their COVID-19 mitigating strategies.

In England, the increased percentage of positive recommendations coincides with the increased median TTR, presumably due to the higher number of ODs in the reimbursement process. With issues identified in previous research prevailing, Spain remains the most challenging market.

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