Orphan Drug Regulation

In response to an EU working document reviewing five years of application of the Regulation on Orphan Medicinal Products, Eurordis welcomes the successful development of 24 orphan drugs and agrees with the main conclusions of the report. Eurordis proposes several actions to improve implementation of the Regulation in the two areas identified as needing progress:

1. Access to authorised orphan drugs

  • EU Committee to help assess Therapeutic Added Value (TAV) and determine a reference price for each orphan drug
  • Open compassionate use programmes.


2. Effectiveness of incentives

  • European Parliament public hearing on orphan drugs
  • Written recommendations addressed by the Commission to Member States.

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