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Orphan and paediatric reform should strive to improve access to medicine for rare disease patients and children


30 Jul 2021


Health & Consumers

Brussels, 30 July 2021

Medicines for Europe submitted its contribution to the European Commission public consultation on the reform of the orphan and paediatric medicines framework.

The legislative framework incentivising research and development in orphan and paediatric medicines is important to encourage scientific development in traditionally underserved populations.  The lack of  incentives for stimulating faster competition in the follow-on orphan field and for incentivising off-patent paediatric innovation leaves a lot of untapped potential.

The reform should pursue two objectives:

  1. ensure effective, immediate competition at exclusivities expiries for generic and biosimilar versions of medicines for rare diseases;
  2. encourage investment in  innovation on off-patent molecules, especially in the paediatric field. 

Medicines for Europe proposed targeted regulatory and market access measures to stimulate and incentivise development in these areas with the ultimate aim of increasing access to medicines for underserved patient populations.

Adrian van den Hoven, Director General of Medicines for Europe stated: “The EU can do more to stimulate innovation in areas of unmet need, including off-patent paediatric development, where there is clearly no commercial viability for industry to invest . We see an opportunity to revise PUMA (the EU incentive for off-patent paediatric innovation) by linking it to new pricing models similar to what is discussed for novel antimicrobials. Together with other targeted incentives, this would encourage investment in off-patent development. This will need to go hand in hand with solid incentives to develop follow-on orphan medicines to bring more sustainability to healthcare and to reduce health inequalities in rare disease treatment.”

Medicines for Europe contribution to public consultation can be found here.