Rare diseases experts join forces in the European Expert Group of Orphan Drug Incentives

In the driving seat of the project there are EURORDIS, the non-governmental rare diseases patient-driven alliance, and EUCOPE, the European association for small to medium-sized companies in the field of pharmaceuticals and medical technologies, many of which focused on rare diseases. The group was established in the light of the upcoming Pharmaceutical Strategy and ongoing Evaluation of the Orphan Medicinal Products (OMP) Regulation.

Six companies from EUCOPE provide expertise and financial resources to support this initiative: Alexion, Biogen, Bristol Myers Squibb, Chiesi, PTC Therapeutics and Takeda.

The co-chairs of the group are former MEP Renate Sommer, and Professor Maurizio Scarpa, coordinator of MetaBERN and Chairman of the European Reference Networks Board of Coordinators (ERN-BC).

The Expert Group is focusing on three areas to develop concrete policy proposals for potential improvements of the European OMP Incentives environment. These areas are:

• This refers to ensuring that therapeutic areas that currently have no treatment are prioritised, as well as ensuring that there is continued innovation for rare disease which do have some available therapies.
  
• The aim is to optimise the delivery of treatments for rare diseases, as well as understand the reasons for the uneven delivery of innovation in areas of unmet medical need.
  

• A large number of development projects are abandoned prior to finalisation, potentially due to inefficient regulatory pathways.
  
• The aim is to analyse the current regulatory landscape and suggest measures to improve development and access to OMPs for rare disease patients.
  

• This group will aim to support OD development through incentives based on international experience to achieve collaboration between academia, patients, industry and regulators in Europe.
  
• It aims to enhance patient access, increase EU competitiveness, and develop a more streamlined approach with the US.
  

The European Expert Group on Orphan Drug Incentives will aim to publish concrete proposals on how to improve the OMP regulation by early 2021.

For further information, visit the OD Expert Group’s website: www.OD-ExpertGroup.eu