EURORDIS release joint statement on patient access to medicines for rare diseases
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Europe: EFPIA-EURORDIS release joint statement on patient access to medicines for rare diseases 


The European Federation of Pharmaceutical Industries and Associations (EFPIA) and Rare Diseases Europe (EURORDIS) have published a joint statement on Patient Access to Medicines for Rare Diseases outlining recommendations to support accelerated and broader access to both currently approved and future innovative medicines. They came up with six proposals, split into three subgroups, aimed at addressing the key barriers to patient access for orphan medicinal products (OMPs) and rare diseases.  

Increasing the equity of access to rare disease medicines 

1:  Equity Based Tiered Pricing (EBTP) 

Based on this proposal, products would be priced differently in different countries based on their ability to pay. In practice, this would be possible through country tiering in which the launch price within lower tiers must be below the lowest price in the tier above. To work effectively there would need to be adaptations to ERP and regulation of parallel trade. 

2: Industry Commitment to File and European Access Portal 

This proposal consists of two parts: 

  • As of April 2022, EFPIA member industry companies commit to submitting P&R applications across all 27 EU member states within two years of EU marketing authorisation. 
  • EFPIA have launched a European Access Portal, which will be used to collect information on the P&R status of medicines in all EU countries and identify causes of delays. 

Improvement of HTA and P&R processes  

3:  Supporting enhancement of EU-level and cross-country collaboration for OMPs 

As part of the recent Regulation (EU) 2021/2282, joint HTA activities across EU member states will be implemented from 2025, including mandatory joint clinical assessments (JCA). EFPIA and EURORDIS support the Regulation but voice concerns regarding the application to OMPs. 

4: Adaptive pathways and Real-World Evidence (RWE) in value assessments 

Due to evidential uncertainties at time of EU market authorisation, it can be more difficult for OMPs to go through conventional HTA and P&R pathways. 

  • EFPIA and EURORDIS propose using adaptive pathways utilising RWE for OMPs, allowing patients earlier access to medicines and companies time for further data collection. 
  • To coordinate the cross-country collection of RWE in rare diseases, EURORDIS have proposed the establishment of an EU Fund for RWE. This would provide a platform for members to align research questions for post-approval data collection and allow collaboration across Europe, mitigating the issue of small patient populations. 

5: Adaptation of country-level HTA and P&R frameworks  

EFPIA has developed several proposals for adaptations to country level systems which aim to overcome the difficulties OMPs face in conventional HTA and P&R frameworks: 

  • More systematic use of novel payment and pricing models (e.g. outcomes-based pricing, over-time payments) to address clinical and financial uncertainty. 
  • A need for HTA and P&R bodies to recognise the inherent challenges faced by OMPs due to the relatively smaller knowledge base behind the diseases and difficulties generating robust evidence. They propose an adaptation to HTA methods to offer flexibility on uncertainties within evidence packages. 
  • Proposals for all patients within the authorised EMA label to be considered for reimbursement to prevent restrictions to the already small patient populations for OMPs. 

Support for access today and innovation in the future 

6: ‘Moonshot’ for basic and translational research for adult and paediatric rare diseases 

Due to the limited understanding behind rare diseases, EFPIA and EURORDIS propose the establishment of a ‘Moonshot’ for rare diseases – shared open-science networks to make knowledge sharing across scientific research transparent and accessible. This would allow research to be more coordinated and encourage further investment, thus contributing to more efficient development of innovative products. 


  2., “EFPIA-EURORDIS Joint statement on access to medicines for rare diseases”, 15/6/2022 

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