Revision of the EU pharmaceutical legislation

In April 2023, the European Commission (EC) published proposals to revise pharmaceutical legislation—including measures relating to medicines for children and rare diseases.  

The proposed reform consists of:  

  • A directive on the EU code for medicinal products for human use  
  • Regulation on the authorization and supervision of medicinal products for human use and on rules governing the European Medicines Agency (EMA)  

You can read the original text of the proposals on the EC site.  

The proposed directive   

For every medicine authorized at EU and national level, the directive covers all requirements for:  

  • Authorization  
  • Monitoring  
  • Labelling and regulatory protection  
  • Placing on the market  
  • Other regulatory procedures.  

The proposed regulation

The regulation:  

  • Defines specific rules for the most innovative medicines authorized at EU level  
  • Creates protocols for management of critical shortages and supply security of critical medicines  
  • Stipulates rules governing the EMA  

Aims

The main aims of this large reform are to:  

  • Ensure EU patients have timely and equitable access to safe, effective, and affordable medicines  
  • Improve security of supply and availability of medicines in the EU  
  • Offer an attractive and innovation-friendly environment for medicine research and development (R&D) and production in Europe  

In addition, the legislative framework will:  

  • Be simplified to reduce administrative burdens and speed up procedures  
  • Become future-proof  
  • Include environmental safeguards  

Why does it matter to EHA and hematology? 

The EU’s pharmaceutical reform directly touches on EHA’s core advocacy priorities:  

  • Affordable and equitable access to innovative therapies  
  • Support for research  
  • Better regulation 

Potential impact on access 

The legislation can address:  

  • Current inequities and delays in access across Europe  
  • Supply problems  
  • High prices 

Potential impact on research

The legislation can:  

  • Stimulate patient-centric and needs-based development of medicines  
  • Create dedicated frameworks for personalized medicine and rare diseases  
  • Incentivize R&D in Europe 


Summary of EHA’s position

EHA has focused its advocacy on the following topics: 

  • Unmet medical need 
  • Hospital exemption 
  • Drug repurposing 
  • Bolar exemption 
  • Incentives 
  • Shortages 
  • Conflicts of interest 
  • Funding transparency requirements 

In April 2025, EHA updated its position on this legislative package. Read the summarized version here, and the complete version here.  

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