Using the Hospital Exemption Pathway to improve patient access to ATMPs in Europe

More alignment is needed to ensure that the Hospital Exemption Pathway is used to fill gaps where commercial ATMPs are lacking, for more sustainable and equal access to safe ATMPs.

Europe is lagging behind Asia-Pacific and North America in providing patient access to Advanced Therapy Medicinal Products (ATMPs). Since 2009, 7 out of 25 European Union (EU) approved ATMPs were withdrawn or not renewed, primarily due to commercial reasons. On the other hand, access to ATMPs through the hospital exemption (HE) pathway has increased among EU Member States. Reports suggest that there are at least 32 ATMPs available in individual Member States via the HE pathway. The HE pathway can be a valuable tool for patient access to ATMPs in certain circumstances. However, if not implemented correctly, it could pose a challenge for long-term development and equal access to safe and effective ATMPs. 

In this two-part blog series, the first part explores the concept of the hospital exemption pathway, along with the challenges and opportunities it entails. The second part  delves into the variability in HE implementation and its impact on the access and pricing of centrally approved ATMPs. 

What is the Hospital Exemption pathway?

The EU Regulation 1394/2007 makes it mandatory for ATMPs to obtain marketing authorisation via the centralised procedure, subjecting them to high regulatory standards. This includes compliance with Good Manufacturing Practice (GMP) standards and conducting pivotal clinical trials to demonstrate safety and efficacy. As a result, ATMP developers, that are often small biotech companies and academic institutions, face a significant regulatory burden to gather sufficient data to support HE approval. 

To address this issue, an ‘HE’ was introduced in the regulation for EU Member States to permit the use of ATMPs without centralised marketing authorisation in their territories under certain circumstances. The HE can only be applied to ATMPs that are: 

  • Prepared for an individual patient 
  • Administered in a hospital setting under the responsibility of a medical practitioner 
  • In compliance with an individual medical prescription 
  • Produced on a non-routine basis 
  • Used within the same Member State 

The regulation requires competent authorities of the Member States to authorise the ATMPs under the HE pathway, ensuring compliance with the equivalent requirements of GMP, traceability and pharmacovigilance, that apply to centrally authorised ATMPs.

Challenges of the Hospital Exemption pathway

Opportunities of the Hospital exemption pathway

 if implemented correctly , the HE pathway offers:

Need for overcoming challenges and seizing the opportunities

In summary, the HE pathway for ATMPs comes with both challenges and opportunities. There are opportunities of meeting the unmet medical need, generate clinical evidence in real-world and pathway for accelerated approval, however, there are challenges of unequal access across Europe, concerns around safety and effectiveness and disincentive for future development of centrally approved ATMPs in few markets. Check out the second blog of this two-part blog series that discuss the disincentive part in detail by examining impact of HE ATMPs on the access and pricing of centrally approved ATMPs in few markets.

More alignment is needed to ensure that the HE ATMP pathway is used to fill gaps where commercial ATMPS are lacking, while harmonising it’s application and interpretation across Europe, to ensure sustainable and equal access to safe and potentially lifesaving treatments. 

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