On April 26, 2023, the European Commission (EC) published a legislative proposal to replace the existing regulations. The proposal aims to introduce a new pharmaceutical strategy to address unmet medical needs, streamline administration, and promote innovation. It includes provisions applicable to all medicinal products, as well as revisions specific to ATMPs.
In a recent commentary published in the June 2023 edition of MedNous, Dr Tatiana Reimer, Principal Consultant at NDA, and Dr Paula Salmikangas, Director of Biopharmaceuticals and ATMPs at NDA, delve into some crucial aspects of the proposal, highlighting potential implications and concerns.
Transformation of Committee Structure
A notable feature of the legislation is the proposed dissolution of three key committees at the European Medicines Agency (EMA): the Committee for Advanced Therapies (CAT), the Paediatric Committee (PDCO), and the Committee for Orphan Medicinal Products (COMP). These committees' responsibilities would be consolidated under the Committee for Medicinal Products for Human Use (CHMP). The CHMP would now take over all the work conducted by these three committees, including classification and assessment of the Marketing Authorisation Applications of the ATMPs. However, concerns are raised regarding the preservation of expertise accumulated by the CAT, which may lead to assessment challenges and potential delays.
Formalization of the Hospital Exemption
The legislative proposal includes the formalization of the hospital exemption (HE) with defined rules. Member states would be required to approve HE procedures and systematically collect safety and efficacy data, which would then be reported to the EMA. However, the proposal falls short in terms of mandating transparency of HE procedures, contrasting with the EMA's general transparency rules for marketing authorization procedures.
Addressing Biosimilar ATMPs
The legislation expands the definition of "similar active substance" to encompass ATMPs. The assessment of similarity between two active substances would be based on biological and functional characteristics. While the feasibility of "biosimilar ATMPs" remains uncertain due to the complexity of many ATMPs and their unique manufacturing processes, the provisions within the proposal could potentially foster increased competition within the industry.
Balancing Patient Access and Data Protection
To enhance patient access to new medicines at affordable prices, the legislation reduces the standard data protection period. The shorter data protection time can be increased if the company is able to make the product available in all EU member states within two years from the authorisation. However, this reduction may present challenges for small companies engaged in the development of novel ATMPs, as ensuring full distribution across the EU could prove difficult due to reimbursement variations and limited manufacturing capacity.
Other Considerations
The proposed legislation addresses various other aspects, including qualified persons (QPs), decentralized manufacturing, the utilization of quality master files, improvements in environmental risk assessment (ERA) for genetically modified organisms (GMOs), and the introduction of a "regulatory sandbox" for testing new regulatory approaches.
Conclusion
The proposed EU pharmaceutical legislation represents a significant transformation of committee structures, the formalization of the hospital exemption, and provisions for biosimilar ATMPs. While the legislation aims to improve patient access to affordable medicines, concerns persist regarding the preservation of expertise, transparency of HE procedures, and the feasibility of biosimilar ATMPs. As the legislative proposal undergoes review and potential amendments, it is imperative to monitor its progress and anticipate the impact on the pharmaceutical industry within the EU.
