The European Union (EU) is currently experiencing transformative regulatory changes that will significantly impact the pharmaceutical and biotech industries. These changes are deeply rooted in the ongoing EU elections, extensive revisions to critical legislation, and a novel approach to health technology assessments (HTA), which are collectively creating a dynamic new landscape for healthcare companies operating across Europe. These topics were thoroughly discussed at a panel during the BIO International conference, held from June 3 to June 6, 2024, emphasizing the widespread relevance and implications of these changes not only within Europe but also for any global company seeking to enter this evolving market.
Amid these regulatory evolutions, a notable development is the growing focus on decentralized clinical trials (DCTs), which are increasingly viewed as a solution to multiple challenges in pharmaceutical research, including improving patient recruitment and retention. However, the primary conclusion from the discussions was the vital necessity for companies to proactively engage with important stakeholders early, ideally two to three years before a product’s planned launch. This proactive early engagement is crucial to successfully navigate the complex regulatory environment and ensure secure product approval and market access.
Revised EU Pharmaceutical Legislation: Impacts on Orphan Drugs and Market Access
A significant focus for many stakeholders in the industry is the revised General Pharmaceutical Legislation (GPL) and the EU’s HTA regulation. The revised GPL aims to broaden access to medicines and foster more equitable healthcare access throughout Europe. However, Claire Skentelbery, director general of EuropaBio, observed that this revision results in a general weakening of data protection and other provisions governing orphan drug designations and market exclusivity. This reduction in protections could particularly impact small-to-mid-sized biotechs by introducing uncertainty and potentially reducing the incentives for developing treatments for rare conditions.
Thomas Bols from PTC Therapeutics underscored additional complexities introduced by the revised GPL, notably the new requirement for orphan drugs to demonstrate an “unmet need” even in the presence of competing products. This significant change could complicate the investment landscape, as uncertainties about a drug’s market potential might lead investors to assign only baseline values to such products. Companies are therefore encouraged to meticulously review their development pipelines and adapt their strategies to align with the new regulatory framework.
EU’s Revised GPL and HTA Implementation: Key Changes for Pharmaceutical Companies
The latest format of the revised GPL, which was approved in March, at least acknowledges the necessity for innovation protection. The subsequent steps involve detailed assessments by national governments within Europe, with the anticipation that the revised GPL will be finalized by 2028. The current EU elections introduce an element of unpredictability to this timeline, emphasizing the need for companies to remain informed and adaptable.
Another major regulatory adjustment involves the implementation of EU-wide Health Technology Assessments (HTAs), designed to reduce redundant work across member states through joint clinical assessments (JCA). From January 2025, applications for oncology and cell and gene therapies will be subjected to JCAs by all EU member states, with orphan drugs included in this regimen by 2028 and all drugs by 2030. This system aims to streamline the approval process throughout Europe, although it operates independently from the European Medicines Agency (EMA) approval process.
Matias Olsen from EUCOPE highlighted the critical need for regulatory and market access teams within pharmaceutical companies to work collaboratively to effectively manage these changes. Dr. Steffen Thirstrup of the EMA reassured that the agency is committed to ensuring a reasonable alignment between the regulatory approvals and HTA processes. The experience in the UK post-Brexit serves as an instructive model for the EU, with Santoke Naal of IGES UK noting that achieving a uniform interpretation of clinical data across various member states is essential. He also emphasized the crucial importance of early and proactive engagement with HTA bodies, pointing out that previous delays in such engagement have led to product rejections in the UK.
Resource: Pharmaceutical Technology, June 06, 2024
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