Implications of New European Union Regulations on Advanced Therapy Medicinal Products

Advanced Therapy Medicinal Products (ATMPs) are innovative drugs based on genes, cells, and tissues, offering potentially curative treatment options for a variety of diseases. ATMPs are associated with high costs and, in some cases, uncertainties regarding their effectiveness or safety, which currently presents a barrier to market access for these medications. 

In 2021, the European Parliament and the Council of the EU approved a new EU Health Technology Assessments Regulation (EU HTAR) (Regulation (EU) 2021/2282) to establish common rules and methodologies for HTAs. Then in 2023, the European Commission published a draft of a new EU Pharma Package to revise and replace the existing general pharmaceutical legislation. This sweeping set of proposals will likely affect numerous aspects of pharmaceutical regulation in the EU.

This white paper provides an overview of the key changes impacting access to innovative medicines in the evolving EU market and details of the EU HTAR and EU Pharma Package, with some considerations for manufacturers.