OBJECTIVES: Advanced Therapy Medicinal Products (ATMPs) are distinct from traditional pharmaceutical products and include high cost and benefits uncertainty. This research aims to identify developments in the regulatory and reimbursement landscape to improve access for patients.

METHODS: In February 2022, we reviewed published ATMPs guidance. We identified 66 products classed as regenerative medicines by the Alliance for Regenerative Medicines. We searched the Food and Drug Administration (FDA) and European Medicines Agency (EMA) websites for regulatory approval status. We reviewed health technology assessment (HTA) bodies in the US, UK, and EU4 (Germany, France, Italy and Spain) for reimbursement information and status.

RESULTS: The FDA and EMA offer accelerated pathways for regulatory assessment but differences exist across ATMPs’ classification and access. Over 25 products are FDA-approved (including cord blood therapies and tissue-engineered products [TEP]). Eleven are available in the US but not in the EU, mostly in the TEP category (9). Fourteen have been formally classified as an ATMP by the EMA and authorized for use, seven of them are not yet FDA-approved. Unlike the regulatory agencies, there are no specialized HTA committees with dedicated ATMPs expertise. Only four HTA guidance documents specific to ATMPs were identified, in the US, Germany, and the UK. Many payers instead acknowledge their desire to improve access for “innovative” products and to explore funding for high-cost treatments. The recently adopted EU Regulation on HTA includes ATMPs within the first group of products it will apply to in 2025. Given the differences in the assessment framework across HTA bodies, the purpose and conclusions of each guidance varied.

CONCLUSIONS: This research reveals differences between EU and US access from a regulatory and HTA perspective. Harmonization of ATMPs assessment procedures and cohesive data collection planning at pre-authorization and post-authorization could help close the reimbursement gap, particularly within the EU.