Expanding Patient Access to Advanced Therapies

Area of Science:

• Biotechnology and Pharmaceutical Sciences
• Regulatory Science
• Health Economics and Outcomes Research

Background:

• Advanced therapy medicinal products (ATMPs) hold transformative clinical potential but face significant barriers to widespread adoption.
• Complex manufacturing processes, high costs, and fragmented market access limit the availability of ATMPs to patients.
• Existing regulatory frameworks may not adequately address the unique characteristics of ATMPs, hindering innovation and patient access.

Purpose of the Study:

• To analyze existing and potential regulatory pathways for increasing patient access to ATMPs.
• To evaluate the suitability of current biologic and biosimilar regulatory principles for ATMPs.
• To identify policy recommendations for facilitating sustainable and equitable access to ATMPs.

Main Methods:

• Product-level analysis of ATMPs and biologic medicines submitted to the European Medicines Agency (EMA) up to May 2026.
• Comprehensive review of current regulatory pathways and relevant policy frameworks.
• Comparative assessment of regulatory strategies for ATMPs and established biologic/biosimilar products.

Main Results:

• While some regulatory principles from biologics and biosimilars are transferable, the standard biosimilar pathway is often unsuitable for ATMPs.
• Alternative regulatory approaches, such as bio-hybrid applications and prior knowledge-based dossiers, show promise for ATMPs.
• The analysis highlights the need for tailored follow-on pathways and supportive industrial policies.

Conclusions:

• Establishing tailored regulatory pathways, including bio-hybrid and prior knowledge-based approaches, is essential for ATMPs.
• Open and well-governed platform technologies can foster innovation and competitive entry for ATMPs.
• Supportive industrial strategies and data-sharing policies are critical for translating the scientific promise of ATMPs into accessible and equitable patient solutions.