Journal Article

ARM’s Perspective on the First Joint Clinical Assessments for ATMPs: Challenges and Opportunities on the Path Ahead

Morgese Paolo, Patel, Dilip, Majors, Stephen

2025

Overview

Advanced Therapy Medicinal Products (ATMPs) are revolutionising modern medicine. By addressing the root cause rather than the symptoms of disease, ATMPs hold the promise of long-lasting benefits or even cures for severe, genetic, and rare diseases—including rare cancers—for patients with few or no viable treatment options. At the same time, the inherent complexities of ATMPs pose challenges to traditional HTA frameworks. Unlike conventional treatments, ATMPs are often one-time therapies with a high magnitude of effect. However, their long-term durability remains uncertain at launch. The Joint Clinical Assessment (JCA), under the EU’s Health Technology Assessment (HTA) Regulation, represents a once-in-a-generation opportunity to consolidate the strengths of national HTA processes into a unified framework that accounts for the specificities of ATMPs and streamlines decision-making, cementing Europe’s position as a pioneer in innovative HTA approaches. While concerns remain regarding the suitability of current JCA methodologies for ATMPs, the HTA Regulation continues to bring the HTA ecosystem closer together, with numerous benefits already emerging from EU-wide collaboration on JCAs. This article outlines the HTA challenges posed for and by ATMPs, and ARM’s perspective on the JCA’s implementation. A ‘fit for purpose’ JCA holds the promise to unlocking these therapies’ benefits for individuals across Europe.

• Publisher: MDPI AG
• Subject: Benefits / Cancer / Clinical assessment / Clinical decision making / Decision making / Design / Disease / Evaluation / Genetics / Health technology assessment / Medical technology / Medical treatment / Medicine / Patients / Rare diseases / Regulation / Stakeholders / Standard of care / Suitability / Technology assessment / Therapy / Treatment methods