Call to Action of Concerned Stakeholders on the Implementation of the EU HTA and Joint Clinical Assessment for ATMPs
This Call to Action, co-signed by 39 patient organisations, clinical societies, research institutions, and industry stakeholders including Cancer Patients Europe, addresses critical concerns regarding the implementation of Joint Clinical Assessment (JCA) methodology under the EU HTA Regulation for Advanced Therapy Medicinal Products (ATMPs). The document argues that adopted guidelines requiring randomised controlled trials risk excluding single-arm trial evidence — on which most ATMPs are authorised — thereby creating barriers to patient access for life-changing therapies in rare and ultra-rare diseases. Signatories urge the HTA Coordination Group to recognise all types of available evidence including real-world data and to limit JCA to description rather than evaluation of uncertainty.