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HomeCall to Action of Concerned Stakeholders on the implementation of the EU HTA and Joint Clinical Assessment for ATMPs

Call to Action of Concerned Stakeholders on the implementation of the EU HTA and Joint Clinical Assessment for ATMPs

About the Call to Action

The new EU Joint Clinical Assessment (JCA) will become an additional barrier for patient access to advanced therapies if the Health Technology Assessment (HTA) Coordination Group does not modernise its approach to evaluating these medicines, warned 33 not-for-profit organisations, including patient groups, scientific societies, research foundations and medical institutes, in a Call to Action announced today.

The organisations urged all those involved in the JCA to recognise and use all types of available clinical evidence, including single-arm trials and real-world evidence, in the assessment of Advanced Therapy Medicinal Products (ATMPs), which include cell and gene therapies. 

Recognising that randomised control trials are often unfeasible and unethical for ATMPs, which often target rare or ultra rare diseases, they called on the EU HTA Coordination Group to:

  • Revise the JCA methodological guidance to avoid discrediting evidence from single-arm trials 
  • Take a more pragmatic approach to running JCAs of ATMPs by using real-world data to fill evidence gaps


Pancreatic Cancer Europe (PCE) is a signatory of this Call to Action. Other endorsing organisations include:

  • Active Citizenship Network (ACN)
  • Alejandro Da Silva Foundation (Spain)
  • Alliance for Regenerative Medicine (ARM)
  • Canadian Organization for Rare Disorders (CORD)
  • CCRM Nordic
  • Community Health Association (Romania)
  • Cystic Fibrosis Europe (CF Europe)
  • Dravet Syndrome Foundation (Spain)
  • European Association for Haemophilia and Allied Disorders (EAHAD)
  • European Association of Urology (EAU)
  • European Brain Council (EBC)
  • European Hematology Association (EHA)
  • European Leukodystrophy Association (ELA International)
  • European Liver Patient Association (ELPA)
  • European Multiple Sclerosis Platform (EMSP)
  • European Society for Blood and Marrow Transplantation (EBMT)
  • European Society for Paediatric Oncology (SIOPE)
  • European Society for Phenylketonuria and Allied Disorders treated like Phenylketonuria (E.S.PKU)
  • European Society of Gene & Cell Therapy (ESGCT)
  • EveryLife Foundation for Rare Diseases
  • Fondazione Telethon
  • Genéthon
  • Gynecological Cancerpatients (Finland)
  • International Patient Organisation for Primary Immunodeficiencies (IPOPI)
  • International Society for Cell and Gene Therapy (ISCT)
  • Italian Federation for Rare Diseases (UNIAMO)
  • Lymphoma Coalition Europe (LCE)
  • Opie Jones Foundation
  • Partners for Patients (PFP) NGO
  • SMA Europe
  • Thalassaemia International Federation (TIF)

Read the full press release for more details on this important initiative.