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EPTRI endorses TRANSFORM’s Position Paper

EPTRI is member of the European Alliance for Transformative Therapies (TRANSFORM), which is a multi-stakeholder Alliance endorsed by Members of the European Parliament and policy-makers, that connects patient groups, medical experts and associations, scientists, researchers, industry actors, networks and other relevant stakeholders.

The aim of the Alliance is to foster dialogue and provide evidence-based policy recommendations to enable safe and timely patient access to cell and gene therapies, whilst ensuring sustainability of healthcare systems. Within the Alliance, EPTRI strengthenes the paediatric point of view proposing paediatric facets of the recommendations.

The members of TRANSFORM with the collaboration of patient groups, clinicians, scientific experts and industry representatives have developed a Position Paper on the recommendations for action in the EU pharmaceutical strategy to inform evidence-based policy-making and to propose actionable solutions along the full patient pathway, from research and clinical development to access to treatment.


The document identify six priorities for action:

  1. Create a patient-centric, innovation ecosystem to address unmet medical needs.
  2. Safeguard patient access by defining appropriate regulatory requirements for ATMPs.
  3. Enable the use of real world patient data through the creation of a European Health Data Space.
  4. Improve infrastructure and enable cross-border patient access to transformative therapies.
  5. Increase patient access and ensure sustainability of healthcare systems through innovative payment models.
  6. Share best practices and recommendations on genetic testing and diagnostics.

The Position paper is available at the following link.