EUCOPE Resources / News - March 17, 2023

Position Paper on Real World Evidence (RWE) across lifecycle of ATMPs

The past 20 years have seen the gradual launch of advanced therapy medicinal products (ATMPs), in the EU, presenting patients with treatment options where, in some cases, no previous treatments were available. This pace in innovation is not expected to slow down, with five gene therapies alone approved in the EU in 2022. To ensure that patients benefit from these transformative therapies as soon as possible, establishing an appropriate Real World Evidence (RWE) framework is key. 

RWE plays a role both in facilitating Marketing Authorisations, as well as in post-authorisation settings, addressing the inherent data uncertainties associated with ATMPs and supporting innovative payment and risk-sharing solutions. It is in the post-authorisation context where more barriers to the use of RWE exist, but major opportunities lay. As the EU revises the General Pharmaceutical Legislation, establishes the EU HTA procedures, and debates the European Health Data Space proposal, it is the opportune moment to update the EU landscape to embrace RWE in the context of ATMPs, building a futureproofed system. 

Our latest position paper explores the role and acceptability of RWE across the lifecycle of ATMPs, and provides the policy solutions that are needed at both EU and national level that promote the uptake of RWE across the ATMP life-cycle to help encourage access and embrace this wave of transformative innovation.


Our Policy Recommendations:

  1. Revise the General Pharmaceutical Legislation to allow for more regular use and acceptance
    of RWE and alternative evidence-generation strategies.
  2. Call for an updated and living framework document including recommendations and methodologies
    for the use of RWE for regulatory purposes.
  3. Recommend the creation of a multi-stakeholder EU learning network on RWE
  4. Remove obstacles to cross-border flow of health and personal data in the EU.
  5. Welcome the ambition behind the Data Analysis and Real World Interrogation Network (DARWIN
    EU) project.
  6. Call for clarity and harmonization regarding the application of GDPR provisions to address the
    fragmented landscape caused by different Member State interpretations and remove barriers to the
    secondary use of electronic health data.
  7. Establish a framework for earlier and more frequent dialogue between developers, regulators, HTA
    bodies, and payers
  8. Call for the Joint Clinical Assessments methodologies to reflect the specificities of innovative
    technologies such as ATMPs,
  9. Call for Joint Scientific Consultations to be offered to all developers under the EU HTA Procedure;
  10. Support investment to establish the necessary IT infrastructure, human resources, data exchange

EUCOPE’s Cell and Gene Therapies Working Group is a forum to discuss developments and collectively find solutions for funding, pricing and reimbursement of advanced therapies medicinal products so that they actually reach the patients.

For more information on EUCOPE’s Cell and Gene Therapies Working Group or how your organisation can contribute to it, please contact Victor Maertens