Position Paper on Real World Evidence (RWE) across lifecycle of ATMPs
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:
- Revise the General Pharmaceutical Legislation to allow for more regular use and acceptance
of RWE and alternative evidence-generation strategies.
- Call for an updated and living framework document including recommendations and methodologies
for the use of RWE for regulatory purposes.
- Recommend the creation of a multi-stakeholder EU learning network on RWE
- Remove obstacles to cross-border flow of health and personal data in the EU.
- Welcome the ambition behind the Data Analysis and Real World Interrogation Network (DARWIN
- 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.
- Establish a framework for earlier and more frequent dialogue between developers, regulators, HTA
bodies, and payers
- Call for the Joint Clinical Assessments methodologies to reflect the specificities of innovative
technologies such as ATMPs,
- Call for Joint Scientific Consultations to be offered to all developers under the EU HTA Procedure;
- Support investment to establish the necessary IT infrastructure, human resources, data exchange