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.
