EMA joins regulatory coalition in calling for RWE to be integrated into regulatory decisions
As more and more biotechs are looking into the potential applications of using real-world evidence in regulatory submissions, the European Medicines Agency is calling for more widespread adoption.
EMA endorsed a statement published Friday by ICMRA, the International Coalition of Medicines Regulatory Authorities, highlighting the use of both RWE and real-world data in development and regulatory authorizations, noting their role
in supporting the development of medicines across their different stages of development and use is evolving rapidly. However, challenges exist, due for example to heterogeneous data sources, different levels of data quality, and various governance models for data sharing and access. Close collaboration between regulators across the world can help address these challenges. ICMRA can play an important role by catalysing increased cooperation on the use of RWE for regulatory decision-making.
RWE has been a particularly nitpicky arena — an FDA pilot program said two months ago that only about half of a select group of clinical trials could be well-emulated with available real-world evidence. RWE was also a focal point in the 21st Century Cures Act, which pushed the FDA in new directions on the evidence.
According to Harvard Medical School associate professor Shirley Wang at the time, challenges were seen in researchers’ attempts to replicate studies in osteoporosis, chronic kidney disease, heart failure, asthma and COPD.
It was noted, according to the pilot program, that other trials could be more closely emulated, such as atrial fibrillation, VTE and hypertension.
In their statement, the 24 member agencies of ICMRA, including the FDA, NMPA and EMA pledged “to foster global efforts and further enable the integration of real-world evidence into regulatory decision-making,” EMA said. As part of that, the agencies noted four focus areas.
First, is the “harmonisation of terminologies for real-world data and real-world evidence.” According to ICMRA, one of the focuses is on generating “common operational definitions of RWD and RWE, with clear scope and level of granularity.” Next, the coalition pointed to a regulatory convergence of “real-world data and real-world evidence guidance and best practice.” The guidance and best practices would include templates, suitable scenarios where real-world evidence can contribute to regulatory decision-making, and metadata.
The third focus area is on readiness — specifically focused on addressing public health challenges and what ICMRA called “emerging health threats.”
That would work by allowing for the quick creation of expert groups on specific topics and collaborating to make studies more efficient. The final focus area is on transparency — defining principles and practices to register pre-specified study protocols and study results in publicly available registries. And on top of that, promoting the publication of results in peer-reviewed and open-source journals.
“These potential areas for regulatory collaboration on RWD and RWE could be taken forward through a variety of existing fora including ICH, international standardisation bodies, and clusters of interested regulators. ICMRA remains committed to steering this work in the interests of patient health and innovation,” ICMRA added in a statement.