Five prominent real-world data and analytics companies are launching the RWE Alliance, a new industry coalition seeking to push the FDA toward using real-world evidence (RWE) to support its regulatory decisions. The founding members are Aetion, Flatiron Health, IQVIA, Syapse, and Tempus.
Working off the foundation established in the 21st Century Cures Act, the group hopes to push for clarity in where and when RWE can be used in lieu of or in addition to clinical trial data to support approval of novel drugs and treatments as well as new indications for existing therapies.
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The RWE alliance plans to provide expert opinion to Congress and engage with FDA on its 2021 Guidance Agenda and 2022 user fee authorization, according to a statement.
RWD is health-related data derived from sources outside of clinical trials, such as electronic health records, administrative claims, product and disease registries, personal devices, wearables, and other health-related applications. By analyzing that data and taking efforts to address bias, RWD can be used as RWE.
RWE has been used to speed access to new treatment options, especially for rare diseases, and served as a resource to better characterize treatment effects, including those in underrepresented populations.
“A lot of effort went into creating an FDA framework to tap into the power of RWE and we’ve made amazing progress in the years since, especially during Covid, which created a need and urgency that catalyze energy in this space,” said Lowell Schiller, chief regulatory and legal officer at Aetion, in a phone interview. “That’s why RWE organizations are coming together and agree the time is right to make sure we have a unified voice that we can present as a resource for legislators and in developing policy.”
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Schiller brings a helpful perspective to this effort, having previously served as Principal Associate Commissioner for Policy at FDA until September 2020, when he joined the team at Aetion. He reasons that the overwhelming pain point the coalition seeks to address is clarity around expectations, given the newness of the field and the need to settle investor anxiety.
“Investors are understandably reluctant to jump in and invest in undertaking RWE-dependent research without knowing if it will be accepted,” Schiller said. “The organizations coming together now to form this coalition hope to help the FDA develop a shared understanding of what ‘good’ looks like, to articulate that and then put it into practice.”
Schiller said the coalition seeks clarity on statistical standards, real-world endpoints, and the requirements for “external control arms,” wherein companies use a natural history study as the control arm of a clinical trial in lieu of having an entire group of people take a placebo.
The FDA has indicated support for external control arms where it would benefit the research, such as phase II oncology trials where no effective standard of care exists, or trials for rare diseases with a very small number of eligible patients. However, the statistical standards to control for bias are still being agreed upon.
“We need to know, at a high level, how tight a fit we need between the real-world population and the study population — there are 50-100 things you need to be looking out for,” said Schiller, “And I’m not sure there’s a shared understanding yet of where we fall on these points.”
The RWE Alliance will collaborate with patient groups, biopharmaceutical and medical device companies and their trade associations, as well as other key stakeholders to support broader efforts around the use of Real-World Data (RWD) to produce RWE, according to the news release.
“We applaud FDA’s ongoing leadership in forging the necessary public and private partnership to advance its use of RWE to monitor post market safety and adverse events and to make regulatory decisions,” said Rob Kotchie, president, Real World Solutions, IQVIA in a statement.
Schiller is particularly hopeful that RWE will help diversify study data.
“RWE is an important tool for achieving greater equity and since leaving the FDA, I have been hugely interested in enhancing the diversity of clinical trials,” Schiller said. “There are a lot of gaps, some of which are structural, and real-world evidence helps fill some of those gaps.”
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