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Using Real World Evidence in Clinical Development to Enhance Regulatory Submissions: Part 2 in a series
How can real world evidence strengthen a regulatory package?
Jan 22, 2021
Is your company ready to consider real world evidence (RWE) for regulatory submissions? If so, it’s imperative to know the right questions to ask as well as the research designs you may consider in a submission package. Asking the right questions will help determine if you are in a place to consider leveraging real world evidence.
Figure 1 shows a set of questions that may arise when dealing with RWE-related business challenges, and that could indicate that the company is ready to explore the use of RWE in regulatory submissions. These questions may come from a therapy lead who is wondering how to address post-approval long-term follow-up requirements for gene therapies, or looking for ways to strengthen the submission package of a single-arm trial. In some cases, the clinical operations leadership is seeking to drive cost and time efficiencies through use of RWE.
Figure 1: Is your company ready to consider RWE for regulatory submissions?
In specific instances, regulatory agencies have signaled openness to using innovative RWE designs to strengthen evidence presented as part of a regulatory package. Figure 2 highlights key RWE innovative designs that can support regulatory submissions.
Innovative extension studies follow patients post-RCT to assess long-term treatment outcomes using novel data collection methods. Depending on how much you can shift data collection from a traditional, site-based scenario to a lite-touch, site-based, decentralized approach—or even a direct-to-patient approach—you can cut cost tremendously and alleviate patient burden, which ultimately helps patient recruitment and retention. These approaches allow the budget to be focused on obtaining validation of events of special interest, rather than in routinely examining every patient, most of whom will not experience adverse events.
Figure 2: Real world innovative designs can create time or cost efficiencies to counter rising drug development costs.
Gene therapy and other therapies with long-term outcomes goals are a good application for innovative extension studies, especially because they have the advantage of not requiring drug provisioning. U.S. and EU regulators have recommended long-term follow-up for monitoring safety (U.S. and EU) and efficacy (EU) after exposure to non-integrating gene therapies. The draft FDA guidance recommends a risk-based monitoring schedule that includes analysis of patient samples at various time points, and in some cases yearly for up to 15 years. Furthermore, the FDA has indicated that post-approval requirements for therapies with regenerative medicine advanced therapy (RMAT) designation and accelerated approval may be satisfied with RWE coming from such sources as “... patient registries, or other sources of [RWE] such as electronic health records.”5
External comparators that use historical RCT data or RWD are sometimes utilized to demonstrate relative clinical benefit information when randomization is not ethical or feasible. External comparators can bring efficiencies in time and cost, and can increase the odds of regulatory success over single-arm trials. Common scenarios for the use of external comparators include rare disease with high unmet need in which a placebo arm could be considered unethical; indications with highly predictable disease progression, which leads to less variability and renders modeling of the active arm more feasible; and, indications with large expected treatment effect such that the use of RWE will not dilute the effect.
Pragmatic trials in which participants are randomized to a treatment of interest or whatever standard of care is being used (physician’s choice) enable clinical outcomes of interest to be observed over time. These designs are used to help evaluate the effectiveness of intervention in real-life conditions and to reflect therapeutic choices that health care providers and patients make every day. This design can include a broader population that otherwise may be under-represented in RCTs.
Figure 3 provides some questions about endpoints to assess feasibility before implementing any of these RWE-RCT designs.
Asking the right questions and implementing the right innovative study design(s) can help save time and create cost efficiencies to help counter rising drug development costs and meet stakeholder needs. Collectively, taking the right approach to RWE can help strengthen your regulatory package.
In case you missed it, read the first of this 2-part blog series here: "Using Real World Evidence in Clinical Development to Enhance Regulatory Submissions: Part 1."
References
1 EMA - HMA-EMA Joint Big Data Taskforce – summary report
2 EMA - HMA-EMA Joint Big Data Taskforce Phase II report: Evolving Data-Driven Regulation
3 FDA - Real-World Evidence
4 National Medical Products Administration, China, Guiding principles for using real-world evidence, released Jan. 8, 2020.
5 FDA, Guidance for Industry, Long Term Follow-up After Administration of Human Gene Therapy Products (Silver Spring, MD, Jan. 30, 2020).
Real World Evidence. Real Confidence. Real Results.
To learn more about how to create an effective RWE strategy, including innovative design models, and technologies to accelerate results, contact us.