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Using Real World Evidence in Clinical Development to Enhance Regulatory Submissions - Part 1 in a series
The growing acceptance by regulators globally
Jan 20, 2021
The use of real world evidence (RWE) to enhance clinical trials and regulatory submissions is gaining more acceptance around the world. Regulatory bodies are encouraging the incorporation of evidence beyond that from randomized control trials (RCT). Following a long history of applying RWE in evaluating safety, it is now recognized that RWE can be leveraged to assess effectiveness and can inform regulatory decisions as well. RWE also provides value in optimizing clinical trial protocols, such as testing the impact of inclusion and exclusion criteria on the size of eligible population for recruitment. Figure 1 shows some highlights of the increasing application of RWE by regulators across the globe.
Figure 1: Growing use for RWE by regulators across the globe.
The 21st Century Cures Act includes a clear mandate that the FDA consider the use of RWE when modifying an indication for a therapeutic, such as adding a new population or new information about comparative effectiveness.1 The European Medicines Agency (EMA) and Heads of Medicines Agencies (HMA) have released two reports on “big data,” a term that is often used as a synonym for RWE.2,3 Despite the fact that these regulatory bodies recognize they have no control over big data, they understand its importance.
To quote this report, “While randomized, double-blind controlled trials will remain the reference standard for most regulatory-use cases, the complementary evidence that big data sources generate may facilitate, inform, and improve decisions.”3
China’s National Medical Products Administration is currently the only regulatory authority that has issued formal guidance around the use of RWE. These guiding principles focus on effectiveness in addition to safety, with an interest in pragmatic randomized control trials.4 Their initial focus has been on rare and unaddressed diseases and recognizes the value of external comparators.
Some real world evidence success stories to date
Recent regulatory decisions have led to label extensions based on RWE. Among these is a notable decision by the FDA to broaden the label for Pfizer’s Ibrance (palbociclib) to include metastatic breast cancer in men.
Another notable circumstance arose in the evaluation of a label expansion for the anti-schizophrenic medication Invega Sustenna (paliperidone palmitate) in a different delivery format. The decision was based on a pragmatic randomized trial that provided a comparison between a new once-monthly injection of paliperidone palmitate and a choice of commonly used oral medications. A trial was conducted among a high-risk population group and revealed significant delays in relapse with the monthly administration compared to daily medication use. Most likely, there will be more of these pragmatic trials as time goes on.
A different example addresses the situation in which early trials have shown new treatments to be quite promising, as was recently encountered by Amgen for Blincyto (blinatumomab). This drug was initially approved for lymphoblastic leukemia. Amgen conducted a Phase 2 trial that it later supplemented with the real world comparators and achieved a label expansion for refractory acute lymphoblastic leukemia.
In China, Avastin (bevacizumab), in combination with a specific set of chemotherapies, was approved in 2015 based on RWE. As new combinations of chemotherapies evolved, regulators looked at evidence for new chemotherapies. They considered RWE from medical record reviews at three different hospitals, all of which showed the benefit of Avastin.5,6,7 As a result, the label was modified to address these newer chemotherapy combinations.
The information above further demonstrates that real world evidence provides valuable data to substantiate label expansions and augment initial approval. The next blog post in this two-part series covers, “How Real World Evidence Can Strengthen a Regulatory Package.”
References
2 EMA - HMA-EMA Joint Big Data Taskforce Phase II report: Evolving Data-Driven Regulation
3 EMA - HMA-EMA Joint Big Data Taskforce – summary report
4 National Medical Products Administration, China - "Guiding principles for using real-world evidence," released Jan. 8, 2020.
5 Xing P, Mu Y, Wang Y, et al. "Real world study of regimen containing bevacizumab as first-line therapy in Chinese patients with advanced non-small cell lung cancer." Thoracic Cancer - 2018;9(7):805-813. doi:10.1111/1759-7714.12650
6 Tang N, Wang Z - "Comparison of bevacizumab plus chemotherapy with chemotherapy alone in advanced non-small lung cancer patients." Onco Targets Ther. 2016;(9):4671-4679. doi:10.2147/OTT.S110339
7 Li X, Abbas M, Li Y, et al. (2019) - "Comparative effectiveness of Pemetrexed-platinum doublet chemotherapy with or without bevacizumab as first-line therapy for treatment-naïve patients with advanced nonsquamous non-small cell lung cancer in China." Clin Ther. 41(51) 8-29. doi: 10.1016/j.clinthera.2019.02.004
eBook: Explore Transformative RWD Sources
Discover how you can leverage RWE to accelerate study timelines, shorten time to market, cut research costs, and more. In this interactive eBook, read case examples on how key client questions were answered in various therapeutic areas and glean insights on how you can use RWD to answer challenging research questions.