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Blog
Regulators Want Real World Evidence
RWE delivers added value to the regulatory submission review process
Nathalie Horowicz-Mehler, PhD, MPH, Senior Principal, Head of Real World Evidence Strategy, IQVIA
Sep 29, 2020

The real world evidence (RWE) trend has gained significant momentum over the past several years as regulators have increasingly signaled interest in these studies. Several regulatory bodies, including the US Food and Drug Administration (FDA), and the European Medicines Agency (EMA), have voiced their willingness to consider RWE in regulatory submissions, particularly for oncology and rare disease drugs where clinical trial sizes are small and it is often unethical to conduct placebo arms.

Whether a submission is based on a small single-arm trial, or a larger clinical study, RWE can add value to the submission review process. These studies can be used to show current standards of care, and to demonstrate effectiveness or safety in real world settings, providing regulators and other decision makers with a more robust view of the benefits and risks of these treatments in the general population.

Over the past few years, several drugs have been approved using evidence from real world studies. These include the accelerated approval of the immunotherapy drug Bavencio in 2017 by the FDA. The approval was based on the results of a multicenter phase 2 trial, combined with data from a real-world retrospective study of patients with metastatic Merkel cell carcinoma treated with chemotherapy.

Similarly, EMA approved Kymriah in 2018 based in part on the use of RWE as an external control to confirm a response rate in a single‐arm trial. Further RWE studies of Kymriah in real-world settings in the U.S. show that the efficacy and safety of the drug are consistent with pivotal trial results.

Can RWE bring more value to you?

When deciding whether to use a real world study, sponsors should be able to answer the following questions:

  1. Is the clinical trial protocol still modifiable?
  2. Do we have enough time (18 months or more) to engage with regulators on the use of RWE, and do they approve of its use?
  3. Will RWE add value for our regulatory submission?
  4. Do we have cross functional buy-in from clinical, data, and regulatory experts, as well as support from senior management?
  5. Is the data we are collecting robust and fit for purpose?
  6. Do the endpoints align with our RCT data collection strategy? 
  7. Can our targeted real world data be captured in the study model you select?

If the answer to these questions is “yes,” a real world study will add significant value to the regulatory submission process.

RWE done right

Industry leaders predict that as drug developers focus more on specialized medicines, the use of RWE in regulatory submissions will steadily increase. 
However, studies that use RWE must follow rigorous standards to be included in any submission package. Every regulatory body has different requirements for their use, and expectations for proof of accuracy and the elimination of bias in study results. Sponsors should factor these preferences into their planning process, and work with RWE experts who can help them craft study designs that meet regulatory requirements.

That planning process begins with determining which real world study formats will support the submission goals, and how to best operationalize these studies to generate significant time and cost savings. There are several RWE models to choose from.

  • External comparators. These studies use real world data to establish a comparator arm for single-arm trials when randomization is not ethical or feasible. Their use can cut the time and cost of approval or label expansion by 50%.1
  • Pragmatic trials. These studies evaluate the effectiveness of an intervention in real life conditions, which provides insights into safety and effectiveness for more diverse populations than are represented in a randomized clinical trial (RCT). Their use can lead to a 50% cost reduction for label expansions.2
  • Extension trials. These studies follow study participants post-RCT, to determine long-term value through novel data collection methods. They can cost up to 60% less than site based studies.3
  • Observational studies. These non-interventional studies leverage existing real world data to support label expansion. They can deliver a 90% cost savings and 75% time savings compared to traditional site studies.4 

IQVIA can help you analyze and understand the ever-expanding ecosystem of clinically-rich data in the context of your organization’s needs. By combining advanced analytics with unparalleled data and scientific expertise, IQVIA provides customers with innovative Real World Evidence approaches to meet stakeholder needs throughout the product lifecycle.

To learn more contact us, or listen to the IQVIA webinar, Real World Evidence to Support Biotech from Milestone to Milestone.

 

1, 3 Source: Access Point, Vol. 7, Issue 14, IQVIA, November 2017
2 Source: Nancy Dreyer, Therapeutic Innovation & Regulatory Science. May 2018.
4 Source: Elliott Levy, Amgen Inc., The National Academy of Sciences, July 2018

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.
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