Dr. Chitra Lele, Chief Scientific Officer, Sciformix

Real World Evidence of Regulatory Operations

Dr. Chitra Lele

[This is the third post in a three part series that discusses and to a certain extent demystifies the phenomenon that is Real World Evidence (RWE) and its influence on the product lifecycle. This post focuses on the regulatory view on RWE and how it can help to fill the knowledge gap between clinical trials and actual clinical practice.]

The healthcare industry now has access to an unprecedented volume of electronic data via a variety of sources including but not limited to smart phones, social media, fitness trackers, health surveys, electronic health records, insurance claim databases and patient registries. Although Randomized Clinical Trials (RCT) remain the primary medium for evaluating the efficacy of pharmaceutical drug and medical device safety, the earlier mentioned sources all hold the potential of delivering vast amounts of accessible patient data from outside the controlled RCT ecosystem.

The real world data (RWD) thus collected can help to fill the knowledge gap between clinical trials and actual clinical practice. Properly analyzed RWD can provide vital information about patient health and offer key insights that can help reduce healthcare costs and improve both the safety and efficacy of drugs.

The US FDA has released multiple pieces of draft guidance on the use of RWE, acknowledging that despite its inherent problems with reliability and relevancy, it provides paths for effective and efficient drug development. The FDA first looked at patient reported outcomes (PROs), highlighting their use in the development process and supporting labeling claims. Similarly guidance was released regarding medical devices in 2016, about sources of RWD and how it should be used while making pre and post-marketing decisions.

The FDA is being called to further clarify the use of RWD to support more effective drug development and post-marketing commitments. The report from the bipartisan policy center claims RWD has rarely been used beyond post-marketing surveillance and that the broader use, for example, in labeling expansions, new indications, or to support new drug applications is not yet routinely considered by the FDA.

The 21st Century Cures Act explores ways to integrate the patient’s perspective into the regulatory process, and support 21st century digital medicine by facilitating data sharing and use of new technologies. Section 3002 on patient-focused drug development requires the FDA to issue guidance about how to collect patient experience data and how FDA plans to use these data to evaluate risks and benefits of drugs.

The 2015 World Health Organization’s (WHO) global survey on HTA by national authorities also found that in addition to the paucity of regulatory guidance, RWD is not currently being utilized due to a global lack of qualified human resources, with the majority of countries not having an academic HTA training program.

Advances in IT & new media have afforded a treasure trove of data from a wide array of sources. RWE collected from these sources is an effective complement to clinical trials and has the potential to greatly influence and improve the quality, safety and efficacy of drugs and medical treatments. RWE will only grow in stature and relevancy in the coming years as it will provide a more holistic view of patient health, their response to medications and disease patterns as well as make data available for economic analyses.
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Want to read the first part in the series? Click here.
Want to read Dr. Chitra’s full article on RWE? Click here.
To learn more about our end-to-end RWE and MA services. Click here.