Real-world data (RWD) are data that come from sources other than traditional clinical trials and are becoming increasingly important to today’s healthcare decisions. Historically, drug developers have been limited to prospective randomized clinical trials as their primary avenue for achieving product approval. Recent technological advances and regulatory expansions, however, have broadened the possibilities available to clinical researchers by enabling the use of real-world data and the evidence that derives from their analysis, which is known as real-world evidence (RWE).
Randomized clinical trials (RCTs) take place in controlled settings and seek to answer narrowly framed questions for selected populations. RCTs are typically the most expeditious pathway for gaining regulatory approval. The data they produce, however, often fall short of informing clinical implementation in a real-world environment. As such, regulatory bodies and healthcare providers often rely on post-marketing studies to guide clinical use.
By contrast, RWD are collected outside of the context of RCTs and as a routine function of modern healthcare. For example, RWD may come in the form of:
- Electronic health records (EHRs)
- Insurance billing and claims
- Disease or medication/device registries
- Patient-reported outcomes
- Biometric monitoring devices (such as phones and watches)
Using these sources of data, therapies can be evaluated under real-world conditions in a broader population and at a much lower cost than is possible with typical RCTs.
Considerations for Real-World Evidence
The FDA and other regulatory bodies consider the totality of evidence when evaluating the safety and efficacy of new therapies. As such, the FDA is developing a framework for evaluating the use of real-world evidence to help support approval of new indications for previously approved therapies and to help satisfy post-marketing study requirements.
The Agency recognizes that the use of EHRs, mobile health technology, and other electronic data-capture technology, together with new trial and study designs using RWD, has the potential to streamline and improve the efficiency of clinical studies. Therefore, they have issued formal guidance on the use of electronic data in clinical investigations and are developing documents to standardize the data used in RWD studies.
Rethinking Study Designs
In contrast to a typical clinical trial in which subjects are recruited and screened to know whether they meet inclusion and exclusion criteria, RWD studies have the potential to leverage EHRs to identify potential subjects while they are in clinical care.
Interrogating EHRs in real time could trigger an on-screen messaging system to advise the clinician that their patient may be eligible to participate in a study and prompt for consent. Using this type of study design, data regarding the subject’s response to the investigational treatment would be recorded in the EHR. Such a design would significantly streamline the clinical trial recruitment and participation process, leading to dramatic increases in efficiency and cost-savings.
Biometric Monitoring Devices
One of the most exciting aspects of RWD studies involves the increasing ubiquity of biometric monitoring devices in the form of smart phones, watches, and other wearable technology. The ability of these devices to collect objective biological data such as heart rate, blood pressure, electrocardiography, mobility, and sleep patterns, has the potential to revolutionize clinical trials.
In typical RCTs, these parameters are recorded in a controlled environment at discrete time points. Using biometric monitoring devices would allow for collection of these parameters continuously and remotely, making the data much more indicative of a therapy’s effectiveness.
Using real-world data can help ensure that patients and providers are better informed, leading to quicker and more definitive incorporation of safe and effective therapies into clinical practice guidelines. Contact Nuventra today for help with the design and analysis of your RWD studies.
References and Related Resources
- FDA Guidance: Submitting Documents Utilizing Real-World Data and Real-World Evidence to FDA for Drugs and Biologics
- FDA Guidance: Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices
- FDA Guidance: Use of Electronic Health Records in Clinical Investigations
- Framework for FDA’s Real-World Evidence Program