Real-world evidence (RWE) is fundamentally changing the future of healthcare. Here’s how it happens:
Let’s take a closer look at RWD, how it becomes RWE, and the value of RWE across the healthcare ecosystem delivered through nine use cases. Specifically, we cover:
In order to understand RWE and its role in healthcare, we must first define RWD.
Real-world data is any data that is collected in the context of the routine delivery of care, as opposed to data collected within a clinical trial where study design controls variability in ways that are not representative of real-world care and outcomes.
There are many different types and sources of RWD. Let’s take a closer look at each type, its source, and its uses.
Clinical and medical record data come from electronic health records, prescriptions, and lab and imaging studies. These data provide patient demographics, vital sign measurements, and lab and imaging results that are collected during clinic visits, procedures, and hospital stays.
Payer and regulatory data come from claims and government datasets, and they provide information regarding healthcare services utilization, population coverage, and prescribing patterns.
Registry data come from disease, device, patient, and specialty clinical registries. These data provide information on treatment outcomes, safety, and patient quality of life.
Patient-sourced data come from patient surveys, biometric wearables, and population health measures. These data provide insights directly from the patient, and they help researchers understand what happens outside of clinic visits, procedures, and hospital stays.
The U.S. Food and Drug Administration (FDA) defines RWE as “the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real-world data."1
Unlike traditional clinical trials, where necessary data elements can be curated and collection mandated, the creation of RWE requires assessing, validating and aggregate various often disparate sources of data available on the routine clinical practice.
The creation of RWE requires a combination of high-powered analytics, a validated approach and a robust knowledge of available RWD sources (e.g. what data is capture within existing quality registries, what data can be capture through an EHR or claims, which patient organizations capture data on relevant patient cohorts).
This process includes several steps:
RWE can provide a holistic view of patients that in many cases cannot be studied through traditional clinical trials.
There’s a gap between research (what we learn) and everyday practice (what we do) in healthcare, and it creates a difference between what is expected to happen and what really happens.
But it’s what really happens that matters. Driving measurable improvements in healthcare requires us all to be rooted in the reality of what actually happens before, during, and after clinical procedures, interventions, and office visits.
RWE is here to fill those gaps and root us in truth. It tells us what really happens when doctors treat a wide range of patients that don’t look like the homogeneous patient groups in a clinical trial.
Because of this, RWE serves many uses and provides many benefits across the healthcare ecosystem.
Pharmaceutical and medical device companies are major consumers of RWE, as it can provide value across the entire product lifecycle.
RWE plays an important role for research across the product lifecycle for both pharmaceutical and device companies. It can inform pre-trial study design by helping researchers identify potential patients and create proper inclusion criteria for clinical trials.
Much of medical innovation is driven by traditional clinical trials, where new pharmaceuticals and devices are rigorously studied and tracked before they can be sold and widely distributed. Although clinical trials are incredibly important to determine the safety and efficacy of new technologies, they do have some limitations.
RWE can help overcome the limitations of clinical trials by providing information about a broader cross-section of society. This can help clinicians, researchers, and industry partners better understand their products and how they work.
Also, during clinical trials, RWE can supplement traditional outcome measurements with patient-generated data such as patient-reported outcomes (PROs) or biometric data that is easier to collect between doctor appointments.
Once a product is approved and marketed, RWE can help a pharmaceutical or device company understand their products’ relative safety, effectiveness, value, off-label use and more. This post-market surveillance is valuable to stakeholders across healthcare. For example, we support several clinical data registries that provide real-time RWE that device manufacturers use to evaluate their products.
Purposes of this post-market surveillance may include:
RWE can help company expand an indication profile for a product. For example, while it may not be feasible to perform a full clinical trial for a product that is commonly used for off-label indications, by collecting RWD from disease or product registries, companies may be able to study safety and outcomes data for their product or device, which can then be used to supplement a submission to the U.S. FDA or European Medicines Agency (EMA).
In addition, the U.S. FDA has indicated that for certain products, a promise of RWE analysis in the post-market period can be used as part of a regulatory approval. For example, it might not be feasible to run a clinical trial on a product that is to be implanted for a long period of time. In a case like this, post-market RWE can help provide that missing long-term data.
Regulatory bodies like the U.S. FDA and the EMA are increasingly using real-world evidence, and in some cases requiring it, to satisfy post-market approvals.
The U.S. FDA has provided a framework that provides insight and instruction on how to utilize RWE to support a new drug or device regulatory filing.2
They specifically note a few use cases where RWD and RWE can be utilized:
Through advanced analytics, RWE can provide individualized decision support tools that can facilitate shared decision-making between a patient and physician. For example, the Michigan Bariatric Surgery Collaborative uses a tool that predicts, based on individual characteristics, how a patient might respond to different types of bariatric surgery.
Through comparing outcomes for different treatment options, it might become evident that particular drugs or dosages are better optimized for different individuals, and treatment guidelines can be refined and optimized accordingly.
With large real-world datasets, researchers can perform observational, retrospective, and prospective studies that help academia advance its understanding of current therapies and technologies. Check out this list of research publications that use RWE from clinical registries to advance knowledge.
RWE can track outcome and quality measures across organizations. For administrators, RWE can provide information about how their entire health system is performing relative to others, and how their individual physicians are benchmarking relative to peers. This information can help inform coverage discussions with payers, and refine quality-based reimbursement strategies that reward solid outcomes.
Clinical registries, including patient, product and claims registries, not only provide a rich source of RWD. They are also critical tools for generating RWE itself and using RWE to support post-market surveillance and research.
If you want to read more about the role of registries in RWE, check out my post that covers how a global medical device registry that delivers RWE is closing the gap between what really happens in healthcare and what is “supposed” to happen.
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