Every few years an idea so powerful is introduced to the healthcare ecosystem that its potential to reinvigorate the field inspires almost every healthcare business. Two megatrends that have earned widespread acceptance in recent years are patient-centricity and digital health. Their appeal is so universal that most organizations have shown interest in embracing them.

 

Real-world evidence (RWE) has emerged as the newest sweeping, celebrated idea that promises to leverage unprecedented patient intelligence to accelerate innovation and improving care, after gathering steam shortly before the COVID-19 crisis. The use of RWE became even more evident as the shock caused by the pandemic led biopharma, healthcare, regulation and health policy authorities to reassess the pace at which they develop, authorize, commercialize and implement at scale innovative treatments and therapies. So let us stop to appreciate why it has garnered so much interest.

RWE is evidence obtained from real-world data (RWD) collected outside of clinical trials and used to demonstrate a therapy’s potential benefits and risks in routine care settings. RWD sources may include electronic health records, patient and disease registries, insurance claims, patient- and clinician-reported outcomes measures, connected medical devices, wearables and digital therapeutics (DTx). This type of evidence can contain qualitative or “unstructured” values, such as free text, that speak to a therapy’s utility to patients beyond just clinical efficacy and safety. Such input is of increasing interest to health technology assessment (HTA), regulatory and reimbursement bodies as they seek to mainstream a more holistic view of patient care than clinical indicators alone can offer.

RWE provides patient-generated insights that are uniquely capable of adding value at different stages of an asset’s life cycle (in the context of pharma) or a care pathway (in the context of care delivery). For example, in clinical product development, RWE can enable more-cost-effective clinical trials by serving as raw material for populating synthetic control arms (SCAs). SCAs are used as a replacement for placebo or standard-of-care control arms that serve as comparators for testing investigational therapies but require a higher total number of trial participants, bigger sponsor budgets and a longer time horizon for conducting studies. In addition, their use can be unethical in the case of randomizing patients with life-threatening diseases in whom standard-of-care treatments have already failed. Still, to date, the most common use of RWE has been for post-marketing safety and effectiveness monitoring, where it is leveraged to maintain market and patient access.

On the patient side, RWE can help uncover new patient demographics (such as breast cancer drug Ibrance’s label expansion to men), unmet clinical needs and novel indications for existing therapies—effectively flagging opportunities that sponsor-driven research objectives may overlook. Similarly, from a clinical and health management perspective, RWE processed in real or near-real-time can reveal blind spots in established care pathways that can be optimized for better patient outcomes.

 

From an investment standpoint, RWE can also reduce spending on developing assets that appear likely to be of low or marginal clinical value. If an asset is of marginal clinical value, this is an important consideration given that private investment is often a decisive factor in bringing new treatments across the finish line (that is, to market approval and commercialization). But this also carries a significant downside risk for investors.

 

Finally, RWE’s value is seen through the prism of it acting as an accelerator for decisions on reimbursement-preferred formulary listings, which can be a make-or-break factor for innovative digital therapies (see our recent article on reimbursement challenges for DTx).

Now that we’ ve taken an express tour of RWE and its many benefits to the pharma and healthcare ecosystems, you may be wondering: “Where do I start in designing my own RWE study to support a research, regulatory approval or reimbursement objective?” I recommend you start by doing a thorough landscape assessment to uncover where the greatest unmet needs of your target user base are and to map early economic models of interest. Then you can work backward from there to determine appropriate measurable outcomes and endpoints. For example, if you are developing a DTx or other connected medical device, an informative endpoint could be one that demonstrates the modulating effect of your technology on the burden of disease as experienced by patients or on standard-of-care clinical workflow and treatment.

 

Once you have shown the utility of your chosen endpoints, you can refine your economic models and do a granular pricing analysis. This, in turn, puts you in a position to start building a data-supported business case, submit your product for health technology assessment (HTA) review and begin communicating value to potential payers. And this last point is where I would like to make a special emphasis, because it’s where many digital health innovators trip: Your product cannot thrive in today’s ultra-competitive commercial setting by addressing unilateral stakeholder needs. Its benefits must cut across and be clear through all stakeholders up and down the value chain. Be sure to bring on board as early as possible a capable communications team steeped in the language of health technology value.

Conducting an RWE study is without a doubt bound to be more challenging in practice than what we can describe in an article. RWE leads must consider complex corporate structures that vary widely across global, regional and local divisions and teams. RWE initiatives may be spearheaded by one or more internal functions, including clinical, regulatory, medical affairs, commercial or drug safety departments. Across different team configurations, there will also be different budget allocations, reporting lines, visions, priorities, data and vendor agreements and access rights. A word to the wise: Internally selling, getting resources for, designing and carrying out a successful RWE study is a fine balancing act—but its inconveniences and risks are almost certain to be outweighed by the eventual benefits.

 

So be bold, learn as much as you can and don’t be afraid to throw your hat in the RWE ring if your product is at any stage of its life cycle that can gain from proof of efficacy and utility beyond a clinical interpretation. Here’s hoping that this overview will encourage you to embrace this innovation.