On the road toward commercialization, RWE puts a spring into your step


Despite healthcare’s notoriously slow adoption of innovation compared to other industries, every few years, an idea so powerful floats up to the surface that even the most change-wary gatekeepers of the healthcare edifice get inspired by its potential to reinvigorate the field. Two such megatrends that have earned widespread acceptance in recent years are patient centricity and digital health, whose appeal is so universal that no organization has wanted to risk being seen as a dinosaur by not embracing them.

Having started to gather steam shortly before the COVID-19 crisis, real-world evidence (RWE) has emerged as the newest sweeping, multi-faceted, celebrated idea that promises to leverage unprecedented patient intelligence into accelerating innovation and improving care. 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, the utility of RWE became even more evident. So let us stop to appreciate why it has garnered so much interest lately.


What is RWE?


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.


The value of RWE across a (digital) product life cycle


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 conduction 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.


The value of RWE from multiple stakeholder perspectives


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. The latter 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 also carries a significant downside risk for investors.

Last but not least, RWE’s value can also be 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 (check out our recent blog on reimbursement challenges for DTx).


How to design and leverage an effective RWE study?


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 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 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 the ultra competitive commercial setting we are immersed in by addressing unilateral stakeholder needs. Its benefits have to cut across – and be clear through – all stakeholders up and down the value chain, so be sure to bring on board as early as possible a capable communications team steeped in the language of health technology value creation.


A word on RWE leadership


Conducting a RWE study is without a doubt bound to be more challenging in practice than described in a blog. RWE leads have to consider complex corporate structures that vary widely across global, regional, and local divisions and teams. Further, 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. Therefore, word to the wise is that 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 to hoping this succinct overview will serve as an encouragement.

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