The impending launch of numerous “next-gen therapies”—inclusive not only of ultra-high-cost cell and gene therapies but also biomarker-based precision treatments as well as digital therapeutics—will disrupt traditional treatment paradigms and require stakeholders to take a more coordinated, expansive approach to data collection, evidence-generation, outcomes monitoring, and value assessment over time.
Life sciences’ pipelines are increasingly shifting toward more narrowly targeted precision therapies, ranging from cell and gene therapies to CRISPR, CAR-T, and other treatments that leverage patients’ biomarkers and genomic makeup to treat disease. In fact, there are over 1,764 cell and gene therapies in development. But beyond such new-in-kind drugs, investors and life sciences organizations are also pouring money into innovative digital therapeutics (DTx) and digital health tools, many of which leverage technology such as apps, telehealth platforms, and even video games to treat disease. In 2021, investors poured $29.1B into U.S.-based digital health startups, and investments in digital therapeutics increased 2.6x between 2020 and 2021.
Despite promising clinical advancements, many of these treatments carry a high price tag (ranging from tens of thousands to millions of dollars). This raises the bar for the evidence and proof of “value” that payers, providers, and regulators will require for product coverage and use. Yet next-gen therapies and digital therapeutics create two novel challenges for innovators focused on evidence generation and outcomes monitoring.
First, many next-gen therapies are durable and/or curative in nature, with safety and efficacy profiles that require long periods of monitoring to prove value for each patient. However, today’s value assessments typically rely on randomized clinical trial (RCT) data spanning shorter (one- to three-year) time horizons. Most health care organizations are just starting to develop the infrastructure needed to track and evaluate more longitudinal outcomes. Additionally, these therapies are pushing payer and provider organizations to expand the scope of their clinical value assessments to consider a greater range of pharmacoeconomic outcomes and impacts on total costs of care.
Second, the broad classes of cell/gene therapies and digital therapeutics are, together, starting to illuminate the industry-wide need for value assessments that consider an expanded range of clinical endpoints, not to mention the need for manufacturers to generate stronger evidence dossiers overall. For example, some customers may place greater weight on patient-reported outcomes (PROs) that help illuminate patient experiences, quality of life impacts, and preferences over time. To help stakeholders evaluate digital therapeutics, manufacturers may need to collect digital-first endpoints via wearable devices or smartphones, and they’ll need to provide greater proof of adherence and clinical impact over time.
It’s also worth noting how cell/gene therapies and digital therapeutics are disrupting the care continuum and dispersing the most common places for treatment, albeit in very different ways. Notably, patients are still funneled to just a handful of Centers of Excellence for initial cell/gene therapy, even if follow-up care and monitoring happens closer to home. Conversely, most digital therapeutics meet the definition of “everywhere care”—supporting patients at home, at the office, virtually, etc. In either case, clinical innovators will need to develop compelling ways to track safety, efficacy, and durability across a wide range of clinical and home-based settings.
Implications for life sciences leaders
Life sciences leaders will need to expand where and from whom they generate evidence. They’ll also need to broaden the kinds of data sources that can provide insight into endpoints customers may require. Doing so will require heightened collaboration across medical affairs, HEOR, market access, and other internal functions, as well as with cross-industry stakeholders. Such efforts will not only enable smarter, more coordinated evidence generation, but they may also pave the way for new opportunities in value-based contract design, especially for high-cost cell and gene therapy drugs.
Life sciences leaders must also recognize how payers and providers are broadening the set of treatment options they consider as comparators. Drugs are no longer evaluated in isolation. With heightened cost pressures and limited insight into longitudinal outcomes, decision-makers may expand their evaluations from just drug vs. drug to drug vs. digital therapeutic or drug vs. non-medical intervention. As a result, customers will have new demands for comparative effectiveness studies or real-world data showing meaningful differences among treatment options.
Questions to consider
- How are you working cross-functionally to identify and prepare for your customers’ future evidence needs? Are you thinking about endpoints that customers might require three to five years from now and incorporating those endpoints into study designs today?
- What real-world data sources (e.g., claims, EHR data, data from wearable devices, patient reported outcomes) can you leverage to better demonstrate the value of your products?
- Are you generating evidence that shows how your products impact a broader range of metrics beyond safety and efficacy, such as total cost of care, absenteeism, or patient satisfaction?