To date, most health care stakeholders define “next-generation” by whether a therapeutic or intervention is curative or durable; high-cost; a cell or gene therapy; and/or indicated to treat rare disease. But the term next-generation should be much broader than that.

Panelists discussed how next-generation therapies should include treatments that change how the health care system—and especially providers—operationalize care delivery. Some next-gen therapies are delivered via new routes of administration, are used by clinical specialties that don’t traditionally manage high-cost or infusion-based treatments, or require a higher level of multi-specialty collaboration. Panelists mentioned Aduhelm for Alzheimer's, and Tepezza, the first infusion-based drug on the market to treat thyroid eye disease, as examples. Both therapies require providers to operate in new ways. For example, ophthalmologists have rarely had to administer infusion-based drugs until the launch of Tepezza.

Many payers, providers, and value assessment organizations have not shifted their mindset about what constitutes value for next-generation therapies. Many still view next-gen treatments through the same framework used for traditional small-molecule drugs: strictly evaluating safety and efficacy over a short time horizon (typically 1-2 years) based on limited data from a pivotal randomized controlled trial (RCT).

However, for many curative or durable therapies, safety and efficacy can only be established after a long period of time, which means value cannot be assessed in the short-term. As result, stakeholders must shift from looking at one-time costs and immediate clinical outcomes to a broader, longer-term set of value drivers. This includes longitudinal outcomes, the opportunity cost of not treating a patient, the overall impact on total cost of care, and patient preferences or quality of life. It can also include new-in-kind value driver For example, stakeholders are starting to consider novelty as a value driver because by making an untreatable condition treatable, there can be beneficial ripple effects in terms of better screening, better diagnosis, and better understanding of the disease etiology.

Panelists discussed how the data and endpoints captured in clinical trials are not the same as those needed to assess value in the real world. To fully assess the value of next-gen therapies, stakeholders need to understand the impact on long-term outcomes and value drivers like quality of life. Capturing those metrics requires leveraging a variety of nontraditional real-world data sources – like data from wearable devices or patient-reported outcomes (PROs).

However, whether FDA and other stakeholders accept this data as evidence of value remains to be determined. Many stakeholders believe PROs or other emerging data sources have either too much or too little granularity, are “squishy,” and are not objective. For example, one panelist noted how it’s difficult to evaluate Aduhelm because “it is difficult to maintain objectivity when endpoints like quality of life and cognitive impairment are inherently subjective.”

During the session, panelists noted how the FDA’s work on patient-focused drug development has encouraged manufacturers to recognize the benefit of capturing patient-centered outcomes. For example, life sciences companies and evidence generators that identify and measure quality-of-life metrics can “completely transform” their ability to prove their medicines’ value. Some can even justify higher price points based on this data.

However, the metrics that payers, providers, and manufacturers collect are not always aligned with what patients value. Additionally, patients often have their own definitions of terms like functional improvement or quality of life, which can impede shared decision-making between patients and their providers.

As next-gen therapies continue to enter the market, patients will be followed for longer time horizons, and information about their experiences will play a crucial role in value assessments. Organizations that don’t start collecting this data risk falling behind, even before product launch.

Today, most payers and providers do not actively capture longitudinal data about the impact of next-gen therapies. One primary reason is the lack of infrastructure or incentives to capture the data in electronic health records. As one panelist noted, “As much as we want to use new-in-kind data for our analyses, we don’t have the data because there is no way to capture it longitudinally in our current medical record ecosystem, and it’s not a requirement.”

Additionally, many hospital systems, centers of excellence, and independent physician groups struggle to identify who is (or should be) responsible for capturing and tracking outcomes data longitudinally. This challenge is made more complex as physicians are already burnt out and not reimbursed for the additional tasks of data collection. Moving forward, stakeholders must identify practical ways to aggregate data and incentivize its collection.

During the session, panelists discussed how providers and payers have struggled to reconcile the tension between population-level analysis and decision-making for individual patients for next-gen therapies. However, some organizations are taking active steps to grapple with this challenge head-on. For example, Novant Health System is evolving its P&T committee to look at the population health impact of a therapy, rather than focusing on individual patient impact, and leveraging ICER reports to help determine population value. It is also starting to evaluate pharmacoeconomics from a broader value and ethical framework.

Moving forward, payers, providers, and value assessment organizations must recognize that value will evolve over time, and that our collective understanding of value will be driven by the data and evidence we measure. Manufacturers must also continue to develop evidence to inform value assessments over time.