The webinar featured:

• John Feore, Associate Principal, Center for Healthcare Transformation
• Brian Leinwand, Senior Research Scientist, Health Economics & Advanced Analytics
• John Neal, Managing Director, Market Access
• Christie Teigland, Principal, Health Economics & Advanced Analytics

Approaches and Challenges in Assessing the Value of Novel Treatments

The traditional methods of evaluating a treatment’s value in the US rely on cost-effectiveness from a payer or health system perspective. However, a gradual shift toward incorporating a patient-centric perspective of value has prompted stakeholders to consider patient-centered metrics in value assessment, resulting in a more holistic representation of value, one that may depart from traditional payer models. Many non-traditional, patient-centric elements of value are often qualitative in nature, introducing difficulties in assessing value in a quantitative fashion.

When applied to novel curative therapies, defining value becomes even more challenging because of therapies’ high cost and the general uncertainty around the real-world effectiveness and sustained benefits of these treatments. Clinical trials only provide a short-term perspective and are conducted in a tightly controlled setting with carefully selected subjects; hence, they may not be generalizable to a larger population over the long term. As a result of these uncertainties, payers often provide hurdles to access curative treatments because of their high cost. Payers value proven, long-term benefits to patients, and manufacturers wish to recoup the cost of development of a potentially curative drug. To resolve these issues around sustained value and shared risk, payers have sought to spread risk across providers and manufacturers through value-based arrangements (VBAs).

Shared risk arrangements between payers and manufacturers include VBAs and outcomes-based contracts (OBCs). The use of these models may help mitigate the effects of patient portability, which occur if patients switch health plans. Payers can also assess the sustainability of a product by employing real-world evidence, which can provide a better understanding of how outcomes accrue in the real world over time. Since 2010, manufacturers have implemented other mechanisms to support payers, including offering payment plans across different payers and establishing measures for their therapeutic products over a period of time to demonstrate clinical outcomes to payers.

Novel Therapy Affordability and Accessibility

Novel curative treatments like CAR-T have generated successful results among the Medicare population. Medicare will track quality and cost as the products are used in routine clinical settings. Subsequent treatment outcomes will drive coverage, reimbursement, and resulting patient access. The Centers for Medicare & Medicaid Services could expand access to curative therapies through greater adoption by the program’s various alternative payment models (APMs). Medicare Fee-for-Service (FFS) APMs can improve accessibility to curative therapies to disadvantaged populations. Medicare Advantage plans are a popular option among Medicare enrollees, and they can enter OBCs with manufacturers without government oversight, which allows for more flexibility when designing arrangements. Medicare has shown interest in designing APMs to spread risk among providers, and the program has the size, scope, and patient data to track downstream benefits that are often difficult to follow for curative treatments.

Importance of Data in Healthcare Decision-Making

While clinical trials are designed to illustrate causality in tightly controlled clinical settings, the data are less applicable to routine clinical practice because they do not provide a holistic picture of long-term benefits and risks or overall impact to the healthcare system and society in general. Given the presumed high prices of novel curative therapies, manufacturers must leverage real-world data to demonstrate sustained benefit and safety, materializing into positive clinical, economic, and humanistic (e.g., quality-of-life) outcomes beyond the short-term trial setting.

Patient portability or churn and a lack of longitudinal tracking for patients presents another challenge to collecting the real-world evidence that can demonstrate the true value of a curative therapy. Avalere has unique access to claims data, particularly robust in the Medicare FFS space. This comprehensive dataset allows Avalere to track patients over time despite patient churn and is currently being used to help stakeholders make modeling predictions on a product’s performance. This type of long-term analysis requires extensive, multi-year data collection and is essential to demonstrating value, however defined.

Access the archived version of the webinar.

To receive Avalere updates, connect with us.