Before and during the first few years of cell and gene therapy (CGT) launches, payer expectations for this market centered on curative one-time treatments, substantial financial impact with low predictability, and a rapidly expanding pipeline of therapies. In 2016, payers were bracing for the onset of single-administration CGT launches. In 2017, tisagenlecleucel (Kymriah) was the first to enter the market, a CGT for the treatment of acute lymphoblastic leukemia in children and young adults up to age 25.
The emergence of a novel group of therapies was perceived by many stakeholders to be a market-shaping event that would lead to substantial healthcare spending, driven by a critical mass of CGT launches as part of a paradigm-shifting tsunami. Actual market experience has been considerably more complex. Available market experience pointed to factors such as clinical safety and durability concerns, lack of uniformity in utilization, and manufacturing constraints. The actual experience also included fewer-than-anticipated approvals from the Food and Drug Administration, and slower-than-expected product uptake. All of these issues have reshaped how payers and employers evaluate CGT risk. For payers and health plan sponsors, the divergence from expectations underscores the need for more nuanced, data-driven forecasting approaches and flexible risk-management strategies as the CGT market continues to evolve.
Ten years later, market experience has revealed that, of all the assumptions, the only one that materialized as expected was the high cost and low predictability. Understanding why the CGT tsunami did not occur is essential for plan sponsors and other payers in evaluating future exposure.
In this paper, we discuss a forecasting approach that leverages the divergence of actual experience from early payer expectations. We cover the following key discussion points.
- CGT market dynamics: lessons for payer forecasting
- The actual experience: three factors regarding early expectations of CGT uptake
- Predicting CGT uptake: limitations of claims-based analysis
- Modeling CGT uptake: using manufacturer revenue
- Case study No. 1: Zolgensma—a blockbuster in decline
- Case study No. 2: Elevidys—a picture of volatility
- Beyond the claims: Forecasting CGT event risk based on market factors