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Evolving cost-effectiveness assessment as gene therapy care evolves

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By Jeremy Schafer, PharmD, MBA, SVP Precision for Value

The potential for gene therapy to revolutionize the treatment of genetic disease cannot be understated. In hemophilia, an ongoing study at the 3-year mark found that gene therapy resulted in a 96% reduction in factor replacement use. Zolgensma, approved for spinal muscular atrophy, is off to a strong start with approximately 100 infants treated to date. However, the revolution comes at a cost. Zolgensma is the most expensive drug in the world at $2.1 million. For payers, managing the upfront cost is a challenge, but the specter of the unknown durability of effect is perhaps most daunting. Will payers need to pay for the treatment a second time? Will chronic therapy creep back if the gene therapy effect wanes? Such unanswered questions require flexibility not only in how gene therapy is paid for but also in how the cost-effectiveness of these drugs is measured.

The Institute for Clinical and Economic Review (ICER) has garnered significant attention for its cost-effectiveness reviews of emerging therapies. ICER has conducted reviews of multiple products in the gene and cell therapy space, including Kymriah, Yescarta, Zolgensma, and Luxturna. After 1 year of stakeholder feedback and deliberation, ICER released an updated framework specific to gene and cell therapy. Titled the Adapted Value Assessment Methods for High-Impact 'Single and Short-Term Therapies' (SSTs), the updated framework contains several important differences compared with ICER’s standard framework. First, the adapted framework includes both “optimistic” and “conservative” scenarios that attempt to accommodate the unknowns of SSTs, including duration of effect and ability to cease chronic therapy. Second, when the SST price is known, a threshold analysis will be done to determine how many years of benefit would be needed to achieve at least a $150,000/QALY result. Finally, the framework will support multiple shared savings scenarios when considering price points.

A survey involving 20 unique health plan respondents found that “durability of response” was the most needed information for payers regarding gene therapy. Payer concerns revealed in the same survey included the potential need for retreatment and continued use of chronic therapy. The SST framework cannot answer these clinical questions, but it can help payers understand cost-effective targets when negotiating outcomes-based arrangements with the manufacturers of gene and cell therapies. Payers could push for annuity payments based on cost-effective thresholds or include “claw back” clauses, which would refund a set value if treatment effect dissipates.

Manufacturers would be wise to review the SST framework. Manufacturers of gene and cell therapies should have a long-term evidence plan that gathers patient outcomes data at regular intervals. With each interval, the manufacturer should consider submitting the data to ICER and request a reevaluation to adjust the value price. Doing so may help manufacturers of efficacious therapies to extract additional value based on longer-term experience.

With hundreds of gene and cell therapies in the pipeline, payers and manufacturers need to explore new models to pay for treatment. The challenge of limited data on durability and degree of effect will remain, particularly for newly released agents. However, patients afflicted with genetic disease have waited long enough. ICER’s SST framework provides an opportunity for payers and manufacturers to negotiate on pricing and payment terms to open cost-effective access while the science tries to catch up.

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