ICER updates methods for assessing potentially curative single, short-term therapies

The Institute for Clinical and Economic Review (ICER) announced that it has finalised a set of adaptations on how it assesses potential cures and other treatments that qualify as high-impact single or short-term therapies (SSTs). The changes will be launched by ICER with reviews starting in January 2020.

"Patients and caregivers are celebrating the first cell and gene therapies to reach the market, and they eagerly anticipate many additional one-time therapies that are poised for FDA review over the next few years," commented ICER president Steven Pearson. He added "we need to think hard about whether the methods of technology assessment and cost-effectiveness analysis are ready to capture the potential for broader benefits of these treatments."

Earlier this year, ICER disputed a Novartis claim that the company's as-yet unapproved Zolgensma (onasemnogene abeparvovec) gene therapy for spinal muscular atrophy would be cost-effective at $4 million to $5 million per patient, saying instead that it should carry a per-treatment cost of up to $1.5 million. When Zolegnsma was approved by the FDA in May, Novartis set the price at around $2.1 million for a single intravenous infusion.

ICER has also said that Spark Therapeutics' Luxturna (voretigene neparvovec), a one-time gene therapy for patients with rare inherited retinal disease, should cost about 75% less "in most cases" than the price of $850,000, or $425,000 per eye, that the company had proposed.

Under the updated methods adaptations, ICER said it will create two specific scenario analyses to reflect an "optimistic" and a "conservative" assumption regarding the benefit of SSTs under review. "These scenarios will test uncertainty regarding the duration of benefit, the magnitude or quality of the benefit, or the proportion of patients achieving clinical benefit over their lifetime," the group said.

Moreover, for all ICER reviews, including non-SSTs, the independent appraisal committees will also now vote on three additional domains of "potential other benefits or disadvantages." These include potential advantages for therapies that offer a new treatment choice or increase access to future therapies that may be approved over patients' lifetimes, as well as a potential disadvantage for therapies that could impair the effectiveness of future treatments.

ICER is also introducing two hypothetical "shared savings" scenarios that take into account how high-impact SSTs may result in "very large cost offsets" by preventing the need for expensive chronic treatments. Under the first scenario, 50% of the lifetime health system cost offsets from a new treatment will be assigned back to the health system. In the second, cost offsets will be assigned entirely to the new treatment, but capped at $150,000 per year. "These two informational scenarios...will not replace ICER's established approach for determining value-based price benchmarks," the group noted.

For related analysis, read ViewPoints: ICER's proactive-ness key to its growing influence, say experts.  

See also ViewPoints: Novartis' Zolgensma sets the new status quo and ViewPoints: Payers hopeful that Zolgensma may spark new thinking in gene therapy cost models.

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