Study: Breakthrough Therapies Can be Cost-Effective


Drugs approved with an FDA breakthrough designation can provide value that offsets their higher costs, finds study conducted by Tufts Center for the Evaluation of Value and Risk in Health.

Drugs approved with the FDA’s breakthrough therapy designation provide greater health gains and are more cost-effective than non-breakthrough therapies, according to new research published in the Journal of Managed Care + Specialty Pharmacy.

Investigators in this study, which was conducted by Tufts Center for the Evaluation of Value and Risk in Health, found that incremental costs and health gains — quality-adjusted-life-year (QALY) — were higher for therapies designated as breakthrough compared with those that didn’t have the designation.

Additionally, breakthrough therapies had more favorable cost-effectiveness ratios and median value of $38,000/QALY compared with $50,000/QALY for non-breakthrough products. Among therapies with the breakthrough designation, products to treat hepatitis C had the most favorable cost-effectiveness ratios. Investigators said this may be because of the curative nature of these therapies.

“Our analysis of available CEAs [cost-effectiveness analyses] showed that breakthrough therapies appear to confer incremental health gains, as compared with previously reported median QALY gains,” investigators wrote. “We also found that CE depends on a study’s comparator type. Analyses comparing drugs with ‘best supportive care’ reported greater incremental costs and QALY gains than analyses comparing drugs with active treatments. As expected, these larger cost and health gain differences reflect the tendency of best supportive care to be cheaper and less effective than an active treatment.”

Natalia Olchanski, Ph.D.

Natalia Olchanski, Ph.D.

Investigators, led by Natalia Olchanski, Ph.D., analyzed the cost-effectiveness of products with breakthrough therapy designation compared with the cost-effectiveness of those without the designation. Olchanski is with the Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies at Tufts Medical Center in Boston. Investigators also measured cost-effectiveness per quality-adjusted-life-year and aimed to identify factors associated with cost-effectiveness using a willingness to pay benchmark.

Investigators used the Tufts Medical Center Cost-Effectiveness Analysis Registry, a database of more than 8,000 cost-utility analyses on a wide variety of diseases and treatments, published from 1976 to 2020.

They assessed drugs approved between 2013 and 2018. During that time, the FDA approved 279 drugs, designating 83 (32%) as breakthrough therapies. Investigators identified published cost-effectiveness studies for 26% of drugs with breakthrough designation (48 studies, 227 cost-effectiveness ratios) and 23% of those without the designation (60 studies, 96 cost-effectiveness ratios).

Investigators calculated mean and median incremental costs and QALY gains for products with and without breakthrough designation. They calculated the mean cost-effectiveness ratios, restricting attention to drugs that improve health but increase care costs, relative to their comparators. They also calculated the proportion of interventions that improve health and reduce costs, and the proportion that improve health, regardless of cost. They used recognized U.S. value benchmarks of $50,000/QALY, $100,000/QALY, $150,000/QALY.

Breakthrough therapies had lower incremental costs and higher incremental QALYs gained, resulting in more favorable cost-effectiveness ratios, compared with those drugs that didn’t have a breakthrough designation. Overall breakthrough therapies had lower, ie, more favorable cost-effectiveness ratios with larger QALY gains. On average, breakthrough therapies had lower incremental costs (means of $31,835 vs $66,282), but they had higher median incremental costs.

Investigators indicated that one limitation was the analysis evaluated a small of newly approved drugs. Cost-effectiveness analyses were not available for many of the therapies. Additionally, they found that cost-effectiveness values used depended on the comparators incorporated into the studies they analyzed.

This research was sponsored by Genentech.

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