Questions Raised About “Breakthrough” Therapies’ Clinical Support

Compared to other drugs, therapeutics given breakthrough status receive FDA approval on the basis of weaker trial evidence, a study finds.

The US Food and Drug Administration’s “breakthrough therapy” designation has helped expedite the development and approval of more than 45 therapeutics in the last 5 years. But a study published in JAMA today (July 17) suggests that these interventions gain Food and Drug Administration (FDA) approval on the basis of weaker clinical evidence than those that don’t have the breakthrough designation do—a finding that contrasts with public optimism about these drugs’ effectiveness.

“Our research suggests that FDA approval of these breakthrough therapies is generally based on shorter and smaller clinical trials than those that support FDA approval of non-breakthrough therapy drugs,” study coauthor Joseph Ross of Yale University School of Medicine writes in an email to CNN. “When approvals are based on shorter and smaller clinical trials there is greater uncertainty at the time of approval.”

Breakthrough therapy status was introduced by the FDA in 2012 with the purpose of speeding up the review of therapeutics designed to treat especially serious medical conditions—particularly those for which no effective treatments are already available. Drugs that gained approval this way include Clovis Oncology’s rucaparib (Rubraca) for the treatment of ovarian cancer and Bristol-Myers Squibb’s nivolumab (Opdivo) for Hodgkin lymphoma.

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