Editorial Ponatinib: Accelerated Disapproval

Disclosures of potential conflicts of interest may be found at the end of this article. Drug development for life-threatening diseases is still a pro-cess in evolution. In oncology, as drugs have become more targeted, the regulatory path to drug approval has short-ened, with increasing reliance on the Accelerated Approval pathway to provide early access to active agents. However, this approach is not without risks, as illustrated by the recent experience with ponatinib, a promising agent in chronic myeloid leukemia (CML). In 1992, responding to the need for rapid access to anti-HIV medication, Congress passed legislation creating a new pathway for early drug approval, called “Accelerated Approval” (AA). From its inception, this approval was considered con-ditional because it relied on a surrogate endpoint, such as response rate, that was considered “reasonably likely t