New FDA Breakthrough-Drug Category—Implications for Patients

Created by Congress in 2012, the FDA’s breakthrough therapy designation was intended to expedite approval of drugs showing “exceptional results for patients,” yet in its first year the FDA received 92 applications, far exceeding expectations of approximately 2 per year, and approved 27 designations, raising concerns about a potential surge in drugs approved on the basis of limited clinical data. The authors trace the history of prior expedited-access programs and examine the clinical, ethical, and regulatory implications of this latest pathway, noting the dangers illustrated by cases like ponatinib, which was briefly suspended after accelerated approval when serious thromboembolic events emerged in up to 48% of patients.