title: Accelerated approval and breakthrough therapy designation: oncology drug development on speed?
abstract: Recent advances in biotechnology have led to discoveries resulting in major improvements in the therapy of refractory malignancies, although most advanced cancers remain incurable. Thus, there is global consensus around the need to streamline the drug approval process for effective agents. Accelerated Approval and Breakthrough Therapy Designation hold the promise of making new treatments available sooner through the use of smaller studies using intermediate endpoints. Here, we consider the inherent limitations of smaller studies and discuss the strategies for hastening oncology drug development while maintaining high-efficacy standards.
authors.affiliation: Department of Gastrointestinal Medical Oncology, Investigational Cancer Therapeutics, and Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, Texas 77030, USA. jyao@mdanderson.org
authors.forename: James C
authors.initials: JC
authors.lastname: Yao
authors.forename: Funda
authors.initials: F
authors.lastname: Meric-Bernstam
authors.forename: J Jack
authors.initials: JJ
authors.lastname: Lee
authors.forename: S Gail
authors.initials: SG
authors.lastname: Eckhardt
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use; D017277:Drug Approval / Q000331:legislation & jurisprudence; D006801:Humans; D008495:Medical Oncology / Q000592:standards; D009369:Neoplasms / Q000188:drug therapy; D012107:Research Design; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D000970:Antineoplastic Agents