The US Food and Drug Administration’s expedited approval programs: Addressing premarket flexibility with enhanced postmarket evidence generation

Wallach JD Ross JS Naci H . The US Food and Drug Administration’s expedited approval programs: Evidentiary standards, regulatory trade-offs, and potential improvements. Clin Trials 2018; 3: 219–229.

Califf R . Expedited and facilitated drug evaluations and evidence of benefit and risk: the cup is half-full. Clin Trials1 2018; 3: 235–239.

Downing NS Aminawung JA Shah ND et al . Clinical trial evidence supporting FDA approval of novel therapeutic agents, 2005–2012. JAMA 2014; 311: 368–377.

Psaty BM Meslin EM Breckenridge A . A lifecycle approach to the evaluation of FDA approval methods and regulatory actions: opportunities provided by a new IOM report. JAMA 2012; 307: 2491–2492.

Naci H Smalley KR Kesselheim AS . Characteristics of preapproval and postapproval studies for drugs granted accelerated approval by the US Food and Drug Administration. JAMA 2017; 318: 626–636.

Naci H Wouters OJ Gupta R et al . Timing and characteristics of cumulative evidence available on novel therapeutic agents receiving Food and Drug Administration accelerated approval. Milbank Q 2017; 95: 261–290.

Pease AM Krumholz HM Downing NS et al . Postapproval studies of drugs initially approved by the FDA on the basis of limited evidence: systematic review. BMJ 2017; 357: j1680.

Woodcock J . Expediting drug development for serious illness: trade-offs between patient access and certainty. Clin Trials 2018; 3: 230–234.

Beaver JA Howie LJ Pelosof L et al . A 25-year experience of US Food and Drug Administration accelerated approval of malignant hematology and oncology drugs and biologics: a review. JAMA Oncol. Epub ahead of print 1 March 2018. DOI: 10.1001/jamaoncol.2017.5618.

Woloshin S Schwartz LM White B et al . The fate of FDA postapproval studies. N Engl J Med 2017; 377: 1114–1117.

Fain K Daubresse M Alexander GC . The Food and Drug Administration Amendments Act and postmarketing commitments. JAMA 2013; 310: 202–204.

Ciani O Buyse M Drummond M et al . Time to review the role of surrogate end points in health policy: state of the art and the way forward. Value Health 2017; 20: 487–495.

Schilsky R . Access versus evidence: the regulators’ dilemma. Clin Trials 2018; 3: 240–242.

Califf RM Robb MA Bindman AB et al . Transforming evidence generation to support health and health care decisions. N Engl J Med 2016; 375: 2395–2400.

Kesselheim AS Wang B Franklin JM et al . Trends in utilization of FDA expedited drug development and approval programs, 1987–2014: cohort study. BMJ 2015; 351: h4633.

Chary KV . Expedited drug review process: fast, but flawed. J Pharmacol Pharmacother 2016; 7: 57–61.

US Food and Drug Administration (FDA). Guidance for industry: fast track drug development programs – designation, development, and application review, https://www.fda.gov/ohrms/dockets/98fr/980813gd.pdf, September 1998 (accessed 10 March 2018). Note: the document has since been removed, but is available on request

US Food and Drug Administration. Guidance for industry: expedited programs for serious conditions-drugs and biologics, May 2014, www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm358301.pdf (accessed 10 March 2018).

Schick A Miller KL Lanthier M et al . Evaluation of pre-marketing factors to predict post-marketing boxed warnings and safety withdrawals. Drug Saf 2017; 40: 497–503.

Mostaghim SR Gagne JJ Kesselheim AS . Safety related label changes for new drugs after approval in the US through expedited regulatory pathways: retrospective cohort study. BMJ 2017; 358: j3837.

Downing NS Shah ND Aminawung JA et al . Postmarket safety events among novel therapeutics approved by the US Food and Drug Administration between 2001 and 2010. JAMA 2017; 317: 1854–1863.

Pinnow E Amr S Bentzen SM et al . Postmarket safety outcomes for new molecular entity (NME) drugs approved by the Food and Drug Administration between 2002 and 2014. Clin Pharmacol Ther. Epub ahead of print 20 December 2017. DOI: 10.1002/cpt.944.

Wallach JD Ross JS . Gabapentin approvals, off-label use, and lessons for postmarketing evaluation efforts. JAMA 2018; 319: 776–778.