Trial design and efficacy thresholds for granting breakthrough therapy designation in oncology

Journal of Oncology Practice

Volumn 12, Issue 8, 2016, Pages e810-e817

  Kern, Kenneth A ^a  

^a PFIZER INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLINATUMOMAB; CERITINIB; IBRUTINIB; IDELALISIB; NIVOLUMAB; OBINUTUZUMAB; OFATUMUMAB; PALBOCICLIB; PEMBROLIZUMAB; ANTINEOPLASTIC AGENT;

ARTICLE; BREAKTHROUGH THERAPY DESIGNATION; CANCER SURVIVAL; CANCER THERAPY; FOOD AND DRUG ADMINISTRATION; HEALTH CARE CONCEPTS; HEMATOLOGIC MALIGNANCY; HUMAN; MEDLINE; ONCOLOGY; PROGRESSION FREE SURVIVAL; SOLID TUMOR; STUDY DESIGN; TREATMENT RESPONSE; CLINICAL TRIAL (TOPIC); DRUG APPROVAL; METHODOLOGY; NEOPLASMS; TIME FACTOR; TREATMENT OUTCOME;

ANTINEOPLASTIC AGENTS; CLINICAL TRIALS AS TOPIC; DRUG APPROVAL; HUMANS; NEOPLASMS; RESEARCH DESIGN; TIME FACTORS; TREATMENT OUTCOME;

EID: 84981727011     PISSN: 15547477     EISSN: 1935469X     Source Type: Journal    
DOI: 10.1200/JOP.2016.012161     Document Type: Article

References (27)

1. Richey EA, Lyons EA, Nebeker JR, et al: Accelerated approval of cancer drugs: Improved access to therapeutic breakthroughs or early release of unsafe and ineffective drugs? J Clin Oncol 27:4398-4405, 2009
2. Sherman RE, Li J, Shapley S, et al: Expediting drug development: The FDA's new "breakthrough therapy" designation. N Engl J Med 369:1877-1880, 2013
3. DiMasi JA, Hansen RW, Grabowski HG: The price of innovation: New estimates of drug development costs. J Health Econ 22:151-185, 2003
4. DiMasi JA, Grabowski HG, Hansen RW: Innovation in the pharmaceutical industry: New estimates of R&D costs. J Health Econ 47:20-33, 2016
5. US Food and Drug Administration: Guidance for industry: Expedited programs for serious conditions-Drugs and biologics: CDER, CBER. http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm358301.pdf
6. Allison M: Can cancer clinical trials be fixed? Nat Biotechnol 29:13-15, 2011
7. US Government Publishing Officer: US Food and Drug Administration Safety and Innovation Act. http://www.gpo.gov/fdsys/pkg/BILLS-112s3187enr/pdf/BILLS-112s3187enr.pdf
8. Woodcock J: Drug development in serious diseases: The new "breakthrough therapy" designation. Clin Pharmacol Ther 95:483-485, 2014
9. Woodcock J: Breakthrough designation: The first two years. http://www.focr.org/breakthrough-therapy-designation-first-two-years-janet-woodcock
10. Bristol-Myers Squibb: Opdivo (nivolumab). http://www.accessdata.fda.gov/drugsatfda-docs/label/2015/125527s000lbl.pdf
11. GlaxoSmithKline: Arzerra (ofatumumab). http://www.accessdata.fda.gov/drugsatfda-docs/label/2014/125326s060lbl.pdf
12. Genentech: Gazyva (obinutuzumab). http://www.accessdata.fda.gov/drugsatfda-docs/label/2013/125486s000lbl.pdf
13. Merck: Keytruda (pembrolizumab). http://www.accessdata.fda.gov/drugsatfda-docs/label/2015/125514s003lbl.pdf
14. Pfizer Pharma: Ibrance (palbociclib). http://www.accessdata.fda.gov/drugsatfda-docs/label/2015/207103s000lbl.pdf
15. Pharmacyclics Pharma: Imbruvica (ibrutinib). http://www.accessdata.fda.gov/drugsatfda-docs/label/2015/205552s002lbl.pdf
16. Novartis Pharma: Zykadia (ceritinib). http://www.accessdata.fda.gov/drugsatfda-docs/label/2015/205755s003s004lbl.pdf
17. Gilead Sciences Pharma: Zydelig (idelalisib). http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm?fuseaction=Search.Overview&DrugName=ZYDELIG
18. Amgen Pharma: Blincyto (blinatumomab). http://www.accessdata.fda.gov/drugsatfda-docs/label/2014/125557lbl.pdf
19. Aggarwal SR: A survey of breakthrough therapy designations. Nat Biotechnol 32:323-330, 2014
20. Friends of Cancer Research: Breakthrough therapies. http://www.focr.org/breakthrough-therapies
21. US Food and Drug Administration: CDER breakthrough therapy designation requests. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/DrugandBiologicApprovalReports/INDActivityReports/ucm373559.htm
22. US Food and Drug Administration: CBER breakthrough therapy designation requests. http://www.fda.gov/regulatoryinformation/legislation/significantamendmentstothefdcact/fdasia/ucm406467.htm
23. US Food and Drug Administration: The CDER handbook. http://druganddevicelaw.net/CDER-handbook.pdf
24. American Association for Cancer Research: Pembrolizumab case study: Accelerating Anticancer Agent Development and Validation Workshop. May 7, 2015. Bethesda, MD. www.acceleratingworkshop.org
25. Jiang K: Caution urged over the FDA's new breakthrough designation. Nat Med 19:1196, 2013
26. Arnardottir AH, Haaijer-Ruskamp FM, Straus SMJ, et al: Additional safety risk to exceptionally approved drugs in Europe? Br J Clin Pharmacol 72:490-499, 2011
27. Center for Health Policy at Brookings: Breakthrough therapy designation: Exploring the qualifying criteria. http://www.brookings.edu/~/media/events/2015/04/24-fda-breakthrough-therapy-designation/breakthrough-therapy-designation-final.pdf