A 25-year experience of us food and drug administration accelerated approval of malignant hematology and oncology drugs and biologics a review

JAMA Oncology

Volumn 4, Issue 6, 2018, Pages 849-856

  Beaver, Julia A ^a   Howie, Lynn J ^a   Pelosof, Lorraine ^a   Kim, Tamy ^a   Liu, Jinzhong ^a   Goldberg, Kirsten B ^a   Sridhara, Rajeshwari ^a   Blumenthal, Gideon M ^a   Farrell, Ann T ^a   Keegan, Patricia ^a   Pazdur, Richard ^a   Kluetz, Paul G ^a  

^a CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTINEOPLASTIC AGENT; BIOLOGICAL PRODUCT; BIOLOGICAL MARKER; NEW DRUG;

ACCELERATED DRUG APPROVAL; CANCER CHEMOTHERAPY; DISEASE FREE SURVIVAL; DRUG APPROVAL; DRUG EFFICACY; DRUG INDICATION; DRUG MARKETING; DRUG SAFETY; FOOD AND DRUG ADMINISTRATION; HEMATOLOGIC MALIGNANCY; HUMAN; POSTMARKETING SURVEILLANCE; PROGRESSION FREE SURVIVAL; RECURRENCE FREE SURVIVAL; REVIEW; BIOASSAY; CLINICAL TRIAL (TOPIC); FACTUAL DATABASE; HEMATOLOGIC DISEASE; HISTORY; NEOPLASM; TREATMENT OUTCOME; UNITED STATES;

ANTINEOPLASTIC AGENTS; BIOLOGICAL PRODUCTS; BIOMARKERS; CLINICAL TRIALS AS TOPIC; DATABASES, FACTUAL; DRUG APPROVAL; DRUGS, INVESTIGATIONAL; ENDPOINT DETERMINATION; HEMATOLOGIC DISEASES; HISTORY, 20TH CENTURY; HISTORY, 21ST CENTURY; HUMANS; NEOPLASMS; PRODUCT SURVEILLANCE, POSTMARKETING; TREATMENT OUTCOME; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 85048201222     PISSN: 23742437     EISSN: 23742445     Source Type: Journal    
DOI: 10.1001/jamaoncol.2017.5618     Document Type: Review

References (21)

1. Code of Federal Regulations. 21-314.126.https://www.ecfr.gov/cgi-bin/text-idx?SID=8eea61dec0ef9b17f1fa0d0d3a7736c6&mc=true&node=se21.5.314-1126&rgn=div8. March 4, 2002. Accessed September 13, 2017.
2. Code of Federal Regulations. 314.510. https://www.ecfr.gov/cgi-bin/text-idx?SID=7e3afa31ba9326a86c1c08ca7cb22f4c&mc=true&node=se21.5.314-1510&rgn=div8. December 11, 1992. Accessed September 13, 2017.
3. Code of Federal Regulations. 601.41.https://www.ecfr.gov/cgi-bin/text-idx?SID=c03234aa22abbed0380923a4cc6ab001&mc=true&node=se21.7.601-141&rgn=div8. December 11, 1992. Accessed September 13, 2017.
4. Food and Drug Administration Safety and Innovation Act, 21 USC ?301. https://www.gpo.gov/fdsys/pkg/PLAW-112publ144/pdf/PLAW-112publ144.pdf. July 9, 2012. Accessed September 13, 2017.
5. US Food and Drug Administration. Guidance for Industry: Expedited Programs for Serious Conditions Drugs and Biologics. May 2014. http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm358301.pdf. Accessed September 13, 2017.
6. US Food and Drug Administration. Center for Drug Evaluation and Research, Oncologic Drugs Advisory Committee (ODAC) [meeting transcript]. February 8, 2011. https://wayback.archive-it.org/7993/20170404153747/https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/OncologicDrugsAdvisoryCommittee/UCM245644.pdf.
7. Dagher R, Johnson J, Williams G, Keegan P, Pazdur R. Accelerated approval of oncology products: A decade of experience. J Natl Cancer Inst. 2004;96(20):1500-1509.
8. Johnson JR, Ning YM, Farrell A, Justice R, Keegan P, Pazdur R. Accelerated approval of oncology products: the Food and Drug Administration experience. J Natl Cancer Inst. 2011; 103(8):636-644.
9. US Food and Drug Administration. Drugs@FDA: FDA approved drug products. http://www.accessdata.fda.gov/scripts/cder/daf/. Accessed March 15, 2017.
10. Johnson JR, Williams G, Pazdur R. End points and United States Food and Drug Administration approval of oncology drugs. J Clin Oncol. 2003;21 (7):1404-1411.
11. 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(7):626-636.
12. Kesselheim AS, Woloshin S, Eddings W, Franklin JM, Ross KM, Schwartz LM. Physicians' knowledge about FDA approval standards and perceptions of the "breakthrough therapy" designation. JAMA. 2016;315(14):1516-1518.
13. Schwartz LM, Woloshin S. Communicating uncertainties about prescription drugs to the public: A national randomized trial. Arch Intern Med. 2011;171(16):1463-1468.
14. US Food and Drug Administration. Guidance for Industry: Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics. May 2007. https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm071590.pdf. Accessed September 13, 2017.
15. Blumenthal GM, Kluetz PG, Schneider J, Goldberg KB, McKee AE, Pazdur R. Oncology drug approvals: evaluating endpoints and evidence in an era of breakthrough therapies. Oncologist. 2017;22 (7):762-767.
16. Blumenthal GM, Pazdur R. Response rate as an approval end point in oncology: back to the future. JAMA Oncol. 2016;2(6):780-781.
17. Califf RM. Balancing the need for access with the imperative for empirical evidence of benefit and risk. JAMA. 2017;318(7):614-616.
18. Kazandjian D, Blumenthal GM, Chen HY, et al. FDA approval summary: crizotinib for the treatment of metastatic non-small cell lung cancer with anaplastic lymphoma kinase rearrangements. Oncologist. 2014;19(10):e5-e11.
19. Kluetz PG, Chingos DT, Basch EM, Mitchell SA. Patient-reported outcomes in cancer clinical trials: measuring symptomatic adverse events with the National Cancer Institute's patient-reported outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE). Am Soc Clin Oncol Educ Book. 2016;35:67-73.
20. Kluetz PG, Papadopoulos EJ, Johnson LL, et al. Focusing on core patient-reported outcomes in cancer clinical trials: response. Clin Cancer Res. 2016;22(22):5618.
21. FDA approves Mylotarg for treatment of acute myeloid leukemia [press release]. Silver Spring, MD: US Food and Drug Administration; September 1, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574507.htm. Accessed September 25, 2017.