Expedited Programs for Serious Conditions: An Update on Breakthrough Therapy Designation

Clinical Therapeutics

Volumn 37, Issue 9, 2015, Pages 2104-2120

  Kwok, Martin ^a,b   Foster, Theresa ^a,b   Steinberg, Michael ^b  

^a BIOGEN   (United States)

^b MASSACHUSETTS COLLEGE OF PHARMACY AND HEALTH SCIENCES   (United States)

Author keywords

Breakthrough Therapy Designation; BTD; CDER; drug development; FDA; NME

Indexed keywords

ARTICLE; BREAKTHROUGH THERAPY DESIGNATION; CLINICAL PHARMACOLOGY; DRUG APPROVAL; DRUG DEVELOPMENT; DRUG INDUSTRY; DRUG LEGISLATION; DRUG SCREENING; FOOD AND DRUG ADMINISTRATION; HEALTH CARE QUALITY; HUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); UNITED KINGDOM; LEGISLATION AND JURISPRUDENCE; TIME FACTOR; UNITED STATES;

DRUG APPROVAL; DRUG DISCOVERY; HUMANS; TIME FACTORS; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84941426631     PISSN: 01492918     EISSN: 1879114X     Source Type: Journal    
DOI: 10.1016/j.clinthera.2015.07.011     Document Type: Review

References (59)

1. Kesselheim A, Darrow J. FDA designations for therapeutics and their impact on drug development and regulatory review outcomes. Clin Pharmacol Ther. 2015;97: 29-36, http://dx.doi.org/10.1002/cpt.1.
2. US Food and Drug Administration. Center for Drug Evaluation and Research. (2014, April 25). FDA Drug Approval Process Infographic. http://www.fda.gov/Drugs/ResourcesForYou/Consumers/ucm295473.htm. Accessed March 19, 2015.
3. Administrative actions, 21 C.F.R. § 312.47 (2014).
4. Early consultation, 21 C.F.R. § 312.82. (2014).
5. US Food and Drug Administration. (2011, September 1). Proposed PDUFA V Reauthorization Performance Goals and Procedures; Fiscal Years 2013 through 2017. http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM270412.pdf. Accessed December 4, 2014.
6. Anonymous. New breakthrough therapy designation program aims to cut clinical trial time. Impact Report. 2014;15:1. http://csdd.tufts.edu/files/uploads/SummaryJanFebIR2014.pdf. Accessed March 29, 2015.
7. Darrow JJ, Avorn J, Kesselheim AS. New FDA breakthrough-drug category-implications for patients. N Engl J Med. 2014;370:1252-1258, http://dx.doi.org/10.1056/NEJMhle1311493.
8. US Food and Drug Administration. Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research. (2014, May). Guidance for Industry: Expedited Programs for Serious Conditions - Drugs and Biologics. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf. Accessed November 12, 2014.
9. Horning SJ, Haber DA, Selig WK, et al. Developing standards for breakthrough therapy designation in oncology. Clin Cancer Res. 2013; 19:4297-4304, http://dx.doi.org/10.1158/1078-0432.CCR-13-0523.
10. Conrad R, Harrington A, Stark G, Taylor K. Breakthrough therapy designation: Two and a half years in. [video] The Brookings Institution. April 24, 2015. http://www.brookings.edu/events/2015/04/24-fda-breakthrough-therapy-criteria. Accessed April 28, 2015.
11. Serebrov M. Breakthrough 'incredible tool,' but challenge for FDA resources. Bioworld, Thomson Reuters. April 24, 2015. http://www.bioworld.com/content/breakthroughincredible-tool-challenge-fda-resources-0. Accessed April 27, 2015.
12. US Food and Drug Administration. Center for Drug Evaluation and Research. (2014, January 31. Approved Drugs 2013. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DrugInnovation/UCM381803.pdf. Accessed March 19, 2015.
13. Aggarwal SR. A survey of breakthrough therapy designations. Nature Biotechnol. 2014;32:323-330.
14. US Food and Drug Administration. Center for Drug Evaluation and Research. (2014, July 29). Good Review Practice: Management of Breakthrough Therapy-Designated Drugs and Biologics. http://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ManualofPoliciesProcedures/UCM407009.pdf. Accessed November 12, 2014.
15. US Food and Drug Administration. Center for Drug Evaluation and Research. (2015, March 9). Good Review Practice: Review of Marketing Applications for Breakthrough Therapy-Designated Drugs and Biologics That are Receiving an Expedited Review. http://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ManualofPoliciesProcedures/UCM437281.pdf. Accessed March 19, 2015.
16. US Food and Drug Administration. Center for Drug Evaluation and Research. (2013, April 29). Good Review Practice: Good Review Management Principles and Practices for Effective IND Development and Review. http://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ManualofPoliciesProcedures/UCM349907.pdf. Accessed November 19, 2014.
17. Woodcock J. Drug development in serious diseases: the new "breakthrough therapy" designation. Clin Pharmacol Ther. 2014;95:483-485, http://dx.doi.org/10.1038/clpt.2014.23.
18. Jenkins J.K. US Foodand Drug Administration. Center for Drug Evaluation and Research. (2014, December 11). CDER New Drug Review: 2014 Update. http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ucm074833.htm. Accessed January 13, 2015.
19. Shah A. Breakthrough Therapy Designation: Bringing innovation swiftly to patients. Ther Innov Regul Sci. 2014;49:256-561.
20. US Food and Drug Administration. Center for Drug Evaluation and Research. (2015, February 5). New Drugs at FDA: CDER's new molecular entities and new therapeutic biological products. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm20025676.htm. Accessed March 6, 2015.
21. Subramanian R, Sheppard T, Rubin B, Kramer C. FDA's New Breakthrough Therapy Designation: What does it mean for Pricing and Market Access? Oncology Business Review. 2013;7(8)1-3. http://obroncology.com/obrgreen/article/FDAs-New-Breakthrough-Therapy-Designation. Accessed March 29, 2015.
22. Burns D.L. US Food and Drug Administration. Center for Biologics Evaluation and Research. (2014, October 29). Summary Basis for Regulatory Action: Trumenba. http://www.fda.gov/downloads/BiologicsBloodVaccines/Vaccines/ApprovedProducts/UCM424125.pdf. Accessed December 4, 2014.
23. Hoffmann-La Roche Ltd. (2014, October 16). FDA approves Esbriet (pirfenidone) for the treatment of idiopathic pulmonary fibrosis (IPF) in the United States. http://www.roche.com/media/store/releases/med-cor-2014-10-16b.htm. Accessed February 17, 2015.
24. Boehringer Ingelheim. "Boehringer Ingelheim's OFEV™ (nintedanib∗) approved by the FDA for the treatment of idiopathic pulmonary fibrosis" (2014, October 16). https://www.boehringer-ingelheim.com/news/news-releases/press-releases/2014/16-october-2014.html. Accessed February 17, 2015.
25. US Food and Drug Administration. (2014, October 29). First vaccine approved by FDA to prevent serogroup B Meningococcal disease. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm420998.htm. Accessed January 6, 2015.
26. Senior M. Drugs with breakthrough status charm investors. Nat Biotechnol. 2013;31:945-947, http://dx.doi.org/10.1038/nbt1113-945.
27. Sherman RE, Li J, Shapley S, et al. Expediting drug development-the FDA's new "breakthrough therapy" designation. N Engl J Med. 2013;369:1877-1880, http://dx.doi.org/10.1056/NEJM/p1311439.
28. Richardson, E. (2014, May 15). Health Policy Brief: Breakthrough Therapy Designation. Health Affairs. http://www.healthaffairs.org/healthpolicybriefs/brief.php?brief-id=115. Accessed March 29, 2015.
29. US Food and Drug Administration. (2015, March 10). Frequently Asked Questions: Breakthrough Therapies. http://www.fda.gov/regulatoryinformation/legislation/federalfooddrugandcosmeticactfdcact/significantamendmentstothefdcact/fdasia/ucm341027.htm. Accessed November 18, 2014.
30. Carroll, J. (2015, February 10). Breakthrough? Not anymore says FDA as Bristol-Meyers joins Merck in hep C's coach section. FierceBiotech. http://www.fiercebiotech.com/story/breakthrough-not-anymoresays-fda-bristol-myers-joins-merckhep-cs-coach-se/2015-02-10. Accessed March 29, 2015.
31. Pierson R. (2015, February 3). Merck says hepatitis C treatment to lose "breakthrough" status. Thompson Reuters. http://www.reuters.com/article/2015/02/04/merck-co-results-idUSL1N0VE0Q920150204. Accessed May 12, 2015.
32. Sutter S. (2015, May 4). Unsure about 'Breakthrough'-Worthiness? Talk to the FDA First. The Pink Sheet. Regulatory Update. https://www.pharmamedtechbi.com/publications/the-pink-sheet/77/18/unsure-about-breakthroughworthiness-talk-to-fda-first. Accessed May 8, 2015.
33. UK Department of Health and Office for Life Sciences. (2015, March 11). Review into medical innovation and technology: further details. https://www.gov.uk/government/news/review-into-medical-innovation-and-technology-further-details. Accessed March 24, 2015.
34. Sutter S. (2013, September 16). Will Breakthrough Therapies Face Reimbursement Challenges? The Pink Sheet. Regulatory Update. https://www.pharmamedtechbi.com/publications/the-pink-sheet/75/37/will-breakthrough-therapies-face-reimbursement-challenges. Accessed January 30, 2015.
35. Humer C. (2014, December 22). Express Scripts Drops Gilead hep C drugs for cheaper AbbVie rival. Thompson Reuters. http://www.reuters.com/article/2014/12/22/us-express-scripts-abbvie-hepatitisc-idUSKBN0K007620141222. Accessed March 20, 2015.
36. Davis, J. (2015, January 29). Hepatitis C Scorecard: Express Scripts Drafts Inferior Drug. MedpageToday. http://www.medpagetoday.com/InfectiousDisease/Hepatitis/49769. Accessed March 20, 2015.
37. US Food and Drug Administration. (2014, August 5). What We Do. http://www.fda.gov/AboutFDA/WhatWeDo/. Accessed May 12, 2015.
38. Diamond P.F. (2013, September 4). FDA Speeds Things Up: What's happened so far with breakthrough therapy designation? Genetic Engineering & Biotechnology News. http://www.genengnews.com/insight-and-intelligenceand153/fda-speeds-things-up/77899888/. Accessed March 24, 2015.
39. US Food and Drug Administration. (2015, March 21). FDA Hepatitis Update-Important Safety Information: Harvoni, and Sovaldi. http://content.govdelivery.com/accounts/USFDA/bulletins/f97c71. Accessed March 24, 2015.
40. Lacana E. US Food and Drug Administration. Office of Biotechnology Products. (2014). Expedited Programs: Regulatory and Quality Implications for Product Development. http://c.ymcdn.com/sites/www.casss.org/resource/resmgr/CMC-No-Am-Jan-Spkr-Slds/2014-CMCJan-AccDev-Lacana-Em.pdf. Accessed March 29, 2015.
41. Farrell AT. US Food and Drug Administration. Center for Biologics Evaluation and Research. (2013, October 25). Summary Review for Regulatory Action: Gazyva. http://www.accessdata.fda.gov/drugsatfda-docs/label/2013/125486Orig1s000SumR.pdf. Accessed April 8, 2015.
42. Janssen Research & Development, LLC. "Ibrutinib Receives Two Oncology Breakthrough Therapy Designations from U.S. Food and Drug Administration." (2013, February 12). http://www.investor.jnj.com/releasedetail.cfm?ReleaseID=739865. Accessed April 8, 2015.
43. GlaxoSmithKline. "FDA grants GSK and Genmab's Arzerras® (ofatumumab) Breakthrough Therapy designation for previously untreated chronic lymphocytic leukaemia." (2013, September 13). http:/r/us.gsk.com/en-us/media/press-releases/2013/fda-grants-gsk-and-genmabs-arzerrarandnbspofatumumab-break through-therapy-designation-for-previously-untreated-chronic-lymphocytic-leukaemia/. Accessed April 8, 2015.
44. Keegan P.US Food and Drug Administration. Center for Drug Evaluation and Research. (2014, April 1). Division Director Summary: Zykadia. http://www.accessdata.fda.gov/drugsatfda-docs/nda/2014/205755Orig1s000SumR.pdf. Accessed April 8, 2015.
45. Gilead Sciences, Inc. U.S. Food and Drug Administration Approves Gilead's Zydelig® (idelalisib) for Relapsed Chronic Lymphocytic Leukemia, Follicular Lymphoma and Small Lymphocytic Lymphoma. (2014, July 23). http://www.gilead.com/news/press-releases/2014/7/us-food-and-drug-administration-approves-gileads-zydelig-idelalisib-for-relapsed-chronic-lymphocytic-leukemia-follicular-lymphoma-and-small-lymphocytic-lymphoma. Accessed April 8, 2015.
46. Theoret M. US Food and Drug Administration. Center for Drug Evaluation and Research. (2014, September 3). Cross-Discipline Team Leader Review: Keytruda. http://www.accessdata.fda.gov/drugsatfda-docs/nda/2014/125514Orig1s000CrossR.pdf. Accessed April 8, 2015.
47. Baird AC, Kolibab K, United States. Food and Drug Administration. Center for Drug Evaluation and Research. (2014, May). Other Review(s): Blincyto. http://www.accessdata.fda.gov/drugsatfda-docs/nda/2014/125557Orig1s000OtherR.pdf. Accessed April 8, 2015.
48. Chuk M and Hazarika M, Theoret M. US Food and Drug Administration. Center for Drug Evaluation and Research. (2014, September 22). Clinical Review: Opdivo. http://www.accessdata.fda.gov/drugsatfda-docs/nda/2014/125554Orig1s000MedR.pdf. Accessed April 8, 2015.
49. Ibrahim A. US Food and Drug Administration. Center for Drug Evaluation and Research. (2015, February 3). Summary Review for Regulatory Action: Ibrance. http://www.accessdata.fda.gov/drugsatfda-docs/nda/2015/207103Orig1s000SumR.pdf. Accessed April 8, 2015.
50. Vertex Pharmaceuticals Incorporated. "Vertex Outlines Corporate Strategy and Defines Key 2013 Business Priorities." (2013, January 6). http://investors.vrtx.com/releasedetail.cfm?ReleaseID=731630. Accessed April 8, 2015.
51. Drugs.com: Esbriet. (2014, July 17). http://www.drugs.com/nda/esbriet-140717.html. Accessed April 8, 2015.
52. Drugs.com: Ofev. (2014, July 16). http://www.drugs.com/nda/nintedanib-140716.html. Accessed April 8, 2015.
53. Birnkrant D. US Food and Drug Administration. Center for Drug Evaluation and Research. (2013, November 27). Summary Review for Regulatory Action: Sovaldi. http://www.accessdata.fda.gov/drugsatfda-docs/nda/2013/204671Orig1s000 SumR.pdf. Accessed April 8, 2015.
54. Birnkrant D. US Food and Drug Administration. Center for Drug Evaluation and Research. (2014, September 23). Summary Review for Regulatory Action: Harvoni. http://www.accessdata.fda.gov/drugsatfda-docs/nda/2014/205834Orig1s000SumR.pdf. Accessed April 8, 2015.
55. Murray JS. US Food and Drug Administration. Center for Drug Evaluation and Research. (2014, December 8). Summary Review for Regulatory Action: Viekira Pak http://www.accessdata.fda.gov/drugsatfda-docs/nda/2014/206619Orig1s000SumR.pdf. Accessed April 8, 2015.
56. Bash M.C. US Food and Drug Administration. Center for Biologics Evaluation and Research. (2015, January 23). Summary Basis for Regulatory Action: Bexsero. http://www.fda.gov/downloads/BiologicsBloodVaccines/Vaccines/ApprovedProducts/UCM434748.pdf. Accessed April 8, 2015.
57. GlaxoSmithKline. "GSK gains FDA Breakthrough Therapy designation for Promacta®/Revolade® (eltrombopag) for severe aplastic anemia." (2014, February 3). http://www.gsk.com/en-gb/media/press-releases/2014/gsk-gains-fda-breakthrough-therapy-designation-for-promactarevolade-eltrombopag-for-severe-aplastic-anaemia/. Accessed April 8, 2015.
58. Terry M. BioSpace. "FDA Breakthrough Status For Genentech (RHHBY)'s Lucentis. (2014, December 16). http://www.biospace.com/News/fda-breakthrough-status-forgenentechs-lucentis/358319. Accessed April 8, 2015.
59. Regeneron. EYLEA® (aflibercept) Injection Receives FDA Breakthrough Therapy Designation for Diabetic Retinopathy in Patients with Diabetic Macular Edema. (2014, September 16). http://investor.regeneron.com/releasedetail.cfm?ReleaseID=871009. Accessed April 8, 2015.