Faster Access to Drugs for Serious or Life-Threatening Illnesses Through use of the Accelerated Approval Regulation in the United States

  • David M. Cocchetto
  • Douglas R. Jones


The Food and Drug Administration issued a final regulation on accelerated approval for human drugs, antibiotics, and biologics on December 11, 1992. This regulation provided a formal regulatory mechanism for a sponsor to seek accelerated approval by submitting a New Drug Application for a drug intended to treat a serious or life-threatening illness. In order to qualify for accelerated approval, a drug must seek an indication for an illness having either a surrogate endpoint which is reasonably likely to predict clinical benefit or a reliable clinical endpoint other than survival or irreversible morbidity. This important and much needed regulation has been used to achieve accelerated approval of several antiretroviral and oncology drugs, as well as several other drugs and biologics. Since this regulation has been in place for almost five years, the purpose of this paper is to summarize all of the drugs that have utilized this regulatory mechanism to attain approval in the United States. From 1992 through the end of July 1997, a total of 20 drugs have received accelerated approval. This summary of these drugs shows that this regulation has been a successful means of providing more rapid regulatory review and approval for drugs intended to treat a serious or life-threatening illness. Regulatory affairs professionals should be familiar with the precedents for approval of drugs under this regulation, both to enable them to follow well-trodden paths and to consider extending the experience to drugs for other indications under appropriate circumstances.

Key Words

Accelerated approval Regulatory affairs Serious or life-threatening illnesses United States 


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  1. 1.
    Food and Drug Administration. Drug Administration. New Drug, Antibiotic, Biological Drug Product Regulations; Accelerated Approval. Proposed rule. Federal Register. April 15, 1992;57:13234–13242.Google Scholar
  2. 2.
    Food and Drug Administration. Drug Administration. New Drug, Antibiotic, Biological Drug Product Regulations; Accelerated Approval. Final rule. Federal Register. December 11, 1992;57:58942–58960.Google Scholar
  3. 3.
    Kessler DA. Issues in approving drugs for AIDS treatment. Regul Aff. 1994;6:189–200.Google Scholar
  4. 4.
    Kessler DA. Feiden KL. Faster evaluation of vital drugs. Scientif Am. March 1995;272 (No. 3):48–54.CrossRefGoogle Scholar
  5. 5.
    Klein R. FDA s role in the treatment of HIV/AIDS. Regul Aff. 1994;6:221–227.Google Scholar
  6. 6.
    Shulman SR. Brown JS. The Food and Drug Administration’s early access and fast-track approval initiatives: how have they worked? Food Drug Law J. 1995;50:503–531.PubMedGoogle Scholar
  7. 7.
    President Bill Clinton, Vice President Al Gore. Reinventing the Regulation of Cancer Drugs. Accelerating Approval and Expanding Access. National Performance Review. March 1996.Google Scholar
  8. 8.
    Glaxo Wellcome Epivir AIDS therapy review complete in four-and-a-half month accelerated approval process; product is first-line for use with AZT. F-D-C Reports—The Pink Sheet, November 27, 1995;6–7.Google Scholar
  9. 9.
    oche Invirase/Hivid combination reduces mortality risk by 68%. F-D-C Reports—The Pink Sheet. October 7, 1996;T&G-5.Google Scholar
  10. 10.
    Agouron Viracept to be compared to Norvir in clinical endpoint study. F-D-C Reports—The Pink Sheet. March 17, 1997;T&G-1.Google Scholar
  11. 11.
    Pharmacia & Upjohn Rescriptor 15.1% rebate available to AIDS Drug Assistance Programs; clinical endpoint trials of combination with proteases planned. F-D-C Reports—The Pink Sheet. April 14, 1997;11–12.Google Scholar
  12. 12.
    Rhone-Poulenc Rorer Taxotere lower doses examined in ongoing studies; FDA committee recommends approval for anthracycline-resistant breast cancer. F-D-C Reports—The Pink Sheet. October 23, 1995;11–12.Google Scholar
  13. 13.
    RPR Taxotere June launch likely following accelerated approval May 14; firm to submit studies of Taxotere v. Bristol’s Taxol, doxorubicin i. breast cancer. F-D-C Reports—The Pink Sheet. May 20, 1996:3–5.Google Scholar
  14. 14.
    US Bioscience’s Ethyol. F-D-C Reports—The Pink Sheet. March 25, 19%.Google Scholar
  15. 15.
    US Bioscience Ethyol to be marketed in US by Alza via $35 mil deal. F-D-C Reports—The Pink Sheet. December 18, 1995;T&G-2 to T&G-3.Google Scholar
  16. 16.
    Zeneca Casodex for advanced prostate cancer receives accelerated approval from FDA; agency awaits completion of Eulexin comparison for full approval. F-D-C Reports—The Pink Sheet. October 9, 1995;7.Google Scholar
  17. 17.
    Pharmacia & Upjohn Camptosar for colorectal cancer to be available within six weeks; data to support full approval to be submitted to FDA in two years. F-D-C Reports—The Pink Sheet. June 24, 1996;7–8.Google Scholar
  18. 18.
    Sequus Doxil for AIDS-related Kaposi’s sarcoma gains accelerated approval Nov. 17; studies in breast, ovarian and liver solid tumors are under way. F-D-C Reports—The Pink Sheet. November 27, 1995;7–8.Google Scholar
  19. 19.
    Pharmacia’s Zinecard full approval depends on Phase IV breast cancer study; cardioprotector must show delayed progression or survival benefit. F-D-C Reports—The Pink Sheet. June 5, 1995;6–7.Google Scholar
  20. 20.
    Abbott Biaxin for MAC recommended for accelerated approval as lead agent in combination therapy by FDA Antiviral Drugs Advisory Committee May 10. F-D-C Reports—The Pink Sheet. May 17, 1993; 10–11.Google Scholar
  21. 21.
    Abbott Biaxin for MAC therapy has $1,800 annual cost. F-D-C Reports—The Pink Sheet. January 3, 1994;T&G-1 to T&G-2.Google Scholar
  22. 22.
    Pulmozyme development may be prototype for other biotech products; Genentech declined to skip Phase III or rush PLA filing; Roche financial security helped. F-D-C Reports—The Pink Sheet. January 3, 1994;8–10.Google Scholar
  23. 23.
    Milestones in Betaseron approval process. Food and Drug Administration. News Release P93-31; July 23, 1993.Google Scholar
  24. 24.
    Serono Serostim hGH approved Aug. 23 for AIDS wasting. F-D-C Reports—The Pink Sheet. August 26, 1996;T&G-1.Google Scholar
  25. 25.
    Roberts ProAmatine to be introduced in mid-October. F-D-C Reports—The Pink Sheet. September 16, 1996;T&G-2 to T&G-3.Google Scholar

Copyright information

© Drug Information Association, Inc 1998

Authors and Affiliations

  • David M. Cocchetto
    • 1
  • Douglas R. Jones
    • 1
  1. 1.Regulatory AffairsGlaxo Wellcome Inc.North CarolinaUSA

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