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Expediting the availability of drugs for US patients with cancer

Nature Clinical Practice Urology volume 5, pages 654–656 (2008)Cite this article

In the US, to obtain marketing approval for a New Drug Application (NDA), an applicant must demonstrate that the drug is safe, and provide substantial evidence from adequate and well-controlled investigations that the drug is effective.1,2,3 The studies must allow a valid comparison of participants receiving the drug with participants from a control group, and must provide a quantitative assessment of the drug's effect. Guidance from the US FDA, published in the 1980s, indicated that efficacy should be demonstrated by a prolongation of life, by an improvement in quality of life, or by an established surrogate outcome measure for at least one of these effects.4,5 The safety requirement for the NDA is derived from the Federal Food, Drug, and Cosmetic Act of 1938, and a 1962 amendment to the Act codifies the efficacy requirement.

In the early 1990s, the FDA finalized its accelerated approval regulations,4 which are intended to encourage the development and expeditious approval of drugs for serious or life-threatening diseases. These regulations allow for the approval of a drug on the basis of a surrogate end point that is reasonably likely (based on epidemiologic, therapeutic, pathophysiologic, or other evidence4) to predict clinical benefit. These surrogates are explicitly less well-established than those commonly used in pivotal trials for regular approval, such as blood pressure or cholesterol level. Since the inception of the accelerated approval regulations, over 35 oncology drugs have been approved in the US via this regulatory mechanism.3,6

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References

  1. US Food and Drug Administration (online 31 December 2005) Code of Federal Regulations—Title 21: Food and Drugs 314.126—Adequate and well-controlled studies [http://vlex.com/vid/19710185] (accessed 11 October 2008)

  2. Temple R (1996) Development of drug law, regulations, and guidance in the United States. In Principles of Pharmacology: Basic Concepts and Clinical Applications, 1643–1665 (Eds Munson PL. et al.) New York: Chapman & Hall

    Google Scholar 

  3. Johnson JR and Temple R (1985) Food and Drug Administration requirements for approval of new anticancer drugs. Cancer Treat Rep 69: 1155–1159

    CAS  PubMed  Google Scholar 

  4. US Food and Drug Administration (online 11 December 1992) Code of Federal Regulations—Title 21: Food and Drugs 314 and 601—New Drug, Antibiotic, and Biologic Product Regulations; Accelerated Approval 57 FR 58942 [http://www.fda.gov/cder/fedreg/fr19921211.txt] (accessed 11 October 2008)

  5. Johnson JR et al. (2003) End points and United States Food and Drug Administration approval of oncology drugs. J Clin Oncol 21: 1404–1411

    Article  Google Scholar 

  6. US Food and Drug Administration (online 31 December 2005) Code of Federal Regulations—Title 21: Food and Drugs 314.530—Withdrawal procedures [http://vlex.com/vid/19708988] (accessed 11 October 2008)

  7. Dagher R et al. (2004) Accelerated approval of oncology products: a decade of experience. J Natl Cancer Inst 96: 1500–1509

    Article  Google Scholar 

  8. US Food and Drug Administration (online 31 May 2007) Guidance for Industry: Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics [http://www.fda.gov/cder/guidance/7478fnl.pdf] (accessed 11 October 2008)

  9. US Food and Drug Administration (online 31 May 1998) Guidance for Industry: Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products [http://www.fda.gov/cder/guidance/1397fnl.pdf] (accessed 11 October 2008)

  10. US Food and Drug Administration (online 31 May 2001) E 10 Choice of Control Group and Related Issues in Clinical Trials [http://www.fda.gov/cder/guidance/4155fnl.pdf] (accessed 11 October 2008)

  11. US Food and Drug Administration (online 31 December 2005) Code of Federal Regulations—Title 21: Food and Drugs 312.34—Treatment use of an investigational new drug [http://vlex.com/vid/19709947] (accessed 11 October 2008)

  12. Farrell A et al. (2008) FDA's role in the development and approval of drugs, biologics, and devices for cancer. In DeVita, Hellman, and Rosenberg's Cancer: Principles & Practice of Oncology, edn 8 2914–2925 (Eds DeVita VT. et al.) Philadelphia: Lippincott Williams & Wilkins

    Google Scholar 

  13. US Food and Drug Administration (online 31 March 2002) Guidance for Industry: Information Program on Clinical Trials for Serious or Life-Threatening Diseases and Conditions [http://www.fda.gov/cder/guidance/4856fnl.pdf] (accessed 11 October 2008)

  14. US Food and Drug Administration (online 14 December 2006) Code of Federal Regulations—Title 21: Food and Drugs 312—Expanded Access to Investigational Drugs for Treatment Use [http://www.fda.gov/OHRMS/DOCKETS/98fr/06-9684.pdf] (accessed 11 October 2008)

  15. US Food and Drug Administration (online 31 July 2004) Guidance for Industry: Fast Track Drug Development Programs—Designation, Development, and Application Review [http://www.fda.gov/cder/guidance/5645fnl.pdf] (accessed 11 October 2008)

  16. US Food and Drug Administration (online 31 December 1998) Guidance for Industry: FDA Approval of New Cancer Treatment Uses for Marketed Drug and Biological Products [http://www.fda.gov/cder/guidance/1484fnl.pdf] (accessed 11 October 2008)

  17. US Food and Drug Administration (online 31 October 2001) Guidance for Industry: Cancer Drug and Biological Products—Clinical Data in Marketing Applications [http://www.fda.gov/cder/guidance/4332fnl.pdf] (accessed 11 October 2008)

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Authors and Affiliations

  1. AT Farrell is the Deputy Director of the Division of Drug Oncology Products, R Pazdur is the Director of the Office of Oncology Drug Products, and AC von Eschenbach is the Commissioner at the US Food and Drug Administration, Rockville, MD, USA.,

    Ann T Farrell, Richard Pazdur & Andrew C von Eschenbach

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  1. Ann T Farrell
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  2. Richard Pazdur
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  3. Andrew C von Eschenbach
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Correspondence to Andrew C von Eschenbach.

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Farrell, A., Pazdur, R. & von Eschenbach, A. Expediting the availability of drugs for US patients with cancer. Nat Rev Urol 5, 654–656 (2008). https://doi.org/10.1038/ncpuro1255

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