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The Expanded Access Cure: A Twenty-First Century Framework for Companies

Abstract

Through expanded access protocols, the Food and Drug Administration (FDA) allows patients with serious or immediately life-threatening diseases access to experimental drugs outside the clinical trial setting when no satisfactory alternative treatment is available. While the FDA has established a mechanism for providing patients with unapproved drug access, the regulations do not require the pharmaceutical company to provide the drug. The drug company’s permission to use its experimental drug is a necessary prerequisite to using the FDA’s expanded access mechanism. Increasingly, drug companies are coming under scrutiny regarding the programs governing that decision-making power. Historically, disclosing whether a company has an expanded access program, and whether or how it would respond to an expanded access request, has been left to discretion of the drug companies themselves. Few manufacturers publish adequate expanded access protocols. As a result, patients were provided with little insight into how companies evaluate expanded access requests and are naturally skeptical as to the ethical integrity of the process. The recently passed 21st Century Cures Act changes that practice by requiring drug companies to have, and make publically available, their expanded access procedures including criteria for evaluating and responding to patient requests. In this article, we contend that complying with the new transparency provisions will require drug companies to respond to several unresolved expanded access issues. Namely, how to reconcile a patient’s desire to access lifesaving experimental therapies alongside the company and society’s interest in the efficient development of new drugs. Even more challenging, how can companies devise practices for evaluating and processing expanded access requests that also fairly and equitably acknowledge those concerns? In addressing these questions, this article explores the legal, regulatory, business, and societal influences that have shaped expanded access policies and practices. From there, we provide companies a framework that balances appropriately the desires of individuals and gaining the requisite approvals ensure access not just for one person but for society.

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Notes

  1. 1.

    21 C.F.R. §312.310; 21 C.F.R. §312. 315, 21 C.F.R. §312.320.

  2. 2.

    21 CFR §312.310.

  3. 3.

    Pub. L. No. 114-255 (2016).

  4. 4.

    Nichols (1991).

  5. 5.

    Young et al. (1988).

  6. 6.

    Young et al. (1988).

  7. 7.

    Eichler et al. (2013). The Risks of Risk Aversion in Drug Regulation, 12 Nature.

  8. 8.

    Buhles (2011).

  9. 9.

    Buhles (2011).

  10. 10.

    Kolata (1989).

  11. 11.

    Buhles (2011).

  12. 12.

    http://www.bwfund.org/history.

  13. 13.

    Sanghavi (2013).

  14. 14.

    Sanghavi (2013).

  15. 15.

    Food and Drug Administration, HHS (2009).

  16. 16.

    21 C.F.R. §312.305 (2105).

  17. 17.

    Food and Drug Administration, HHS (2009).

  18. 18.

    Food and Drug Administration, HHS (2009).

  19. 19.

    U.S. Department Health and Human Services (2013).

  20. 20.

    US Food and Drug Administration (2013). IND Activity Reports. Expanded Access INDs and Protocols.http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/DrugandBiologicApprovalReports/INDActivityReports/ucm373560.htm.

  21. 21.

    Kim et al. (2015).

  22. 22.

    United States v. Rutherford (1979).

  23. 23.

    Id. at 556.

  24. 24.

    United States v. Rutherford (1979).

  25. 25.

    Abigail Alliance v. von Eschenhach (2007).

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    Abigail Alliance v. von Eschenhach (2007).

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    Abigail Alliance v. von Eschenhach (2007).

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    Mackey and Schoenfeld (2016).

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    Flatten (2016).

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    Caplan and Moch (2014).

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    Caplan and Moch (2014).

  43. 43.

    Gaffney (2015).

  44. 44.

    H.R. 1281 (2014).

  45. 45.

    Zettler and Greely (2014).

  46. 46.

    H.R. 1281 (2014).

  47. 47.

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  48. 48.

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  49. 49.

    Bateman-House (2016).

  50. 50.

    Edney (2017).

  51. 51.

    S.2912 Trickett Wendler Right to Try Act (2016).

  52. 52.

    Zettler and Greely (2014).

  53. 53.

    Zettler and Greely (2014).

  54. 54.

    Darrow et al. (2015).

  55. 55.

    Darrow et al. (2015).

  56. 56.

    Zettler and Greely (2014).

  57. 57.

    Mutual Pharmaceutical v. Bartlett (2013).

  58. 58.

    Food and Drug Admin. v. Brown & Williamson Tobacco Corp. (2000).

  59. 59.

    Zettler and Greely (2014).

  60. 60.

    Darrow et al. (2015).

  61. 61.

    Cameron (2014).

  62. 62.

    Darrow et al. (2015).

  63. 63.

    Walker et al. (2014).

  64. 64.

    Walker et al. (2014) and Okie (2006).

  65. 65.

    Proposed new drug (1983).

  66. 66.

    Docker Marcus (2014).

  67. 67.

    McDiarmid (2014).

  68. 68.

    Jarow et al. (2016).

  69. 69.

    Food and Drug Administration, HHS (2009).

  70. 70.

    Darrow et al. (2015).

  71. 71.

    Food and Drug Administration, HHS (2009).

  72. 72.

    Food and Drug Administration, HHS (2009).

  73. 73.

    In Glaser v. Enzo Biochem Inc., 303 F. Supp. 2d 724 (E.D. Va. 2003),.

  74. 74.

    Food and Drug Administration, HHS (2009).

  75. 75.

    Darrow et al. (2015).

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    Huneycutt et al. (2016).

  79. 79.

    Food and Drug Administration, HHS (2009).

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    "Shire Policies: Expanded access." Shire Policies: Expanded access. N.p., n.d. Web. 13 June 2016..

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    "Shire Policies: Expanded access." Shire Policies: Expanded access. N.p., n.d. Web. 13 June 2016..

  83. 83.

    Food and Drug Administration, HHS (2009).

  84. 84.

    Shire Policies: no access (2016).

  85. 85.

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    "Shire Policies: Expanded access." Shire Policies: Expanded access. N.p., n.d. Web. 13 June 2016..

  87. 87.

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    Caplan and Ray (2016).

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    Caplan and Ray (2016).

    .

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    Caplan and Ray (2016).

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Lee, S.B., Murata, A.Y. The Expanded Access Cure: A Twenty-First Century Framework for Companies. J Bus Ethics 156, 155–171 (2019). https://doi.org/10.1007/s10551-017-3533-3

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Keywords

  • Expanded access
  • Compassionate use
  • Corporate frameworks
  • Experimental drugs
  • Investigational drugs
  • FDA
  • Pharmaceutical manufacturers
  • Resource allocation