Access to Investigational Drugs: FDA Expanded Access Programs or "Right-to-Try" Legislation?

M. E Blair Holbein, Jelena P. Berglund, Kevin Weatherwax, David E. Gerber, Joan E. Adamo

Research output: Contribution to journalArticle

15 Citations (Scopus)

Abstract

Purpose: The Food and Drug Administration Expanded Access (EA) program and "Right-to-Try" legislation aim to provide seriously ill patients who have no other comparable treatment options to gain access to investigational drugs and biological agents. Physicians and institutions need to understand these programs to respond to questions and requests for access. Methods: FDA EA programs and state and federal legislative efforts to provide investigational products to patients by circumventing FDA regulations were summarized and compared. Results: The FDA EA program includes Single Patient-Investigational New Drug (SP-IND), Emergency SP-IND, Intermediate Sized Population IND, and Treatment IND. Approval rates for all categories exceed 99%. Approval requires FDA and Institutional Review Board (IRB) approval, and cooperation of the pharmaceutical partner is essential. "Right-to-Try" legislation bypasses some of these steps, but provides no regulatory or safety oversight. Conclusion: The FDA EA program is a reasonable option for patients for whom all other therapeutic interventions have failed. The SP-IND not only provides patient access to new drugs, but also maintains a balance between immediacy and necessary patient protection. Rather than circumventing existing FDA regulations through proposed legislation, it seems more judicious to provide the knowledge and means to meet the EA requirements.

Original languageEnglish (US)
Pages (from-to)526-532
Number of pages7
JournalClinical and Translational Science
Volume8
Issue number5
DOIs
StatePublished - Oct 1 2015

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Investigational Drugs
Legislation
Biological Factors
Pharmaceutical Preparations
Research Ethics Committees
United States Food and Drug Administration
Emergencies
Therapeutics
Physicians
Safety

Keywords

  • FDA
  • New agents
  • Phase I
  • Trials

ASJC Scopus subject areas

  • Medicine(all)
  • Biochemistry, Genetics and Molecular Biology(all)
  • Neuroscience(all)
  • Pharmacology, Toxicology and Pharmaceutics(all)

Cite this

Access to Investigational Drugs : FDA Expanded Access Programs or "Right-to-Try" Legislation? / Holbein, M. E Blair; Berglund, Jelena P.; Weatherwax, Kevin; Gerber, David E.; Adamo, Joan E.

In: Clinical and Translational Science, Vol. 8, No. 5, 01.10.2015, p. 526-532.

Research output: Contribution to journalArticle

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