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Pre-approval Access Terminology: A Cause for Confusion and a Danger to Patients

  • Regulatory Science: Original Research
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Abstract

Background

Patients who are seriously ill and have run out of available treatment options may seek access to investigational agents that have not yet been fully vetted by regulatory agencies for safety and efficacy and approved for use in human subjects. Over time, a variety of terms have evolved internationally to denote mechanisms for providing access to such unapproved investigational agents. The lack of consistency in terminology used to describe this process is confusing at best and, at worst, possibly even detrimental to patients.

Methods

To highlight variation around the globe in terminology denoting pre-approval access to investigational agents, we conducted extensive Internet searches to locate specific legislation, guidance, or policy documents describing access mechanisms in numerous countries. We created a table of results intended to convey a sampling of international terminological diversity.

Results

The profusion of terms used internationally to indicate pre-approval access to investigational agents is evident. We recommend a shift toward the use of “pre-approval access” as an umbrella term encompassing all forms of access to unapproved agents. We also recommend use of the phrases “individual/named patient regulatory routes for pre-approval access” and “group/cohort regulatory routes for pre-approval access” to differentiate between pre-approval access programs designed for single patients, versus those designed for groups of patients.

Conclusions

There is a pressing need to revisit and better align pre-approval access terminology at the international level. Adopting the umbrella term “pre-approval access” may be a useful strategy for initiating and promoting harmonization of terms to reduce potential confusion by patients and health care decision makers regarding experimental treatment options.

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Authors and Affiliations

  1. School of Social Work, Columbia University School of Social Work, 1255 Amsterdam Avenue, 9th Floor, New York, NY, 10027, USA

    Laura L. Kimberly MSW, MBE

  2. Division of Medical Ethics, NYU Langone Medical Center, New York, NY, USA

    Marc M. Beuttler MA, Michael Shen BS, Arthur L. Caplan PhD & Alison Bateman-House PhD, MPH, MA

Authors
  1. Laura L. Kimberly MSW, MBE
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  2. Marc M. Beuttler MA
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  3. Michael Shen BS
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  4. Arthur L. Caplan PhD
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  5. Alison Bateman-House PhD, MPH, MA
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Corresponding author

Correspondence to Laura L. Kimberly MSW, MBE.

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Kimberly, L.L., Beuttler, M.M., Shen, M. et al. Pre-approval Access Terminology: A Cause for Confusion and a Danger to Patients. Ther Innov Regul Sci 51, 494–500 (2017). https://doi.org/10.1177/2168479017696267

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  • DOI: https://doi.org/10.1177/2168479017696267

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