Pre-Approval Access to Drugs in Development:
Navigating the Changing Regulatory, Ethical, and Legal Landscape

Pre-Approval Access to Drugs in Development

Navigating the Changing Regulatory, Ethical, and Legal Landscape


Over the last several years, an increasing interest in expanded access, which is treatment access to experimental drugs (also referred to as "compassionate use" or "pre-approval access"), has sparked discussion and debate. Currently, US federal regulations allow manufacturers to provide investigational drugs to patients with serious diseases or conditions who have exhausted approved therapeutic options, and cannot participate in a clinical trial.

Regulations require IRBs to review and approve expanded access protocols, making them a key player in the pre-approval access process. Moreover, as federal and state "Right to Try" laws are enacted, and the FDA continues to evolve its guidance, there is an increasing need for IRB members and other key stakeholders to stay current with legal and regulatory developments, as well as the shifting ethical landscape.

During this 90-minute webinar, the former director of the Patient Liaison Program at FDA and a senior attorney and health policy expert will discuss the following:

  • Societal developments leading to increased attention on pre-approval access
  • The regulatory and ethical background of the FDA regulations, and the requirements for expanded access, including a summary of FDA’s Guidance for Industry: Expanded Access to Investigational Drugs for Treatment Use
  • Ethical tensions stakeholders must confront when considering pre-approval access programs or compassionate use requests, including the distinction between treatment and clinical research
  • The role of the IRB, including considerations for informed consent, protocol review for cohort programs, individual Investigational New Drugs (INDs), emergency use protocols, and the recent FDA exemption from full board review requirements for individual patient INDs
  • Potential ethical risks raised by access to investigational drugs outside of controlled clinical trials, including therapeutic misconception, distribution of unsafe drugs, and the potential harm from unknown side effects
  • Recent federal legislation, including the 21st Century Cures Act and the Hatch Amendment to the FDA Reauthorization Act, and the legal and ethical implications of "Right to Try" laws

What will I learn?

After attending this webinar, attendees will be able to:

  • Define pre-approval access and differentiate it from clinical research
  • Distinguish between the processes, procedures, and requirements for expanded access cohort protocols and individual INDs, including emergency individual INDs
  • Understand the evolving role of the IRB in reviewing pre-approval access protocols, including differences in membership review of large protocols vs. individual INDs, and informed consent objectives

Who should attend?

This webinar will benefit a wide audience of HRPP professionals who are presently involved with or may become involved with expanded access programs in the future, as well as research sponsors, patient groups, healthcare attorneys and regulatory professionals, and institutions engaged in clinical trials.

Continuing Education

Webinar participants holding the Certified IRB Professional (CIP®) credential may apply 1.5 continuing education credits towards CIP recertification.


Richard Klein

Richard Klein

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Beth E. Roxland, JD, MBioethics

Beth E. Roxland, JD, MBioethics

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Additional Resources

A collection of relevant background reading, further reading, links, templates, checklists, and/or charts accompany each PRIM&R webinar. Groups that register for webinars receive a discussion guide containing thought-provoking questions and facilitation tips that may help in using the webinar as an educational tool for IRBs.

Background Reading:

US Food and Drug Administration. (2016). Guidance for Industry: Expanded Access to Investigational Drugs for Treatment Use.


If you are not yet a PRIM&R member, we encourage you to join today, and take advantage of your very first membership benefit immediately: a discounted registration fee to this webinar.

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Nonmember $200
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