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

March 7, 2018 | 1:00-2:30 PM ET

View the webinar recording:


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

CIP Credit labelWebinar participants holding the Certified IRB Professional (CIP®) credential may apply 1.5 continuing education credits towards CIP recertification. Learn More »


Richard KleinRichard Klein is the former director of the FDA's Patient Liaison Program in the Office of Health and Constituent Affairs, the primary interface between the FDA and patient and patient advocate communities. The program interacts extensively with outside communities and within the agency's scientific offices to facilitate patient engagement and to address issues and concerns of patients in a variety of areas, including treatment access to unapproved drugs, product safety, and clinical trial design. The program ensures that patient communities are represented in approval and policy decisions, and at FDA advisory committee meetings. Mr. Klein worked with the FDA for many years in various capacities, giving him a well-rounded understanding of the regulatory issues that affect patients. He helped develop revised expanded access regulations and guidelines. He also developed the agency's patient web pages, and disease-specific electronic listservs. Prior to his role at FDA, he managed the agency's HIV/AIDS Program and, earlier, was involved in human subject protection policy development for the agency, working closely with other federal agencies and DHHS to ensure consistent and meaningful policy for the protection of human subjects in biomedical research.

Beth RoxlandBeth E. Roxland, JD, MBioethics is an Attorney and Bioethicist with unique experience across industry, law, government and academia. She is a Senior Consultant on Law, Health Policy and Ethics to law firms, life science and medical institutions, and professional and patient associations. She frequently lectures and publishes on a variety of cutting-edge regulatory and bioethical topics, and serves on several scientific and academic oversight boards at leading institutions.

Ms. Roxland previously served as Johnson & Johnson's Bioethics and Strategy Leader, in the Office of the Chief Medical Officer. In this industry-leading role, Ms. Roxland re-launched and chaired Johnson & Johnson's Ethics Committee; created an enterprise-wide research ethics consultation service for teams and individuals confronting complex ethical issues; led the first-of-its-kind major colloquium fostering public dialog between industry, academia, governmental entities, and patients; and provided guidance on issues such as post-trial access for former research participants, conducting clinical trials in war-torn regions, testing of a potential Ebola vaccine during a public health crisis, commercialization of stem cell therapies, subject recruitment and withdrawal, and expanded access/compassionate use.

Prior to joining Johnson & Johnson, Ms. Roxland was the Executive Director of the New York State (NYS) Task Force on Life and the Law, where she led formulation of policy recommendations for conducting research with adults who lack consent capacity, revision of NYS’s Brain Death guidelines, proxy consent for health care decisions, and allocating scarce ventilators during a pandemic.

Ms. Roxland concurrently served as the Special Advisor to the Commissioner of Health on Stem Cell Research Ethics, where she spearheaded creation of state-wide rules on embryonic stem cell protocols, human-animal chimera research, compensation of women who donate their oocytes to stem cell research, informed consent processes, re-contact for return of research results and incidental findings, and downstream uses of biological samples.

Ms. Roxland was previously a Senior Litigation Associate at Simpson Thacher & Bartlett LLP, and a Federal Judicial Law Clerk in the Southern District of New York. She graduated from Columbia University with a Bachelor's degree in Biology, and received a joint JD - Master's in Bioethics magna cum laude from the University of Pennsylvania, where she served as a Senior Editor of the University of Pennsylvania's Law Review.

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.


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