Working Group on Compassionate Use & Preapproval Access Resources | NYU Langone Health

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Division of Medical Ethics Working Group on Compassionate Use & Preapproval Access Working Group on Compassionate Use & Preapproval Access Resources

Working Group on Compassionate Use & Preapproval Access Resources

The Working Group on Compassionate Use and Preapproval Access (CUPA) at NYU Langone has developed and identified reliable resources to help patients, healthcare professionals, journalists, researchers, and the general public learn more about compassionate use and preapproval access, relevant U.S. Food and Drug Administration (FDA) regulations, and right to try laws. Our resources mainly focus on U.S. policy, but we have also provided international resources for more information on regulatory issues outside the United States. These resources are not meant to constitute or replace medical advice and are provided for informational purposes only.

Our Resources

Access to Drugs Before FDA Approval: An Explainer (VIDEO)

NYU Langone Health Working Group on Compassionate Use and Preapproval Access (CUPA) 2019 Annual Report

Compassionate Use Lesson Plan by the High School Bioethics Project

Fact Sheet and Recommendations from the NYU Langone Health Working Group on Compassionate Use and Preapproval Access

A Primer on Compassionate Use (via Cancer Knowledge Network)

Frequently Asked Questions About Preapproval Access

Publications and Media Contributions from CUPA

Federal Government Resources

Expanded Access to Experimental Biologics

FDA’s Expanded Access (includes information for patients, physicians, and industry)

FDA’s Expanded Access Contact Information

FDA’s Project Facilitate

FDA’s Right to Try

Expanded Access to Investigational Drugs for Treatment Use—Questions and Answers

Charging for Investigational Drugs Under an IND—Questions and Answers

National Cancer Institute: Access to Experimental Cancer Drugs

U.S. Government Accountability Office’s 2019 Report on Investigational New Drugs

Relevant Legislation

21st Century Cures Act’s Expanded Access Policy, Subtitle E—Expediting Patient Access

FDA Reauthorization Act of 2017

Right to Try Act of 2017

Expanded Access Navigators

Reagan-Udall Foundation’s Expanded Access Navigator

Kids v Cancer’s Compassionate Use Navigator

Patient Advocacy Position Papers and Other Information

The Foundation for Compassionate Use Reform (CURE) Compassionate Use Frequently Asked Questions

National Organization for Rare Disorders Frequently Asked Questions on Expanded Access

Pharmaceutical Industry Resources

The pharmaceutical company resources listed here are not exhaustive and meant to serve as examples of preapproval access policies for informational purposes only.

Pharmaceutical Researchers and Manufacturers of America (PhRMA) Principles on Conduct of Clinical Trials: Communication of Clinical Trial Results

Biotechnology Innovation Organization’s Points to Consider: Early Access to Experimental Products Programs

Janssen’s Compassionate Use and Pre-Approval Access

Pfizer’s Compassionate Use and Expanded Access

Novartis’s Managed Access Programs

International Resources

Nuffield Council on Bioethics Briefing Note on Patient Access to Experimental Treatments in the United Kingdom

European Patients’ Academy (EUPATI): Compassionate Use

The European Organisation for Rare Diseases (EURORDIS) Position Paper on Compassionate Use

Other External Resources

American Action Forum’s Primer: Introduction to the FDA Drug Approval Process and Off Label Use

FDA Expanded Access Program at the University of Michigan

The New York Academy of Sciences’ Symposium Pre-Approval Access: Can Compassion, Business, and Medicine Coexist?

Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) Educational Webinar: Right to Try? Historical, Legal and Ethical Considerations

Reagan-Udall Foundation’s Expanded Access Resources

Avalere Health’s 2017 Report on Manufacturers’ Compassionate Use Policies