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 at NYU Langone has developed and identified reliable resources to help patients and doctors, members of the media, researchers, and the general public learn more about compassionate use and preapproval access, U.S. Food and Drug Administration (FDA) regulations, and right-to-try laws.

Our Resources

NYU Langone Health Working Group on Compassionate Use and Preapproval Access (CUPA) 2017 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

Frequently asked questions about preapproval access and right to try laws

Publications and media contributions from the Working Group

Federal Government Resources

Right to Try Act of 2017

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

FDA Reauthorization Act of 2017

FDA’s definition of Expanded Access

Individual Patient Expanded Access Applications: Form FDA 3926

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

Charging for Investigational Drugs Under an IND—Questions and Answers

National Cancer Institute’s description of Access to Experimental Cancer Drugs

ClinicalTrials.gov

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

Patient and Physician Navigator Resources

Regan-Udall Foundation’s Expanded Access Navigator

Kids v Cancer’s Compassionate Use Navigator

Patient Advocacy Resources

Principles for Access to Unapproved Therapies statement from 18 patient groups

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

Pharmaceutical Industry Resources

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

Other External Resources

American Society of Clinical Oncology (ASCO) University’s Expanded Access: Introduction

The Policy Surveillance Program’s Right to Try Laws

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

WideTrial

FDA Expanded Access Program at the University of Michigan

Nuffield Council on Bioethics briefing note on patient access to experimental treatments in the UK

EURORDIS Rare Diseases Europe position paper on early access to medicines in Europe

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