Working Group on Compassionate Use & Preapproval Access Publications & Media | 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 Publications & Media

Working Group on Compassionate Use & Preapproval Access Publications & Media

The Working Group on Compassionate Use and Preapproval Access at NYU Langone has published hundreds of articles about compassionate use and preapproval access, U.S. Food and Drug Administration (FDA) regulations, and right-to-try laws. Below is a selection of our contributions to peer-reviewed academic journals and other media outlets, and publications from other reliable resources.

Peer-Reviewed Publications

Djordjevic D, McFadyen A, and Anderson JA. Ethical challenges and opportunities in the development and approval of novel therapeutics for rare diseases. J Med Access. 2023. DOI.

Bateman-House A and Kearns L. Individualized therapeutics development for rare diseases: The current ethical landscape and policy responses. Nucleic Acid Ther. 2022. DOI.

Gould P … Lynch HF. Perspectives of academic oncologists about offering expanded access to investigational drugs. JAMA Netw Open. 2022. DOI.

Polak TB … Darrow JJ. Generating evidence from expanded access use of rare disease medicines: Challenges and recommendations. Front Pharmacol. 2022. DOI.

Kang S … Miller JE. Implementation of 21st Century Cures Act expanded access policies requirements. Clin Pharmacol Ther. 2021. DOI.

Miller JE … Bach PB. Evaluation of drug trials in high-, middle-, and low-income countries and local commercial availability of newly approved drugs. JAMA Netw Open. 2021. DOI.

Caplan AL … Maree A. Drugs of unproven benefit for COVID-19: A pharma perspective on ethical allocation of available therapies. J Clin Invest. 2020. DOI.

Lynch HF, Bateman-House A, and Joffe S. Emergency approvals for COVID-19: Evolving impact on obligations to patients in clinical care and research. Ann Intern Med. 2020. DOI.

Nakada H, Folkers KM, and Takashima K. Comparative assessment of non-trial access to investigational medical products in the U.S. and Japan. Ther Innov Regul Sci. 2020. DOI.

Snyder J, Bateman-House A, and Turner L. Is right to try being tried? Using crowdfunding data to better understand usage of nontrial pre-approval access pathways. Regen Med. 2020. DOI.

See our additional peer-reviewed publications.

Articles and Opinion Pieces

Belli H. Real-world data collected from expanded access programs. Association for Clinical Research Professionals Blog. February 7, 2023.

Bateman-House A and Kearns L. Investigational medical products for rare diseases: Ethical concerns and unresolved issues with expanded access. Inside Precision Medicine. December 1, 2021.

Dal-Ré R and Caplan AL. Current Covid-19 vaccine trials in high-income countries: Are placebo-controlled trials ethical? Clin Microbiol Infect. 2021. DOI.

Kearns L … Bateman-House A. Gene therapy companies have an ethical obligation to develop expanded access policies. Mol Ther. 2021. DOI.

Lynch HF, Folkers KM, and Caplan AL. Private equity and Right to Try: A dangerous combination. Health Affairs Blog. July 6, 2020.

Caplan AL and Moch KI. ‘Rescue Me’ revisited: A five-year perspective on preapproval access to experimental medicines. Health Affairs Blog. November 1, 2019.

Folkers KM and Bateman-House A. Glioblastoma patient is first to receive treatment under Right to Try. Our question is why? The Cancer Letter. February 1, 2019.

Klein R. Compassion and safety drive FDA expanded access program. Clinical Research Pathways blog. March 4, 2019.

Bateman-House A. “Right to Try” is law: Now what?: Part 2. Health Affairs Blog. October 26, 2018.

Bateman-House A. “Right to Try” is law: Now what?: Part 1. Health Affairs Blog. October 25, 2018.

See our additional articles and opinion pieces.

Selected External Publications

Berlanga P … Vassal G. Measuring safety and outcomes for the use of compassionate and off-label therapies for children, adolescents, and young adults with cancer in the Sacha-France study. JAMA Netw Open. 2023.  DOI.

Fultinaviciute U. Managed access programmes: Expanding clinical development plans for patients in need. Clinical Trials Arena. March 22, 2023.

Lee SS … Appelbaum PS. Individualized interventions for rare genetic conditions and the research-treatment spectrum: Stakeholder perspectives. Genet Med. 2023. DOI.

Zettler PJ … Roxland BE. An International Society for Cell & Gene Therapy working group short report on the future of expanded access to unapproved cell and gene therapies. Cytotherapy. 2023. DOI.
Aliu P … Scosyrev E. International country-level trends, factors, and disparities in compassionate use access to unlicensed products for patients with serious medical conditions. JAMA Health Forum. 2022. DOI.

EFPIA-EURORDIS. Joint statement on patient access to medicines for rare diseases. June 15, 2022.

U.S. House of Representatives Committee on Oversight Reform and the Committee on Energy and Commerce. The high price of Aduhelm’s approval: An investigation into FDA’s atypical review process and Biogen’s aggressive launch plans. December 29, 2022.

Greenbaum D. Making compassionate use more useful: Using real-world data, real-world evidence and digital twins to supplement or supplant randomized controlled trials. Pac Symp Biocomput. 2021. DOI.

Kane PB, Bittlinger M, and Kimmelman J. Individualized therapy trials: Navigating patient care, research goals and ethics. Nat Med. 2021. DOI.

Stout J … Master Z. Oncologists’ reflections on patient rights and access to compassionate use drugs: A qualitative interview study from an academic cancer center. PLoS One. 2021. DOI.

See our additional selected external publications.

Bioethics Responses to Coronavirus Disease (COVID-19)   

McCarthy MW, Oshinsky D, and Caplan A. Make preapproval COVID-19 vaccines available through expanded access, not an EUA. STAT First Opinion. November 9, 2020.

Miller JE, Ross JS, and Mello MM. Far more transparency is needed for COVID-19 vaccine trials. STAT First Opinion. November 5, 2020.

Perspectives on Ethical and Regulatory Implications of Recent Events (Webinar). VOZ Advisors and CUPA Working Group members answer questions about the use of investigational products to treat or prevent COVID-19. October 16, 2020.

Moch KI. Bioethics and the need for ethical leadership during the COVID-19 pandemic. In: Levin JM, ed. Biotechnology in the Time of COVID-19: Commentaries from the Front Line. New York: RosettaBooks; 2020:145-152.

Bateman-House A. Many patients, little drugs: Who should get scarce COVID-19 treatments?. The Hill. May 29, 2020.

Loike JD and Miller JE. Opinion: Ethically accessing experimental therapies for COVID-19. The Scientist. April 8, 2020.

Robertson C … Joiner K. In the rush to innovate for COVID-19 drugs, sound science is still essential. The Conversation. April 8, 2020.

Lynch HF, Bateman-House A, and Caplan AL. ‘Panic prescribing’ untested coronavirus treatments: A danger to patients today and tomorrow. Health Affairs Blog. March 31, 2020.