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.

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.

Bateman-House A, Gustafson MS, and Caplan A. Trump's Regeneron treatment is a tangled ethical mess. Barron's Commentary. October 8, 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.

Folkers KM and Caplan AL. False hope about coronavirus treatments. The Hastings Center. March 20, 2020.

Peer-Reviewed Publications

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

Nakada H, Folkers KM, and Takashima K. Comparative assessment of non-yrial 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

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 and Bateman-House A. Facilitating both evidence and access: Improving FDA’s accelerated approval and expanded access pathways. J Law Med Ethics. 2020. DOI

Chapman CR, Eckman J, and Bateman-House AS. Oversight of right‐to‐try and expanded access requests for off‐trial access to investigational drugs. Ethics & Human Research. 2020. DOI.

Folkers KM, Leone S, and Caplan A. Patient advocacy organizations’ information for patients on pre-approval access to investigational treatments. BMC Res Notes. 2019. DOI.

Chapman CR … Bateman-House A. Single-patient expanded access requests: IRB professionals' experiences and perspectives. AJOB Empir Bioeth. 2019. DOI.

Chapman CR … Bateman-House A. What compassionate use means for gene therapies. Nature Biotechnol. 2019. DOI.

Folkers K, Chapman C, and Redman B. Federal right to try: Where is it going? Hastings Cent Rep. 2019. DOI.

Salgado R … Massard C. Addressing the dichotomy between individual and societal approaches to personalised medicine in oncology. Eur J Cancer. 2019. DOI.

Thomas S and Caplan A. The orphan drug act revisited. JAMA. 2019. DOI.

Bateman-House A and Robertson CT. The federal Right to Try Act of 2017—A wrong turn for access to investigational drugs and the path forward. JAMA Intern Med. 2018. DOI.

Borysowski J … Górski A. Expanded access: Growing importance to public health. J Epidemiol Community Health. 2018. DOI.

Caplan AL … Gardner SL. Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials. J Med Ethics. 2018. DOI.

Folkers KM and Bateman-House A. Improving expanded access in the United States: The role of the institutional review board. Ther Innov Regul Sci. 2018. DOI.

Kearns L, Bateman-House A, and Caplan A. Ensuring justice in access to investigational neurological drugs. Semin Neurol. 2018. DOI.

Puthumana J … Ross JS. Availability of investigational medicines through the U.S. Food and Drug Administration’s expanded access and compassionate use programs. JAMA Network Open. 2018. DOI.

Vox F … Caplan AL. Medical crowdfunding for scientifically unsupported or potentially dangerous treatments. JAMA. 2018. DOI.

Caplan A and Folkers KM. Charlie Gard and the limits of parental authority. Hastings Cent Rep. 2017. DOI.

Kearns L and Bateman-House A. Who stands to benefit? Right to try law provisions and implications. Ther Innov Regul Sci. 2017. DOI.

Kearns L and Caplan AL. Hard choices for vulnerable patients: Some lessons learned that may apply. Am J Bioeth. 2017. DOI.

Kimberly LL … Bateman-House A. Pre-approval access terminology: A cause for confusion and a danger to patients. Ther Innov Regul Sci. 2017. DOI.

Miller JE … Caplan AL. Characterizing expanded access and compassionate use programs for experimental drugs. BMC Res Notes. 2017. DOI.

Moch KI. Ethical crossroads: Expanded access, patient advocacy, and the #SaveJosh social media campaign. Med Access @ Point Care. 2017. DOI.

Watson T. A global perspective on compassionate use and expanded access. Ther Innov Regul Sci. 2017. DOI.

Caplan AL and Ray A. The ethical challenges of compassionate use. JAMA. 2016. DOI.

Redman BK and Bateman-House AS. Institutional review boards as arbiters of expanded access to unapproved drugs: Time for a change? Ther Innov Regul Sci. 2016. DOI.

Robertson C and Kesselheim AS. Regulating off-label promotion—A critical test. N Engl J Med. 2016. DOI.

Bateman-House A … Caplan A. Right-to-try laws: Hope, hype, and unintended consequences. Ann Intern Med. 2015. DOI.

Caplan AL and Bateman-House A. Should patients in need be given access to experimental drugs? Expert Opin Pharmacother. 2015. DOI.

Articles and Opinion Pieces

Bateman-House A, Caplan AL, and Kearns L. Trump gave patients a 'Right to Try' experimental drugs. It hasn't helped them. Barron's Commentary. November 3, 2020.

McNair L and Gustafson S. Expanded access in the time of COVID-19: What do IRBs need to know? Ampersand, the PRIM&R Blog. October 19, 2020.

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

Klein R. Counterpoint: 3 reasons Right to Try is less flexible than expanded access. October 29, 2019.

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

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

Caplan A, Folkers KM, and McFadyen A. A bizarre claim of Right to Try. The Health Care Blog. January 18, 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.

Moch KI, McFadyen A, and Caplan A. Here’s how to structure successful right-to-try laws. The Hill. March 11, 2018.

Folkers KM and Bateman-House A. Will new FDA regulation on IRB review speed patient access to experimental drugs? Health Affairs Blog. December 11, 2017.

Chapman CR. Is it time for the FDA to consider a differentiated approval system? Health Affairs Blog. December 4, 2017.

Klein R. Dispelling the myths and misconceptions around expanded access. Inside Digital Health. December 4, 2017.

Bateman-House A. What I told Congress about patient access to experimental medicines. Forbes. October 17, 2017.

Caplan A and Bateman-House A. The FDA is an integral part of compassionate use. Forbes. July 6, 2017.

Bateman-House A and Caplan AL. All hat, no cattle—The false hope of right to try laws. Harvard Health Policy Review. November 12, 2016.

Caplan A and Moch K. Rescue me: The challenge of compassionate use in the social media era. Health Affairs Blog. August 27, 2014.

Selected External Publications

Borysowski J and Górski A. Compassionate use of unauthorized drugs: Legal regulations and ethical challenges. Eur J Intern Med. 2019. DOI.

Feit NZ … Hyman DM. Use, safety, and efficacy of single-patient use of the US Food and Drug Administration expanded access program. JAMA Oncol. 2019. DOI.

Florko N. A year after Trump touted ‘right to try,’ patients still aren’t getting treatment. STAT News. January 29, 2019.

Kelman A, Kang A, and Crawford B. Continued access to investigational medicinal products for clinical trial participants—An industry approach. Cambridge Quarterly of Healthcare Ethics. 2019. DOI.

Lanzel AF and Lavery JV. Unintended consequences of the Right to Try Act for palliative care in pediatric oncology. JAMA Oncol. 2019. DOI.

Lawrence L. A look at the clinical benefits of the FDA’s expanded access program. CancerNetwork. March 14, 2019.

Moerdler S ... Weiser DA. Physician perspectives on compassionate use in pediatric oncology. Pediatr Blood Cancer. 2019. DOI.

O'Dell R and Penzenstadler N. You elected them to write new laws. They’re letting corporations do it instead. USA Today. April 4, 2019.

Spector-Bagdady K … Shuman AG. The critical role of medical institutions in expanding access to investigational interventions. Hastings Cent Rep. 2019. DOI.

Usdin S. Compassionate use of gene therapies raises ethical, commercial quandaries. BioCentury. April 12, 2019.

Bunnik EM, Aarts N, and van de Vathorst S. Little to lose and no other options: Ethical issues in efforts to facilitate expanded access to investigational drugs. Health Policy. 2018. DOI.

Cossu G … Wilson J. Lancet Commission: Stem cells and regenerative medicine. Lancet. 2018. DOI.

Eaton ES. WideTrial seeks to increase expanded access programs by helping companies recover costs. BioCentury. October 18, 2018.

Fernandez Lynch H, Zettler PJ, and Sarpatwari A. Promoting patient interests in implementing the federal Right to Try Act. JAMA. 2018. DOI.

Fountzilas E, Said R, and Tsimberidou AM. Expanded access to investigational drugs: Balancing patient safety with potential therapeutic benefits. Expert Opin Investig Drugs. 2018. DOI.

Joffe S and Fernandez Lynch H. Federal Right-to-Try Legislation—Threatening the FDA’s Public Health Mission. N Engl J Med. 2018. DOI.

Kapczynski A. Dangerous times: The FDA’s role in information production, past and future. Minnesota Law Review. 2018.

Landsmann MA. Will Right to Try increase access to experimental treatments? CancerToday. June 15, 2018.

London AJ. Social value, clinical equipoise, and research in a public health emergency. Bioethics. 2018. DOI.

Miseta E. Now that Right to Try is law, what does it mean to you? Clinical Leader. November 29, 2018.

Neuhaus CP and Zacharias RL. Compassionate use of gene therapies in pediatrics: An ethical analysis. Semin Perinatol. 2018. DOI.

Roxland B and Hurley EA. Taking a closer look at the new federal “Right to Try” law. Ampersand: The PRIM&R Blog. August 21, 2018.

Thomas K. Why can’t dying patients get the drugs they want? The New York Times. March 23, 2018.

Jarow JP and Moscicki R. Impact of expanded access on FDA regulatory action and product labeling. Ther Innov Regul Sci. 2017. DOI.

McKee AE … Lurie P. How often are drugs made available under the Food and Drug Administration’s expanded access process approved? J Clin Pharmacol. 2017. DOI.

Woollett G and Jackson J. Manufacturer’s compassionate use policies: Companies with posted policies more than doubled since September 2016. Avalere Health. March 27, 2017.

Jarow JP … Moscicki R. Expanded access of investigational drugs: The experience of the Center of Drug Evaluation and Research over a 10-year period. Ther Innov Regul Sci. 2016. DOI.

Piel J. Informed consent in right-to-try cases. J Am Acad Psychiatry Law. 2016;44(3)290–296.

Darow JJ … Kesselheim AS. Practical, legal, and ethical issues in expanded access to investigational drugs. N Engl J of Med. 2015. DOI.

Dresser R. The ‘right to try’ investigational drugs: Science and stories in the access debate. Texas Law Review. 2015.

Klopfenstein M, Van Campen LE, and Garnett T. Expanded access programs: Ethical and practical considerations for biopharmaceutical sponsors. Ther Innov Regul Sci. 2015. DOI.

Adriance S. Fighting for the “Right to Try” unapproved drugs: Law as persuasion. The Yale Law Journal. December 4, 2014.

Zettler PJ and Greely HT. The strange allure of state “right-to-try” laws. JAMA Inter Med. 2014. DOI.

Jacobson PD and Parmet WE. A new era of unapproved drugs: The case of Abigail Alliance v Von Eschenbach. JAMA. 2007. DOI.

Groopman J. The right to a trial. The New Yorker. December 10, 2006.