Investigational Medical Products for Rare Diseases: Ethical Concerns and Unresolved Issues with Expanded Access

Kimberly LL , Beuttler MM , Shen M , Caplan AL , Bateman-House A . (2017). Pre-approval access terminology: a cause for confusion and a danger to patients. Ther Innov Regul Sci. Jul; 51(4):494-500. doi: 10.1177/2168479017696267. Epub 2017 Mar 3. PMID: 30227050.

Bunnik EM , Aarts N , van de Vathorst S . (2018). Little to lose and no other options: ethical issues in efforts to facilitate expanded access to investigational drugs. Health Policy. Sep; 122(9):977–983. doi: 10.1016/j.healthpol.2018.06.005. Epub 2018 Jun 18. PMID: 29935731.

Folkers K , Chapman C , Redman B . (2019) Federal Right to Try: where is it going? Hastings Cent Rep. Mar;49(2):26–36. doi: 10.1002/hast.990. PMID: 3099828.1

Bateman-House A , Kimberly L , Redman B , Dubler N , Caplan A . Right-to-try laws: hope, hype, and unintended consequences. (2015) Ann Intern Med. Nov 17; 163(10):796–7. doi: 10.7326/M15-0148. Epub 2015 Sep 29. PMID: 26413841.

Chapman CR , Shearston JA , Folkers KM , Redman BK , Caplan A , Bateman-House A . Single-patient expanded access requests: IRB professionals' experiences and perspectives. AJOB Empir Bioeth. 2019 Apr-Jun; 10(2):88–99. doi: 10.1080/23294515.2019.1577192. Epub 2019 Apr 9. PMID: 30964737.

Chapman CR , Moch KI , McFadyen A , Kearns L , Watson T , Furlong P , Bateman-House A . (2019). What compassionate use means for gene therapies. Nat Biotechnol. Apr; 37(4):352–355. doi: 10.1038/s41587-019-0081-7.

Chapman CR , Belli H , Leach D , Shah L , Bateman-House A . (2021). A survey of pediatric hematologists/oncologists' perspectives on single patient Expanded Access and Right to Try. Med Access Point Care. 5. 239920262110059. 10.1177/23992026211005991.

Project Facilitate: https://www.fda.gov/about-fda/oncology-center-excellence/project-facilitate. Accessed Sep 3, 2021.

Kang S , Chang S , Ross JS , Miller JE . 2021. Implementation of 21st Century Cures Act expanded access policies requirements. Clin Pharmacol Ther. Aug 24. doi:10.1002/cpt.2401. Online ahead of print.

Moerdler S , Zhang L , Gerasimov E , Zhu C , Wolinsky T , Roth M , Goodman N , Weiser DA . (2019). Physician perspectives on compassionate use in pediatric oncology. Pediatr Blood Cancer. Mar; 66(3):e27545. doi: 10.1002/pbc.27545. Epub 2018 Nov 8. PMID: 30408307.

97th Congress (1981-1983). Public Law 97-414. https://www.fda.gov/media/99546/download. Accessed Sep 13, 2021.

U.S. Food and Drug Administration. (2016, updated 2017). Expanded access to investigational drugs for treatment use—questions and answers: guidance for industry. https://www.fda.gov/media/85675/download. Accessed Aug 28, 2021.

Kearns L , Chapman CR , Moch KI , Caplan AL , Watson T , McFadyen A , Furlong P , Bateman-House A . (2021). Gene therapy companies have an ethical obligation to develop expanded access policies. Mol Ther. Apr 7; 29(4):1367–1369. doi: 10.1016/j.ymthe.2021.03.008. Epub 2021 Mar 13. PMID: 33714373; PMCID: PMC8058481.

McBride Folkers K , Bateman-House A , Robertson C . (2020). Wake Forest Law Rev. Vol11:1.

Kim J , Hu C , El Achkar C Moufawad , Black LE , Douville J , Larson A , Pendergast MK , Goldkind SF , Eunjung AL , et al. (2019). Patient-customized oligonucleotide therapy for a rare genetic disease. N Engl J Med, 381(17):1644–1652.

Hayden EC . This girl's dramatic story shows hyper-personalized medicine is possible—and costly. https://www.technologyreview.com/2019/10/09/102551/this-girls-dramatic-story-shows-hyper-personalized-medicine-is-possibleand-costly/. Accessed Sep 4, 2021.

Bateman-House A , Kearns L .(forthcoming) The Ethics of N=1 Development for Rare Diseases. Nucleic Acid Ther.

Snyder J , Bateman-House A , Turner L . Is right to try being tried? Using crowdfunding data to better understand usage of nontrial pre-approval access pathways. (2020). Regen Med. Aug; 15(8):1979-1985. doi: 10.2217/rme-2020-0043. Epub 2020 Oct 7. PMID: 33023369.

Kenworthy NJ . Crowdfunding and global health disparities: an exploratory conceptual and empirical analysis. (2019) Global Health. Nov 28; 15(Suppl 1):71. doi: 10.1186/s12992-019-0519-1. PMID: 31775896; PMCID: PMC6882318.