Chapter 12 242 REFERENCES 1. Darrow JJ, Sarpatwari A, Avorn J, Kesselheim AS. Practical, Legal, and Ethical Issues in Expanded Access to Investigational Drugs. Hamel MB, ed. N Engl J Med. 2015;372(3):279-286. doi:10.1056/ NEJMhle1409465 2. CHMP/ EMEA/27170/2006. Guideline on Compassionate Use of Medicinal Products, Pursuant to Article 83 of Regulation (EC) No 726/2004. Emea/27170/2006. 2007;(October 2006). 3. Authorisation for early access to medicinal products: HAS assessment doctrine. Published online 2021. 4. Federal Agency for Medicines and Health. FAQ - Unmet Medical Need V1.11.; 2022. 5. Medicines and Healthcare products Regulatory Agency. Guidance Apply for the early access to medicines scheme (EAMS). https://www.gov. uk/guidance/apply-for-the-early-access-tomedicines-scheme-eams#fees 6. BASG. Information on Named Patient Use (Heilversuch) in Austria - Definition and Framework.; 2015. 7. Aliu P, Sarp S, Reichenbach R, et al. International Country-Level Trends, Factors, and Disparities in Compassionate Use Access to Unlicensed Products for Patients With Serious Medical Conditions. JAMA Health Forum. 2022;3(4):e220475. doi:10.1001/ jamahealthforum.2022.0475 8. Polak TB, Cucchi DGJ, van Rosmalen J. [Expanded Access in The Netherlands: prescribing unregistered medicine]. Ned Tijdschr Geneeskd. 2021;165:1-5. 9. Zeverijn LJ, van Waalwijk van Doorn-Khosrovani SB, van Roy AAMGP, et al. Harmonising patientaccess programmes: the Dutch DRUG Access Protocol platform. Lancet Oncol. 2022;23(2):198201. doi:10.1016/S1470-2045(21)00707-5 10. Haute Autorité de Santé. Authorisation for Early Access to Medicinal Products: HAS Assessment Doctrine.; 2021. https://www.has-sante. fr/upload/docs/application/pdf/2021-08/ authorisation_for_early_access_to_medicinal_ products_has_assessment_doctrine.pdf 11. Polak TB, Cucchi DG, van Rosmalen J, Uyl-de Groot CA. Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals. BMJ Open. 2022;12(1):e052186. doi:10.1136/bmjopen-2021-052186 12. Chapman CR, Moch KI, McFadyen A, et al. What compassionate use means for gene therapies. Nat Biotechnol. 2019;37(4):352-355. doi:10.1038/s41587-019-0081-7 13. Ferreira CR. The burden of rare diseases. Am J Med Genet A. 2019;179(6):885-892. doi:10.1002/ ajmg.a.61124 14. Kimberly LL, Beuttler MM, Shen M, Caplan AL, Bateman-House A. Pre-approval Access Terminology. TherInnovRegulSci.2017;51(4):494500. doi:10.1177/2168479017696267 15. Office of the Federal Register National Archives and Records Administration. Federal Register Vol. 52, No.105, June 2, 1987.; 1987:2059120694. https://www.govinfo.gov/app/details/ FR-1987-06-02 16. Grein J, Ohmagari N, Shin D, et al. Compassionate Use of Remdesivir for Patients with Severe Covid-19. N Engl J Med. 2020;382(24):23272336. doi:10.1056/NEJMoa2007016 17. Joyner MJ, Carter RE, Senefeld JW, et al. Convalescent Plasma Antibody Levels and the Risk of Death from Covid-19. N Engl J Med. 2021;384(11):1015-1027. doi:10.1056/ NEJMoa2031893 18. Borysowski J, Lewis ACFF, Górski A. Conflicts of interest in oncology expanded access studies. Int J Cancer. 2021;149(10):1809-1816. doi:10.1002/ijc.33733 19. Gore ME, Szczylik C, Porta C, et al. Final results from the large sunitinib global expandedaccess trial in metastatic renal cell carcinoma. Br J Cancer. 2015;113(1):12-19. doi:10.1038/ bjc.2015.196 20. Huemer F, Melchardt T, Jansko B, et al. Durable remissions with venetoclax monotherapy in secondary AML refractory to hypomethylating agents and high expression of BCL-2 and/ or BIM. Eur J Haematol. 2019;102(5):437-441. doi:10.1111/EJH.13218
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