Return on Investment from the Prevention of Orphan Diseases in Kuwait
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Keywords

Spinal Muscular Atrophy
Inherited Retinal Disease
Premarital Screen
Preimplantation Genetic Diagnosis
In Vitro Fertilization

How to Cite

Abuhadida, S., Bastaki, L. ., Bash, B. ., & Alhindal, B. (2022). Return on Investment from the Prevention of Orphan Diseases in Kuwait. Annals of Public Health. https://doi.org/10.55085/aph.2022.637

Abstract

Spinal Muscular Atrophy type 1 and RPE65 mutation-associated Inherited Retinal dystrophy are two well-known Orphan diseases for expensive Orphan drugs. Being inheritable disorders, they can be prevented through a program that includes Premarital Genetic Screening to detect the defective gene carriers followed by Preimplantation Genetic Diagnosis to identify healthy gametes and In Vitro Fertilization. We developed a stochastic financial model to assess the Return on Investment over five years of implementing a prevention program to tackle these conditions from the financial perspective of the Ministry of Health in Kuwait. The ROI from the prevention program was shown to be highly cost-saving, with a probabilistic average of 9,710,311 USD (2,930,727 KWD). Every 1 USD or KWD spent on prevention would return 1.5 USD or KWD in savings. Meanwhile, not implementing the prevention program could cost the MOH a probabilistic average of 71,431,037 USD (21,555,325 KWD) within the same period. The findings of this study strongly support the adoption and implementation of the prevention program from the financial perspective of the MOH.

https://doi.org/10.55085/aph.2022.637
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References

Useful information on rare diseases from an EU perspective. [Accessed 2022 Mar 12]. Available from: https://ec.europa.eu/health/ph_information/documents/ev20040705_rd05_en.pdf

Rare Disease Act of 2002, Pub. L. No. 107–280, 116 Stat. 1988 (2002). [Accessed 2022 Mar 12]. Available from: https://history.nih.gov/research/downloads/pl107-280.pdf

Hayashi S, Umeda T. 35 years of Japanese policy on rare diseases. Lancet. 2008;372(9642):889–90. https://doi.org/10.1016/S0140-6736(08)61393-8.

Broekhoff TF, Sweegers CCG, Krijkamp EM, Mantel-Teeuwisse AK, Leufkens HGM, Goettsch WG, et al. Early cost-effectiveness of Onasemnogene Abeparvovec-xioi (Zolgensma) and Nusinersen (Spinraza) treatment for spinal muscular atrophy I in the Netherlands with relapse scenarios. Value Health. 2021;24(6):759–69. https://doi.org/10.0.3.248/j.jval.2020.09.021

Ana S, Jimmy L. Rare Diseases, Diagnosis, Therapies, and Hope. St. Louis, MO: Rare Genomics Institute. 2014;6. [Accessed 2022 Mar 12]. Available from: https://static1.squarespace.com/static/55899403e4b0dbf7728075b1/t/55db714de4b04e4d229c580d/1440444749559/Rare-Diseases-Book-Diagnosis-Therapies-and-Hope-V5-330b.pdf

U.S. Food and Drug Administration. Package Insert-Zolgensma. [Accessed 2022 Mar 12]. Available from: https://www.fda.gov/media/126109/download

Al‐Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, et al. Health Outcomes in spinal muscular atrophy type 1 following AVXS‐101 Gene Replacement therapy. Pediatr Pulmonol. 2018;54(2):179–85. https://doi.org/10.1002/ppul.24203

Johnson S, Buessing M, O’Connell T, Pitluck S, Ciulla TA. Cost-effectiveness of Voretigene Neparvovec-Rzyl vs standard care for RPE65-mediated inherited retinal disease. JAMA Ophthalmol. 2019;137(10):1115. https://doi.org/10.1001/jamaophthalmol.2019.2512

U.S. Food and Drug Administration. Package Insert-Luxturna. [Accessed 2022 Mar 12] Available from: https://www.fda.gov/media/109906/download

Verhaart IE, Robertson A, Wilson IJ, Aartsma-Rus A, Cameron S, Jones CC, et al. Prevalence, incidence and carrier frequency of 5q–linked spinal muscular atrophy – a literature review. Orphanet J Rare Dis. 2017;12(1). https://doi.org/10.1186/s13023-017-0671-8

Sciorio R, Tramontano L, Catt J. Preimplantation Genetic diagnosis (PGD) and genetic testing for aneuploidy (PGT-A): Status and future challenges. Gynecol Endocrinol. 2019;36(1):6–11. https://doi.org/10.1080/09513590.2019.1641194

Ministry of Health in Kuwait. Kuwait Premarital Screening Program. [Accessed 2022 Mar 12]. Available from: https://web.archive.org/web/20130302211614/http://www.moh.gov.kw/tcenter/ENGLISH%20BOOKLET.pdf

Kuwait times newspaper. 2019. [Accessed 2022 Mar 12]. Available from: http://news.kuwaittimes.net/pdf/2019/nov/05/p01.pdf

Cantoni E, Ronchetti E. A robust approach for skewed and heavy-tailed outcomes in the analysis of Health Care Expenditures. J Health Econ. 2006;25(2):198–213. https://doi.org/10.1016/j.jhealeco.2005.04.010

Hu C, Zhou H, Sharma A. Applying beta distribution in analyzing bounded outcome score data. AAPS J. 2020;22(3). https://doi.org/10.1208/s12248-020-00441-4

Kuwait Medical Genetic Center. [Accessed 2022 Mar 12]. Available from: https://www.kmgc.gov.kw/en/

Basille C, Frydman R, Aly AE, Hesters L, Fanchin R, Tachdjian G, et al. Preimplantation Genetic diagnosis: State of the art. Eur J Obstet Gynecol Reprod Biol. 2009;145(1):9–13. https://doi.org/10.1016/j.ejogrb.2009.04.004

Bertram MY, Lauer JA, Stenberg K, Edejer TT. Methods for the economic evaluation of Health Care Interventions for priority setting in the health system: An update from WHO choice. Int J Health Policy Manag. 2021. https://doi.org/10.34172/ijhpm.2020.244

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Copyright (c) 2022 Abuhadida S et al.

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