150
Views
0
CrossRef citations to date
0
Altmetric
Research Article

Cost/effectiveness of elivaldogene autotemcel vs. hematopoietic stem cells transplantation in patients with cerebral adrenoleukodystrophy (CALD)

, &
Article: 2342888 | Received 13 Dec 2023, Accepted 09 Apr 2024, Published online: 18 Apr 2024

References

  • Berger J, Forss-Petter S, Eichler FS. Pathophysiology of X-linked adrenoleukodystrophy. Biochimie. 2014;98(100):1–10. doi: 10.1016/j.biochi.2013.11.023.
  • Berger J, Gärtner J. X-linked adrenoleukodystrophy: clinical, biochemical and pathogenetic aspects. Biochim Biophys Acta. 2006;1763(12):1721–1732. doi: 10.1016/j.bbamcr.2006.07.010.
  • Jardim LB, da Silva ACF, Blank D, et al. X-linked adrenoleukodystrophy: clinical course and minimal incidence in South Brazil. Brain Dev. 2010;32(3):180–190. doi: 10.1016/j.braindev.2009.02.002.
  • Zhu J, Eichler F, Biffi A, et al. The changing face of adrenoleukodystrophy. Endocr Rev. 2020;41(4):577–593. doi: 10.1210/endrev/bnaa013.
  • Raymond GV, Aubourg P, Paker A, et al. Survival and functional outcomes in boys with cerebral adrenoleukodystrophy with and without hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2019;25(3):538–548. doi: 10.1016/j.bbmt.2018.09.036.
  • Miller WP, Rothman SM, Nascene D, et al. Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report. Blood. 2011;118(7):1971–1978. doi: 10.1182/blood-2011-01-329235.
  • Eichler F, Duncan C, Musolino PL, et al. Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy. N Engl J Med. 2017;377(17):1630–1638. doi: 10.1056/NEJMoa1700554.
  • Keam SJ. Elivaldogene autotemcel: first approval. Mol Diagn Ther. 2021;25(6):803–809. doi: 10.1007/s40291-021-00555-1.
  • Larkin HD. Autologous gene therapy approved for childhood CALD. JAMA. 2022;328(17):1679–1680. doi: 10.1001/jama.2022.19800.
  • Skysona | European Medicines Agency [Internet]; [cited 2024 Feb 24]. Available from: https://www.ema.europa.eu/en/medicines/human/EPAR/skysona#ema-inpage-item-news-on
  • Schreiber-Katz O, Klug C, Thiele S, et al. Comparative cost of illness analysis and assessment of health care burden of Duchenne and Becker muscular dystrophies in Germany. Orphanet J Rare Dis. 2014;9(1):210. doi: 10.1186/s13023-014-0210-9.
  • Winquist E, Bell CM, Clarke JTR, et al. An evaluation framework for funding drugs for rare diseases. Value Health. 2012;15(6):982–986. doi: 10.1016/j.jval.2012.06.009.
  • Karnon J, Haji Ali Afzali H. When to use discrete event simulation (DES) for the economic evaluation of health technologies? A review and critique of the costs and benefits of DES. Pharmacoeconomics. 2014;32(6):547–558. doi: 10.1007/s40273-014-0147-9.
  • Gutić M, Milosavljević MN, Janković SM. Cost-effectiveness of miglustat versus symptomatic therapy of Niemann–Pick disease type C. Int J Clin Pharm. 2022;44(6):1442–1453. doi: 10.1007/s11096-022-01491-8.
  • Republic Health Insurance Fund of Serbia. Rulebook on the list of medicines prescribed and issued at the expense of compulsory health insurance funds. Off Gaz RS. 440/2022, 144/2022, 40/2023, 57/2023, 66/2023, 67/2023, 86/2023, 104/2023 and 17/2024.
  • The Government of the Republic of Serbia. Decision on the highest prices of prescription medicines for human use. Off Gaz RS. 48/2021.
  • Eichler F, Duncan C, Orchard P, et al. Disease stabilization following treatment with elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy for the treatment of cerebral adrenoleukodystrophy: interim results from phase 2/3 (ALD-102) and phase 3 (ALD-104) studies (2064). Neurology. 2021;96(15 Supplement).
  • Suzuki Y, Imamura A, Shimozawa N, et al. The clinical course of childhood and adolescent adrenoleukodystrophy before and after Lorenzo’s oil. Brain Dev. 2001;23(1):30–33. doi: 10.1016/s0387-7604(00)00196-0.
  • Kühl JS, Suarez F, Gillett GT, et al. Long-term outcomes of allogeneic haematopoietic stem cell transplantation for adult cerebral X-linked adrenoleukodystrophy. Brain. 2017;140(4):953–966. doi: 10.1093/brain/awx016.
  • Pierpont EI, Nascene DR, Shanley R, et al. Neurocognitive benchmarks following transplant for emerging cerebral adrenoleukodystrophy. Neurology. 2020;95(5):e591–e600. doi: 10.1212/WNL.0000000000009929.
  • Pereira FS, Matte U, Habekost CT, et al. Mutations, clinical findings and survival estimates in South American patients with X-linked adrenoleukodystrophy. PLoS One. 2012;7(3):e34195. doi: 10.1371/journal.pone.0034195.
  • Matos T, Costa C, Araújo AN, et al. Clinical course and endocrine dysfunction in X-linked adrenoleukodystrophy: a case series. Endocrinol Diabetes Nutr. 2023;70(6):421–428. doi: 10.1016/j.endinu.2022.01.007.
  • Kanakis G, Kaltsas G. Adrenal Insufficiency Due to X-Linked Adrenoleukodystrophy. [Updated 2018 Oct 12]. In: Feingold KR, Anawalt B, Blackman MR, et al., editors. Endotext [Internet]. South Dartmouth (MA): MDText.com, Inc.; 2000-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK278944/.
  • Tordrup D, Smith R, Kamenov K, et al. WHO HEAR group. Global return on investment and cost-effectiveness of WHO’s HEAR interventions for hearing loss: a modelling study. Lancet Glob Health. 2022;10(1):e52–e62. doi: 10.1016/S2214-109X(21)00447-2.
  • Lloyd A, Nafees B, Gavriel S, et al. Health utility values associated with diabetic retinopathy. Diabet Med. 2008;25(5):618–624. doi: 10.1111/j.1464-5491.2008.02430.x.
  • Fattori A, Neri L, Aguglia E, et al. Estimating the impact of workplace bullying: humanistic and economic burden among workers with chronic medical conditions. Biomed Res Int. 2015;2015:708908–708912. doi: 10.1155/2015/708908.
  • Chittrakul J, Siviroj P, Sungkarat S, et al. Multi-system physical exercise intervention for fall prevention and quality of life in pre-frail older adults: a randomized controlled trial. Int J Environ Res Public Health. 2020;17(9):3102. doi: 10.3390/ijerph17093102.
  • Ozkan Y. Child’s quality of life and mother’s burden in spastic cerebral palsy: a topographical classification perspective. J Int Med Res. 2018;46(8):3131–3137. doi: 10.1177/0300060518772758.
  • Zhang P, Brown MB, Bilik D, et al. Health utility scores for people with type 2 diabetes in U.S. managed care health plans: results from translating research into action for diabetes (TRIAD). Diabetes Care. 2012;35(11):2250–2256. doi: 10.2337/dc11-2478.
  • López-Bastida J, Perestelo-Pérez L, Montón-Alvarez F, et al. Social economic costs and health-related quality of life in patients with degenerative cerebellar ataxia in Spain. Mov Disord. 2008;23(2):212–217. doi: 10.1002/mds.21798.
  • McDaid D, Park AL, Chadha S. Estimating the global costs of hearing loss. Int J Audiol. 2021;60(3):162–170. doi: 10.1080/14992027.2021.1883197.
  • Marques AP, Ramke J, Cairns J, et al. The economics of vision impairment and its leading causes: a systematic review. EClinicalMedicine. 2022;46:101354. doi: 10.1016/j.eclinm.2022.101354.
  • Shahat ARS, Greco G. The economic costs of childhood disability: a literature review. Int J Environ Res Public Health. 2021;18(7):3531. doi: 10.3390/ijerph18073531.
  • de Lissovoy G, Matza LS, Green H, et al. Cost-effectiveness of intrathecal baclofen therapy for the treatment of severe spasticity associated with cerebral palsy. J Child Neurol. 2007;22(1):49–59. doi: 10.1177/0883073807299976.
  • Beecham J, O’Neill T, Goodman R. Supporting young adults with hemiplegia: services and costs. Health Soc Care Community. 2001;9(1):51–59. doi: 10.1046/j.1365-2524.2001.00279.x.
  • Happich M, John J, Stamenitis S, et al. The quality of life and economic burden of neuropathy in diabetic patients in Germany in 2002–results from the diabetic microvascular complications (DIMICO) study. Diabetes Res Clin Pract. 2008;81(2):223–230. doi: 10.1016/j.diabres.2008.03.019.
  • Liu A. A $3M gene therapy: bluebird bio breaks its own pricing record with FDA approval of Skysona [Internet]. Fierce Pharma. 2022; [cited 2023 Mar 30]. Available from: https://www.fiercepharma.com/pharma/3m-gene-therapy-bluebird-breaks-own-record-fda-approval-skysona
  • Statistical Office of the Republic of Serbia 2022. [Internet]. [cited 2023 Apr 04]. Available from: https://data.stat.gov.rs/Home/Result/09020101?languageCode=sr-Cyrl
  • National Bank of Serbia. Interest rates [Internet]. [cited 2023 Apr 04]. Available from: https://www.nbs.rs/sr/ciljevi-i-funkcije/monetarna-politika/kamatne-stope/
  • Republic Health Insurance Fund of Serbia Rulebook on the prices of health services at the secondary and tertiary levels of health care. Off Gaz RS. 88/2021, 97/2021, 109/2021, 132/2021, 47/2022, 82/2022, 123/2022, 128/2022, 1/2023, 59/2023, 96/2023 and 15/2024.
  • National Bank of Serbia. Average exchange rates [Internet]. [cited 2024 Mar 29]. Available from: https://nbs.rs/en/finansijsko_trziste/medjubankarsko-devizno-trziste/kursna-lista/prosecni-kursevi/index.html
  • Gutić M, Milosavljević MN, Safiye T, et al. Economic analysis of cerliponase alfa for treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Expert Rev Pharmacoecon Outcomes Res. 2023;23(5):561–570. doi: 10.1080/14737167.2023.2197213.
  • Eunethta. PTJA17 – Core Submission Dossier for Elivaldogene autotemcel (eli-cel) for treatment of Cerebral Adrenoleukodystrophy (CALD) [Internet]. [cited 2023 May 20]. Available from: https://www.eunethta.eu/wp-content/uploads/2021/08/PTJA17-Eli-cel-Core-Submission-Dossier-v1.0.pdf
  • Kuratsubo I, Suzuki Y, Shimozawa N, et al. Parents of childhood X-linked adrenoleukodystrophy: high risk for depression and neurosis. Brain Dev. 2008;30(7):477–482. doi: 10.1016/j.braindev.2007.12.012.
  • Paramore C, Dietz A, Boulanger L. PND48 costs of episodes of care for allogeneic transplant for the the treatment of cerebral adrenoleukodystrophy in the U.S. Value Health. 2020;23(suppl. 1):S268. doi: 10.1016/j.jval.2020.04.941.
  • Kanters TA, van der Ploeg AT, Kruijshaar ME, et al. Cost- effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease. Orphanet J Rare Dis. 2017;12(1):179. doi: 10.1186/s13023-017-0731-0.
  • Iskrov G, Stefanov R. Criteria for drug reimbursement decision-making: an emerging public health challenge in Bulgaria. Balkan Med J. 2016;33(1):27–35. doi: 10.5152/balkanmedj.2015.15185.