Publications

Sidhu MK, Miller B, Jensen I, Rabiner P. 2025. Abstract EE171: Conceptual framework for a de novo cost-effectiveness model in glycogen storage disease type Ia (GSDIa). Value in Health 28(6-Sup1):S93-S94; doi: 10.1016/j.jval.2025.04.463.

Duong T, Haselkorn T, Miller B, Coats J, Jensen I, Ward E, Wood M, Graham RJ, Servais L. 2025. A real-world analysis of the impact of X-linked myotubular myopathy on caregivers in the United States. Orphanet J Rare Dis 20(1):224. doi: 10.1186/s13023-025-03583-w. PMID: 40355957.

Haselkorn T, Hughes W, Schara-Schmidt U, Lennox A, Roca A, Miller B, Jensen I, Solomon F, et al. 2024. Abstract PCR257: Real-world impact of x-linked myotubular myopathy (XLMTM) on caregivers in the United Kingdom (UK), Germany, and Spain. Value in Health 27(12-Sup):S557; doi: 10.1016/j.val.2024.10.3499.

Olaye A, Bean K, Velikanova R, Wolters S, Miller B, Jensen I, Pang F. Cost-effectiveness of atidarsagene autotemcel (ARSA-CEL) gene therapy for treating metachromatic leukodystrophy (MLD) in Ireland, Belgium, and the Netherlands as a part of the Beneluxa Initiative. Abstract EE693, ISPOR Europe 2024. Value in Health 27(12 Sup):S191; doi: 10.1016/j.jval.2024.10.973. Barcelona, November 2024.

Pang F, Dean R, Jensen I, Bean K, Fields C, Miller B. The cost-effectiveness of atidarsagene autotemcel (ARSACEL) for the treatment of metachromatic leukodystrophy (MLD) in Spain. ISPOR Europe 2024, November 2024.

Bean K, Jones SA, Chakrapani A, Vijay S, Wu T, Church H, Chanson C, Olaye A, Miller B, et al. 2024. Exploring the cost-effectiveness of newborn screening for metachromatic leukodystrophy (MLD) in the UK. Int J Neonatal Screen 10(3):45; doi: 10.3390/ijns10030045. PMID: 39051401.

Bean K, Miller B, Jensen I, Howle K, Wilds A, Walz M, et al. The societal costs of metachromatic leukodystrophy (MLD) in the United States. Abstract EE547, ISPOR EU 2023.

Miller B, Russel-Szymczyk M, Jensen I, Shah A, Alexopoulos T, Herbert A, et al. Cost-consequence of cladribine tablets for the treatment of highly-active relapsing-remitting multiple sclerosis (RRMS) in the UK. Abstract EE658, ISPOR EU 2023.

Majhail NS, Miller B, Dean R, Manghani R, Shin H, Sivaraman S, Maziarz RT. 2023. Hospitalization and healthcare resource utilization of omidubicel-only versus umbilical cord blood transplantation for hematologic malignancies: Secondary analysis from a pivotal phase 3 clinical trial. Transplant Cell Ther 29(12):749.e1-749.e5; doi: 10.1016/j.jtct.2023.09.004. PMID: 37703995.

Bean K, Miller B, Jensen I, Fields C, Pang F. Evaluating the face validity of health state utility values (HSUVS) for metachromatic leukodystrophy (MLD). Abstract EE285, ISPOR 2023.

Bean K, Olaye A, Miller B, Jensen I, Dean R, Pang F. Direct cost analysis associated with the management of patients with metachromatic leukodystrophy (MLD) across nine European countries. Abstract EE408, ISPOR 2023, Boston, MA, May 2023.

Pang F, Dean R, Jensen I, Bean K, Fields C, Miller B. 2023. The cost-effectiveness of OTL-200 for the treatment of metachromatic leukodystrophy (MLD) in the US. Mol Gene Metab 138(2):107258; doi: 10.1016/j.ymgme.2022.107258.

Pang F, Dean R, Jensen I, Tehard B, Roze S, Olaye A, Bean K, Miller B. The cost-effectiveness of atidarsagene autotemcel for the treatment of metachromatic leukodystrophy (MLD) in France. Abstract EE24, ISPOR 2022.

Majhail NS, Miller B, Dean R, Manghani R, Sivaraman S, Galamidi-Cohen E, Maziarz RT. 2021. Hospitalization and healthcare resource use of omidubicel vs cord blood transplantation for hematological malignancies in a global randomized phase III clinical trial. Blood 138(Sup 1):4036; doi: 10.1182/blood-2021-147480.

Pang F, Dean R, Jensen I, Olaye A, Miller B. The cost-effectiveness of OTL-200 for the treatment of metachromatic leukodystrophy (MLD) in the US. Abstract PRO30, ISPOR 2021.

Dean R, Jensen IS, Cyr PL, Miller B, Maru B, Sproule DM, Feltner DE, et al. 2021. An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER). J Mark Access Health Pol 9(1):1889841; doi: 10.1080/20016689.2021.1889841. PMID: 33708361.

Miller B, Yao W, Dean R, Jensen IS, Cyr PL, Slocomb T. 2020. Abstract PMS57: Understanding the impact of XLMTM on parents and caregivers in the US: An analysis of survey results. Value in Health 23(Sup 2):S601; doi: 10.1016/j.jval.2020.08.1188.

Dean R, Arjunji R, Awano H, Igarash A^, Tanaka A, Jensen IS, Cyr PL, Miller B, et al. Cost-effectiveness of onasemnogene abeparvocec (ZOLGENSMA) for spinal muscular atrophy type 1 against nusinersen in Japan. ISPOR 25th Annual International Meeting, Orlando FL, 2020.

Dean R, Miller B, Arjunji R, Awano H, Igarashi A, Tanaka S, Feltner DE, Sproule DM, Jensen I, Dabbous O. 2020. Abstract PMU36: Cost-utility analysis of single dose gene-replacement therapy for spinal muscular atrophy type 1 compared to chronic nusinersen treatment in Japan. Value in Health 23(Sup 1):S239; doi: 10.1016/j.val.2020.04.810.

Miller B, Jensen I, Dean R, Slocomb T, James ES, Beggs AH. 2020. Abstract PRO77: Health resource use in patients with x-linked myotubular myopathy (XLMTM): Data from the RECENSUS study. Value in Health 23(Sup 1):S342; doi: 10.1016/j.jval.2020.04.1298.