Economic Evaluation of Stem Cell Therapies in Neurological Diseases: A Systematic Review

Anjali Nagpal, Rachel Milte, Susan W. Kim, Susan Hillier, Monica A. Hamilton-Bruce, Julie Ratcliffe, Simon A. Koblar

Research output: Contribution to journalReview article

Abstract

Objectives: To examine economic evaluation studies of stem cell therapies (SCTs) in neurological disorders and to provide an overview of the quality of the economic evidence available on this topic. Methods: The review examined studies that performed an economic evaluation of the use of stem cells in adult patients with neurological diseases and that were published in English during the period 2007 to 2017. Data analyzed and reported included study population, disease indication, main analytical approaches for the economic analysis and perspective, key assumptions made or tested in sensitivity analyses, cost outcomes, estimates of incremental cost effectiveness, and approaches to quantifying decision uncertainty. Results: A total of three studies reporting on the findings of the economic evaluation of the use of SCT in stroke, Parkinson disease, and secondary progressive multiple sclerosis, respectively, were identified. All three studies conducted a cost-utility analysis using decision-analytic models and reported an incremental cost per quality-adjusted life-years gained (incremental cost-effectiveness ratio) versus standard care. These studies reported meaningful cost savings in stroke, Parkinson disease, and secondary progressive multiple sclerosis in the base-case scenarios. Conclusions: Despite significant progress in clinical research in the use of SCT in neurological diseases, economic evaluation of these therapies is still at a nascent stage. Given the early stage of research inputs (clinical and cost outcomes data) into the models per se, further research is urgently needed to enable meaningful assessment of the cost effectiveness of these advanced therapies and to ensure sustainable access for population groups most likely to benefit in clinical practice.

LanguageEnglish
Pages254-262
Number of pages9
JournalValue in Health
Volume22
Issue number2
DOIs
Publication statusPublished - 1 Feb 2019

Keywords

  • economic evaluation
  • neurological disease
  • stem cells
  • stroke

ASJC Scopus subject areas

  • Health Policy
  • Public Health, Environmental and Occupational Health

Cite this

Nagpal, A., Milte, R., Kim, S. W., Hillier, S., Hamilton-Bruce, M. A., Ratcliffe, J., & Koblar, S. A. (2019). Economic Evaluation of Stem Cell Therapies in Neurological Diseases: A Systematic Review. Value in Health, 22(2), 254-262. https://doi.org/10.1016/j.jval.2018.07.878
Nagpal, Anjali ; Milte, Rachel ; Kim, Susan W. ; Hillier, Susan ; Hamilton-Bruce, Monica A. ; Ratcliffe, Julie ; Koblar, Simon A. / Economic Evaluation of Stem Cell Therapies in Neurological Diseases : A Systematic Review. In: Value in Health. 2019 ; Vol. 22, No. 2. pp. 254-262.
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Nagpal, A, Milte, R, Kim, SW, Hillier, S, Hamilton-Bruce, MA, Ratcliffe, J & Koblar, SA 2019, 'Economic Evaluation of Stem Cell Therapies in Neurological Diseases: A Systematic Review', Value in Health, vol. 22, no. 2, pp. 254-262. https://doi.org/10.1016/j.jval.2018.07.878

Economic Evaluation of Stem Cell Therapies in Neurological Diseases : A Systematic Review. / Nagpal, Anjali; Milte, Rachel; Kim, Susan W.; Hillier, Susan; Hamilton-Bruce, Monica A.; Ratcliffe, Julie; Koblar, Simon A.

In: Value in Health, Vol. 22, No. 2, 01.02.2019, p. 254-262.

Research output: Contribution to journalReview article

TY - JOUR

T1 - Economic Evaluation of Stem Cell Therapies in Neurological Diseases

T2 - Value in Health

AU - Nagpal, Anjali

AU - Milte, Rachel

AU - Kim, Susan W.

AU - Hillier, Susan

AU - Hamilton-Bruce, Monica A.

AU - Ratcliffe, Julie

AU - Koblar, Simon A.

PY - 2019/2/1

Y1 - 2019/2/1

N2 - Objectives: To examine economic evaluation studies of stem cell therapies (SCTs) in neurological disorders and to provide an overview of the quality of the economic evidence available on this topic. Methods: The review examined studies that performed an economic evaluation of the use of stem cells in adult patients with neurological diseases and that were published in English during the period 2007 to 2017. Data analyzed and reported included study population, disease indication, main analytical approaches for the economic analysis and perspective, key assumptions made or tested in sensitivity analyses, cost outcomes, estimates of incremental cost effectiveness, and approaches to quantifying decision uncertainty. Results: A total of three studies reporting on the findings of the economic evaluation of the use of SCT in stroke, Parkinson disease, and secondary progressive multiple sclerosis, respectively, were identified. All three studies conducted a cost-utility analysis using decision-analytic models and reported an incremental cost per quality-adjusted life-years gained (incremental cost-effectiveness ratio) versus standard care. These studies reported meaningful cost savings in stroke, Parkinson disease, and secondary progressive multiple sclerosis in the base-case scenarios. Conclusions: Despite significant progress in clinical research in the use of SCT in neurological diseases, economic evaluation of these therapies is still at a nascent stage. Given the early stage of research inputs (clinical and cost outcomes data) into the models per se, further research is urgently needed to enable meaningful assessment of the cost effectiveness of these advanced therapies and to ensure sustainable access for population groups most likely to benefit in clinical practice.

AB - Objectives: To examine economic evaluation studies of stem cell therapies (SCTs) in neurological disorders and to provide an overview of the quality of the economic evidence available on this topic. Methods: The review examined studies that performed an economic evaluation of the use of stem cells in adult patients with neurological diseases and that were published in English during the period 2007 to 2017. Data analyzed and reported included study population, disease indication, main analytical approaches for the economic analysis and perspective, key assumptions made or tested in sensitivity analyses, cost outcomes, estimates of incremental cost effectiveness, and approaches to quantifying decision uncertainty. Results: A total of three studies reporting on the findings of the economic evaluation of the use of SCT in stroke, Parkinson disease, and secondary progressive multiple sclerosis, respectively, were identified. All three studies conducted a cost-utility analysis using decision-analytic models and reported an incremental cost per quality-adjusted life-years gained (incremental cost-effectiveness ratio) versus standard care. These studies reported meaningful cost savings in stroke, Parkinson disease, and secondary progressive multiple sclerosis in the base-case scenarios. Conclusions: Despite significant progress in clinical research in the use of SCT in neurological diseases, economic evaluation of these therapies is still at a nascent stage. Given the early stage of research inputs (clinical and cost outcomes data) into the models per se, further research is urgently needed to enable meaningful assessment of the cost effectiveness of these advanced therapies and to ensure sustainable access for population groups most likely to benefit in clinical practice.

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KW - neurological disease

KW - stem cells

KW - stroke

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U2 - 10.1016/j.jval.2018.07.878

DO - 10.1016/j.jval.2018.07.878

M3 - Review article

VL - 22

SP - 254

EP - 262

JO - Value in Health

JF - Value in Health

SN - 1098-3015

IS - 2

ER -

Nagpal A, Milte R, Kim SW, Hillier S, Hamilton-Bruce MA, Ratcliffe J et al. Economic Evaluation of Stem Cell Therapies in Neurological Diseases: A Systematic Review. Value in Health. 2019 Feb 1;22(2):254-262. https://doi.org/10.1016/j.jval.2018.07.878