IDEAS home Printed from https://ideas.repec.org/a/spr/pharme/v40y2022i1d10.1007_s40273-021-01115-5.html
   My bibliography  Save this article

Systematic Literature Review to Identify Utility Values in Patients with Spinal Muscular Atrophy (SMA) and Their Caregivers

Author

Listed:
  • C. Simone Sutherland

    (F. Hoffmann-La Roche Ltd)

  • Pollyanna Hudson

    (Mtech Access Limited)

  • Stephen Mitchell

    (Mtech Access Limited)

  • Noman Paracha

    (F. Hoffmann-La Roche Ltd)

Abstract

Background Spinal muscular atrophy (SMA) is a progressive neuromuscular disorder that has a substantial impact on health-related quality of life for patients with SMA and their caregivers. Utility values (‘utilities’) are used in health economic analyses to incorporate individual or societal perspectives regarding the desirability of health outcomes such as a certain health state or change in health states over time. Objectives The primary objective of this systematic literature review (SLR) was to identify published utilities associated with patients with SMA and their caregivers and to determine the extent to which Health Technology Assessment (HTA) requirements of methods used to generate utilities are met. A secondary objective was to broaden the scope to identify utilities associated with other (non-SMA) neuromuscular disorders. Methods A comprehensive search to capture published utilities associated with patients with SMA and their caregivers was performed in 2019 and updated in 2021 using several electronic databases in addition to supplementary sources. As we anticipated that few published utilities associated with SMA would be identified, the search also captured utilities for other (non-SMA) neuromuscular disorders that may serve as useful surrogate values for studies of SMA and other rare diseases. Electronic searches were performed in Embase, MEDLINE, Evidence-Based Medicine Reviews, and EconLit via the Ovid platform and were supplemented by searches of the grey literature (reference lists, conference proceedings, global HTA body websites, and other relevant sources). Study eligibility criteria were based on the population, interventions, comparators, and outcomes (PICO) framework. The quality of the full-text publications was assessed using a checklist based on UK National Institute for Health and Care Excellence technical support documents. Results In total, 14 publications that reported SMA-related patient or caregiver utilities or disutilities met the eligibility criteria to be included in the SLR; the included studies demonstrate the substantial health-related quality-of-life burden of SMA on both patients with SMA and their caregivers. A variety of preference-based measures were used to derive utilities for patients with SMA and their caregivers. Different methods for collecting utility data included patient and proxy assessment of health states using questionnaires, vignette methodologies, structured forms of expert elicitation, and mapped data from results of clinical trials. A range of utilities was reported from both patient- and proxy-reported data, which reflects the degree of disability associated with early- and later-onset SMA. Methods for deriving utilities were assessed with respect to three reference cases from HTA bodies in the UK, the USA, and Canada. None of the 14 publications met the requirements of all three HTA bodies because of differing tariff requirements between countries; one study met the requirements of HTA bodies in Canada and the UK. Also, six studies did not report the method of valuation, which precluded analysis with respect to the HTA reference cases. Conclusions This SLR provides a comprehensive repository of the currently available utilities relevant to patients with SMA and their caregivers. This SLR provides recommendations for establishing consensus on the approach to generating utility values for the SMA patient population and their caregivers for health economic decisions.

Suggested Citation

  • C. Simone Sutherland & Pollyanna Hudson & Stephen Mitchell & Noman Paracha, 2022. "Systematic Literature Review to Identify Utility Values in Patients with Spinal Muscular Atrophy (SMA) and Their Caregivers," PharmacoEconomics, Springer, vol. 40(1), pages 39-67, April.
    • Handle: RePEc:spr:pharme:v:40:y:2022:i:1:d:10.1007_s40273-021-01115-5
    DOI: 10.1007/s40273-021-01115-5
    as

    Download full text from publisher

    File URL: http://link.springer.com/10.1007/s40273-021-01115-5
    File Function: Abstract
    Download Restriction: Access to the full text of the articles in this series is restricted.
    File URL: https://libkey.io/10.1007/s40273-021-01115-5?utm_source=ideas
    LibKey link: if access is restricted and if your library uses this service, LibKey will redirect you to where you can use your library subscription to access this item
    ---><---

    As the access to this document is restricted, you may want to search for a different version of it.

    References listed on IDEAS

    as
    1. Patrick W. Sullivan & Vahram Ghushchyan, 2006. "Preference-Based EQ-5D Index Scores for Chronic Conditions in the United States," Medical Decision Making, , vol. 26(4), pages 410-420, July.
    2. Drummond, Michael F. & Sculpher, Mark J. & Claxton, Karl & Stoddart, Greg L. & Torrance, George W., 2015. "Methods for the Economic Evaluation of Health Care Programmes," OUP Catalogue, Oxford University Press, edition 4, number 9780199665884.
    3. Chris Sampson;Martina Garau, 2019. "How Should We Measure Quality of Life Impact in Rare Disease? Recent Learnings in Spinal Muscular Atrophy," Briefing 002146, Office of Health Economics.
    4. Luz María Peña-Longobardo & Isaac Aranda-Reneo & Juan Oliva-Moreno & Svenja Litzkendorf & Isabelle Durand-Zaleski & Eduardo Tizzano & Julio López-Bastida, 2020. "The Economic Impact and Health-Related Quality of Life of Spinal Muscular Atrophy. An Analysis across Europe," IJERPH, MDPI, vol. 17(16), pages 1-12, August.
    Full references (including those not matched with items on IDEAS)

    Most related items

    These are the items that most often cite the same works as this one and are cited by the same works as this one.
    1. Andrew J. Palmer & Julie A. Campbell & Barbara de Graaff & Nancy Devlin & Hasnat Ahmad & Philip M Clarke & Mingsheng Chen & Lei Si, 2021. "Population norms for quality adjusted life years for the United States of America, China, the United Kingdom and Australia," Health Economics, John Wiley & Sons, Ltd., vol. 30(8), pages 1950-1977, August.
    2. Irina Pokhilenko & Luca M. M. Janssen & Aggie T. G. Paulus & Ruben M. W. A. Drost & William Hollingworth & Joanna C. Thorn & Sian Noble & Judit Simon & Claudia Fischer & Susanne Mayer & Luis Salvador-, 2023. "Development of an Instrument for the Assessment of Health-Related Multi-sectoral Resource Use in Europe: The PECUNIA RUM," Applied Health Economics and Health Policy, Springer, vol. 21(2), pages 155-166, March.
    3. Chiranjeev Sanyal & Don Husereau, 2020. "Systematic Review of Economic Evaluations of Services Provided by Community Pharmacists," Applied Health Economics and Health Policy, Springer, vol. 18(3), pages 375-392, June.
    4. Andrew J. Mirelman & Miqdad Asaria & Bryony Dawkins & Susan Griffin & Richard Cookson & Peter Berman, 2020. "Fairer Decisions, Better Health for All: Health Equity and Cost-Effectiveness Analysis," World Scientific Book Chapters, in: Paul Revill & Marc Suhrcke & Rodrigo Moreno-Serra & Mark Sculpher (ed.), Global Health Economics Shaping Health Policy in Low- and Middle-Income Countries, chapter 4, pages 99-132, World Scientific Publishing Co. Pte. Ltd..
    5. Christopher M Doran & Irina Kinchin, 2020. "Economic and epidemiological impact of youth suicide in countries with the highest human development index," PLOS ONE, Public Library of Science, vol. 15(5), pages 1-11, May.
    6. Boniface Oyugi & Olena Nizalova & Sally Kendall & Stephen Peckham, 2024. "Does a free maternity policy in Kenya work? Impact and cost–benefit consideration based on demographic health survey data," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 25(1), pages 77-89, February.
    7. Muchandifunga Trust Muchadeyi & Karla Hernandez-Villafuerte & Gian Luca Tanna & Rachel D. Eckford & Yan Feng & Michela Meregaglia & Tessa Peasgood & Stavros Petrou & Jasper Ubels & Michael Schlander, 2024. "Quality Appraisal in Systematic Literature Reviews of Studies Eliciting Health State Utility Values: Conceptual Considerations," PharmacoEconomics, Springer, vol. 42(7), pages 767-782, July.
    8. Lili Wang & Lei Si & Fiona Cocker & Andrew J. Palmer & Kristy Sanderson, 2018. "A Systematic Review of Cost-of-Illness Studies of Multimorbidity," Applied Health Economics and Health Policy, Springer, vol. 16(1), pages 15-29, February.
    9. Etienne Nédellec & Judith Pineau & Patrice Prognon & Nicolas Martelli, 2018. "Level of Evidence in Economic Evaluations of Left Atrial Appendage Closure Devices: A Systematic Review," Applied Health Economics and Health Policy, Springer, vol. 16(6), pages 793-802, December.
    10. Qi Cao & Erik Buskens & Hans L. Hillege & Tiny Jaarsma & Maarten Postma & Douwe Postmus, 2019. "Stratified treatment recommendation or one-size-fits-all? A health economic insight based on graphical exploration," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 20(3), pages 475-482, April.
    11. Hensher, Martin & Canny, Ben & Zimitat, Craig & Campbell, Julie & Palmer, Andrew, 2020. "Health care, overconsumption and uneconomic growth: A conceptual framework," Social Science & Medicine, Elsevier, vol. 266(C).
    12. Dongzhe Hong & Lei Si & Minghuan Jiang & Hui Shao & Wai-kit Ming & Yingnan Zhao & Yan Li & Lizheng Shi, 2019. "Cost Effectiveness of Sodium-Glucose Cotransporter-2 (SGLT2) Inhibitors, Glucagon-Like Peptide-1 (GLP-1) Receptor Agonists, and Dipeptidyl Peptidase-4 (DPP-4) Inhibitors: A Systematic Review," PharmacoEconomics, Springer, vol. 37(6), pages 777-818, June.
    13. Werner Brouwer & Kaya Verbooy & Renske Hoefman & Job Exel, 2023. "Production Losses due to Absenteeism and Presenteeism: The Influence of Compensation Mechanisms and Multiplier Effects," PharmacoEconomics, Springer, vol. 41(9), pages 1103-1115, September.
    14. Simon Pol & Paula Rojas Garcia & Fernando Antoñanzas Villar & Maarten J. Postma & Antoinette D. I. Asselt, 2021. "Health-Economic Analyses of Diagnostics: Guidance on Design and Reporting," PharmacoEconomics, Springer, vol. 39(12), pages 1355-1363, December.
    15. Paul Revill & Simon Walker & Valentina Cambiano & Andrew Phillips & Mark J Sculpher, 2018. "Reflecting the real value of health care resources in modelling and cost-effectiveness studies—The example of viral load informed differentiated care," PLOS ONE, Public Library of Science, vol. 13(1), pages 1-13, January.
    16. Kim Edmunds & Penny Reeves & Paul Scuffham & Daniel A. Galvão & Robert U. Newton & Mark Jones & Nigel Spry & Dennis R. Taaffe & David Joseph & Suzanne K. Chambers & Haitham Tuffaha, 2020. "Cost-Effectiveness Analysis of Supervised Exercise Training in Men with Prostate Cancer Previously Treated with Radiation Therapy and Androgen-Deprivation Therapy," Applied Health Economics and Health Policy, Springer, vol. 18(5), pages 727-737, October.
    17. Knott, R. & Lorgelly, P. & Black, N. & Hollingsworth, B., 2016. "Differential item functioning in the EQ-5D: An exploratory analysis using anchoring vignettes," Health, Econometrics and Data Group (HEDG) Working Papers 16/14, HEDG, c/o Department of Economics, University of York.
    18. Omar B. Da'ar & Abdi A. Gele, 2023. "Tuberculosis in a weak health system, conflict and fragile zone: The monetary value of human lives lost associated with deaths of persons older than 14 years in Somalia," International Journal of Health Planning and Management, Wiley Blackwell, vol. 38(1), pages 53-68, January.
    19. Fredrik Salvesen Haukaas & Audun Ohna & Tania Krivasi, 2018. "Cost-Effectiveness of Obinutuzumab in Combination with Bendamustine Followed by Obinutuzumab Maintenance versus Bendamustine Alone in Treatment of Patients with Rituximab-Refractory Follicular Lymphom," Applied Health Economics and Health Policy, Springer, vol. 16(4), pages 569-577, August.
    20. Thai, Thao & Lancsar, Emily & Spinks, Jean & Freeman, Christopher & Chen, Gang, 2024. "Understanding Australian pharmacy degree holders’ job preferences through the lens of motivation-hygiene theory," Social Science & Medicine, Elsevier, vol. 348(C).

    More about this item

    Statistics

    Access and download statistics

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:spr:pharme:v:40:y:2022:i:1:d:10.1007_s40273-021-01115-5. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    If CitEc recognized a bibliographic reference but did not link an item in RePEc to it, you can help with this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: Sonal Shukla or Springer Nature Abstracting and Indexing (email available below). General contact details of provider: http://www.springer.com .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.