The value-based healthcare approach to haemophilia: Development of outcome measures for the evaluation of care of people with haemophilia.
haemophilia
outcome assessment
patient reported outcome measures
quality indicators
treatment outcome
value-based purchasing
Journal
Haemophilia : the official journal of the World Federation of Hemophilia
ISSN: 1365-2516
Titre abrégé: Haemophilia
Pays: England
ID NLM: 9442916
Informations de publication
Date de publication:
05 Feb 2024
05 Feb 2024
Historique:
revised:
16
10
2023
received:
30
04
2023
accepted:
12
11
2023
medline:
5
2
2024
pubmed:
5
2
2024
entrez:
5
2
2024
Statut:
aheadofprint
Résumé
Considering the advances in haemophilia management and treatment observed in the last decades, a new set of value-based outcome indicators is needed to assess the quality of care and the impact of these medical innovations. The Value-Based Healthcare in Haemophilia project aimed to define a set of clinical outcome indicators (COIs) and patient-reported outcome indicators (PROIs) to assess quality of care in haemophilia in high-income countries with a value-based approach to inform and guide the decision-making process. A Value-based healthcare approach based on the available literature, current guidelines and the involvement of a multidisciplinary group of experts was applied to generate a set of indicators to assess the quality of care of haemophilia. A final list of three COIs and five PROIs was created and validated. The identified COIs focus on two domains: musculoskeletal health and function, and safety. The identified PROIs cover five domains: bleeding frequency, pain, mobility and physical activities, Health-Related Quality of Life and satisfaction. Finally, two composite outcomes, one based on COIs, and one based on PROIs, were proposed as synthetic outcome indicators of quality of care. The presented standard set of health outcome indicators provides the basis for harmonised longitudinal and cross-sectional monitoring and comparison. The implementation of this value-based approach would enable a more robust assessment of quality of care in haemophilia, within a framework of continuous treatment improvements with potential added value for patients. Moreover, proposed COIs and PROIs should be reviewed and updated routinely.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
© 2024 John Wiley & Sons Ltd.
Références
Iorio A, Stonebraker JS, Chambost H, et al. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries. Ann Intern Med. 2019;171(8):540.
Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia. Haemophilia. 2020;26(S6):1-158.
Balkaransingh P, Young G. Novel therapies and current clinical progress in hemophilia A. Therapeutic Adv Hematol. 2018;9:49-61.
Ling G, Nathwani AC, Tuddenham EGD. Recent advances in developing specific therapies for haemophilia. Br J Haematol. 2018;181(2):161-172.
Abrantes JA, Solms A, Garmann D, Nielsen EI, Jönsson S, Karlsson MO. Relationship between factor VIII activity, bleeds, and individual characteristics in severe hemophilia A patients. Haematologica. 2020;105(5):1443-1453.
Petrini P, Valentino LA, Gringeri A, Re WM, Ewenstein B. Individualizing prophylaxis in hemophilia: a review. Expert Rev Hematol. 2015;8(2):237-246.
Hazendonk HCAM, Van Moort I, Mathôt RAA, et al. Setting the stage for individualized therapy in hemophilia: what role can pharmacokinetics play? Blood Rev. 2018;32(4):265-271.
Delavenne X, Ollier E, Lienhart A, Dargaud Y. A new paradigm for personalized prophylaxis for patients with severe haemophilia A. Haemophilia. 2020;26(2):228-235.
Ling G, NathwaniAC TuddenhamEGD. Recent advances in developing specific therapies for haemophilia. Br J Haematol. 2018;181(2):161-172.
Mullard A. FDA approves first haemophilia B gene therapy. Nat Rev Drug Discov. 2022. doi:10.1038/d41573-022-00199-8
Fitch K, Bernstein SJ, Aguilar MD, et al. The RAND/UCLA Appropriateness Method User's Manual. Santa Monica, CA: RAND Corporation. 2001. https://www.rand.org/pubs/monograph_reports/MR1269.html
WHO. Quality of care. Available at: https://www.who.int/health-topics/quality-of-care#tab=tab_1
Donabedian A. The quality of medical care. Science. 1978;200(4344):856-864.
Donabedian A. The quality of care. How can it be assessed? JAMA. 1988;260(12):1743-1748.
Porter ME, Olmsted-Teisberg E. Redefining Health Care: Creating Value-Based Competition on Results. Harvard Business School Press; 2006.
Porter ME. What is value in health care? N Engl J Med. 2010;363(26):2477-2481.
Boulkedid R, Abdoul H, Loustau M, Sibony O, Alberti C. Using and reporting the Delphi method for selecting healthcare quality indicators: a systematic review. PLoS One. 2011;6(6):e20476.
Armstrong PW, Westerhout CM. Composite end points in clinical research: a time for reappraisal. Circulation. 2017;135(23):2299-2307. doi:10.1161/CIRCULATIONAHA.117.026229
Manco-Johnson MJ, Warren BB, Buckner TW, Funk SM, Wang M. Outcome measures in haemophilia: beyond ABR (Annualized Bleeding Rate). Haemophilia. 2021(3):87-95. Suppl.
Strazzabosco M, Cortesi PA, Conti S, et al. Clinical outcome indicators in chronic hepatitis B and C: a primer for value-based medicine in hepatology. Liver Int. 2020;40(1):60-73.
Kolovos S, Bosmans JE, van Dongen JM, et al. Utility scores for different health states related to depression: individual participant data analysis. Qual Life Res. 2017;26(7):1649-1658.
ATS Committee on Proficiency Standards for Clinical Pulmonary Function Laboratories. ATS statement: guidelines for the six-minute walk test. Am J Respir Crit Care Med. 2002;166(1):111-117. Erratum in: Am J Respir Crit Care Med. 2016; 193(10):1185.
Martinoli C, Della Casa Alberighi O, Di Minno G, et al. Development and definition of a simplified scanning procedure and scoring method for Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US). Thromb Haemost. 2013;109(6):1170-1179.
Feldman BM, Funk S, Lundin B, Doria AS, Ljung R, Blanchette V. International Prophylaxis Study Group (IPSG). Musculoskeletal measurement tools from the International Prophylaxis Study Group (IPSG). Haemophilia. 2008;14(Suppl 3):162-169. doi:10.1111/j.1365-2516.2008.01750.x
Cleeland CS. The brief pain inventory user guide. 2009.
van Genderen FR, Westers P, Heijnen L, et al. Measuring patients' perceptions on their functional abilities: validation of the haemophilia activities list. Haemophilia. 2006;12(1):36-46. doi:10.1111/j.1365-2516.2006.01186.x
Groen WG, van der Net J, Helders PJ, Fischer K. Development and preliminary testing of a paediatric version of the haemophilia activities list (PedHAL). Haemophilia. 2010;16(2):281-289. doi:10.1111/j.1365-2516.2009.02136.x. Epub 2009 Nov 11
Herdman M, Gudex C, Lloyd A, et al. Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L). Qual Life Res. 2011;20:1727-1736. doi:10.1007/s11136-011-9903-x
Von Mackensen S, Gringeri A, Mantovani L. Development, and validation of the first treatment satisfaction scale for adult haemophiliacs (Hemo-Sat A). Haemophilia. 2004;10(Suppl 3):126.
Geiger R, Strasak A, Treml B, et al. Six-minute walk test in children and adolescents. J Pediatr. 2007;150(4):395-399.
Lammers AE, Hislop AA, Flynn Y, Haworth SG. The 6-minute walk test: normal values for children of 4-11 years of age. Arch Dis Child. 2008;93(6):464-468.
De la Corte-Rodriguez H, Rodriguez-Merchan EC, Alvarez-Roman MT, Martin-Salces M, Martinoli C, Jimenez-Yuste V. The value of HEAD-US system in detecting subclinical abnormalities in joints of patients with hemophilia. Expert Rev Hematol. 2018;11(3):253-261.
Måseide RJ, Berntorp E, Astermark J, et al. Haemophilia early arthropathy detection with ultrasound and haemophilia joint health score in the moderate haemophilia (MoHem) study. Haemophilia. 2021;27(2):e253-e259.
Zwagemaker AF, Kloosterman FR, Hemke R, et al. Joint status of patients with nonsevere hemophilia A. J Thromb Haemost. 2022;20(5):1126-1137.
Lapane KL, Quilliam BJ, Benson C, Chow W, Kim MS. Impact of noncancer pain on health-related quality of life. Pain Pract. 2015;15(4):333-342.
Younger J, McCue R, Mackey S. Pain outcomes: a brief review of instruments and techniques. Curr Pain Headache Rep. 2009;13(1):39-43.
Timmer MA, Gouw SC, Feldman BM, et al. Measuring activities and participation in persons with haemophilia: a systematic review of commonly used instruments. Haemophilia. 2018;24(2):e33-e49.
Brewer JR, Harris S, Zakarija A. Hemophilia activities list questionnaire detects functional limitations in young adults with hemophilia a prior to physical examination. Blood. 2009;114(22):1300.
Kuijlaars IAR, van der Net J, van Vulpen LFD, et al. Validation of the pedHALshort and HALshort in Dutch children and adults with haemophilia. Haemophilia. 2022;28(6):1007-1015.
van Balen EC, O'Mahony B, Cnossen MH, et al. Patient-relevant health outcomes for hemophilia care: development of an international standard outcomes set. Res Pract Thromb Haemost. 2021;5(4):e12488.
O'Hara J, Hughes D, Camp C, Burke T, Carroll L, Diego DG. The cost of severe haemophilia in Europe: the CHESS study. Orphanet J Rare Dis. 2017;12:106.
Dover S, Blanchette VS, Srivastava A, Fischer K, Abad A, Feldman BM. Clinical outcomes in hemophilia: towards development of a core set of standardized outcome measures for research. Res Pract Thromb Haemost. 2020;4(4):652-658.
Konkle BA, Skinner M, Iorio A. Hemophilia trials in the twenty-first century: defining patient important outcomes. Res Pract Thromb Haemost. 2019;3(2):184-192.
Hermans C, Klamroth R, Richards M, de Moerloose P, Garrido RP; EHTSB. Outcome measures in European patients with haemophilia: survey of implementation in routine clinical practice, perception of relevance and recommendations by European treaters in the EHTSB. Haemophilia. 2017;23(2):222-229.