{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,5]],"date-time":"2026-06-05T10:19:13Z","timestamp":1780654753043,"version":"3.54.1"},"reference-count":23,"publisher":"BMJ","issue":"Suppl 2","license":[{"start":{"date-parts":[[2024,5,6]],"date-time":"2024-05-06T00:00:00Z","timestamp":1714953600000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000865","name":"Bill and Melinda Gates Foundation","doi-asserted-by":"publisher","award":["INV-003239"],"award-info":[{"award-number":["INV-003239"]}],"id":[{"id":"10.13039\/100000865","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["bmj.com"],"crossmark-restriction":true},"short-container-title":["BMJ EBM"],"accepted":{"date-parts":[[2024,4,7]]},"published-print":{"date-parts":[[2025,12]]},"abstract":"<jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>To assess the cost-effectiveness of emicizumab prophylaxis for patients having haemophilia A with inhibitors in the Indian context using an adaptive health technology assessment (aHTA) methodology.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design<\/jats:title>\n                    <jats:p>Economic evaluation using multiple approaches aimed at adjusting previously generated cost-effectiveness results based on (1) price differences only (\u2018simple\u2019) and (2) differences in cost and expected treatment duration (\u2018moderate\u2019) and differences in cost, inflation and life expectancy (\u2018complex\u2019).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Setting<\/jats:title>\n                    <jats:p>Typical haemophilia care in India.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Participants<\/jats:title>\n                    <jats:p>Patients with haemophilia A and inhibitors.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Intervention<\/jats:title>\n                    <jats:p>Emicizumab prophylaxis using two vial strengths (30 or 150 mg\/mL) in comparison to no prophylaxis.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Main outcome measures<\/jats:title>\n                    <jats:p>Adjusted incremental cost-effectiveness ratio (ICERa), incremental costs and incremental quality-adjusted life years associated with emicizumab prophylaxis from both the health system and societal perspectives.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Using the simple ICER adjustment method, emicizumab prophylaxis resulted in potential cost savings from the payers\u2019 perspective for both vial strengths in patients aged \u226512 and &lt;12 years. However, from a societal perspective, emicizumab prophylaxis was not cost-effective. Using the moderate adjustment method, emicizumab prophylaxis showed potential cost saving from the health system perspective. The complex adjustment method also revealed cost savings for emicizumab prophylaxis from the health system and societal perspectives across different age groups.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>We found that implementing emicizumab prophylaxis for patients with haemophilia A and inhibitors in India has the potential to result in cost savings. This study highlights the feasibility of using the expanded aHTA methodology for rapid evidence generation in the Indian context. However, it is crucial to address certain research gaps, including data limitations, challenges in translating international evidence to Indian context and associated uncertainties. Additionally, conducting a comprehensive budget impact analysis is necessary. These findings hold significant implications for decision-making regarding the potential provision of emicizumab prophylaxis through federal or\/and state government-funded programmes and institutions in India.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1136\/bmjebm-2023-112492","type":"journal-article","created":{"date-parts":[[2024,5,6]],"date-time":"2024-05-06T13:20:44Z","timestamp":1715001644000},"page":"s11-s19","update-policy":"https:\/\/doi.org\/10.1136\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":9,"title":["Cost-effectiveness of emicizumab prophylaxis for haemophilia A with inhibitors: an adaptive health technology assessment for the Indian setting"],"prefix":"10.1136","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7122-523X","authenticated-orcid":false,"given":"Sitanshu Sekhar","family":"Kar","sequence":"first","affiliation":[{"name":"Preventive and Social Medicine, Jawaharlal Institute of Postgraduate Medical Education and Research (JIPMER), Puducherry, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Parthibane","family":"Sivanantham","sequence":"additional","affiliation":[{"name":"Preventive and Social Medicine, Jawaharlal Institute of Postgraduate Medical Education and Research (JIPMER), Puducherry, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vanessa","family":"Ravel","sequence":"additional","affiliation":[{"name":"Preventive and Social Medicine, Jawaharlal Institute of Postgraduate Medical Education and Research (JIPMER), Puducherry, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3045-1649","authenticated-orcid":false,"given":"Abha","family":"Mehndiratta","sequence":"additional","affiliation":[{"name":"Center for Global Development, Washington, District of Columbia, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kirti","family":"Tyagi","sequence":"additional","affiliation":[{"name":"Department of Community Medicine and School of Public Health, Post Graduate Institute of Medical Education & Research (PGIMER), Chandigarh, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel A","family":"Ollendorf","sequence":"additional","affiliation":[{"name":"Center for the Evaluation of Value and Risk in Health, Tufts University School of Medicine, Tufts University, Boston, Massachusetts, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"239","published-online":{"date-parts":[[2024,5,6]]},"reference":[{"key":"2025112607301101000_30.Suppl_2.s11.1","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1007\/s12288-011-0084-0","article-title":"Hemophilia care in India: a review and experience from a tertiary care centre in Uttar Pradesh","volume":"27","author":"Phadke","year":"2011","journal-title":"Indian J Hematol Blood Transfus"},{"key":"2025112607301101000_30.Suppl_2.s11.2","unstructured":"World Federation of Hemophilia . Canada WHF; Report on the Annual Global Survey 2016, 2017. Available: https:\/\/www1.wfh.org\/publication\/files\/pdf-1690.pdf [Accessed 4 Apr 2023]."},{"key":"2025112607301101000_30.Suppl_2.s11.3","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1111\/j.1600-0609.1998.tb01103.x","article-title":"Early treatment with recombinant factor Viia results in greater efficacy with less product","volume":"63","author":"Lusher","year":"1998","journal-title":"Eur J Haematol Suppl"},{"key":"2025112607301101000_30.Suppl_2.s11.4","doi-asserted-by":"publisher","DOI":"10.1177\/2040620712464509"},{"key":"2025112607301101000_30.Suppl_2.s11.5","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1516437"},{"key":"2025112607301101000_30.Suppl_2.s11.6","doi-asserted-by":"crossref","first-page":"398","DOI":"10.1111\/hae.13747","article-title":"Recombinant factor VIII products and inhibitor development in previously untreated patients with severe Haemophilia A: combined analysis of three studies","volume":"25","author":"Volkers","year":"2019","journal-title":"Haemophilia"},{"key":"2025112607301101000_30.Suppl_2.s11.7","doi-asserted-by":"crossref","first-page":"67","DOI":"10.1111\/hae.13633","article-title":"Prevalence of FVIII inhibitors in severe Haemophilia A patients: effect of treatment and genetic factors in an Indian population","volume":"25","author":"David","year":"2019","journal-title":"Haemophilia"},{"key":"2025112607301101000_30.Suppl_2.s11.8","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1703068"},{"key":"2025112607301101000_30.Suppl_2.s11.9","doi-asserted-by":"crossref","first-page":"e295","DOI":"10.1016\/S2352-3026(19)30054-7","article-title":"Efficacy, safety, and pharmacokinetics of Emicizumab prophylaxis given every 4 weeks in people with Haemophilia A (HAVEN 4): a Multicentre, open-label, non-randomised phase 3 study","volume":"6","author":"Pipe","year":"2019","journal-title":"Lancet Haematol"},{"key":"2025112607301101000_30.Suppl_2.s11.10","doi-asserted-by":"crossref","first-page":"2127","DOI":"10.1182\/blood.2019001869","article-title":"A multicenter, open-label phase 3 study of Emicizumab prophylaxis in children with Hemophilia A with inhibitors","volume":"134","author":"Young","year":"2019","journal-title":"Blood"},{"key":"2025112607301101000_30.Suppl_2.s11.11","doi-asserted-by":"crossref","DOI":"10.1002\/rth2.12670","article-title":"Prophylactic Emicizumab for Hemophilia A in the Asia-Pacific region: A randomized study (HAVEN 5)","volume":"6","author":"Yang","year":"2022","journal-title":"Res Pract Thromb Haemost"},{"key":"2025112607301101000_30.Suppl_2.s11.12","doi-asserted-by":"crossref","first-page":"216","DOI":"10.1055\/s-0039-3401822","article-title":"Cost-effectiveness and budget impact of Emicizumab prophylaxis in Haemophilia A patients with inhibitors","volume":"120","author":"Cortesi","year":"2020","journal-title":"Thromb Haemost"},{"key":"2025112607301101000_30.Suppl_2.s11.13","doi-asserted-by":"crossref","first-page":"e1","DOI":"10.1111\/hae.14129","article-title":"Cost-effectiveness of Emicizumab vs Bypassing agents in the prevention of bleeding episodes in Haemophilia A patients with anti-FVIII inhibitors in France","volume":"27","author":"Polack","year":"2021","journal-title":"Haemophilia"},{"key":"2025112607301101000_30.Suppl_2.s11.14","unstructured":"Vargas ER , Ali S , Lee K . CADTH Ottawa; Hemlibra (Emicizumab) Economic Review Report, 2019. Available: https:\/\/www.cadth.ca\/sites\/default\/files\/hta-he\/ob0005-emicizumab-eeconomic-report.pdf [Accessed 2 Apr 2023]."},{"key":"2025112607301101000_30.Suppl_2.s11.15","unstructured":"Institute for Clinical and Economic Review . United States of America: ICER; Emicizumab for Hemophilia A with Inhibitors: Effectiveness and Value, 2018. Available: https:\/\/icer.org\/wp-content\/uploads\/2020\/10\/ICER_Hemophilia_Final_Evidence_Report_041618.pdf [Accessed 1 Apr 2023]."},{"key":"2025112607301101000_30.Suppl_2.s11.16","doi-asserted-by":"crossref","first-page":"e12","DOI":"10.1111\/hae.14143","article-title":"Cost-utility analysis of Emicizumab prophylaxis in Haemophilia A patients with factor VIII inhibitors in Korea","volume":"27","author":"Lee","year":"2021","journal-title":"Haemophilia"},{"key":"2025112607301101000_30.Suppl_2.s11.17","doi-asserted-by":"crossref","first-page":"460","DOI":"10.1007\/s10729-022-09594-4","article-title":"What is the value of explicit priority setting for health interventions? A simulation study","volume":"25","author":"Barlow","year":"2022","journal-title":"Health Care Manag Sci"},{"key":"2025112607301101000_30.Suppl_2.s11.18","doi-asserted-by":"crossref","first-page":"461","DOI":"10.1017\/S0266462319000199","article-title":"From design to evaluation: applications of health technology assessment in Myanmar and lessons for low or lower middle-income countries","volume":"35","author":"Dabak","year":"2019","journal-title":"Int J Technol Assess Health Care"},{"key":"2025112607301101000_30.Suppl_2.s11.19","unstructured":"Department of Health Research . DHR New Delhi; Health Technology Assessment in India: A Manual, 2018. Available: https:\/\/htain.icmr.org.in\/virtual-library\/htain-manuals [Accessed 16 Apr 2023]."},{"key":"2025112607301101000_30.Suppl_2.s11.20","doi-asserted-by":"crossref","DOI":"10.1136\/bmjgh-2020-004549","article-title":"Adaptive health technology assessment to facilitate priority setting in Low- and middle-income countries","volume":"6","author":"Nemzoff","year":"2021","journal-title":"BMJ Glob Health"},{"key":"2025112607301101000_30.Suppl_2.s11.21","unstructured":"National Cancer Grid . NCG Mumbai; NCG AHTA Process and Methods Guide, 2022. Available: https:\/\/tmc.gov.in\/ncg\/docs\/pdf\/AHTA%20process%20guide%20v2.0_final.pdf [Accessed 5 Apr 2023]."},{"key":"2025112607301101000_30.Suppl_2.s11.22","doi-asserted-by":"crossref","DOI":"10.1186\/s13643-022-02106-z","article-title":"Rapid evidence synthesis to enable innovation and adoption in health and social care","volume":"11","author":"Norman","year":"2022","journal-title":"Syst Rev"},{"key":"2025112607301101000_30.Suppl_2.s11.23","unstructured":"Applied Research Collaboration Greater Manchester . The NIHR ARC-GM and Health Innovation Manchester approach to rapid evidence synthesis to support health system decision making, 2020. Available: https:\/\/osf.io\/https:\/\/osf.io\/munzh [Accessed 25 Oct 2023]."}],"container-title":["BMJ Evidence-Based Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/syndication.highwire.org\/content\/doi\/10.1136\/bmjebm-2023-112492","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,11,26]],"date-time":"2025-11-26T15:30:43Z","timestamp":1764171043000},"score":1,"resource":{"primary":{"URL":"https:\/\/ebm.bmj.com\/lookup\/doi\/10.1136\/bmjebm-2023-112492"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,5,6]]},"references-count":23,"journal-issue":{"issue":"Suppl 2","published-online":{"date-parts":[[2025,11,26]]},"published-print":{"date-parts":[[2025,12]]}},"alternative-id":["10.1136\/bmjebm-2023-112492"],"URL":"https:\/\/doi.org\/10.1136\/bmjebm-2023-112492","relation":{},"ISSN":["2515-446X","2515-4478"],"issn-type":[{"value":"2515-446X","type":"print"},{"value":"2515-4478","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,5,6]]}}}