{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T02:44:58Z","timestamp":1791945898698,"version":"4.3.4"},"reference-count":30,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2026,10,9]],"date-time":"2026-10-09T00:00:00Z","timestamp":1791504000000},"content-version":"vor","delay-in-days":8,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pharmacoepidemiology and Drug"],"published-print":{"date-parts":[[2026,10]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Purpose<\/jats:title>\n                    <jats:p>Gaucher disease (GD) requires lifelong high\u2010cost treatment within the Brazilian Unified Health System (SUS). In Brazil, treatment eligibility and monitoring are defined by Clinical Protocol and Therapeutic Guidelines (PCDT), while pharmaceutical prices are regulated by the Drug Market Regulation Chamber (CMED). Exploratory price\u2013value approaches may help make explicit the relationship between drug prices and measurable clinical benefit. We explored the applicability of a Pareto\u2010based Efficiency Frontier (EF) as a descriptive hematologic price\u2013value framework for GD therapies in Brazil.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>An exploratory Pareto\u2010based EF analysis compared annual drug acquisition prices with standardized hematologic benefits, defined as hemoglobin and platelet improvements, derived from a published systematic review. Annual prices were obtained from CMED, Maximum Price of Sale to the Government (PMVG), and median public procurement prices from the Health Price Database (BPS). Prices are presented in Brazilian reais and USD adjusted by purchasing power parity. Directly dominated technologies were excluded, and all remaining directly non\u2010dominated therapies were connected by linear interpolation to form a Pareto\u2010based 12\u2010month reference EF. This construction retains alternatives that would be excluded by extended dominance under a conventional convex EF. The analysis was restricted to treatment\u2010na\u00efve patients.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Annual costs ranged from approximately USD\u2010PPP 94900 to 763\u2009200. Under the primary 12\u2010month analysis, imiglucerase was directly dominated across all three pricing scenarios, whereas taliglucerase alfa, velaglucerase alfa, and miglustat defined the Pareto\u2010based frontier. In the BPS hemoglobin analysis and in all three platelet analyses, taliglucerase alfa would be subject to extended dominance under a conventional convex EF but was retained as a directly non\u2010dominated therapy. This classification was conditional on indirect comparisons from heterogeneous evidence and the selected 12\u2010month hematologic point estimates. In the descriptive scenario analysis, direct dominance of imiglucerase was less stable when maximum effects across longer follow\u2010up durations were considered.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>This exploratory application of a Pareto\u2010based Efficiency Frontier describes the relationship between current price scenarios and comparative hematologic benefit for GD therapies. Findings should not be interpreted as definitive comparative value evidence, an ICER\u2010based optimization rule, or a stand\u2010alone pricing instrument. The approach may complement broader value\u2010based assessment alongside QALY\u2010based modeling, long\u2010term outcomes, formal uncertainty analysis, and non\u2010hematologic clinical dimensions.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/pds.70497","type":"journal-article","created":{"date-parts":[[2026,10,10]],"date-time":"2026-10-10T03:31:58Z","timestamp":1791603118000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Applying a Pareto\u2010Based Efficiency Frontier to Inform Value\u2010Based Pricing for Gaucher Disease Therapies in Brazil"],"prefix":"10.1002","volume":"35","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0918-6505","authenticated-orcid":false,"given":"Marcus Carvalho","family":"Borin","sequence":"first","affiliation":[{"name":"Postgraduate Program in Medicines and Pharmaceutical Assistance, Department of Social Pharmacy, Faculty of Pharmacy Federal University of Minas Gerais (UFMG)  Belo Horizonte Minas Gerais Brazil"},{"name":"Collaborating Center for Health Technology Assessment and Excellence in Health, Faculty of Pharmacy Federal University of Minas Gerais (UFMG)  Belo Horizonte Minas Gerais Brazil"},{"name":"Division of Biological and Life Sciences, Visiting Associate Professor of Epidemiology Mohamed Bin Zayed University of Artificial Intelligence  Abu Dhabi UAE"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0666-7776","authenticated-orcid":false,"given":"Ludmila Peres","family":"Gargano","sequence":"additional","affiliation":[{"name":"Postgraduate Program in Medicines and Pharmaceutical Assistance, Department of Social Pharmacy, Faculty of Pharmacy Federal University of Minas Gerais (UFMG)  Belo Horizonte Minas Gerais Brazil"},{"name":"Collaborating Center for Health Technology Assessment and Excellence in Health, Faculty of Pharmacy Federal University of Minas Gerais (UFMG)  Belo Horizonte Minas Gerais Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francisco de Assis","family":"Acurcio","sequence":"additional","affiliation":[{"name":"Postgraduate Program in Medicines and Pharmaceutical Assistance, Department of Social Pharmacy, Faculty of Pharmacy Federal University of Minas Gerais (UFMG)  Belo Horizonte Minas Gerais Brazil"},{"name":"Collaborating Center for Health Technology Assessment and Excellence in Health, Faculty of Pharmacy Federal University of Minas Gerais (UFMG)  Belo Horizonte Minas Gerais Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0210-0721","authenticated-orcid":false,"given":"Juliana","family":"\u00c1lvares\u2010Teodoro","sequence":"additional","affiliation":[{"name":"Postgraduate Program in Medicines and Pharmaceutical Assistance, Department of Social Pharmacy, Faculty of Pharmacy Federal University of Minas Gerais (UFMG)  Belo Horizonte Minas Gerais Brazil"},{"name":"Collaborating Center for Health Technology Assessment and Excellence in Health, Faculty of Pharmacy Federal University of Minas Gerais (UFMG)  Belo Horizonte Minas Gerais Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5256-0577","authenticated-orcid":false,"suffix":"Junior","given":"Augusto Afonso","family":"Guerra","sequence":"additional","affiliation":[{"name":"Postgraduate Program in Medicines and Pharmaceutical Assistance, Department of Social Pharmacy, Faculty of Pharmacy Federal University of Minas Gerais (UFMG)  Belo Horizonte Minas Gerais Brazil"},{"name":"Collaborating Center for Health Technology Assessment and Excellence in Health, Faculty of Pharmacy Federal University of Minas Gerais (UFMG)  Belo Horizonte Minas Gerais Brazil"},{"name":"Division of Biological and Life Sciences, Visiting Associate Professor of Epidemiology Mohamed Bin Zayed University of Artificial Intelligence  Abu Dhabi UAE"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,10,9]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1080\/10245332.2016.1240391"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms18020441"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.3390\/diagnostics14242840"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.25875"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.3389\/fphar.2024.1433970"},{"key":"e_1_2_10_7_1","volume-title":"Aplicaci\u00f3n de La Evidencia Econ\u00f3mica En La Evaluaci\u00f3n de Tecnolog\u00edas Sanitarias y La Toma de Decisiones Sobre Asignaci\u00f3n de Recursos Sanitarios En Am\u00e9rica Latina: Siete Temas Clave y Una Propuesta Preliminar de Implementaci\u00f3n","author":"Pichon\u2010Riviere A.","year":"2021"},{"key":"e_1_2_10_8_1","unstructured":"Brasil. 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