{"id":"https://openalex.org/W4223493443","doi":"https://doi.org/10.1186/s13023-022-02283-z","title":"Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose?","display_name":"Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose?","publication_year":2022,"publication_date":"2022-04-05","ids":{"openalex":"https://openalex.org/W4223493443","doi":"https://doi.org/10.1186/s13023-022-02283-z","pmid":"https://pubmed.ncbi.nlm.nih.gov/35382853","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/8981887"},"language":"en","primary_location":{"id":"doi:10.1186/s13023-022-02283-z","doi":"https://doi.org/10.1186/s13023-022-02283-z","is_oa":true,"landing_page_url":"https://doi.org/10.1186/s13023-022-02283-z","pdf_url":"https://ojrd.biomedcentral.com/track/pdf/10.1186/s13023-022-02283-z","source":{"id":"https://openalex.org/S71291065","display_name":"Orphanet Journal of Rare Diseases","issn_l":"1750-1172","issn":["1750-1172"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-scientific","cwts-core","doaj","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","oarl","poland-100","russia-white-list-2","vabb-shw"],"host_organization":"https://openalex.org/P4310320256","host_organization_name":"BioMed Central","host_organization_lineage":["https://openalex.org/P4310320256","https://openalex.org/P4310319965"],"host_organization_lineage_names":["BioMed Central","Springer Nature"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Orphanet Journal of Rare Diseases","raw_type":"journal-article"},"volume":null,"type":"review","is_traditional":true,"indexed_in":["crossref","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"gold","oa_url":"https://ojrd.biomedcentral.com/track/pdf/10.1186/s13023-022-02283-z","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5016565734","display_name":"Maarten Jacobus Postma","orcid":"https://orcid.org/0000-0002-6306-3653","observed_orcids":["https://orcid.org/0000-0002-6306-3653"]},"institutions":[{"id":"https://openalex.org/I1334415907","display_name":"University Medical Center Groningen","ror":"https://ror.org/03cv38k47","country_code":"NL","type":"healthcare","lineage":["https://openalex.org/I1334415907"]},{"id":"https://openalex.org/I169381384","display_name":"University of Groningen","ror":"https://ror.org/012p63287","country_code":"NL","type":"education","lineage":["https://openalex.org/I169381384"]}],"countries":["NL"],"is_corresponding":false,"raw_author_name":"Maarten J. Postma","raw_affiliation_strings":["Department of Economics, Econometrics and Finance, Faculty of Economics and Business, University of Groningen, Groningen, The Netherlands","Department of Health Sciences, University Medical Center, University of Groningen, Groningen, The Netherlands"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Economics, Econometrics and Finance, Faculty of Economics and Business, University of Groningen, Groningen, The Netherlands","institution_ids":["https://openalex.org/I169381384"]},{"raw_affiliation_string":"Department of Health Sciences, University Medical Center, University of Groningen, Groningen, The Netherlands","institution_ids":["https://openalex.org/I1334415907","https://openalex.org/I169381384"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5000561395","display_name":"Declan Noone","orcid":"https://orcid.org/0000-0003-2183-4277","observed_orcids":["https://orcid.org/0000-0003-2183-4277"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Declan Noone","raw_affiliation_strings":["European Haemophilia Consortium, Brussels, Belgium"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"European Haemophilia Consortium, Brussels, Belgium","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5080737514","display_name":"Mark Hermannes Rozenbaum","orcid":"https://orcid.org/0000-0001-6209-4370","observed_orcids":["https://orcid.org/0000-0001-6209-4370"]},"institutions":[{"id":"https://openalex.org/I4210122616","display_name":"Pfizer (Netherlands)","ror":"https://ror.org/02bzf1224","country_code":"NL","type":"company","lineage":["https://openalex.org/I180857899","https://openalex.org/I4210122616"]}],"countries":["NL"],"is_corresponding":false,"raw_author_name":"Mark H. Rozenbaum","raw_affiliation_strings":["Pfizer, Inc., Rotterdam, The Netherlands"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Pfizer, Inc., Rotterdam, The Netherlands","institution_ids":["https://openalex.org/I4210122616"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5065512022","display_name":"John A. Carter","orcid":"https://orcid.org/0000-0001-8539-5352","observed_orcids":["https://orcid.org/0000-0001-8539-5352"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"John A. Carter","raw_affiliation_strings":["OPEN Health - Evidence & Access, Bethesda, USA"],"raw_orcid":"https://orcid.org/0000-0001-8539-5352","affiliations":[{"raw_affiliation_string":"OPEN Health - Evidence & Access, Bethesda, USA","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5087184017","display_name":"Marc F Botteman","orcid":"https://orcid.org/0000-0003-2808-7163","observed_orcids":["https://orcid.org/0000-0003-2808-7163"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Marc F. Botteman","raw_affiliation_strings":["OPEN Health - Evidence & Access, Bethesda, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"OPEN Health - Evidence & Access, Bethesda, USA","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5108656268","display_name":"Elisabeth A. L. Fenwick","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Elisabeth Fenwick","raw_affiliation_strings":["OPEN Health - Evidence & Access, Oxford, UK"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"OPEN Health - Evidence & Access, Oxford, UK","institution_ids":[]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5060938706","display_name":"Louis P. Garrison","orcid":"https://orcid.org/0000-0002-9468-4989","observed_orcids":["https://orcid.org/0000-0002-9468-4989"]},"institutions":[{"id":"https://openalex.org/I201448701","display_name":"University of Washington","ror":"https://ror.org/00cvxb145","country_code":"US","type":"education","lineage":["https://openalex.org/I201448701"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Louis P. Garrison","raw_affiliation_strings":["Comparative Health Outcomes, Policy, and Economics Institute, University of Washington, Seattle, USA. lgarrisn@uw.edu","Comparative Health Outcomes, Policy, and Economics Institute, University of Washington, Seattle, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Comparative Health Outcomes, Policy, and Economics Institute, University of Washington, Seattle, USA. lgarrisn@uw.edu","institution_ids":["https://openalex.org/I201448701"]},{"raw_affiliation_string":"Comparative Health Outcomes, Policy, and Economics Institute, University of Washington, Seattle, USA","institution_ids":["https://openalex.org/I201448701"]}]}],"institutions":[],"countries_distinct_count":2,"institutions_distinct_count":4,"corresponding_author_ids":["https://openalex.org/A5060938706"],"corresponding_institution_ids":["https://openalex.org/I201448701"],"apc_list":{"value":2680,"currency":"USD","value_usd":2680},"apc_paid":{"value":2680,"currency":"USD","value_usd":2680},"fwci":1.5365,"has_fulltext":true,"cited_by_count":51,"citation_normalized_percentile":{"value":0.84125145,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":96,"max":99},"biblio":{"volume":"17","issue":"1","first_page":"157","last_page":"157"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10804","display_name":"Health Systems, Economic Evaluations, Quality of Life","score":0.8407999873161316,"subfield":{"id":"https://openalex.org/subfields/2002","display_name":"Economics and Econometrics"},"field":{"id":"https://openalex.org/fields/20","display_name":"Economics, Econometrics and Finance"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}},"topics":[{"id":"https://openalex.org/T10804","display_name":"Health Systems, Economic Evaluations, Quality of Life","score":0.8407999873161316,"subfield":{"id":"https://openalex.org/subfields/2002","display_name":"Economics and Econometrics"},"field":{"id":"https://openalex.org/fields/20","display_name":"Economics, Econometrics and Finance"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}},{"id":"https://openalex.org/T11792","display_name":"Pharmaceutical Economics and Policy","score":0.15639999508857727,"subfield":{"id":"https://openalex.org/subfields/2002","display_name":"Economics and Econometrics"},"field":{"id":"https://openalex.org/fields/20","display_name":"Economics, Econometrics and Finance"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}},{"id":"https://openalex.org/T10841","display_name":"Economic and Environmental Valuation","score":0.00019999999494757503,"subfield":{"id":"https://openalex.org/subfields/2002","display_name":"Economics and Econometrics"},"field":{"id":"https://openalex.org/fields/20","display_name":"Economics, Econometrics and Finance"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/orphan-drugs","display_name":"orphan drugs","score":1.0},{"id":"https://openalex.org/keywords/rare-diseases","display_name":"rare diseases","score":0.8309999704360962},{"id":"https://openalex.org/keywords/cost-effectiveness-analysis","display_name":"cost-effectiveness analysis","score":0.9860000014305115},{"id":"https://openalex.org/keywords/quality-adjusted-life-years","display_name":"quality-adjusted life years","score":0.9309999942779541},{"id":"https://openalex.org/keywords/drug-pricing","display_name":"drug pricing","score":0.8539999723434448},{"id":"https://openalex.org/keywords/drug-reimbursement","display_name":"drug reimbursement","score":0.7049999833106995},{"id":"https://openalex.org/keywords/access-to-medicines","display_name":"access to medicines","score":0.8069999814033508}],"concepts":[{"id":"https://openalex.org/C75480439","wikidata":"https://www.wikidata.org/wiki/Q1367466","display_name":"Orphan drug","level":2,"score":0.9428247213363647},{"id":"https://openalex.org/C2779703844","wikidata":"https://www.wikidata.org/wiki/Q760125","display_name":"Reimbursement","level":3,"score":0.8616458177566528},{"id":"https://openalex.org/C515549039","wikidata":"https://www.wikidata.org/wiki/Q1754768","display_name":"Cost-effectiveness analysis","level":3,"score":0.6343550682067871},{"id":"https://openalex.org/C524218345","wikidata":"https://www.wikidata.org/wiki/Q31218","display_name":"Health economics","level":3,"score":0.4980895519256592},{"id":"https://openalex.org/C162118730","wikidata":"https://www.wikidata.org/wiki/Q1128453","display_name":"Actuarial science","level":1,"score":0.48605018854141235},{"id":"https://openalex.org/C2776291640","wikidata":"https://www.wikidata.org/wiki/Q2912517","display_name":"Value (mathematics)","level":2,"score":0.4518943130970001},{"id":"https://openalex.org/C2779530757","wikidata":"https://www.wikidata.org/wiki/Q1207505","display_name":"Quality (philosophy)","level":2,"score":0.4426063895225525},{"id":"https://openalex.org/C21333345","wikidata":"https://www.wikidata.org/wiki/Q1519843","display_name":"Health technology","level":3,"score":0.4358361065387726},{"id":"https://openalex.org/C3019080777","wikidata":"https://www.wikidata.org/wiki/Q1754768","display_name":"Cost effectiveness","level":2,"score":0.43169647455215454},{"id":"https://openalex.org/C112930515","wikidata":"https://www.wikidata.org/wiki/Q4389547","display_name":"Risk analysis (engineering)","level":1,"score":0.4212720990180969},{"id":"https://openalex.org/C100001284","wikidata":"https://www.wikidata.org/wiki/Q2248246","display_name":"Public economics","level":1,"score":0.4210565686225891},{"id":"https://openalex.org/C2908647359","wikidata":"https://www.wikidata.org/wiki/Q2625603","display_name":"Population","level":2,"score":0.41215604543685913},{"id":"https://openalex.org/C162324750","wikidata":"https://www.wikidata.org/wiki/Q8134","display_name":"Economics","level":0,"score":0.3855915367603302},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.37497061491012573},{"id":"https://openalex.org/C160735492","wikidata":"https://www.wikidata.org/wiki/Q31207","display_name":"Health care","level":2,"score":0.3672776222229004},{"id":"https://openalex.org/C144133560","wikidata":"https://www.wikidata.org/wiki/Q4830453","display_name":"Business","level":0,"score":0.3622131943702698},{"id":"https://openalex.org/C41008148","wikidata":"https://www.wikidata.org/wiki/Q21198","display_name":"Computer science","level":0,"score":0.2668406367301941},{"id":"https://openalex.org/C60644358","wikidata":"https://www.wikidata.org/wiki/Q128570","display_name":"Bioinformatics","level":1,"score":0.15620708465576172},{"id":"https://openalex.org/C99454951","wikidata":"https://www.wikidata.org/wiki/Q932068","display_name":"Environmental health","level":1,"score":0.09636500477790833},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.08039933443069458},{"id":"https://openalex.org/C111472728","wikidata":"https://www.wikidata.org/wiki/Q9471","display_name":"Epistemology","level":1,"score":0.0},{"id":"https://openalex.org/C119857082","wikidata":"https://www.wikidata.org/wiki/Q2539","display_name":"Machine learning","level":1,"score":0.0},{"id":"https://openalex.org/C138885662","wikidata":"https://www.wikidata.org/wiki/Q5891","display_name":"Philosophy","level":0,"score":0.0},{"id":"https://openalex.org/C50522688","wikidata":"https://www.wikidata.org/wiki/Q189833","display_name":"Economic growth","level":1,"score":0.0}],"mesh":[{"descriptor_ui":"D003362","descriptor_name":"Cost-Benefit Analysis","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D009965","descriptor_name":"Orphan Drug Production","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D019057","descriptor_name":"Quality-Adjusted Life Years","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D035583","descriptor_name":"Rare Diseases","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true}],"locations_count":6,"locations":[{"id":"doi:10.1186/s13023-022-02283-z","doi":"https://doi.org/10.1186/s13023-022-02283-z","is_oa":true,"landing_page_url":"https://doi.org/10.1186/s13023-022-02283-z","pdf_url":"https://ojrd.biomedcentral.com/track/pdf/10.1186/s13023-022-02283-z","source":{"id":"https://openalex.org/S71291065","display_name":"Orphanet Journal of Rare Diseases","issn_l":"1750-1172","issn":["1750-1172"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-scientific","cwts-core","doaj","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","oarl","poland-100","russia-white-list-2","vabb-shw"],"host_organization":"https://openalex.org/P4310320256","host_organization_name":"BioMed Central","host_organization_lineage":["https://openalex.org/P4310320256","https://openalex.org/P4310319965"],"host_organization_lineage_names":["BioMed Central","Springer Nature"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Orphanet Journal of Rare Diseases","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:8981887","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/8981887","pdf_url":null,"source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Orphanet J Rare Dis","raw_type":"Text"},{"id":"pmid:35382853","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/35382853","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Orphanet journal of rare diseases","raw_type":"Journal Article"},{"id":"pmh:oai:pure.rug.nl:openaire/dbc4a82a-1d26-44b1-ab98-1021ecdca3fe","doi":null,"is_oa":true,"landing_page_url":"https://research.rug.nl/en/publications/dbc4a82a-1d26-44b1-ab98-1021ecdca3fe","pdf_url":null,"source":{"id":"https://openalex.org/S4306400420","display_name":"University of Groningen research database (University of Groningen / Centre for Information Technology)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I169381384","host_organization_name":"University of Groningen","host_organization_lineage":["https://openalex.org/I169381384"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Postma, M J, Noone, D, Rozenbaum, M H, Carter, J A, Botteman, M F, Fenwick, E & Garrison, L P 2022, 'Assessing the value of orphan drugs using conventional cost-effectiveness analysis : Is it fit for purpose?', Orphanet journal of rare diseases, vol. 17, no. 1, 157. https://doi.org/10.1186/s13023-022-02283-z","raw_type":"info:eu-repo/semantics/review"},{"id":"pmh:oai:doaj.org/article:19eaa71b6ab44aa9a3c08df0b7fd8ca7","doi":null,"is_oa":true,"landing_page_url":"https://doaj.org/article/19eaa71b6ab44aa9a3c08df0b7fd8ca7","pdf_url":null,"source":{"id":"https://openalex.org/S4306401280","display_name":"DOAJ (DOAJ: Directory of Open Access Journals)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-sa","license_id":"https://openalex.org/licenses/cc-by-sa","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-8 (2022)","raw_type":"article"},{"id":"pmh:rug:oai:pure.rug.nl:publications/dbc4a82a-1d26-44b1-ab98-1021ecdca3fe","doi":null,"is_oa":true,"landing_page_url":"https://research.rug.nl/en/publications/dbc4a82a-1d26-44b1-ab98-1021ecdca3fe","pdf_url":null,"source":{"id":"https://openalex.org/S4306400420","display_name":"University of Groningen research database (University of Groningen / Centre for Information Technology)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I169381384","host_organization_name":"University of Groningen","host_organization_lineage":["https://openalex.org/I169381384"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Orphanet journal of rare diseases, 17(1):157. BMC","raw_type":"info:eu-repo/semantics/other"}],"best_oa_location":{"id":"doi:10.1186/s13023-022-02283-z","doi":"https://doi.org/10.1186/s13023-022-02283-z","is_oa":true,"landing_page_url":"https://doi.org/10.1186/s13023-022-02283-z","pdf_url":"https://ojrd.biomedcentral.com/track/pdf/10.1186/s13023-022-02283-z","source":{"id":"https://openalex.org/S71291065","display_name":"Orphanet Journal of Rare Diseases","issn_l":"1750-1172","issn":["1750-1172"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-scientific","cwts-core","doaj","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","oarl","poland-100","russia-white-list-2","vabb-shw"],"host_organization":"https://openalex.org/P4310320256","host_organization_name":"BioMed Central","host_organization_lineage":["https://openalex.org/P4310320256","https://openalex.org/P4310319965"],"host_organization_lineage_names":["BioMed Central","Springer Nature"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Orphanet Journal of Rare Diseases","raw_type":"journal-article"},"sustainable_development_goals":[{"score":0.6240000128746033,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"study_designs":[],"awards":[],"funders":[{"id":"https://openalex.org/F4320307765","display_name":"Pfizer","ror":"https://ror.org/01xdqrp08"},{"id":"https://openalex.org/F4320333645","display_name":"Pfizer Pharmaceuticals","ror":"https://ror.org/02bzf1224"}],"has_content":{"grobid_xml":true,"pdf":true},"content_urls":{"pdf":"https://content.openalex.org/works/W4223493443.pdf","grobid_xml":"https://content.openalex.org/works/W4223493443.grobid-xml"},"referenced_works_count":76,"referenced_works":["https://openalex.org/W186638250","https://openalex.org/W1519384742","https://openalex.org/W1561263655","https://openalex.org/W1578255890","https://openalex.org/W1590828610","https://openalex.org/W1788182075","https://openalex.org/W1983965561","https://openalex.org/W1994884206","https://openalex.org/W1999697554","https://openalex.org/W2005990846","https://openalex.org/W2014759405","https://openalex.org/W2015388811","https://openalex.org/W2042217012","https://openalex.org/W2051593700","https://openalex.org/W2052391811","https://openalex.org/W2065306083","https://openalex.org/W2077204318","https://openalex.org/W2097649441","https://openalex.org/W2105845113","https://openalex.org/W2118432205","https://openalex.org/W2124257155","https://openalex.org/W2127164016","https://openalex.org/W2130083918","https://openalex.org/W2131080517","https://openalex.org/W2136793894","https://openalex.org/W2150246838","https://openalex.org/W2160488963","https://openalex.org/W2161515080","https://openalex.org/W2162877219","https://openalex.org/W2194206520","https://openalex.org/W2319630131","https://openalex.org/W2345849873","https://openalex.org/W2397390372","https://openalex.org/W2470373670","https://openalex.org/W2545421535","https://openalex.org/W2571094594","https://openalex.org/W2580366914","https://openalex.org/W2593395519","https://openalex.org/W2606049844","https://openalex.org/W2752702796","https://openalex.org/W2769892380","https://openalex.org/W2788249140","https://openalex.org/W2788701892","https://openalex.org/W2788999563","https://openalex.org/W2794316606","https://openalex.org/W2796704470","https://openalex.org/W2802935287","https://openalex.org/W2804322580","https://openalex.org/W2890617002","https://openalex.org/W2892087921","https://openalex.org/W2895900438","https://openalex.org/W2899430609","https://openalex.org/W2900146638","https://openalex.org/W2906616062","https://openalex.org/W2916723858","https://openalex.org/W2921498279","https://openalex.org/W2940687015","https://openalex.org/W2973267506","https://openalex.org/W2973809551","https://openalex.org/W3010072720","https://openalex.org/W3022171525","https://openalex.org/W3023587264","https://openalex.org/W3024347837","https://openalex.org/W3033992773","https://openalex.org/W3042370659","https://openalex.org/W3049155515","https://openalex.org/W3092321807","https://openalex.org/W3094200995","https://openalex.org/W3097059098","https://openalex.org/W3118389339","https://openalex.org/W3124075406","https://openalex.org/W3129982381","https://openalex.org/W3135384692","https://openalex.org/W3160726731","https://openalex.org/W3204158394","https://openalex.org/W4211198509"],"related_works":["https://openalex.org/W2155602538","https://openalex.org/W3160726731","https://openalex.org/W2062180874","https://openalex.org/W2113487108","https://openalex.org/W3015792696","https://openalex.org/W2906057837","https://openalex.org/W4383900934","https://openalex.org/W4405751782","https://openalex.org/W3031746769","https://openalex.org/W2766162975"],"abstract_inverted_index":{"Conventional":[0],"cost-effectiveness":[1,87],"analysis-i.e.,":[2],"assessing":[3,92],"pharmaceuticals":[4],"through":[5],"a":[6,15],"cost":[7],"per":[8],"quality-adjusted":[9],"life":[10],"year":[11],"(QALY)":[12],"framework-originated":[13],"from":[14],"societal":[16],"commitment":[17],"to":[18,51,75,91],"maximize":[19],"population":[20],"health":[21],"given":[22],"limited":[23],"resources.":[24],"This":[25,46],"\"extra-welfarist\"":[26],"approach":[27],"has":[28,48,67],"produced":[29],"pricing":[30,99],"and":[31,100,109,112],"reimbursement":[32,101],"systems":[33],"that":[34],"are":[35],"not":[36],"well-":[37],"aligned":[38],"with":[39,54],"the":[40,59,69,83,98],"unique":[41],"considerations":[42],"of":[43,58,61,71,85],"orphan":[44,72,103],"drugs.":[45],"framework":[47],"been":[49],"slow":[50],"evolve":[52],"along":[53],"our":[55],"increased":[56],"understanding":[57],"impact":[60],"rare":[62,77],"diseases,":[63],"which":[64],"in":[65],"turn":[66],"complicated":[68],"assessment":[70],"drugs":[73],"meant":[74],"treat":[76],"diseases.":[78],"Herein,":[79],"we":[80],"(i)":[81],"discuss":[82],"limitations":[84],"conventional":[86],"analysis":[88],"as":[89,95,97],"applied":[90],"access":[93],"to,":[94],"well":[96],"of,":[102],"drugs,":[104],"(ii)":[105],"critically":[106],"appraise":[107],"alternative":[108],"supplemental":[110],"approaches,":[111],"(iii)":[113],"offer":[114],"insights":[115],"on":[116],"plausible":[117],"steps":[118],"forward.":[119]},"counts_by_year":[{"year":2026,"cited_by_count":12},{"year":2025,"cited_by_count":12},{"year":2024,"cited_by_count":13},{"year":2023,"cited_by_count":10},{"year":2022,"cited_by_count":4}],"updated_date":"2026-11-07T11:25:07.637180","created_date":"2022-04-15T00:00:00"}