المؤلفون: Grand TS; Sheffield Centre for Health and Related Research (SCHARR), University of Sheffield, 30 Regent Street, Sheffield, S1 4DA, UK. tsgrand1@sheffield.ac.uk.; Lundbeck A/S, Copenhagen, Denmark. tsgrand1@sheffield.ac.uk., Ren S; Sheffield Centre for Health and Related Research (SCHARR), University of Sheffield, 30 Regent Street, Sheffield, S1 4DA, UK., Hall J; Institute of Applied Health Research, Health Economics Unit, University of Birmingham, Edgbaston, Birmingham, UK., Åström DO; Lundbeck A/S, Copenhagen, Denmark., Regnier S; Lundbeck A/S, Copenhagen, Denmark., Thokala P; Sheffield Centre for Health and Related Research (SCHARR), University of Sheffield, 30 Regent Street, Sheffield, S1 4DA, UK.

المصدر: PharmacoEconomics [Pharmacoeconomics] 2024 Jun; Vol. 42 (6), pp. 619-631. Date of Electronic Publication: 2024 Apr 14.

نوع المنشور: Journal Article; Review

بيانات الدورية: Publisher: Adis, Springer International Country of Publication: New Zealand NLM ID: 9212404 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1179-2027 (Electronic) Linking ISSN: 11707690 NLM ISO Abbreviation: Pharmacoeconomics Subsets: MEDLINE

مواضيع طبية MeSH: Rare Diseases*/drug therapy , Rare Diseases*/economics , Orphan Drug Production*/economics , Cost-Benefit Analysis* , Technology Assessment, Biomedical*, Humans ; Models, Economic

المؤلفون: Man A; BC Children's Hospital Research Institute, 950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada.; Division of Translational Therapeutics, Department of Pediatrics, Faculty of Medicine, University of British Columbia, Vancouver, BC, Canada.; Michael G. DeGroote School of Medicine, McMaster University, Hamilton, ON, Canada., Groeneweg GSS; Division of Translational Therapeutics, Department of Pediatrics, Faculty of Medicine, University of British Columbia, Vancouver, BC, Canada.; Pharmaceutical Outcomes Programme, British Columbia Children's Hospital, Vancouver, BC, Canada., Ross CJD; BC Children's Hospital Research Institute, 950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada.; Department of Medical Genetics, Faculty of Medicine, University of British Columbia, Vancouver, BC, Canada.; Faculty of Pharmaceutical Sciences, University of British Columbia, Vancouver, BC, Canada., Carleton BC; BC Children's Hospital Research Institute, 950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada. bcarleton@popi.ubc.ca.; Division of Translational Therapeutics, Department of Pediatrics, Faculty of Medicine, University of British Columbia, Vancouver, BC, Canada. bcarleton@popi.ubc.ca.; Pharmaceutical Outcomes Programme, British Columbia Children's Hospital, Vancouver, BC, Canada. bcarleton@popi.ubc.ca.; Department of Medical Genetics, Faculty of Medicine, University of British Columbia, Vancouver, BC, Canada. bcarleton@popi.ubc.ca.

المصدر: Drug safety [Drug Saf] 2024 Jun; Vol. 47 (6), pp. 521-528. Date of Electronic Publication: 2024 Mar 14.

نوع المنشور: Journal Article; Review

بيانات الدورية: Publisher: Adis, Springer International Country of Publication: New Zealand NLM ID: 9002928 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1179-1942 (Electronic) Linking ISSN: 01145916 NLM ISO Abbreviation: Drug Saf Subsets: MEDLINE

مواضيع طبية MeSH: Rare Diseases*/genetics , Rare Diseases*/drug therapy , Pharmacogenetics* , Orphan Drug Production*, Humans

المؤلفون: Felippini A; Department of Pharmacy, Faculty of Pharmaceutical Sciences, University of São Paulo, Av. Prof. Lineu Prestes, 580 - Conj. das Químicas - Bloco 13 - Cidade Universitária Butantã, São Paulo, Brazil., Biglia LV; Department of Pharmacy, Faculty of Pharmaceutical Sciences, University of São Paulo, Av. Prof. Lineu Prestes, 580 - Conj. das Químicas - Bloco 13 - Cidade Universitária Butantã, São Paulo, Brazil., Lima TM; Department of Pharmacy and Pharmaceutical Administration, Faculty of Pharmacy, Fluminense Federal University, R. Dr. Mario Vianna, 523 - Santa Rosa, Niteroi, Brazil., Aguiar PM; Department of Pharmacy, Faculty of Pharmaceutical Sciences, University of São Paulo, Av. Prof. Lineu Prestes, 580 - Conj. das Químicas - Bloco 13 - Cidade Universitária Butantã, São Paulo, Brazil. Electronic address: aguiar.pm@usp.br.

المصدر: Health policy (Amsterdam, Netherlands) [Health Policy] 2024 Jun; Vol. 144, pp. 105080. Date of Electronic Publication: 2024 May 06.

نوع المنشور: Journal Article; Review

بيانات الدورية: Publisher: Elsevier Scientific Publishers Country of Publication: Ireland NLM ID: 8409431 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1872-6054 (Electronic) Linking ISSN: 01688510 NLM ISO Abbreviation: Health Policy Subsets: MEDLINE

مواضيع طبية MeSH: Orphan Drug Production*/economics , Technology Assessment, Biomedical* , Rare Diseases*/drug therapy , Delivery of Health Care*, Humans ; National Health Programs

المؤلفون: Costa E; Division of Pharmacoepidemiology and Clinical Pharmacology, World Health Organization Collaborating Centre for Pharmaceutical Policy and Regulation, Utrecht University, Utrecht, The Netherlands., Isgrò A; Centralized Procedures Office, Innovation and Pharmaceutical Strategy Division, Italian Medicines Agency, Rome, Italy., de Montalembert M; Department of Pediatrics, Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris Centre, Paris, France., Leufkens HGM; Emeritus Professor Regulatory Science and Pharmaceutical Policy, Division of Pharmacoepidemiology and Clinical Pharmacoepidemiology, Utrecht University, Utrecht, The Netherlands., Ware RE; Division of Hematology and Global Health Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH., De Franceschi L; Department of Medicine, University of Verona & AOUI Verona, Verona, Italy.

المصدر: Blood advances [Blood Adv] 2024 May 28; Vol. 8 (10), pp. 2455-2465.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: American Society of Hematology Country of Publication: United States NLM ID: 101698425 Publication Model: Print Cited Medium: Internet ISSN: 2473-9537 (Electronic) Linking ISSN: 24739529 NLM ISO Abbreviation: Blood Adv Subsets: MEDLINE

مواضيع طبية MeSH: Anemia, Sickle Cell*/drug therapy , Orphan Drug Production* , Drug Development*, Humans ; United States ; Europe ; Drug Approval

المؤلفون: Duarte DM; INFARMED, National Authority of Medicines and Health Products, I.P.Lisboa, Portugal. Electronic address: dinah.duarte@infarmed.pt., da Silva Lima MB; Universidade de Lisboa, Faculdade de Farmácia, Lisbon, Portugal., Sepodes B; Universidade de Lisboa, Faculdade de Farmácia, Lisbon, Portugal.

المصدر: Journal of the neurological sciences [J Neurol Sci] 2024 May 15; Vol. 460, pp. 122989. Date of Electronic Publication: 2024 Apr 03.

نوع المنشور: Journal Article; Observational Study; Review

بيانات الدورية: Publisher: Elsevier Country of Publication: Netherlands NLM ID: 0375403 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1878-5883 (Electronic) Linking ISSN: 0022510X NLM ISO Abbreviation: J Neurol Sci Subsets: MEDLINE

مواضيع طبية MeSH: Neuromuscular Diseases*/drug therapy , Orphan Drug Production* , Rare Diseases*/drug therapy, Child ; Humans ; Cross-Sectional Studies ; Retrospective Studies

المؤلفون: Lexchin J; School of Health Policy and Management, York University, 4700 Keele St, Toronto, ON, M3J 1P3, Canada. jlexchin@yorku.ca.; Department of Family and Community Medicine, University of Toronto, Toronto, ON, Canada. jlexchin@yorku.ca.

المصدر: Journal of general internal medicine [J Gen Intern Med] 2024 May; Vol. 39 (7), pp. 1276-1279. Date of Electronic Publication: 2024 Feb 07.

نوع المنشور: Letter; Journal Article

بيانات الدورية: Publisher: Springer Country of Publication: United States NLM ID: 8605834 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-1497 (Electronic) Linking ISSN: 08848734 NLM ISO Abbreviation: J Gen Intern Med Subsets: MEDLINE

مواضيع طبية MeSH: Orphan Drug Production*/economics , Drug Approval*, Humans ; Canada ; Cross-Sectional Studies ; Rare Diseases/drug therapy

المؤلفون: Saltonstall P; National Organization for Rare Disorders., Ross H; National Organization for Rare Disorders., Kim PT; Kendall Square Policy Strategies LLC.

المصدر: The Milbank quarterly [Milbank Q] 2024 Mar; Vol. 102 (1), pp. 83-96. Date of Electronic Publication: 2023 Dec 12.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Blackwell Publishers Country of Publication: United States NLM ID: 8607003 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1468-0009 (Electronic) Linking ISSN: 0887378X NLM ISO Abbreviation: Milbank Q Subsets: MEDLINE

مواضيع طبية MeSH: Orphan Drug Production* , Drug Approval*, United States ; Humans ; Rare Diseases/drug therapy ; United States Food and Drug Administration

المؤلفون: Bouwman L; Faculdade de Farmácia, Universidade de Lisboa, Lisbon, Portugal. mlbouwman@edu.ulisboa.pt.; Laboratory of Systems Integration Pharmacology, Clinical and Regulatory Science, Research Institute for Medicines of the University of Lisbon (iMED.ULisboa), Lisbon, Portugal. mlbouwman@edu.ulisboa.pt., Sepodes B; Faculdade de Farmácia, Universidade de Lisboa, Lisbon, Portugal.; Laboratory of Systems Integration Pharmacology, Clinical and Regulatory Science, Research Institute for Medicines of the University of Lisbon (iMED.ULisboa), Lisbon, Portugal., Leufkens H; Faculdade de Farmácia, Universidade de Lisboa, Lisbon, Portugal.; Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences (UIPS), Utrecht University, Utrecht, The Netherlands., Torre C; Faculdade de Farmácia, Universidade de Lisboa, Lisbon, Portugal.; Laboratory of Systems Integration Pharmacology, Clinical and Regulatory Science, Research Institute for Medicines of the University of Lisbon (iMED.ULisboa), Lisbon, Portugal.

المصدر: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2024 Feb 27; Vol. 19 (1), pp. 91. Date of Electronic Publication: 2024 Feb 27.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE

مواضيع طبية MeSH: Orphan Drug Production* , Rare Diseases*/drug therapy, Humans ; European Union ; Europe ; Pharmaceutical Preparations ; Drug Approval

المؤلفون: Belot A; Centre International de Recherche en Infectiologie, Inserm U1111, Université Claude Bernard Lyon 1, CNRS UMR5308, ENS de Lyon, Lyon, France; Pediatric Nephrology, Rheumatology, Dermatology, Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Bron 69677, France. Electronic address: alexandre.belot@chu-lyon.fr., Benezech S; Centre International de Recherche en Infectiologie, Inserm U1111, Université Claude Bernard Lyon 1, CNRS UMR5308, ENS de Lyon, Lyon, France; Pediatric Immunology, Haematology, Oncology, Hospices Civils de Lyon, Lyon, France., Tusseau M; Centre International de Recherche en Infectiologie, Inserm U1111, Université Claude Bernard Lyon 1, CNRS UMR5308, ENS de Lyon, Lyon, France; Genetic Department, Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Bron, France.

المصدر: Lancet (London, England) [Lancet] 2024 Feb 17; Vol. 403 (10427), pp. 592-593. Date of Electronic Publication: 2024 Jan 23.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Elsevier Country of Publication: England NLM ID: 2985213R Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1474-547X (Electronic) Linking ISSN: 01406736 NLM ISO Abbreviation: Lancet Subsets: MEDLINE

مواضيع طبية MeSH: Rare Diseases*/drug therapy , Orphan Drug Production*, Humans ; Antibodies, Monoclonal ; Drug Approval

المؤلفون: Seoane-Vazquez E; Department of Pharmaceutical Economic and Policy, Chapman University School of Pharmacy, Irvine, CA, USA. seoanevazquez@chapman.edu.; Economic Science Institute, Argyros School of Business and Economics, Chapman University, Orange, CA, USA. seoanevazquez@chapman.edu., Rodriguez-Monguio R; Department of Clinical Pharmacy, School of Pharmacy, University of California San Francisco, San Francisco, USA.; Medication Outcomes Center, University of California San Francisco, San Francisco, USA.; Philip R. Lee Institute for Health Policy Studies, University of California San Francisco, San Francisco, USA., Powers JH 3rd; Department of Medicine, George Washington University School of Medicine, Washington, DC, USA.

المصدر: Scientific reports [Sci Rep] 2024 Feb 09; Vol. 14 (1), pp. 3325. Date of Electronic Publication: 2024 Feb 09.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 101563288 Publication Model: Electronic Cited Medium: Internet ISSN: 2045-2322 (Electronic) Linking ISSN: 20452322 NLM ISO Abbreviation: Sci Rep Subsets: MEDLINE

مواضيع طبية MeSH: Orphan Drug Production* , Biological Products*/therapeutic use, United States ; Humans ; United States Food and Drug Administration ; Pharmaceutical Preparations ; Biological Factors ; Drug Approval