Selection from HEOR Bibliography
| Search criteria | ||||
| Rare disease |
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| Physician awareness and understanding of hereditary angioedema: a web-based study in Japan | ||||
| Atsushi Fukunaga 1,2, Miwa Kishimoto 3*, Akinori Oh 3, Takeshi Akiyama 4, Ippei Kotera 3, Yoichi Inoue 3, Junichi Maehara 5 (rwesheorj 4) | ||||
| 1) Division of Dermatology, Department of Internal Related, Kobe University Graduate School of Medicine, Hyogo, Japan 2) Division of Medicine for Function and Morphology of Sensory Organs, Department of Dermatology, Faculty of Medicine, Osaka Medical and Pharmaceutical University, Takatsuki, Japan 3) Japan Medical Office, Takeda Pharmaceutical Company Limited, Tokyo, Japan 4) Real World Evidence Solutions & HEOR, IQVIA Solutions Japan K.K., Tokyo, Japan 5) Department of Acute Care and General Medicine, Emergency and Critical Care Center, Saiseikai Kumamoto Hospital, Kumamoto, Japan | ||||
| Journal of Cutaneous Immunology and Allergy. |
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| , Article | ||||
| , Japan | ||||
| Abstract: https://onlinelibrary.wiley.com/doi/10.1002/cia2.12265 | ||||
| Condition | Year | Language | Analysis type | |
| , Rare disease | 2022 | English | , Survey research | |
| CO71 Investigating the Secondary Care System Burden of Glycogen Storage Disease Type IA (GSDIA) Using the HES Database | ||||
| E Kruger S Chavda RN Gupta V Valayannopoulos | ||||
| Ultragenyx Pharmaceutical Inc., Novato, CA, USA IQVIA, London, UK Ultragenyx Pharmaceutical Inc., Cambridge, MA, USA | ||||
| Kruger, E et al. Value in Health, Volume 25, Issue 7, S317 |
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| , Abstract, Article | ||||
| , England | ||||
| Abstract: https://www.valueinhealthjournal.com/article/S1098-3015(22)00370-9/pdf#%20 | ||||
| Condition | Year | Language | Analysis type | |
| , Metabolic Disorders , Rare disease, Rare Diseases | 2022 | English | , Burden of illness, clinical setting: Secondary care, Quality of life | |
| Leveraging unstructured data to identify hereditary angioedema patients in electronic medical records | ||||
| Emily S Brouwer 1 , Emily W Bratton 2 , Aimee M Near 2 , Lynn Sanders 1 , Christina D Mack 2 | ||||
| 1 Takeda Pharmaceutical Company Limited, 300 Shire Way, Lexington, MA, USA. 2 IQVIA, Durham, NC, USA. | ||||
| Allergy Asthma Clin Immunol . 2021 Apr 20;17(1):41. doi: 10.1186/s13223-021-00541-6. |
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| , Article | ||||
| , USA | ||||
| Abstract: https://pubmed.ncbi.nlm.nih.gov/33879228/ | ||||
| Condition | Year | Language | Analysis type | |
| , Rare disease | 2021 | English | , Clinical setting: Primary care, clinical setting: Secondary care, Epidemiological study, Observational study, Retrospective database analysis | |
| A budget impact analysis of anti-inhibitor coagulant complex use in pediatric hemophilia A patients with inhibitors across kingdom of Saudi Arabia | ||||
| Al Jedai A1, AL-Mudaiheem H1, Al daamah S2, Al kasim F3, Wasri A4, Shaheen N5, Dawood E6, El-Hamaidi I7, Mohamed O8 | ||||
| 1Ministry of Health, Riyadh, NJ, USA, 2Ministry of Health, Damam, Saudi Arabia, 3Ministry of Health, Riyadh, Saudi Arabia, 4Ministry of National Guards, Jeddah, Saudi Arabia, 5King Faisal Specialized Hospitals, Damam, Saudi Arabia, 6Ministry of Health, Mekkah, Saudi Arabia, 7Ministry of National Guards, Riyadh, Saudi Arabia, 8IQVIA, Dubai, DU, United Arab Emirates | ||||
| 2019-11, ISPOR Europe 2019, Copenhagen, Denmark |
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| , Poster | ||||
| , Saudi Arabia | ||||
| Abstract: https://www.ispor.org/heor-resources/presentations-database/presentation/euro2019-3122/96011 | ||||
| Condition | Year | Language | Analysis type | |
| , Hematology, Rare disease | 2019 | English | , Budget impact | |
| Cost-utility analysis of life-long prophylaxis with recombinant factor VIIIFc vs recombinant factor VIII for the management of severe hemophilia A in Sweden. | ||||
| Henry N1, Jovanović J1, Schlueter M1, Kritikou P2, Wilson K2, Myrén KJ2. | ||||
| Henry N1, Jovanović J1, Schlueter M1, Kritikou P2, Wilson K2, Myrén KJ2. 1 IQVIA , London , UK. 2 Swedish Orphan Biovitrum AB , Stockholm , Sweden. | ||||
| , Article | ||||
| , Nordic, UK | ||||
| Condition | Year | Language | Analysis type | |
| , Hematology, Rare disease | 2018 | English | , Retrospective database analysis | |
| Angiotensin II receptor blockers and intestinal malabsorption: an observational cohort study in Germany and Italy | ||||
| Malfertheiner P1, Heiman F2, Ripellino C3, Cataldo N2 | ||||
| 1 Otto-von-Guericke Universität, Klinik für Gastroenterologie, Hepatologie und Infektiologie 2 QuintilesIMS, Milan, Italy 3 HEOR Consultant, Freelance, Milan, Italy | ||||
| ISPOR 20th Annual European Congress, 4-8 November 2017, Glasgow, Scotland |
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| , Poster | ||||
| , Germany, Italy | ||||
| Condition | Year | Language | Analysis type | |
| , Drug safety, Gastrointestinal disorders, Rare disease | 2017 | English | , Database Study, Epidemiological study, Longitudinal study, Observational study, Population Based Study, Real World Data, Real World Evidence, Retrospective cohort analysis, Retrospective database analysis | |
| Real-world Assessment of Renal Involvement in Tuberous Sclerosis Complex (TSC) Patients in the United Kingdom (UK): A Retrospective Cohort Study in the Clinical Practice Research Datalink (CPRD) | ||||
| Kingswood, Chris1, Nasuti, Paola2, Demuth, Dirk2, Patel, Keyur2, Myland, Melissa2, Erhard, Clement3, Gray, Elizabeth4, Pinnegar, Alun4, Magestro, Matthew5 | ||||
| The Royal Sussex County Hospital, Brighton, England 2 IMS Health, London, UK 3 CE Analytics, Cambridge, UK 4 Novartis Pharmaceuticals UK Ltd, Frimley, UK 5 Novartis Pharmaceuticals Corporation, East Hanover NJ, USA | ||||
| International TSC Research Conference (Windsor, 11th Sept 2015). |
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| , Poster | ||||
| , England | ||||
| Condition | Year | Language | Analysis type | |
| , Oncology, Pediatrics, Rare disease | 2015 | English | , Retrospective cohort analysis | |
| Association of sweat chloride concentration at time of diagnosis and CFTR genotype with mortality and cystic fibrosis phenotype | ||||
| McKone EF, Goss C, Velentgas P, Swenson A | ||||
| Journal of Cystic Fibrosis. JCF1155. Feb 2015. 10.1016(j.jcf.2015) 01.005 |
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| , Article | ||||
| Condition | Year | Language | Analysis type | |
| , Rare disease | 2015 | English | , RWLPR (Real-World Late Phase Research-quintiles | |
| Fifth interim analysis of the Kuvan® Adult Maternal Paediatric European Registry (KAMPER): Insights into management/practice in patients with PKU. | ||||
| Lagler FB, Muntau AC, Feillet F, Rogoff D, Moreau-Stucker F, Alm J, Burlina A, Trefz FK. | ||||
| Quintiles Real-World and Late Phase Research | ||||
| Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Congress, 1–4 September 2015, Lyon, France, Abstract No. P-160 |
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| , Abstract | ||||
| Condition | Year | Language | Analysis type | |
| , Patient Registries & Real-World Study Methods, Rare disease | 2015 | English | , RWLPR (Real-World Late Phase Research-quintiles | |
| Impact of Acute Bleeding on Daily Activities of Patients with Congenital Hemophilia with Inhibitors and Their Caregivers and Families: Observations from the Dosing Observational Study in Hemophilia (DOSE). Science Direct | ||||
| Recht N, Neufeld EJ, Sharma VR, Solem CT, Pickard S, Gut RZ, MD, Cooper DL | ||||
| , Article | ||||
| Condition | Year | Language | Analysis type | |
| , Rare disease | 2014 | English | , RWLPR (Real-World Late Phase Research-quintiles | |
| The Budget Impact of Orphan Drugs in the U.S.: A 2007-2013 MIDAS Sales Data Analysis | ||||
| Victoria Divino, Mitch Dekoven, Weiying Wang, Michael Kleinrock, R. Donald Harvey, Rolin L Wade, and Satyin Kaura | ||||
| Celgene, IMS authors: Victoria Divino, Mitch Dekoven, Michael Kleinrock, Rolin L Wade | ||||
| 2014 American Society of Hematology (ASH) Annual Meeting |
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| , Oral presentation | ||||
| , USA | ||||
| Abstract: http://www.bloodjournal.org/content/124/21/736 | ||||
| Condition | Year | Language | Analysis type | |
| , Rare disease | 2014 | English | , Budget impact | |
| Health-related Quality of Life in Patients with Hemophilia and Inhibitors on Prophylaxis with an Activated Prothrombin Complex Concentrate: Results From the Pro-FEIBA Study | ||||
| Gringeri A, Leissinger C,,Cortesi P, Jo H, Fusco F, Riva F, Bulent A, Berntorp E, Carpenter S, Kavakli K, Morfini M, Negrier C, Rocino A, Schramm W, Uscatescu M, Windyga J, Zulfikar B, Mantovani LG | ||||
| Haemophilia 2013 Sep;19(5):736-43 |
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| , Article | ||||
| Condition | Year | Language | Analysis type | |
| , Rare disease | 2013 | English | , RWLPR (Real-World Late Phase Research-quintiles | |
| Impact of pregnancy on women with cystic fibrosis | ||||
| McMullen AH, Pasta DJ, Frederick PD, Konstan MW, Morgan WJ, Schechter MS, Wagener JS | ||||
| Chest 2006; 129(3):706-711 |
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| , Article | ||||
| Condition | Year | Language | Analysis type | |
| , Rare disease | 2006 | English | , RWLPR (Real-World Late Phase Research-quintiles | |