Selection from HEOR Bibliography

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Condition: Rare disease
 
Physician awareness and understanding of hereditary angioedema: a web-based study in Japan
Authors: Atsushi Fukunaga 1,2, Miwa Kishimoto 3*, Akinori Oh 3, Takeshi Akiyama 4, Ippei Kotera 3, Yoichi Inoue 3, Junichi Maehara 5 (rwesheorj 4)
Affiliations: 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
Publications: 
Journal of Cutaneous Immunology and Allergy.
Document Types: , Article
Countries: , 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
Authors: E Kruger S Chavda RN Gupta V Valayannopoulos
Affiliations: Ultragenyx Pharmaceutical Inc., Novato, CA, USA IQVIA, London, UK Ultragenyx Pharmaceutical Inc., Cambridge, MA, USA
Publications: 
Kruger, E et al. Value in Health, Volume 25, Issue 7, S317
Document Types: , Abstract, Article
Countries: , 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
Authors: Emily S Brouwer 1 , Emily W Bratton 2 , Aimee M Near 2 , Lynn Sanders 1 , Christina D Mack 2
Affiliations: 1 Takeda Pharmaceutical Company Limited, 300 Shire Way, Lexington, MA, USA. 2 IQVIA, Durham, NC, USA.
Publications: 
Allergy Asthma Clin Immunol . 2021 Apr 20;17(1):41. doi: 10.1186/s13223-021-00541-6.
Document Types: , Article
Countries: , 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
Authors: Al Jedai A1, AL-Mudaiheem H1, Al daamah S2, Al kasim F3, Wasri A4, Shaheen N5, Dawood E6, El-Hamaidi I7, Mohamed O8
Affiliations: 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
Publications: 
2019-11, ISPOR Europe 2019, Copenhagen, Denmark
Document Types: , Poster
Countries: , 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.
Authors: Henry N1, Jovanović J1, Schlueter M1, Kritikou P2, Wilson K2, Myrén KJ2.
Affiliations: Henry N1, Jovanović J1, Schlueter M1, Kritikou P2, Wilson K2, Myrén KJ2. 1 IQVIA , London , UK. 2 Swedish Orphan Biovitrum AB , Stockholm , Sweden.
Publications: 
Document Types: , Article
Countries: , 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
Authors: Malfertheiner P1, Heiman F2, Ripellino C3, Cataldo N2
Affiliations: 1 Otto-von-Guericke Universität, Klinik für Gastroenterologie, Hepatologie und Infektiologie 2 QuintilesIMS, Milan, Italy 3 HEOR Consultant, Freelance, Milan, Italy
Publications: 
ISPOR 20th Annual European Congress, 4-8 November 2017, Glasgow, Scotland
Document Types: , Poster
Countries: , 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)
Authors: Kingswood, Chris1, Nasuti, Paola2, Demuth, Dirk2, Patel, Keyur2, Myland, Melissa2, Erhard, Clement3, Gray, Elizabeth4, Pinnegar, Alun4, Magestro, Matthew5
Affiliations: 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
Publications: 
International TSC Research Conference (Windsor, 11th Sept 2015).
Document Types: , Poster
Countries: , 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
Authors: McKone EF, Goss C, Velentgas P, Swenson A
Affiliations: 
Publications: 
Journal of Cystic Fibrosis. JCF1155. Feb 2015. 10.1016(j.jcf.2015) 01.005
Document Types: , Article
Countries: 
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.
Authors: Lagler FB, Muntau AC, Feillet F, Rogoff D, Moreau-Stucker F, Alm J, Burlina A, Trefz FK.
Affiliations: Quintiles Real-World and Late Phase Research
Publications: 
Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Congress, 1–4 September 2015, Lyon, France, Abstract No. P-160
Document Types: , Abstract
Countries: 
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
Authors: Recht N, Neufeld EJ, Sharma VR, Solem CT, Pickard S, Gut RZ, MD, Cooper DL
Affiliations: 
Publications: 
Document Types: , Article
Countries: 
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
Authors: Victoria Divino, Mitch Dekoven, Weiying Wang, Michael Kleinrock, R. Donald Harvey, Rolin L Wade, and Satyin Kaura
Affiliations: Celgene, IMS authors: Victoria Divino, Mitch Dekoven, Michael Kleinrock, Rolin L Wade
Publications: 
2014 American Society of Hematology (ASH) Annual Meeting
Document Types: , Oral presentation
Countries: , 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
Authors: 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
Affiliations: 
Publications: 
Haemophilia 2013 Sep;19(5):736-43
Document Types: , Article
Countries: 
Condition Year Language Analysis type
, Rare disease 2013 English , RWLPR (Real-World Late Phase Research-quintiles
 
 
Impact of pregnancy on women with cystic fibrosis
Authors: McMullen AH, Pasta DJ, Frederick PD, Konstan MW, Morgan WJ, Schechter MS, Wagener JS
Affiliations: 
Publications: 
Chest 2006; 129(3):706-711
Document Types: , Article
Countries: 
Condition Year Language Analysis type
, Rare disease 2006 English , RWLPR (Real-World Late Phase Research-quintiles
 
 
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