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For: Contesse MG, Valentine JE, Wall TE, Leffler MG. The Case for the Use of Patient and Caregiver Perception of Change Assessments in Rare Disease Clinical Trials: A Methodologic Overview. Adv Ther 2019;36:997-1010. [PMID: 30879250 DOI: 10.1007/s12325-019-00920-x] [Cited by in Crossref: 6] [Cited by in F6Publishing: 4] [Article Influence: 2.0] [Reference Citation Analysis]
Number Citing Articles
1 Peláez Bejarano A, de las Aguas Robustillo Cortés M, Guzman Ramos MI. Patient experience in a rare disease: application of the IEXPAC questionnaire in hereditary transthyretin-mediated amyloidosis. Expert Opinion on Orphan Drugs 2021;9:175-80. [DOI: 10.1080/21678707.2021.1953468] [Reference Citation Analysis]
2 Zimmermann BM, Eichinger J, Baumgartner MR. A systematic review of moral reasons on orphan drug reimbursement. Orphanet J Rare Dis 2021;16:292. [PMID: 34193232 DOI: 10.1186/s13023-021-01925-y] [Reference Citation Analysis]
3 Staunton H, Trennery C, Arbuckle R, Guridi M, Zhuravleva E, Furlong P, Fischer R, Hall R. Development of a Clinical Global Impression of Change (CGI-C) and a Caregiver Global Impression of Change (CaGI-C) measure for ambulant individuals with Duchenne muscular dystrophy. Health Qual Life Outcomes 2021;19:184. [PMID: 34311756 DOI: 10.1186/s12955-021-01813-w] [Reference Citation Analysis]
4 Whittal A, Meregaglia M, Nicod E. The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment. Patient 2021;14:485-503. [PMID: 33462774 DOI: 10.1007/s40271-020-00493-w] [Cited by in Crossref: 2] [Cited by in F6Publishing: 2] [Article Influence: 2.0] [Reference Citation Analysis]
5 Tambuyzer E, Vandendriessche B, Austin CP, Brooks PJ, Larsson K, Miller Needleman KI, Valentine J, Davies K, Groft SC, Preti R, Oprea TI, Prunotto M. Therapies for rare diseases: therapeutic modalities, progress and challenges ahead. Nat Rev Drug Discov 2020;19:93-111. [PMID: 31836861 DOI: 10.1038/s41573-019-0049-9] [Cited by in Crossref: 46] [Cited by in F6Publishing: 41] [Article Influence: 15.3] [Reference Citation Analysis]
6 Katz EG, McNulty P, Levitan B, Treichler P, Martynowicz J, Jamieson C. U.S. Food and Drug Administration's Patient-Focused Drug Development Initiative: Experience with Integration of Patient-Experience Data in a New Drug Application for Esketamine Nasal Spray Plus a Newly Initiated Oral Antidepressant for Treatment-Resistant Depression. Ther Innov Regul Sci 2021. [PMID: 34623613 DOI: 10.1007/s43441-021-00340-6] [Reference Citation Analysis]