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1.
A practical approach for adoption of a hub and spoke model for cell and gene therapies in low- and middle-income countries: framework and case studies.
Gene Ther
; 31(1-2): 1-11, 2024 Jan.
Artículo
en Inglés
| MEDLINE | ID: mdl-37903929
2.
Value-Based Pricing for Patent-Protected Medicines Over the Product Life Cycle: Pricing Anomalies in the "Age of Cures" and Their Implications for Dynamic Efficiency.
Value Health
; 26(3): 336-343, 2023 03.
Artículo
en Inglés
| MEDLINE | ID: mdl-36336584
3.
Analytic Considerations in Applying a General Economic Evaluation Reference Case to Gene Therapy.
Value Health
; 22(6): 661-668, 2019 06.
Artículo
en Inglés
| MEDLINE | ID: mdl-31198183
4.
Body of Evidence for Onasemnogene Abeparvovec in Spinal Muscular Atrophy Supports Long-Term Duration of Effect Without Relapse.
Value Health
; 2022 Feb 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-35181208
5.
Real-world evidence for coverage determination of treatments for rare diseases.
Orphanet J Rare Dis
; 19(1): 47, 2024 Feb 07.
Artículo
en Inglés
| MEDLINE | ID: mdl-38326894
6.
Patient and Caregiver Outcomes After Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey.
Adv Ther
; 40(12): 5315-5337, 2023 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-37776479
7.
Health Care Resource Utilization and Costs for Patients with Spinal Muscular Atrophy: Findings from a Retrospective US Claims Database Analysis.
Adv Ther
; 40(10): 4589-4605, 2023 10.
Artículo
en Inglés
| MEDLINE | ID: mdl-37587305
8.
Valuation of Treatments for Rare Diseases: A Systematic Literature Review of Societal Preference Studies.
Adv Ther
; 40(2): 393-424, 2023 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-36451072
9.
Recommendations for economic evaluations of cell and gene therapies: a systematic literature review with critical appraisal.
Expert Rev Pharmacoecon Outcomes Res
; 23(5): 483-497, 2023 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-37074838
10.
Hyperuricemia in young adults and risk of insulin resistance, prediabetes, and diabetes: a 15-year follow-up study.
Am J Epidemiol
; 176(2): 108-16, 2012 Jul 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-22753829
11.
International reference pricing of pharmaceuticals in the United States: Implications for potentially curative treatments.
J Manag Care Spec Pharm
; 28(5): 566-572, 2022 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-35471069
12.
Systematic Literature Review of Clinical and Economic Evidence for Spinal Muscular Atrophy.
Adv Ther
; 39(5): 1915-1958, 2022 05.
Artículo
en Inglés
| MEDLINE | ID: mdl-35307799
13.
Development and psychometric evaluation of 2 age-stratified versions of the Pediatric GERD Symptom and Quality of Life Questionnaire.
J Pediatr Gastroenterol Nutr
; 52(5): 514-22, 2011 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-21464757
14.
Impact of chronic constipation on health-related quality of life, work productivity, and healthcare resource use: an analysis of the National Health and Wellness Survey.
Dig Dis Sci
; 56(9): 2688-95, 2011 Sep.
Artículo
en Inglés
| MEDLINE | ID: mdl-21380761
15.
Chronic kidney disease in gout in a managed care setting.
BMC Nephrol
; 12: 36, 2011 Aug 03.
Artículo
en Inglés
| MEDLINE | ID: mdl-21812963
16.
Gene therapy may not be as expensive as people think: challenges in assessing the value of single and short-term therapies.
J Manag Care Spec Pharm
; 27(5): 674-681, 2021 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-33908277
17.
Matching-adjusted indirect treatment comparison of onasemnogene abeparvovec and nusinersen for the treatment of symptomatic patients with spinal muscular atrophy type 1.
Curr Med Res Opin
; 37(10): 1719-1730, 2021 10.
Artículo
en Inglés
| MEDLINE | ID: mdl-34236007
18.
Nusinersen for Spinal Muscular Atrophy in the United States: Findings From a Retrospective Claims Database Analysis.
Adv Ther
; 38(12): 5809-5828, 2021 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-34713391
19.
Variation in market access decisions for cell and gene therapies across the United States, Canada, and Europe.
Health Policy
; 125(12): 1550-1556, 2021 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-34763929
20.
An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER).
J Mark Access Health Policy
; 9(1): 1889841, 2021 Feb 28.
Artículo
en Inglés
| MEDLINE | ID: mdl-33708361