Use of real‐world evidence in regulatory decisions for rare diseases in the United States—Current status and future directions-Reference-Cited by-同舟云学术

Use of real‐world evidence in regulatory decisions for rare diseases in the United States—Current status and future directions

Published:2020-01-30 Issue:10 Volume:29 Page:1213-1218
ISSN:1053-8569
Container-title:Pharmacoepidemiology and Drug Safety
language:en
Short-container-title:Pharmacoepidemiol Drug Saf

Author:

Wu Jasmanda¹^ORCID,Wang Cunlin²,Toh Sengwee³^ORCID,Pisa Federica Edith⁴,Bauer Larry⁵^ORCID

Affiliation:

1. Real‐World Evidence Sanofi Bridgewater New Jersey

2. Pharmacovigilance and Safety Ascentage Pharma Group, Inc. Rockville Maryland

3. Department of Population Medicine Harvard Medical School and Harvard Pilgrim Health Care Institute Boston Massachusetts

4. Real World Evidence Practice UCB Biopharma Sprl Brussels Belgium

5. Hyman, Phelps, & McNamara, P.C. Washington District of Columbia

Publisher

Wiley

Subject

Pharmacology (medical),Epidemiology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/pds.4962

Reference7 articles.

1. Framework for FDA’s Real‐World Evidence Program [Internet]. Food & Drug Administration; [cited2018Dec]. Available from:https://www.fda.gov/media/120060/download.

2. GottliebS. FDA Voices: Perspectives from FDA Leadership and Experts [Internet]. Food & Drug Administration – [cited2018Feb 26]. Available from:https://www.fda.gov/news‐events/fda‐voices‐perspectives‐fda‐leadership‐and‐experts/taking‐new‐steps‐meet‐challenges‐rare‐diseases‐fda‐marks‐11th‐rare‐disease‐day.

3. CTTI Recommendations: Registry Trials [Internet]. Clinical Trials Transformation Initiative; [cited2017May]. Available from:https://www.ctti‐clinicaltrials.org/files/recommendations/registrytrials‐recs.pdf.

4. Draft Guidance: Rare Diseases: Natural History Studies for Drug Development [Internet]. Food & Drug Administration; [cited2019Mar]. Available from:https://www.fda.gov/media/122425/download.

5. Study of Intraventricular Cerliponase Alfa for CLN2 Disease

Cited by 80 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Framework for Multistakeholder Patient Registries in the Field of Rare Diseases;Neurology;2024-09-24

2. The CARMEN-France registry of adult patients with immune thrombocytopenia and autoimmune hemolytic anemia in France;La Revue de Médecine Interne;2024-09

3. Real‐world evidence to support regulatory submissions: A landscape review and assessment of use cases;Clinical and Translational Science;2024-08

4. Comparative effectiveness of lazertinib in patients with EGFR T790M-positive non-small-cell lung cancer using a real-world external control;Scientific Reports;2024-06-25

5. Propensity score weighted multi‐source exchangeability models for incorporating external control data in randomized clinical trials;Statistics in Medicine;2024-06-24