Using real world data to support regulatory approval of drugs in rare diseases: A review of opportunities, limitations & a case example-Reference-Cited by-同舟云学术

Using real world data to support regulatory approval of drugs in rare diseases: A review of opportunities, limitations & a case example

Published:2021-08 Issue:4 Volume:45 Page:100769
ISSN:0147-0272
Container-title:Current Problems in Cancer
language:en
Short-container-title:Current Problems in Cancer

Author:

Gross Andrea M.

Publisher

Elsevier BV

Subject

Cancer Research,Oncology

Reference32 articles.

1. de Vrueh R, Baekelandt, E.R.F., de Haan, J.M.H. Background paper 6.19: rare diseases. priority medicines for europe and the world "a public health approach to innovation" 2013.

2. Virtual controls as an alternative to randomized controlled trials for assessing efficacy of interventions;Strayhorn;BMC Med Res Methodol,2021

3. using electronic health records to derive control arms for early phase single-arm lung cancer trials: proof-of-concept in randomized controlled trials;Carrigan;Clin Pharmacol Therap,2020

4. Healthcare outcomes assessed with observational study designs compared with those assessed in randomized trials;Anglemyer;Cochrane Database Syst Rev,2014

5. Comparison of evidence of treatment effects in randomized and nonrandomized studies;Ioannidis;Jama,2001

Cited by 11 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. Causal estimators for incorporating external controls in randomized trials with longitudinal outcomes;Journal of the Royal Statistical Society Series A: Statistics in Society;2024-08-26

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

3. Paediatric Drug Development in China: Current Status and Future Prospects;Pediatric Drugs;2024-06-05

4. Longitudinal Natural History Study of Children and Adults with Rare Solid Tumors: Initial Results for First 200 Participants;Cancer Research Communications;2023-12-06

5. Using Continuous Glucose Monitoring Values for Bolus Size Calculation in Smart Multiple Daily Injection Systems: No Negative Impact on Post-bolus Glycemic Outcomes Found in Real-World Data;Journal of Diabetes Science and Technology;2023-09-24