References
- E11(R1) Addendum: Clinical Investigation of Medicinal Products in the Pediatric Population. https://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E11/E11_R1_GuidelineWithAddendum_Step4.pdf. Accessed 3 Dec 2018
- New Pediatric Labeling Information Database. https://www.accessdata. fda.gov/scripts/sda/sdNavigation.cfm?filter=&sortColumn=1d&sd=labelin gdatabase&page=1. Accessed 3 Dec 2018
- Eguale T, Buckeridge DL, Verma A, Winslade NE, Benedetti A, Hanley JA, et al. Association of Off-label drug use and adverse drug events in an adult population. JAMA Intern Med 2016;176:55-63. doi: 10.1001/jamainternmed.2015.6058.
- Gupta SK, Nayak RP. Off-label use of medicine: Perspective of physicians, patients, pharmaceutical companies and regulatory authorities. J Pharmacol Pharmacother 2014;5: 88-92. doi: 10.4103/0976-500X.130046.
- Mulugeta LY, Yao L, Mould D, Jacobs B, Florian J, Smith B, et al. Leveraging big data in pediatric development programs. Proceedings from the 2016 American college of clinical pharmacology annual meeting symposium. Clin Pharmacol Ther 2018;104:81-87. doi: 10.1002/cpt.975.
- US FDA Guidance for Industry- General Clinical Pharmacology Considerations for Pediatric Studies for Drugs and Biological Products 2014, available at https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM425885.pdf. Accessed 30 Nov, 2018
- Frieden TR. Evidence for health decision making - Beyond randomized, controlled trials. N Engl J Med 2017;377:465-475. doi: 10.1056/NEJMra1614394.
- US Food and Drug Administration. FDA D.I.S.C.O.: Avelumab in Merkel cell carcinoma transcript. https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm559876.htm. Accessed 30 Nov, 2018
- Alexion Receives Approval for Important Updates to the European Label for Soliris (eculizumab). Business Wire. http://www.businesswire.com/news/home/20150409006360/en/Alexion-Receives-Approval-Important-Updates-European-Label. Accessed 30 Nov, 2018
- Califf RM, Sugarman J. Exploring the ethical and regulatory issues in pragmaticclinicaltrials. Clin Trials2015;12:436-441. doi: 10.1177/1740774515598334.
- Dreyer NA. Advancing a framework for regulatory use of real-World evidence: When real is reliable. Ther Innov Regul Sci 2018;52:362-368. doi: 10.1177/2168479018763591.
- Liu JB, D'Angelica MI, Ko CY. The randomized registry trial: Two birds, One stone. AnnSurg2017;265:1064-1065. doi: 10.1097/SLA.0000000000002166.
- Lauer MS, D'Agostino RB, Sr. The randomized registry trial -- the next disruptive technology in clinical research? N Engl J Med 2013;369:1579-1581. doi: 10.1056/NEJMp1310102.
- James S, Rao SV, Granger CB. Registry-based randomized clinical trials--a new clinical trial paradigm. Nat Rev Cardiol 2015;12:312-316. doi: 10.1038/nrcardio.2015.33.
- Frobert O, Lagerqvist B, Gudnason T, Thuesen L, Svensson R, Olivecrona GK, et al. Thrombus Aspiration in ST-Elevation myocardial infarction in Scandinavia (TASTE trial): a multicenter, prospective, randomized, controlled clinical registry trial based on the Swedish Angiography and Angioplasty Registry (SCAAR) platform: study design and rationale. Am Heart J 2010;160:1042-1048. doi: 10.1016/j.ahj.2010.08.040.