References
- Food and Drug Administration (FDA)Use of real-world evidence to support regulatory decision-making for medical devices Available from: https://www.fda.gov/downloads/medicaldevices/deviceregulationand-guidance/guidancedocuments/ucm513027.pdfAccessed April 5, 2017
- GarrisonLPJrNeumannPJEricksonPUsing real-world data for coverage and payment decisions: the ISPOR Real-world Data Task Force reportValue Health200710532633517888097
- US Congress [webpage on the Internet]H.R.34–21st Century Cures Act. 114th Congress (2015–2016) Available from: https://www.congress.gov/bill/114th-congress/house-bill/34/Accessed August 28, 2017
- CohenAGotoSSchreiberKWhy do we need observational studies of everyday patients in the real-life setting?Eur Heart J Suppl201517Suppl DD2D8
- BergerMDanielGFrankKA framework for regulatory use of real-world evidence Available from: https://healthpolicy.duke.edu/sites/default/files/atoms/files/rwe_white_paper_2017.09.06.pdfAccessed August 24, 2017
- BhattAEvolution of clinical research: a history before and beyond James LindPerspect Clin Res20101161021829774
- World Medical AssociationWorld Medical Association Declaration of Helsinki: ethical principles for medical research involving human subjectsJAMA2013310202191219424141714
- DaviesMJKermaniFFDA and clinical drug trials: a short historyDaviesMJKermaniFA Quick Guide to Clinical Trials1st edRockville, MDBioPlan Associates2008
- BoothCMTannockIFRandomised controlled trials and population-based observational research: partners in the evolution of medical evidenceBr J Cancer2014110355155524495873
- NallamothuBKHaywardRABatesERBeyond the randomized clinical trial: the role of effectiveness studies in evaluating cardiovascular therapiesCirculation2008118121294130318794402
- GuyattGHSackettDLSinclairJCUsers’ guides to the medical literature. IX. A method for grading health care recommendations. Evidence-Based Medicine Working GroupJAMA199527422180018047500513
- BurnsPBRohrichRJChungKCThe levels of evidence and their role in evidence-based medicinePlast Reconstr Surg2011128130531021701348
- PriceDChisholmAvan der MolenTReassessing the evidence hierarchy in asthma: evaluating comparative effectivenessCurr Allergy Asthma Rep201111652653821927929
- GuyattGHOxmanADVistGEGRADE: an emerging consensus on rating quality of evidence and strength of recommendationsBMJ2008336765092492618436948
- EvansDHierarchy of evidence: a framework for ranking evidence evaluating healthcare interventionsJ Clin Nurs2003121778412519253
- BartonSWhich clinical studies provide the best evidence? The best RCT still trumps the best observational studyBMJ2000321725625525610915111
- National Institute for Health and Clinical Excellence [webpage on the Internet]Developing NICE guidelines: the manual Available from: https://www.nice.org.uk/process/pmg20/chapter/introduction-and-overviewAccessed May 11, 2018
- LisYGuoJJRobertsMHKambleSRaischDWA comparison of US Food and Drug Administration and European Medicines Agency regulations for pharmaceutical risk management: report of the International Society for Pharmacoeconomic and Outcomes Research Risk Management Working Group Available from: https://www.ispor.org/sigs/riskbenefitmanagement/A-comparison-of-US-Food-and-Drug-Administration-and-European-Medicines-Agency.pdfAccessed May 11, 2018
- BellHWailooAJHernandezMThe use of real world data for the estimation of treatment effects in NICE decision making Available from: http://nicedsu.org.uk/wp-content/uploads/2018/05/RWD-DSU-REPORT-Updated-DECEMBER-2016.pdfAccessed June 1, 2018
- Forum on Drug Discovery, Development, and TranslationBoard on Health Sciences PolicyHealth and Medicine DivisionNational Academies of Sciences, Engineering and Medicine [webpage on the Internet]Examining the impact of real-world evidence on medical product development: IIncentives: proceedings of a workshop—in brief Available from: http://www.ncbi.nlm.nih.gov/pubmed/29431954Accessed March 3, 2018
- HoPMPetersonPNMasoudiFAEvaluating the evidence: is there a rigid hierarchy?Circulation2008118161675168418852378
- SunXTanJTangLReal-world evidence: experience and lessons from ChinaBMJ2018360j526229437644
- European Medicines AgencyUpdate on real world evidence data collection Available from: http://ec.europa.eu/health/files/committee/stamp/2016-03_stamp4/4_real_world_evidence_ema_presentation.pdfAccessed April 5, 2017
- MianiCRobinEVeronika HorvathV [webpage on the Internet]Health and Healthcare: Assessing the real-world data policy landscape in Europe Available from: https://www.rand.org/pubs/research_reports/RR544.htmlAccessed May 11, 2018
- SedgwickPProspective cohort studies: advantages and disadvantagesBMJ2013347f6726
- Medicines and Healthcare Products Regulatory Agency (MHRA) [webpage on the Internet]The Clinical Practice Research Datalink (CPRD) Available from: https://www.cprd.com/home/Accessed August 2, 2017
- CooneyA [webpage on the Internet]Examining the importance of IDNs: where their influence matters and where it doesn’t Available from: https://www.pm360online.com/examining-the-importance-of-idns-where-their-influence-matters-and-where-it-doesnt/Accessed November 5, 2017
- IBM Big Data and Analytics HubThe four V’s of big data Available from: http://www.ibmbigdatahub.com/infographic/four-vs-big-dataAccessed November 3, 2017
- SchaumbergDAMcDonaldLShahSEvaluation of comparative effectiveness research: a practical toolJ Comp Eff Res Epub2018 221
- BirnbaumHGCremieuxPYGreenbergPEUsing healthcare claims data for outcomes research and pharmacoeconomic analysesPharmacoeconomics199916118
- DanielRMCousensSNDe StavolaBLMethods for dealing with time-dependent confoundingStat Med20133291584161823208861
- AnnemansLAristidesMKubinM [webpage on the Internet]Real-life data: a growing need Available from: https://www.ispor.org/news/articles/oct07/rld.aspAccessed May 11, 2018
- ChatterjeeSDaviesMJKhuntiKWhat have we learnt from “real world” data, observational studies and meta-analysesDiabetes Obes Metab201820Suppl 1475829364585
- Bipartisan Policy CenterUsing real-world evidence to accelerate safe and effective cures Available from: https://bipartisanpolicy.org/wp-content/uploads/2016/06/BPC-Health-Innovation-Safe-Effective-Cures.pdfAccessed June 1, 2018
- BairdJReal-world evidence in Europe Available from: http://fgcasal.org/LibroRWD/Libro_RWD_Jesammy_Baird.pdfAccessed June 6, 2018
- MakadyAvan VeelenAJonssonPUsing real-world data in health technology assessment (HTA) practice: a comparative study of five HTA agenciesPharmacoeconomics201836335936829214389
- WiseJMollerAChristieDThe positive impacts of real-world data on the challenges facing the evolution of biopharmaDrug Discov Today201823478880129337204
- National Academies of Sciences, Engineering, and Medicine2017Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a WorkshopWashington, DCThe National Academies Press https://doi.org/10.17226/24685. Accessed May 11, 2018
- European VoiceFast-tracking medicines innovation: a question of uncertainties Available from: http://www.politico.eu/wp-content/uploads/2015/03/Fast-tracking-medicines-PDF.pdfAccessed April 5, 2017
- AlemayehuDCappelleriJCEmirBZouKHStatistical Topics in Health Economics and Outcomes ResearchBoca Raton, FLCRC PressTaylor and Francis Group2017
- The Network for Excellence in Health InnovationReal-world evidence: a new era for health care innovation Available from: http://www.nehi.net/writable/publication_files/file/rwe_issue_brief_final.pdfAccessed May 11, 2018
- ResnicFSMathenyMEMedical devices in the real worldN Engl J Med2018378759559729443668
- ABPIThe vision for real world data–harnessing the opportunities in the UK–demonstrating value with real world data Available from: http://www.abpi.org.uk/media/1378/vision-for-real-world-data.pdfAccessed May 11, 2018
- LisYRobertsMHKambleSComparisons of Food and Drug Administration and European Medicines Agency risk management implementation for recent pharmaceutical approvals: report of the International Society for Pharmacoeconomics and outcomes research risk benefit management working groupValue Health20121581108111823244814
- European Medicines Agency [webpage on the Internet]Adaptive pathways Available from: http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000601.jspAccessed April 6, 2017
- HoltorfAPWatkinsJBMullinsCDIncorporating observational data into the formulary decision-making process–summary of a roundtable discussionJ Manag Care Pharm200814330230818439052
- RocheNReddelHMartinRQuality standards for real-world research. Focus on observational database studies of comparative effectivenessAnn Am Thorac Soc201411Suppl 2S99S10424559028
- WillkeRJMullinsCD“Ten commandments” for conducting comparative effectiveness research using “real-world data”J Manag Care Pharm2011179 Suppl AS10S1522074669
- HernánMARobinsJMUsing big data to emulate a target trial when a randomized trial is not availableAm J Epidemiol2016183875876426994063