Real World Data in Adaptive Biomedical Innovation: A Framework for Generating Evidence Fit for Decision-Making.
Analyses of healthcare databases (claims, electronic health records [EHRs]) are useful supplements to clinical trials for generating evidence on the effectiveness, harm, use, and value of medical products in routine care. A constant stream of data from the routine operation of modern healthcare systems, which can be analyzed in rapid cycles, enables incremental evidence development to support accelerated and appropriate access to innovative medicines. Evidentiary needs by regulators, Health Technology Assessment, payers, clinicians, and patients after marketing authorization comprise (1) monitoring of medication performance in routine care, including the materialized effectiveness, harm, and value; (2) identifying new patient strata with added value or unacceptable harms; and (3) monitoring targeted utilization. Adaptive biomedical innovation (ABI) with rapid cycle database analytics is successfully enabled if evidence is meaningful, valid, expedited, and transparent. These principles will bring rigor and credibility to current efforts to increase research efficiency while upholding evidentiary standards required for effective decision-making in healthcare.
Division of Pharmacoepidemiology (DoPE), Department of Medicine, Brigham & Women's Hospital, Boston, Massachusetts, USA.
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Schneeweiss
European Medicines Agency (EMA), London, United Kingdom.
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HG
Eichler
Agència de Qualitat i Avaluació Sanitàries de Catalunya (AQuAS), Barcelona, Spain.
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Garcia-Altes
Sanofi, Paris, France.
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C
Chinn
Bluebird Bio, Inc., Cambridge, Massachusetts, USA.
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Eggimann
National Institute for Health and Care Excellence (NICE), London, United Kingdom.
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Garner
National Health Care Institute, Diemen and Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht, The Netherlands.
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Goettsch
Health Products and Food Branch, Health Canada, Ottawa, Ontario, Canada.
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Lim
Gemeinsamer Bundesausschuss (GBA), Abteilung Arzneimittel, Berlin, Germany.
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Löbker
Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
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Martin
Gemeinsamer Bundesausschuss (GBA), Abteilung Arzneimittel, Berlin, Germany.
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Müller
Seoul National University, College of Medicine, Department of Preventive Medicine, Seoul, South Korea.
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Park
Department of Population Medicine, Harvard Medical School and Harvard Pilgrim Healthcare Institute, Boston, Massachusetts, USA.
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Platt
Comprehensive Health Insights (CHI), Humana, Louisville, Kentucky, USA.
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Priddy
Aetion Inc., New York, NY, USA.
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Ruhl
Health Products Regulatory Authority (HPRA), Dublin, Ireland.
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Spooner
Innovative Medicine Initiative - European Medical Information Framework, Janssen Pharmaceutica Research and Development, Beerse, Belgium.
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Vannieuwenhuyse
International Society for Pharmacoeconomics and Outcomes Research, Lawrenceville, New Jersey, USA.
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Willke
D035843:Biomedical Research / Q000458:organization & administration*; D016208:Databases, Factual / Q000706:statistics & numerical data*; D003657:Decision Making; D003695:Delivery of Health Care / Q000458:organization & administration* / Q000592:standards; D004059:Diffusion of Innovation; D017598:Efficiency, Organizational; D057286:Electronic Health Records; D006297:Health Services Accessibility; D006801:Humans; D013673:Technology Assessment, Biomedical