Journal of Comparative Effectiveness ResearchVol. 1, No. 1s ForewordFree AccessR&D transformation and value-based innovationRobert S EpsteinRobert S EpsteinDepartment of Advanced Clinical Science and Research, Medco Health Solutions, Franklin Lakes, NJ, USASearch for more papers by this authorPublished Online:24 Oct 2011https://doi.org/10.2217/cer.11.5AboutSectionsPDF/EPUB ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareShare onFacebookTwitterLinkedInRedditEmail In the current healthcare environment, bringing a new drug to market infers that it has not only received regulatory approval, but also a favorable decision from payers and health technology assessors. As healthcare costs across the globe escalate and the demand for better control of spending and balanced budgets increase, it has become a necessity that the therapeutic value of an innovative technology be demonstrated. Payers play an increasingly important role in the decision-making process of the use of new technologies by determining coverage and reimbursement, which is based largely on evidence that demonstrates the value over current therapies. However, the standard approach to drug development has been geared towards regulatory requirements – demonstration of efficacy and safety, generally against placebo – leaving a gap in evidence required by payers and health technology assessors.From the payer’s perspective, comparative data against alternative treatments are essential information needed for reimbursement decisions, yet a recent study found that only about half of new drugs approved in the USA since 2000 had active comparative studies as part of the pivotal data included in the US FDA approval package [1]. Similarly, an analysis of the literature from six high-impact general medicine journals found that commercially funded studies were less likely to be comparative effectiveness trials than other types of trials [2]. Because regulatory requirements related to research design have been fairly clearly outlined for drug approval, the R&D model within the pharmaceutical industry has grown up around it – into a well-honed systematic generation of safety and efficacy and supporting data. By contrast, the evidentiary requirements for drug reimbursement by payers have only recently been clarified and the existing R&D model has not uniformly changed to incorporate these needs. With the increasing demand for comparative data from payers, physicians and patients, it is evident that pharmaceutical companies need to reshape their R&D model to adapt to this changing environment.This supplement addresses a new approach to value-based drug development and provides a framework for a general approach to obtain the patient-centric information for incorporation into the clinical development of any new technology. The three articles explore novel ways to incorporate these needs into drug development, and outline external parallel efforts to examine and incorporate comparative effectiveness into the equation.This supplement forms an accompaniment to the first issue of the new Journal of Comparative Effectiveness Research launching in January 2012. Through rigorous evaluation and comprehensive coverage, the Journal of Comparative Effectiveness Research provides stakeholders (including patients, clinicians, pharma, payers and health policy makers) with the key data and opinions to make informed and specific decisions on policy and clinical practice.Bibliography1 Goldberg NH, Schneeweiss S, Kowal MK, Gagne JJ. Availability of comparative efficacy data at the time of drug approval in the United States. JAMA305(17),1786–1789 (2011).Crossref, Medline, CAS, Google Scholar2 Hochman M, McCormick D. Characteristics of published comparative effectiveness studies of medications. JAMA303(10),951–958 (2010).Crossref, Medline, CAS, Google ScholarFiguresReferencesRelatedDetailsCited ByBusiness analysis for a sustainable, multi-stakeholder ecosystem for leveraging the Electronic Health Records for Clinical Research (EHR4CR) platform in EuropeInternational Journal of Medical Informatics, Vol. 97Cost-benefit assessment of using electronic health records data for clinical research versus current practices: Contribution of the Electronic Health Records for Clinical Research (EHR4CR) European ProjectContemporary Clinical Trials, Vol. 46The Current Drug Development Paradigm: Responding to US and European Demands for Evidence of Comparative Effectiveness and Relative EffectivenessSSRN Electronic JournalElectronic health records: new opportunities for clinical research18 October 2013 | Journal of Internal Medicine, Vol. 274, No. 6 Vol. 1, No. 1s Follow us on social media for the latest updates Metrics History Published online 24 October 2011 Published in print January 2012 Information© Future Medicine LtdPDF download
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