REAL WORLD DATA – IMPROVING APPROACHES FOR DEMONSTRATING BENEFITS AND RISKS OF NEW DRUGS

Author(s)

Lucas F1, Arenson SM21Pope Woodhead and Associates, St. Ives, United Kingdom, 2Cambridge University, Cambridge, United Kingdom

OBJECTIVES: Regulators and payers view randomized controlled trials (RCT) as the gold standard for establishing the benefit/risk of new drugs. However, they are increasingly interested in real-world data (RWD) due to their external validity. This survey explored stakeholders’ perceptions and emerging trends in the area of RWD. METHODS: We identified relevant literature since 2006 via Google Scholar and manual search, and reviewed it based on several topics: types of RWD, pros and cons of different approaches, and impact of new statistical techniques and technology on availability and quality of RWD.  We then conducted 45-60 min in depth, semi-structured discussions with 17 experts from Academia, HTA bodies, health insurance, research organisations and pharmaceutical industry – from the UK, France, Germany, the Netherlands and the USA. Their views about value and future directions of RWD approaches were elicited. RESULTS: Experts unanimously thought that RCTs would remain a mandatory approach for the foreseeable future due to the limitations of RWD, mainly potential for confounding. New study designs (e.g. randomised database studies) and statistical techniques (e.g. high-dimensional propensity scoring) remove confounding only partially and need to gain credibility. There was a strong view that, while registries have been the reference source of observational data, there is an opportunity for (claims) database and electronic medical records to form an efficient platform for automatic, real-time analysis of naturalistic data. Despite a few good examples, it will, however, require time to resolve technical difficulty of linking databases and, crucially, the challenges of data ownership and privacy issues. Several experts predict the short-term rise of at-home monitors, ‘smart pills’ and ‘smart phones’ that automatically feed into databases, and the increased use of data from Google Health and Microsoft HealthVault.  CONCLUSIONS: RWD may eventually become the new gold standard in drug development, but this will occur only through incremental progress.

Conference/Value in Health Info

2010-11, ISPOR Europe 2010, Prague, Czech Republic

Value in Health, Vol. 13, No. 7 (November 2010)

Code

HT1

Topic

Health Policy & Regulatory, Health Service Delivery & Process of Care, Health Technology Assessment, Study Approaches

Topic Subcategory

Decision & Deliberative Processes, Post Marketing Studies, Prescribing Behavior, Registries, Reimbursement & Access Policy, Risk-sharing Approaches

Disease

Multiple Diseases

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