SUPPORTING PRODUCT CLAIMS IN DRUG DEVELOPMENT- A FRAMEWORK FOR INTEGRATING PATIENT REPORTED OUTCOMES AND HEALTH ECONOMICS CONCEPTS AND ENDPOINTS

Author(s)

Christina Donatti, MSc, PsyD, Senior Outcomes Researcher1, Diane Wild, Msc, Director1, Andrew Briggs, DPhil, Professor2, Mark J Sculpher, PhD, Professor31Oxford Outcomes Ltd, Oxford, United Kingdom; 2 University of Glasgow, Glasgow, United Kingdom; 3 University of York, York, United Kingdom

To introduce, define and differentiate between conceptual models, endpoint models and conceptual frameworks, and to discuss how they fit together in the context of supporting product claims in drug development. In addition, to explore the added benefit of including health economic concepts and endpoints within the same framework. Using alcohol dependence (AD) as an illustrative example, a conceptual model, hypothetical endpoint model and conceptual framework (for a selected AD-specific PRO measure) were developed, following the newly issued guidance from the FDA relating to label claims. The potential for combining each of these elements together with related documents, such as the Target Product Profile to support a product claim, was assessed as was the feasibility of including health economic claims within a similar framework. The development of a holistic framework to support not only label claims, but also value claims is presented. The framework develops the recent guidance by the FDA for PROs and suggests that it may be possible to include health economic outcomes within the general framework. Doing so offers potential benefits in relation to applying the same rigorous treatment now required for PROs, encourages genuine collaboration between different stakeholders and provides a framework for communicating value concepts outside the discipline of outcomes research. In particular, clear specification of health economic outcomes within an endpoint model may assist with the design of prospective studies. Nevertheless, it is clear that value claims remain the responsibility of reimbursement authorities rather than regulators. Each of the models discussed are useful in supporting product claims in drug development and, from a PRO perspective, endpoint models and conceptual frameworks are essential. From a health economics perspective, employing a similar framework encourages scientific rigour, facilitates communication and could help design of appropriate data collection in clinical trials.

Conference/Value in Health Info

2008-05, ISPOR 2008, Toronto, Ontario, Canada

Value in Health, Vol. 11, No. 3 (May/June 2008)

Code

PMH87

Topic

Health Policy & Regulatory

Topic Subcategory

Approval & Labeling

Disease

Mental Health

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