PROGRESSIVE LICENSING AND VALUE FOR MONEY- USE OF RESPONDER ANALYSIS IN ECONOMIC EVALUATION
Author(s)
Heather P McDonald, MSc, Senior Manager, Health Economics and Outcomes Research1, Mayvis Rebeira, MSc, Manager, Health Economics1, Pierre Isogai, HBSc, Statistician2, N Mittmann, PhD, Director, HOPE Research Centre2, Barbara Jaszewski, HonBSc, MBA, Director, Reimbursement11Bayer Healthcare, Toronto, ON, Canada; 2 HOPE Research Centre, Toronto, ON, Canada
Objective: In responder-based reimbursement scenarios, patients who have demonstrated benefit after a test period are reimbursed for continued use of a therapy. Benefit is evaluated based on a priori criteria regarding what constitutes a clinically important improvement. Payers incur costs for the patients who meet established responder criteria for a given therapy. Methods: An economic evaluation of Sativex® vs. standard analgesic care in adults with Multiple Sclerosis and neuropathic pain is used to illustrate the application of a responder-based reimbursement scenario (N=66). Sativex® response was defined as a reduction in pain score (2-points on BS-11) on the 10th day of treatment based on clinician opinion. Base case and responder-based incremental cost-utility ratios (ICUR) were determined. Efficacy and safety data were based on a phase III pivotal trial. Costs (CND$ 2006) were based on provincial sources. Direct medical resources were taken from a burden of illness study. Results: In the base case, the incremental cost was $5339 and incremental QALY was 0.13. The ICUR was $70,103/QALY. In the responder-based economic analysis, 55.9% of Sativex® patients were defined as responders, incremental costs was $9352 and incremental QALY was 0.21, resulting in an ICUR of $44,917/QALY. Conclusion: Progressive licensing, in the form of conditional approvals, allows for earlier market access to pharmaceutical products. Based on the conditional nature of these approvals, the evidence for a new therapy may not be definitive. However, their potential benefit, by filling a void for previously unmet medical need, is significant. As payers are interested in maximizing value for money, they may not feel compelled to fund a conditionally-licensed product, when the data supporting its value is considered uncertain. To increase the confidence in the value for money proposition, responder-based reimbursement scenarios may be an option.
Conference/Value in Health Info
2008-05, ISPOR 2008, Toronto, Ontario, Canada
Value in Health, Vol. 11, No. 3 (May/June 2008)
Code
PHP81
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
Multiple Diseases