USING VALUE OF INFORMATION METHODS WHEN THE DISEASE IS RARE AND THE TREATMENT IS EXPENSIVE – THE EXAMPLE OF HEMOPHILIA A

Monday, October 21, 2013
Key Ballroom Foyer (Hilton Baltimore)
Poster Board # P2-2
Applied Health Economics (AHE)

Lusine Abrahamyan, MD, MPH, PHD, University of Toronto, Toronto, ON, Canada, Andrew R. Willan, PhD, SickKids Research Institute, Toronto, ON, Canada, Joseph Beyene, MSc, PhD, McMaster University, Hamilton, ON, Canada, Marjorie Mclimont, MSc, The Hospital for Sick Children Research Institute, Toronto, ON, Canada, Victor S. Blanchette, MD, FRCPC, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada and Brian M. Feldman, MD, MSc, FRCPC, The Hospital for Sick Children, Toronto, ON, Canada
Purpose: To illustrate, in a decision context, the application of value of information (VOI) methods for assessing current evidence for the use of expensive health care interventions in rare diseases, in particular – recombinant Factor VIII (FVIII) for children with severe hemophilia A. The objective of the study was to use VOI methods to evaluate the current evidence in support of using (i) alternate day prophylaxis (AP), (ii) tailored prophylaxis (TP) or (iii) on-demand treatment (OD) with FVIII to prevent arthropathy in children with severe hemophilia A. 

Method: To apply VOI methods, several parameters such as disease incidence, trial enrollment fraction, time horizon for the decision, costs and threshold values to avoid arthropathy were defined. Two baseline threshold values (willingness to pay) for avoiding arthropathy – $200,000 and $400,000 – were selected for comparing the treatment strategies. 

Result: For threshold values < $200,000 OD had a higher estimated net benefit than either prophylaxis strategy. For threshold values > $400,000 prophylaxis strategies had higher estimated net benefit. At a threshold value of $200,000 OD was superior to both AP and TP. At a threshold value of $400,000, both prophylaxis strategies were superior to OD; however, a new trial with 38 patients per arm was needed to compare AP and TP, yielding an expected net gain of over $17 million. 

Conclusion: In health policy decision-making, the recommendation for the adoption of new health interventions is made on the basis of a positive expected incremental net benefit. However, in rare diseases often the evidence is scarce and insufficient for decision making. In considering the funding of new research and patient reimbursement in rare diseases, VOI methodology provides more relevant determinations of the value and costs of additional research. The results of this study can help in decision making for treatment strategies and in the planning of future studies for hemophilia and other rare diseases.