PROMPT VALUE ASSESSMENT OF BREAKTHROUGH INTERVENTIONS WITH LIMITED OUTCOME DATA
Author(s)
Arbel R1, Hammerman A2, Triki N3, Greenberg D4
1Sapir College, Sderot, Israel, 2Clalit Health Services, Tel-Aviv, Israel, 3Maccabi Healthcare Services, Tel-Aviv, Israel, 4Ben-Gurion University of the Negev, Beer-Sheva, Israel
OBJECTIVES: Many new breakthrough health-interventions are approved only according to favorite surrogate outcomes. Performing a traditional cost-effectiveness analysis with limited clinical outcome data at time of approval requires extensive modeling and many assumptions, making it vulnerable to scrutiny. A recent example is the Proprotein Convertase Subtilisin/Kexin type 9 inhibitors (PCSK9i) that were found to be very effective in reducing levels of LDL cholesterol, with only early evidence of reduction of major adverse cardiovascular events (MACE). Our objective was to suggest an alternative model for value assessment of breakthrough interventions, in the stage in which they have only limited clinical-outcome data, before a full economic-evaluation is feasible. METHODS: We propose to focus an initial value assessment on three straightforward economic implications. First, present the "number-needed to treat" (NNT) and the expected cost to prevent one major adverse event, using the available data at the time of approval. Second, evaluate adoption projections of the new technology and the related budget-impact. Finally, identify sub-groups of patients that would benefit the most from the new intervention. RESULTS: We assimilated the proposed model on PCSK9i as a case study, using outcome and follow-up data from published phase III trials and the official list prices (all data as of March 2016). We found that the cost of preventing any one MACE would be higher than $2,000,000 (NNT 140). The potential total budget-impact of adopting PCSK9 according to the approved indications could be over $100 billion dollars annually in the USA. Identifying sub-groups of patients that would benefit the most from PCSK9 treatment is still required for consideration in reimbursement decisions. CONCLUSIONS: Using our proposed model may enable to provide meaningful initial value assessments to breakthrough interventions, despite severe outcome data constraints. Prompt assessments are essential to guide and support reimbursement and funding decisions for such Interventions.
Conference/Value in Health Info
2016-10, ISPOR Europe 2016, Vienna, Austria
Value in Health, Vol. 19, No. 7 (November 2016)
Code
PHP190
Topic
Health Service Delivery & Process of Care
Topic Subcategory
Health Care Research
Disease
Multiple Diseases