MedicalResearch.com Interview with:
Marcus S. Shaker, MD
Associate Professor of Pediatrics
Associate Professor of Community and Family Medicine
Dartmouth-Hitchcock Medical Center
MedicalResearch.com: What is the background for this study?
Response: There are two peanut allergy treatments that are being evaluated for potential FDA approval—an orally administered treatment and an epicutaneous (skin based) treatment. Both have tremendous potential benefit. The focus of our study was to explore the range of health and economic benefits in terms of establishing pathways for how each therapy could be cost effective.
We want to be clear that our purpose was not to suggest one therapy is or is not cost effective at present. That would be a ridiculous statement to make regarding two treatments that not only lack FDA approval, but do not have established pricing. Rather, we used preliminary inputs that are presently available to create as robust a model as we could to better determine the individual paths that would make them more or less cost-effective.
MedicalResearch.com: What are the main findings?
Response: Cost-effectiveness research involves a lot of uncertainty, and that is clearly the case here as just mentioned. However, a key first step was to identify the instrumental levers in this process.
We show that, aside from price (which is the obvious lever), that the degree of change in health utility (e.g., the relative value of the health state that being allergic to peanut and then undergoing successful peanut allergy treatment represents) is highly critical, as is the likelihood that someone using therapy may eventually develop what is called “sustained unresponsiveness” (e.g., no longer clinically reactive to peanut) and can stop therapy. Interestingly, the range/degree of change of each of these attributes that makes each therapy potentially cost-effective is slightly different for each therapy.
But simply, how much net beneficial change therapy delivers, as well as the chances that you could eventually stop therapy matter a lot. At present, there are significant knowledge gaps to these inputs—health utility of individuals with peanut allergy at baseline and then after treatment has not been established or formally studied (so surrogate measures were used), and there is little data on the likelihood of reaching sustained unresponsiveness with either therapy. So, instead of trying to create too specific of a present-case scenario, we approached this from the standpoint of trying to show the key variables and the ranges under which these therapies could work.
MedicalResearch.com: What should readers take away from your report?
Response: The take home message is this—either therapy can absolutely be cost-effective, and there are wide ranges of key assumptions that influence that outcome. When therapies do not have a price or are approved, there is advantage to the approach we take because this analysis helps establish the targets necessary for cost-effective practice. Nothing is set in stone and it would be pennywise and pound foolish to reach a firm conclusion that one product is more or less cost effective than the other. If you look at our model, the ranges overlap considerably, and there are multiple ways for each to therapy to reach cost-effectiveness. Our analysis provides a road map to better understand these issues.
MedicalResearch.com: What recommendations do you have for future research as a result of this work?
Response: This exercise will need to be repeated a few times, as we learn more about health utility, rates of sustained unresponsiveness that the products can deliver, the relative safety profiles of these products that may influence a parent to prefer one therapy over the other (or stay on a therapy once started), and what parents really hope and expect from these therapies. This is the start of this process, not the end, and we caution against making firm conclusions about these products before they are approved, priced, and sold on the market.
Our disclosures are as stated in the article.
Shaker M, Greenhawt M. Estimation of Health and Economic Benefits of Commercial Peanut Immunotherapy Products: A Cost-effectiveness Analysis. JAMA Netw Open. 2019;2(5):e193242. doi:10.1001/jamanetworkopen.2019.3242
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