Dr. Julia Snider’s research on the impact of delays in patient access to new CAR-T therapies for patients with pediatric lymphoma gained substantial media attention at ASCO’s 2018 annual conference, [including from HemOnc Today and the Journal of Clinical Pathways]. High Leverage Outlier sits down with Dr. Snider to discuss the importance of this study.
Q1: What were the takeaway conclusions from your research on delays in access to new CAR-T treatments?
Patients taking older therapies while waiting for the approval of CAR-T have very high mortality rates so, the longer they wait, the less likely it is that they will live to try CAR-T. CAR-T therapies are new and, in many cases, access and reimbursement policies are still being decided. The important thing is to ensure that patients gain timely access to these therapies given that many patients truly cannot afford to wait. The current data suggest the potential for long-term survival gains for many patients treated with CAR-T, and our results show that these gains in survival and quality of life translate to a large social value.
Q2: What is the concept of social value and why is it important?
Social value represents the concept of the total economic value generated by a treatment, including the value of health and productivity gains to patients, caregivers, and other impacted stakeholders. This concept is related to cost-effectiveness, which captures the clinical benefit of a treatment (often in terms of quality-adjusted life years, or QALYs) in comparison with the net costs of the treatment. Social value takes the perspective of society at large rather than that of a specific stakeholder, such as a payer, to quantify the total value of a therapy. At the same time, social value calculations allow for a decomposition of the sources of value to enable an understanding of, for example, what proportion of the value of a treatment is retained by a manufacturer through sales compared with the proportion of the value that patients and other stakeholders impacted by the treatment receive.
Q3: Delays in access to medicines reflects a trend in insurance benefit design to restrict the ability of patients to obtain high-cost treatments without demonstrated need. What are the promises and risks of this trend?
Insurance benefit designs have become more complicated in an attempt by payers to reduce wasteful spending and shift financial accountability to patients. In some ways, these changes incentivize the use of value-based care, but careful attention needs to be paid to ensuring that cost containment does not result in discouraging the use of higher price treatments when they provide value.
There is increasing acceptance among healthcare stakeholders of the importance of personalized and precision medicine in value-based care delivery. Perhaps too much attention has been paid to the high list prices of some personalized therapies, rather than their value. In the case of CAR-T treatments, cost-effectiveness analyses have affirmed that the price is aligned with the value they provide to patients. Payment systems that currently accommodate years of chronic disease treatments like statins need to adapt to the unique challenges and benefits provided by high-price, one-time treatments that can cure a patient of a disease. It’s crucial to maintain incentives for companies to continue to develop potential cures.