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Predictive Methodologies to Better Manage Costs of Innovative Therapies

WongNovel innovations in cell and gene therapies mark a new era in medicine whereby durable remission, and potentially cures, are within reach for several diseases. These advancements are clearly beneficial and have been shown to be worthwhile through cost-effectiveness analysis. However, they present challenges to a health care payment system that historically has been created to pay for and incentivize disease treatment that typically align payments and benefits over time. The columns and articles in this issue discuss how the US health system and its stakeholders need to evolve to make these therapeutic advancements feasible financially. 

Historically, pharmaceutical breakthroughs have been made possible through the alignment of market incentives to encourage investment in risky innovation. For example, the “miracle drug” penicillin was able to be studied and produced in enough quantities, in part through investment by American pharmaceutical companies, to support the war effort and later the wider public. The development of statins further demonstrates the importance of incentives to encourage investment and payment systems to encourage broad access. In their column, Sean McElligott, MS, and Ira Klein, MD, MBA, FACP, discuss how curative therapies are disrupting the system, what alternate payment and financing mechanisms are being proposed and created, and how decision makers are addressing affordability. 

The two features in this issue echo the above discussion, providing strategies and insight into practical ways to create a clearer financial picture to potentially inform alternative reimbursement strategies. Defining drug value has been elusive with perspectives differing across patients, society, providers, and drug companies. Generally, value is either based on cost-effectiveness analysis or outcome per dollar spent. In his study, Helmy M Guirgis, MD, assessed previously published outcome studies in first- and second-line treatment of advanced/metastatic non–small cell lung cancer lacking the epidermal growth factor receptor mutations and anaplastic lymphoma kinase. The main objective was to weigh costs vs probability of survival and crude dollar value of the immune checkpoint inhibitors. A secondary objective was to investigate the impact that dosage, duration, and drug combinations have on costs.

The Oncology Care Model (OCM) uses payment incentives and practice redesign requirements toward the goal of improving oncology care quality while controlling costs. January 2019 represented a major inflection point for the OCM, marking the halfway point of the 5-year program. Based on their sustained interactions and collaboration with many of the practices participating in the OCM, Charles Saunders, MD, and Jennifer Webster, MS, explain why 2019 is a seminal year for the OCM and discuss what strategic areas participating practices are focusing on in pursuit of success under the program.