Targeting drug development

Muthiah Vaduganathan and Vinay Prasad in a JAMA Viewpoint:

From a pragmatic standpoint, drug development programs conducted in broad populations poorly prioritize which patients should start therapy first. If provided regardless of risk, expensive new first-in-class agents may overwhelm health care budgets. In hepatitis C management, novel drug therapies broadly indicated for most patients with chronic hepatitis C, such as sofosbuvir, cost approximately $1000 per pill, presenting major cost challenges to drug implementation and distribution. In an effort to balance access and affordability, recent hepatitis C clinical practice guidelines have encouraged use of these agents primarily in the sickest subgroup of patients. In the current financial environment, emerging clinical trials should consider selecting the groups at highest risk to guide an economically viable and practical approach to drug utilization.

This gets at the point that even a very cost-effectiveness therapy can overwhelm budgets. The allocative efficiency problem still needs to be addressed.


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