Supporting Formulary Decisions: The Discovery of New Facts or Constructed Evidence?
A critical question, given the growing importance of more targeted therapies to support personalized and precision medicine, is the credibility of the evidence base to support formulary decisions and pricing. On the one hand, for those who subscribe to the reference case model of the National Institute of Health and Care Excellence (NICE) in the UK, the decision rests upon the creation of modeled or simulated imaginary worlds and the application of threshold willingness-to-pay cost-per-QALY thresholds. On the other hand, for those who subscribe to the standards of normal science, the decision rests upon the ability to evaluate competing claims, both clinical and cost-effective, in a timeframe that is meaningful to a formulary committee. If we subscribe to the scientific method where the focus is on the discovery of new facts, untestable claims for clinical benefit and cost-effectiveness, such as created claims for lifetime cost per-quality-adjusted discounted life years (QALYs), are properly relegated to the category of pseudoscience. We have no idea, and will never know, whether the claims are right or even if they are wrong. If formulary decisions are to respect the standards of normal science then there has to be a commitment to claims evaluation. A willingness to accept new products provisionally, subject to an agreed protocol to support the evaluation of clinical and cost-effectiveness claims. This dichotomy between the standards of normal science and pseudoscience is explored in the context of published claims for cost-effectiveness and recommendations for product pricing in the US.