Formulary Submissions: Value Claims, Protocols and Outcomes Based Contracting in Rare Disease.

Outcomes based payments contracting is in its infancy. The increased attention being given to rare disease place a premium on the ability to engage with payers to ensure that there is an analytical framework relevant to value claims contracting. Rare disease is not, of course, alone; many other chronic disease states may be suitable candidates and have been over the past 10 years or more. Rare disease, however stands apart: (i) the evidence base at product launch is limited; (ii) the therapy costs are often considered prohibitive; and (iii) the target patient population is small. At the same time, those seeking to implement an evidence-based engagement with health systems to support innovative rare disease interventions face a substantive technology assessment barrier. The focus in health technology assessment on assumption driven modeled cost-effectiveness simulations that support imaginary recommendations for cost-effective pricing and access is, however, an avoidable barrier. In the US, this barrier is the business model of the Institute for Clinical and Economic Review (ICER) and one endorsed by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Rare disease can be better served with other tools at our disposal with a proposed new start analytical framework in health technology assessment. The purpose of this brief note is to make the case that this proposed new start focused on single attribute value claims that meet the standards of normal science and fundamental evidence can not only dispense with the ICER imaginary modeling but, with a new start formulary submission package, integrate value claims with assessment protocols to set the stage for effective outcome-based contracting as the default standard for future payer negotiations.