OBJECTIVES

To evaluate the modeling approaches taken in Institute for Clinical and Economic Review (ICER) assessments of pharmacological interventions and stakeholder perception of less common modeling approaches.

METHODS

The Modeling Analysis Plans (MAPs) published by ICER were searched in December 2019; all published MAPs were reviewed and key information relevant to the modeling approach was extracted by a single reviewer. For MAPs that described “less common” modeling approaches, defined as those that were not built in Microsoft Excel^® and/or were individual-patient level simulations, public comments on the Draft Scoping Document (DSD) and responses to comments on the Draft Evidence Report (DER) were reviewed to determine stakeholder perception of these modeling approaches.

RESULTS

Thirty-three MAPs were published between February 8, 2017 and November 21, 2019. Most MAPs assessed multiple interventions in a given disease area (72.7%), rather than a single intervention, and most were cost-effectiveness analyses (97.0%). The most common model structures planned were cohort Markov models (66.7%) and most were built in Excel^®(81.8%). Six analyses were identified which described “less common” modeling approaches and were investigated in further detail: arthritis (2019), diabetes (2019), opioid use disorder (2018), cystic fibrosis (2018), diabetes (2016) and non-alcoholic steatohepatitis (2016). In the public comments on the DSD and DER, stakeholders frequently did not comment on the generic modeling approach. Patient organizations and manufacturers were largely in support of the use of individual patient-level simulation models as these were thought to enable more accurate representations of heterogeneous disease areas. Patient organizations criticized the use of the cost per quality-adjusted life year (QALY) framework as under-representing benefits associated with the interventions.

CONCLUSIONS

Most models proposed in MAPs published by ICER follow common modeling approaches; however, use of less common modeling approaches was generally not challenged by manufacturers and patient organizations.