Online Program

The use of Simulation Modeling in the Protocol Development of an Outcomes Trial Involving Multiple Collaborators

Keywords: Simulation, Outcomes Trial, Collaborators

The development of a large-scale outcomes trial requires an enormous commitment from all parties involved in the execution of a study. Decisions towards the appropriate inclusion and hierarchy of potential study endpoints, early stopping criteria for overwhelming efficacy / futility, multiplicity adjustment, and appropriate conservatism towards the study power are all highly influential towards designing, sizing and estimating the completion of the study. Importantly, when multiple organizations are involved in an outcomes study, reaching consensus on these components can be especially challenging. Through the use of detailed simulation results, we show how these critical design components were made more transparent so eventual agreement could be achieved. Ultimately, achieving consensus among the partnership principals does not necessarily require strict agreement on all simulation assumptions. Instead, simulations need to include a level of conservatism and range of assumptions such that the concerns of all parties are reasonably represented and acknowledged. In the end, give and take by all partners is required for a successful collaboration.