ABSTRACT
In many situations, pharmaceutical sponsors are considering development at risk. One example is having to address multiple stakeholders’ requirements, such as regulators and payers. Sponsors’ dilemma in this situation is which approach to take to address these requirements, sequential or parallel. In the sequential approach sponsors are focused on addressing regulatory requirements first. Any additional payers’ requirements are addressed if and when the regulatory approval is achieved. In the parallel approach sponsors are addressing regulatory and payers’ requirements simultaneously, therefore they are developing at risk. We will be using a decision-analytic approach to provide an optimal solution to this problem. We will also present an example to illustrate the optimal solution. Investing at risk, however, has an impact not only on the development program itself, but on the company’s portfolio as a whole. Given that budgets are set at the portfolio level, additional funds invested at risk in one program have to be taken away from other programs. We will be addressing this as a knapsack problem to provide the optimal solution at the portfolio level. Finally, we will be providing an optimal solution to addressing multiple stakeholders’ requirements for a basket design in a confirmatory setting.
