Assessing the Value of Integrated Evidence Approaches in Drug Development.

Background: The use of Integrated Evidence Plans (IEPs) by the pharmaceutical industry has expanded in recent years with the aim of optimizing healthcare and patient outcomes. The evidence base of IEPs goes beyond traditional randomized controlled trials to provide holistic evidence suitable for all stakeholders and allows for consideration of different packages in different regions. However, this approach to drug development is not systematically adopted by all sponsors because of perceived uncertainty in its investment value.

Methods: We introduce the concept of value drivers to which we apply an expected net present value (eNPV) model of the cash flows for drug development and commercialization. The approach is outlined for two, typical, hypothetical lifecycle management IEPs. The measure of IEP value is defined as the increment in eNPV that occurs when IE programs are employed in comparison to when they are not.

Results: We found substantial value for IEPs. One example incorporated a plan to conduct an observational study that could be used as a basis for approval in lieu of a classical phase II trial for a supplemental indication. In the other example, increased adoption of the new treatment leads to a highly positive increment in eNPV based on the critical evidence generated in a phase IIIb study.

Conclusions: Use of value drivers and eNPV-based value models when planning for IEPs can provide objective guidance for project teams. The value can be estimated through formal economic analysis that considers planned timelines, R&D costs, estimates of the likelihood of regulatory approval, patient access and clinical adoption if development is successful.