Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI's Patient Groups and Clinical Trials Project

Abstract

Background: While patient groups, regulators, and sponsors are increasingly considering engaging with patients in the design and conduct of clinical development programs, sponsors are often reluctant to go beyond pilot programs because of uncertainty in the return on investment. We developed an approach to estimate the financial value of patient engagement.

Methods: Expected net present value (ENPV) is a common technique that integrates the key business drivers of cost, time, revenue, and risk into a summary metric for project strategy and portfolio decisions. We assessed the impact of patient engagement on ENPV for a typical oncology development program entering phase 2 or phase 3.

Results: For a pre-phase 2 project, the cumulative impact of a patient engagement activity that avoids one protocol amendment and improves enrollment, adherence, and retention is an increase in net present value (NPV) of $62MM ($65MM for pre-phase 3) and an increase in ENPV of $35MM ($75MM for pre-phase 3). Compared with an investment of $100,000 in patient engagement, the NPV and ENPV increases can exceed 500-fold the investment. This ENPV increase is the equivalent of accelerating a pre-phase 2 product launch by 2½ years (1½ years for pre-phase 3).

Conclusions: Risk-adjusted financial models can assess the impact of patient engagement. A combination of empirical data and subjective parameter estimates shows that engagement activities with the potential to avoid protocol amendments and/or improve enrollment, adherence, and retention may add considerable financial value. This approach can help sponsors assess patient engagement investment decisions.

Keywords: expected net present value; patient engagement; risk-adjusted financial model; therapeutic development.

Figure 1.

Example of a simple ENPV model for a project in phase 2. Circles represent uncertain events; values adjacent to each path from the circles indicate the probability that the project will follow that path. Values on the right indicate the probability that the project will terminate following that path and the NPV for that path (eg, probability of technical and regulatory success = 22%). ENPV is calculated by totaling the product of the NPV and probability of each path. In this case, ENPV = (0.22 × $400) + (0.02 × –$45) + (0.16 × –$40) + (0.60 × –$3) = $77 MM [details in S1].) ENPV, expected net present value.