Delivering the precision oncology paradigm: reduced R&D costs and greater return on investment through a companion diagnostic informed precision oncology medicines approach

Abstract

Background: Precision oncology medicines represent a paradigm shift compared to non-precision oncology medicines in cancer therapy, in some situations delivering more clinical benefit, and potentially lowering healthcare costs. We determined whether employing a companion diagnostic (CDx) approach during oncology medicines development delivers effective therapies that are within the cost constraints of current health systems. R&D costs of developing a medicine are subject to debate, with average estimates ranging from $765 million (m) to $4.6 billion (b). Our aim was to determine whether precision oncology medicines are cheaper to bring from R&D to market; a secondary goal was to determine whether precision oncology medicines have a greater return on investment (ROI).

Method: Data on oncology medicines approved between 1997 and 2020 by the US Food and Drug Administration (FDA) were analysed from the Securities and Exchange Commission (SEC) filings. Data were compiled from 10-K, 10-Q, and 20-F financial performance filings on medicines' development costs through their R&D lifetime. Clinical trial data were split into clinical trial phases 1-3 and probability of success (POS) of trials was calculated, along with preclinical costs. Cost-of-capital (CoC) approach was applied and, if appropriate, a tax rebate was subtracted from the total.

Results: Data on 42 precision and 29 non-precision oncology medicines from 56 companies listed by the National Cancer Institute which had complete data available were analysed. Estimated mean cost to deliver a new oncology medicine was $4.4b (95% CI, $3.6-5.2b). Costs to bring a precision oncology medicine to market were $1.1b less ($3.5b; 95% CI, $2.7-4.5b) compared to non-precision oncology medicines ($4.6b; 95% CI, $3.5-6.1b). The key driver of costs was POS of clinical trials, accounting for a difference of $591.3 m. Additional data analysis illustrated that there was a 27% increase in return on investment (ROI) of precision oncology medicines over non-precision oncology medicines.

Conclusion: Our results provide an accurate estimate of the R&D spend required to bring an oncology medicine to market. Deployment of a CDx at the earliest stage substantially lowers the cost associated with oncology medicines development, potentially making them available to more patients, while staying within the cost constraints of cancer health systems.

Box 1

Data grading system

Fig. 1

Flowchart of identified studies and data grading. Flowchart showing the identification, screening and grading of oncology medicines used to estimate the difference in the estimated R&D spend between precision and non-precision oncology medicines. From the dataset, CAR T-cell therapies, radiopharmaceuticals, and hormonal blockers, etc., were excluded and the remaining 112 oncology medicines split into precision and non-precision oncology medicines. Oncology medicines without clinical trial data were removed (Grade E and F). Finally, precision medicines which did not consistently use a CDx in their trials were excluded (n = 7), as were non-precision medicines which did use a CDx in their clinical trials (n = 8). CDx companion diagnostic, R&D research and development

Fig. 2

Estimated research and development expenditures of oncology medicines. A Precision oncology medicines. Panel B Non-precision oncology medicines. R&D research and development

Fig. 3

Return on investment of precision and non-precision oncology medicines, restricted from 1997 to 2015. A Precision oncology medicines. B Non-precision oncology medicines