Use of Clinical Trial Characteristics to Estimate Costs of New Drug Development.

IMPORTANCE

Despite their importance to patients, health, and industry, the magnitude of investments in drug research and development (R&D) remain nebulous. New policies require more granular and transparent R&D cost estimates to better balance incentives for innovation and returns to developers.

OBJECTIVE

To estimate per-drug R&D costs using a novel, reproduceable approach and to describe firm-level R&D costs per discrete unit of R&D activity (1 patient-month).

DESIGN, SETTING, AND PARTICIPANTS: This economic evaluation used cross-sectional data to estimate 2014 to 2019 costs per patient-month. Costs per patient-month were calculated using data from 268 US publicly traded drug developers, contributing 1311 firm-year observations, that were highest ranked by assets or market capitalization, after exclusions. Per-drug costs were calculated from all R&D activity through approval for a cohort of 38 new drugs approved by the US Food and Drug Administration in 2019. Data were analyzed from January 2022 to July 2024.

EXPOSURE

R&D activity, measured in terms of clinical trial patient-months.

MAIN OUTCOMES AND MEASURES

This study used a 2-step approach to estimate R&D costs, first allocating firm-year-level total R&D spending across similarly aggregated patient-months, and then aggregating these incremental costs to estimate drug-level R&D costs per new drug.

RESULTS

Among 268 developers assessed, 20 large firms accounted for 80.8% of all patient-months and had 27.4% lower mean and 26.7% lower median costs per patient-month compared with other firms. Each 1% increase in patient-months was associated with a 0.9% increase in R&D costs. R&D costs per new drug were highly skewed, with a lower median (IQR), at $708 million ($247 million to $1.42 billion) than mean (SD), at $1.31 ($1.92) billion, after adjusting for the cost of capital and discontinued products. Without these adjustments, direct costs per new drug were a median (IQR) of $150 ($67.6-$453) million and a mean (SD) $369 of ($684) million. While estimated R&D costs varied in sensitivity analyses, mean costs were always substantially greater than median costs.

CONCLUSIONS AND RELEVANCE

This economic evaluation found median per-drug R&D costs toward the lower end of the range from prior studies, with a mean closer to the middle of the existing range despite the broad scope of included costs. These findings suggest parallel development across indications, adjustment for discontinued products, and a small number of expensive development programs are particularly important drivers of R&D costs.