The study – published this week in JAMA Internal Medicine – was conducted by a team of researchers from Johns Hopkins Bloomberg School of Public Health and included a comprehensive assessment of the costs of pivotal clinical trials.
According to the researchers, clinical trials that support US Food and Drug Administration (FDA) approvals of new drugs have a median cost of $19m – representing less than one percent of the average drug development cost, which is estimated at between $2 and $3bn.
Half of the trials examined were estimated to cost between $12m and $33m.
This price tag came as a surprise to the researchers who are the first to conduct such an assessment, said study senior author G. Caleb Alexander, MD, MS, associate professor of epidemiology and medicine at Johns Hopkins Bloomberg School of Public Health.
“In planning the study, we were really surprised that such an analysis has not yet been performed, since these trials generate fundamental new knowledge not only for the FDA, but also that clinicians and patients rely upon to understand drug safety and effectiveness,” Alexander told Outsourcing-Pharma.com.
“Interestingly, the costs were relatively low, given the total costs of drug development and the price tag that consumers pay,” he added.
Additionally, as it pertains to the quality of the information derived from these studies, the researchers found “you get what you pay for,” said Alexander.
Approximate costs were calculated with standard cost-estimation software used by contract research organizations (CROs) and others in the pharmaceutical industry.
The researchers examined 138 trials that led to FDA approvals from 2015 to 2016. The trials covered 59 different drugs, with some tested in multiple trials for multiple disorders.
Alexander said the results “challenge the commonly held belief that it enormous sums of money are required to study the safety and effectiveness of prescription drugs in clinical trials.”
"It’s true that these trials aren’t cheap, but they represent just a small fraction of the total tab of drug development."
The lowest estimated trial cost was $2m for a four-patient trial of a treatment for a rare metabolic disorder. At $347m, the highest estimate was for a large heart-failure drug study.
The median cost of trials that test a drug's ability to prevent a clinically meaningful outcome, such as a heart attack, rang in at $65m.The median cost of trials using a surrogate outcome, such as high serum cholesterol, was $24m.
Costs also were higher for trials comparing a new drug to an existing standard rather than a placebo and when longer in duration or enrolled with more patients, according to the researchers.
Trials with fewer than 100 patients had an average cost of just $6m; those with more than 1,000 patients had an average cost of $77m.
Across treatment areas, pivotal cardiovascular drug trials had a mean cost of $157m, while trials in endocrine and metabolic disease patients averaged $21m.
“Our study generates fundamental new knowledge regarding the costs of a crucial step in drug development and provides a platform from which to base further work examining what it would cost to build better trials,” said Alexander, “trials that provide higher quality information regarding the outcomes that matter most of patients and providers.”
Alexander and his colleagues, including first author Thomas Moore from the Milken Institute of Public Health at George Washington University, plan to study further the characteristics of drug trials and how they affect costs.
Source: JAMA Internal Medicine
Estimated Costs of Pivotal Trials for Novel Therapeutic Agents Approved by the US Food and Drug Administration, 2015-2016
Authors: Thomas Moore, Hanzhe Zhang, Gerard Anderson, and G. Caleb Alexander