New drug trials for hospital-acquired pneumonia cost $89,600 per patient

Phase 3 clinical trials of new drugs for hospital-acquired and ventilator-associated pneumonia cost nearly $90,000 per patient, study data showed.

Researchers wrote that biopharmaceutical companies and regulatory agencies should improve screening and recruitment strategies to reduce study costs.

“New antibiotics are needed to treat MDR bacteria, and several entities are working to increase drug development efforts,” Stella Stergiopoulos, MS, MPH, a research fellow at the Tufts Center for the Study of Drug Development at Tufts University in Boston, and colleagues wrote. “However, despite the critical need, biopharmaceutical organization investment in new antibiotics for [hospital-acquired bacteria pneumonia (HABP) and ventilator-associated bacterial pneumonia (VABP)] and other infectious diseases, and new drug approvals have not kept up with the need.”

Two independent surveys from 2015 showed that high costs and low returns were major reasons for a lack of new antibiotic development.

“To our knowledge, studies identifying key cost drivers of clinical trials have been conducted, but no studies have calculated the specific total cost of HABP/VABP drug development in the U.S. using a comprehensive cost model to identify opportunities for decreasing overall costs,” the researchers wrote.

Stergiopoulos and colleagues used clinician input, as well as input from industry representatives and the FDA, to evaluate cost drivers for trials of new bacterial pneumonia drugs. The researchers used proprietary and commercial databases to create a model that would calculate the direct and indirect costs of a typical phase 3 HABP or VABP study compared with an oncology or endocrinology clinical trial. Based on feedback from those who participated in the studies, the researchers assumed a phase 3 HABP or VABP trial would include 1,000 patients from 200 study sites worldwide, and that the randomization rate would be one patient for every 10 patients screened.

The model showed that a 1,000-patient, 200-site phase 3 trial costs $89,600 per patient, the researchers reported. The overall cost was an estimated $89.6 million, compared with $39.1 million for a typical phase 3 oncology trial and $33.6 million for a typical phase 3 endocrine trial. The oncology and endocrine trials cost a projected $87,400 and $57,500 per patient, respectively, Stergiopoulos and colleagues reported.

The biggest driver of cost was the screen failure rate, followed by the cost of screen failures, Stergiopoulos and colleagues wrote. When the model was adjusted to show one patient in 90 being randomly assigned to a study drug — instead of one patient in 100 — the cost of the trial fell by $5,700 per patient and $5.7 million overall. Similarly, when researchers lowered the cost of a screen failure by $60 per patient, the cost fell by $4,200 per patient and $4.2 million overall.

“By applying the insights gathered from this cost model, pharmaceutical and biotechnology companies — along with other clinical research stakeholders and programs — can move closer to creating a more efficient and financially viable drug development process for HABP/VAPB,” the researchers wrote.

In particular, Stergiopoulos and colleagues encouraged stakeholders to decrease screen failures to lower costs.

“This will allow the clinical trials and regulatory communities to address the need for new HABP/VABP antibiotics and provide the medical community with more clinically informative data with which to treat these desperately ill patients.” – by Andy Polhamus

Disclosure: Stergiopoulos reports no relevant financial disclosures. Please see the study for all other authors’ relevant financial disclosures.