Better, faster, cheaper is the aspiration for many innovations including those affecting pharmaceutical trials. A recent study from the respected Tufts Center for the Study of Drug Development (CSDD) has shown that decentralized clinical trials can deliver results more quickly with both improved savings and quality compared to most traditional trials.
Decentralized clinical trials (DCTs), sometimes also called virtual, siteless or mobile trials, take advantage of technological advances in connected devices, mobile communications platforms and integrated electronic medical records (EMRs) with the goals of addressing costs, enrollment and diversity issues in drug development. DCTs have gained increased attention recently due to the challenges of testing new medicines.
Drug development relies on lengthy, expensive stages with estimates of over $2 Billion to bring a molecule through approval according to Tufts. Additionally, fewer than 10% of drug development programs are successful, which has led to unsustainably low pharmaceutical research and development (R&D) returns on investment (ROI). Furthermore, clinical trials lack representative population diversity with regard to different genetic ethnicity that can cause variability in effects of medications.
The Tufts study shows that DCTs are associated with reduced clinical trial timelines and financial benefits from five to 14 times for Phase II and Phase III trials. The financial impact is higher “expected net present value” (eNPV) that equates to a roughly $10 million return on a $2 million investment for phase 2 trials and $39 million return on a $3 million investment for phase 3 studies, according to Ken Getz, professor and executive director of the Tufts CSDD.
Lead author Joseph DiMasi who is director of economic analysis at Tufts CSDD has said, “Our investigation found that, on average, the financial returns to drug sponsors from shorter development times, lower clinical trial screen failure rates, and fewer clinical trial protocol amendments associated with DCTs substantially exceeded the costs of investing in DCT technologies.”
Using the common financial return on investment (ROI) measure, eNPV integrates the key business drivers of cost, time, revenue, and risk into one summary metric for project strategy and portfolio decision-making by pharmaceutical companies, according to Pamela Tenaerts, a clinical development expert who has worked with academia and the Food and Drug Administration (FDA) and now Chief Scientific Officer at Medable. The metric is widely used by portfolio managers and financial analysts across most industry sectors.
The Tufts analysis included the key findings relating to net financial benefit, shorter clinical trial times, lower screen failure rates, and fewer protocol amendments. Researchers studied over 200 protocols, the majority of which were in late stage trials.
Investigators found that the typical Phase II DCT deployment results in a one to three month time savings that yields a net benefit that is up to five times greater than the upfront investment required. In Phase III studies, a similar time savings yields a net benefit that is up to 14 times greater than the upfront investment.
Cycle time reductions associated with DCT deployments had a substantial impact on net financial benefits. Nearly 85 percent of all clinical trials will experience some sort of delay, with the cost of $600,000 to $8 million per day of delay. Faster trial completion can drive significant cost savings.
Patient engagement and ethnic diversity is a valuable characteristic of successful clinical trials. However, less than 5 percent of the U.S. population participates in clinical research, and up to 50 percent of trials are not completed because of insufficient enrollment. An expected benefit of DCTs is to enable greater patient participation, reduced time and travel burden, faster screening, more convenient consent and enrollment. One notable trial demonstrated a four-fold increase in patient diversity.
Another factor is protocol amendments, which often cause delays and increase costs. Researchers indicated that the potential for fewer sites in a DCT leads to a reduction in regulatory costs and increased flexibility around protocol changes.
The study findings are “very compelling,” says Getz. He noted that this initial economic investigation had some limitations such as sample size and pooling types of DCT solutions. The Tufts researchers plan is to look at specific trails by individual disease conditions in further studies.