Increasing the efficiency of clinical trials

It's impossible to talk about "faster cures" without talking about significantly reducing the time and cost of the clinical trials process. The challenges are well known, and many solutions have been proposed, but transformation has been elusive.

A panel of diverse experts discussed current innovations that could feed systems change, and agreed that universally interconnected electronic medical records will greatly increase the efficiency of many aspects of the clinical trial process.

Clinical trials are a critical component to the development of innovative therapies; however, trial duration has increased by nearly 25 percent and the compound annual growth rate in cost has risen by 12 percent over the past three decades. Moderator Melissa Stevens of FasterCures began the discussion by asking the panelists their thoughts on how to introduce business process improvements into the clinical trials process.

According to Robert Califf of the Duke Translational Medicine Institute, a key incremental change that can be made to the business process to improve the overall system is the development of FDA guidance on wasteful monitoring. Something as simple as efficiently handling and reducing the number of reported adverse events can save time and money by preventing investigators from becoming inundated with data that do not have significant meaning. “Every trial should be designed to answer the [clinical] question, and we need to throw out the excess baggage that comes along with it,” he said. Califf also noted that the development of the www.clinicaltrials.gov database has been extremely useful in that it has allowed more transparency into the number of clinical trials that are poorly designed and not asking clinically meaningful questions. This transparency will help to trim the fat to improve overall efficiency and allocation of resources.

Lawrence Lesko of the Center for Pharmacometrics and Systems Pharmacology at the University of Florida pointed out that figuring out how to access and leverage large datasets – “big data” – will also be critical to driving down costs and improving clinical trial outcomes. Lesko proposed that conducting metadata analysis across large data sets, such as those housed at the FDA, would enable the development of disease progression and dose response models that could then correlate to early predictors of success or failure of investigational drugs. Lesko stated, “We need more predictors to design clinical trials, especially in therapeutic areas where no one size fits all … the FDA has the expertise to do this [metadata analysis] but not the time, therefore we need to farm that task out to collaborators and consortia that don’t have vested interest in the approval process.”

Clifford Hudis of the Memorial Sloan-Kettering Cancer Center presented the “big data” issue from a different perspective. Hudis pointed out that a plethora of patient data exists; albeit in silos of paper files or unconnected electronic filing systems. This problem of disconnected data has placed the research community at a huge disadvantage, particularly when it comes to clinical trials. If patient electronic medical records were housed in an interoperable network of databases, researchers would be able to use these data to design smarter clinical trials that address more clinically meaningful questions, more rapidly enroll clinical trials, and quickly identify safety signals. This would lead to what is commonly referred to as a rapid learning healthcare system.

Susan Solomon of the New York Stem Cell Foundation highlighted the importance of embedding patients into the process of designing better clinical trials. Solomon shared her difficulty in retaining patient participation in the organization’s stem cell research. She explained that patients were excited and wanted to donate stem cells; however, participation enthusiasm was overshadowed by “little things like making sure that their calls were returned quickly or that [patients] did not have to wait too long at appointments at the doctor’s office.” Solomon added that this customer service problem could have been avoided if they had embedded patients into the process during the planning stages.

Gary Neil of Apple Tree Partners punctuated this point by stating, “As we step back and look at this ecosystem, we really have to remember that the focus is the patients … the patients are the real heroes of the entire ecosystem, because they are willing to participate in these trials and take the risk … so we need to make certain that their efforts amount to something.”

The panel agreed that in order to effectively integrate patients into the process to ensure success of these trials, the clinical community needs to invest in creating an infrastructure that increases the number of eligible patients that are enrolled in these trials. In addition, patient participation should be simple, and patients should be involved in formulating key clinical questions.

In closing, the panelists agreed that the factor that will have the most impact on the cost, duration, and intelligent design of clinical trials will be an investment in developing an infrastructure of universally interconnected electronic medical records to create a rapid learning healthcare system.