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.
