21st Century Cures: What it could mean for CROs

Among those provisions tied to CROs include the use of master protocols to speed drug development, a move to use less IRBs (institutional review boards), and the modernization of ClinicalTrials.gov, the online database of trial information.

In addition, the bill calls for a $10bn boost to NIH funding over five years, as well as proposing new tasks for the FDA in connection with developing guidance documents related to trials and drug development.

Paula Brown Stafford, president of clinical development at Quintiles, testified before the House Energy and Commerce Committee last year and presented on ways the bill should focus on patients, processes and pathways.

Stafford spoke with Outsourcing-Pharma.com on Tuesday at DIA’s annual conference in Washington, DC, explaining what some believe to be a necessary shift away from the use of two randomized late-phase trials to obtain FDA approval for a new drug.

“There was testimony from many of us on moving toward registries and real-world trials as quickly as possible when you know there’s a product that has great benefit to the public​,” Stafford said. “It has to depend on some amount of data before we go immediately to registries, but I think what we’re moving away from is the traditional two randomized clinical trials, and that one randomized clinical trial may be enough, or one master protocol -- there may be enough evidence -- especially as we move to more personalized medicine​.”

In a recent editorial in the New England Journal of Medicine​, two Harvard University professors took issue with the way the bill may give the FDA too much discretion to approve drugs without conventional clinical trials.

But Stafford said she doesn’t think the bill gives the FDA any more authority than it already has.

“The idea is about finding the right patients for the right trials – without personalized medicine we’ve been going after a larger population that fit into exclusion and inclusion criteria but the specificity of that is not what it can now be with biomarkers and gene sequencing​,” Stafford said.

On the master protocol front, Quintiles presented one to the FDA around diabetes and cardiovascular outcomes trials, and although they haven’t proceeded with that, “we’ve had a number of conversations with our customers, and for the right indication, it’s a nice way to bring patients into a larger trial​,” Stafford said.

Stafford also spoke to the committee about the adoption of CDISC data standards, which the FDA has been supportive of but which they’ve yet to make any firm requirements on.

“It doesn’t have to be CDISC but that’s what we have right now. CDISC standards are for demographic data and safety data but for the efficacy data, they want 55 therapeutic areas in five years and we’re two years in… We need to get those standards developed​,” she added.

As far as pushing industry to using less IRBs, which the NIH has also recently called for​, Stafford said she doesn’t think “it’s ever going to get to single IRBs, but there should be some consortium where they come together and have one IRB represent a number of the universities – the amount of time and cost that it adds, I’m not sure the value is there​.

“In the US, I think we can come down to three or four, instead of 20 to 50 IRBs. When we’re running a trial in 50 sites in the US, and I have to wait for weeks to get a group together to review it…there’s a lot of redundancy​,” she added.