Do As I Say, And As I Do

Lisa Henderson

What is more tiring for anyone in this industry to hear? Is it the most oft-quoted statistic from the Tufts Center for the Study of Drug Development that the average cost of getting a drug developed through to marketing approval is $2.87 billion; or the blaming of the FDA? Or is it that FDA is the major hurdle to trying anything new in clinical trials? That the fear industry has of doing something differently in a clinical trial is that the agency will reject the data and, ultimately, the drug. It’s clear the costs and industry practices behind clinical trials are not escaping the FDA, and they aren’t pleased.

For the FDA’s sake, let’s put aside the argument that many clinical trials fail in Phase III before they even see the light of submission and face that FDA scrutiny. Let’s also reject the notion that FDA is unwilling to work with pharma. The agency is more than willing to meet with sponsors very early on in their clinical trial planning processes to discuss options. These formal meetings also have their own guidance, so it’s not as if FDA is shutting everyone out of any potential discussions.

CDER and the FDA have backed up their support of basket trials, master protocols, and more collaborative types of trials by initiating for comment on Oct. 1 the draft guidance, “Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics.’’

FDA also put forth for comment its draft guidance, intending to replace one from 2010, ‘‘Adaptive Designs for Clinical Trials of Drugs and Biologics,’’ which provides direction on “the basic principles for designing, conducting, and reporting the results from an adaptive clinical trial.”

It looks as if FDA supports changes in trial designs-as it has for the past eight years-to promote more efficient and timely review of results for drugs being tested in clinical trials. However, the FDA estimates that 40 sponsors will submit 240 plans for trials utilizing an adaptive design, and only 15 will prepare and submit to FDA 20 marketing applications that rely on an adaptive design trial.

In another article from Bloomberg Law, FDA Commissioner Scott Gottlieb hinted that CROs may need to reflect on their own processes that may be unnecessarily bloating drug development costs without adding value. In the same article, attorney Mark Barnes at Ropes & Gray pointed to monitoring activities as one of those processes. While he acknowledged that monitoring could be more risk-based, he then blamed the FDA, saying, “this goal of perfection in trial monitoring is a product of the FDA’s own expectations of monitoring.” No matter that FDA’s guidance on the topic, “Oversight of Clinical Investigations-A Risk-Based Approach to Monitoring,” or its complementary partner, “Electronic Source Data in Clinical Investigations,” have been available for five full years­-since August and September of 2013, respectively.

From clinical trials to the commercial, FDA (and HHS), as well as smaller biopharma, are quickly tiring of these shenanigans. If you aren’t part of the solution, you are going to be considered the problem. Being part of the solution isn’t just lip service, it’s putting that information out there transparently for everyone to measure and make decisions.

Lisa Henderson is Editor-in-Chief of Applied Clinical Trials. She can be reached at lisa.henderson@ubm.com. Follow Lisa on Twitter: @trialsonline