Scott Gottlieb


New FDA Commissioner Scott Gottlieb has hit headlines again this week, following a speech at the RAPS Regulatory Convergence conference yesterday.

Fierce Biotech reports, Gottlieb wants to target drug development costs as yet another study asks how much it really costs to make a medicine. During his speech he focused most of his attention on driving efficiencies into the biomedical R&D model and thinking about the traditional three phases of trials.

“To address these issues, our Centre for Drug Evaluation and Research, under the leadership of Janet Woodcock, is taking steps to modernize its Office of New Drugs,” he said. “The goal is to make sure that our workflows and policies are rooted in the best science and management principles, and that our staff has the support and tools they need to fully achieve their public health mission. I plan to talk more about some of these steps, later this month, at the National Press Club.

“I’ll also advance a Strategic Policy Roadmap that will detail additional steps we’re pursuing. These, and similar, efforts are aimed, in part, at making sure that FDA is able to adopt the modern scientific tools we need to maintain the rigor of our programs.

“We need to make sure that our approach to regulation is efficient, and doesn’t become an obstacle to the translation of scientific discoveries into practical solutions for patients. We need to make sure that we’re using the best science so we maintain our gold standard for determining safety and benefit.”

Furthermore, he went on to explain a way in which costs and time can be cut is through the use of combined-phase studies, otherwise known as seamless trials.

He commented, “Instead of conducting the usual three phases of study, seamless trials encompass one adaptive study where the phases are separated by interim looks. By using one large, continuous trial, it saves time and reduces costs. It also reduces the number of patients that have to be enrolled in a trial.”

Under Rick Pazdur, the FDA’s Oncology Centre of Excellence is “taking steps to better evaluate and cultivate these new approaches as one part of our ongoing efforts to modernize our approaches,” Gottlieb said.

Also in the pipeline are plans to modernise, via advanced computing methods. The agency is also working on the use of ‘Master Protocols” to enable greater coordinated ways to use the same trials structure to evaluate treatments in more than one subtype of a disease or type of patient.

He also warned the industry that “by front-loading the cost of drug discovery, the broader biomedical community is making it harder to advance new ideas. It’s economically harder to capitalise the cost of an early stage drug program, relative to funding a later stage project. So, front-loading the costs is a recipe for reducing the amount of new ideas that can be advanced.

“We need to do these things to make sure we’re providing an efficient path for the translation of cutting-edge science into practical treatments that are going to benefit patients,” he said. “We need to do these things because the rising cost of drug development is unsustainable.

“Unless we find ways to modernize how we approach our work, and make more efficient use of our resources, then we’re going to get fewer medicines, and higher costs.”

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