FDA's Gottlieb Has Big Plans to Get Drugs Approved Faster, Tackle Drug Development Costs

September 12, 2017
By Mark Terry, BioSpace.com Breaking News Staff

Scott Gottlieb, the commissioner of the U.S. Food and Drug Administration (FDA), gave a talk at the Regulatory Affairs Professionals Society (RAPS)’ Regulatory Convergence conference yesterday, where he outlined some approaches the agency is taking to make clinical drug development faster and more efficient.

“There’s been criticism of the various estimates of how much it costs to develop a new drug,” Gottlieb said in his speech. “Moreover, on a relative basis, in many cases the costs of early stage drug development has grown at a proportionally faster rate than the cost of late stage drug development. In other words, inflation in early stage drug trials is rising faster than the inflation in late stage development.”

He also took a moment to mention the high costs of drugs and what the agency is doing to promote generic competition. But the bulk of his talk was on why the FDA is working “to address the other side of the continuum, where the costs start: With the high and rising expense of developing a novel drug.” Then, after bolstering his argument, he described the things the agency is doing.

1. Janet Woodcock, head of the FDA’s Center for Drug Evaluation and Research, is working to modernize its Office of New Drugs. “The goal,” Gottlieb said, “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.”

2. Gottlieb will advance a Strategic Policy Roadmap to further detail the agency’s steps, which he argues will allow the FDA to adopt the most modern scientific tools available. It’s hard to say if that infers the FDA isn’t currently doing so, but it seems to.

3. Modernize the way the company collects clinical information. “For example,” Gottlieb said, “we’re seeing wider use of adaptive approaches, which allow scientists to enrich trials for patient characteristics that correlate with benefits, or that help predict which patients are least likely to suffer a certain side effect.”

4. Increased use of combined-phase studies, or so-called “seamless trials.” This would involve not using the usual Phase I, II and III trials, but, Gottlieb said, “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.”

5. Adapting new guidance and policy work, including common control studies and the wider use of large simple trials.

6. Advancing the use of Master Protocols, Gottlieb said, “to enable more coordinated ways to use the same trial structure to evaluate treatments in more than one subtype of a disease or type of patient.”

7. Improve communications between sponsors, investigators, IRBs and other stakeholders and the FDA.

8. Modernize how sponsors can evaluate clinical information and how the FDA reviews the data.

9. Use of better, more advanced computing tools and more sophisticated statistical and computational methodologies, including more modeling and simulation.

10. The development and implementation of at least 10 new disease-specific guidance documents over the next year.

John Carroll, writing for Endpoints News, points out that much of what Gottlieb is proposing appears based on trends in the oncology field, where biopharma companies have been able to test new drugs on patients with advanced diseases who are involved in clinical trials with little hope for survival. Can those same approaches be used for diseases like diabetes and cardiology and illnesses with a less desperate population? It’s an open question.

Carroll writes, “Another big question is whether biopharma companies will actually pass along any savings they get from a more efficient development pathway to payers and consumers. The entire industry has been tipping more and more of its development dollars to cancer in part because of the big rewards that come from fast approvals. And the FDA has no control whatsoever over the final price drug developers use for their new drugs.”