'Patient-focused' drug development guidance coming, FDA chief says
The Food and Drug Administration will soon issue a guidance document on how the industry can do more "patient-focused drug development," Commissioner Scott Gottlieb said at the BIO International convention in Boston last week.
"The guidance is about creating a formal structure to give industry a framework for how they can drive patient input in a way that they can inform our process. This is becoming an important element in how we evaluate drugs," he said.
While formal changes to the process and structure at the FDA would have to be approved by Congress, Gottlieb said he has proposed some changes internally that are under discussion, including a "flattening" of the organization and creating more review areas around certain diseases.
"There are a lot of reasons why we would want to do that, but one of the reasons is to give an identifiable point-of-contact for providers, patients and others in the external community to be able to have a relationship with a specific therapeutic division that is focused on a specific therapeutic area," he said.
Gottlieb explained that currently, the FDA has structured the review process to balance workloads, but doesn't always align therapeutic areas.
"Now, we're going to create an alignment that is optimal from a therapeutic standpoint, from a clinical standpoint, and would balance the workload just by balancing the number of people. So rather than making sure there are 25% of people in each division, there might be 10 people in one and 60 people in another depending on what the workload is," he added.
The FDA head predicted these and other changes will help drive further efficiencies for the regulator. He says the office will put out more product-specific guidance, many of which will be in the bulleted format. He assured the audience assembled in Boston that such guidance will also be updated, "maybe even annually."
Since beginning his tenure at the head of the agency, Gottlieb has been outspoken about creating more competition and more efficient drug reviews. He has taken a number of steps to increase transparency within the agency, as well as call out industry bad actors.
There will be a "discrete policy office within the Office of New Drugs" to focus on product-specific guidance and for new drugs. Gottlieb pointed out there are currently thousands of guidance documents for generic drugs but not as many product-specific guidance documents.
"Historically, there was a view at the FDA — and this goes back ten years — that the medical policy function shouldn't sit within the Office of New Drugs. But I think that the medical policy function should absolutely sit inside the Office of New Drugs. That's where it should be. That policy function is going to have much more proximity to the review process and the reviewers."
Another potential change will include a central review document. Drug reviews will now center around a single "virtual" report.
"There are a lot of different elements that go into the review that are basically distinct scientific disciplines, like clinical pharmacology, epidemiology, biostatistics, modeling and simulation. Historically, they functioned as consultants to review division, where they write consult reports," Gottlieb said. "It sounds sort of trivial, but being able to collaborate around a single document will eliminate a lot of redundancy."
Restructuring how the FDA divvies up work isn't the only change Gottlieb is contemplating. Also at the BIO meeting, the FDA chief previewed forthcoming guidance on cell and gene therapies that could come by July.
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