We are growing our ability to understand these products and their impact on patients in a way that helps us manage both safety
and efficacy simultaneously—so we see the science of safety emerging as well as the science of efficacy. Even in experimental
models, we are now able to determine the effectiveness of a drug by understanding its on-target effects—for example, in the
tumor for which it is designed—and to understand at the molecular level its off-target effects in terms of what might be happening
to the liver or the endothelium of a heart or to the nervous system.
As we bring those two pieces of the equation closer together through the prism of science, we're going to be able to more
rapidly approve drugs and do it more reliably with regard to understanding the risk/benefit ratio. I see things going rapidly
and safely all at the same time.
A Brief History
The parameters around safety seem to be growing and changing at the same time. Where have you seen progress?
Protecting patients when they receive these drugs is something that really begins at the very front end of the process—and
it carries through the whole continuum. FDA is going to be engaged in the full life cycle of that product, whether it's providing
good agricultural practices for farmers to protect food from contamination, or whether it's collaborating with the vaccine
developers to help create pathways to develop vaccines, like we did this past year for vaccines for influenza. Similarly,
the ability to develop biomarkers in our Critical Path initiative helps us work more effectively on that front end.
And we don't simply disengage after we've made a decision about an application and the drug goes out into the market. We're
going to be even more actively engaged in the postmarket aspect. By collaborating with CMS and VA and private companies like
UnitedHealth, we'll get access to databases that will help us track and learn about these drugs as they're used in diverse
populations. And by using modern information technologies, we're able to understand issues of safety earlier, but also—and
more importantly—to see where these products are having unexpected efficacy. There's tremendous opportunity for us to understand
how drugs can be used even better to get the right outcome, not only how to contain them to avoid complications or adverse
You're talking about using and collecting more information on drug safety, but does FDA have the authority it needs to accomplish
all these things?
At least in the 18 months that I've been here—and following on our previous efforts—we've been working to modernize FDA and
better equip the agency to meet a new vision of our role. That will require us to define the plan: Where will we be engaged—like
in postmarket surveillance? What kind of resources will be required to support that effort? And then we'll ask if there's
the need for additional authority to be able to accomplish those goals.
That's been an iterative process we're engaged in as we speak. I've been trying to maximize utilization of what we already
have, both with regard to resources and authority. And we're talking with Congress as it is addressing these issues and providing
technical assistance, where it's been requested, to inform things like the Kennedy-Enzi bill.
How is FDA reorganizing to make the best use of its current authority?