FDA commissioner Scott Gottlieb has sounded a crystal clear warning over the high — and growing — cost of drug development. And in a speech to regulatory execs on Monday, Gottlieb committed the FDA to backing up more efficient drug development programs with new measures to clear the regulatory path for developers barreling ahead to relatively swift pivotal data in search of an accelerated OK.
Gottlieb started by outlining a bleak picture in drug R&D, noting that the economic model for drug development is broken. It costs too much to develop a drug so it can be approved for marketing. And costs are swelling fast at the discovery end of the business, which will help swamp a system that already doesn’t work particularly well.
As he has in the past, Gottlieb held up some of the rapid-fire clinical trials we’ve been seeing in the cancer field as a model for what can work, paving the way to the accelerated approval pathway at the FDA. And he believes — though there is precious little evidence to back it up — that moving drug development into the fast lane can reduce R&D costs and thereby allow biopharma companies to pass on savings to patients through lower costs.
To help developers, Gottlieb vowed that the FDA, through CDER chief Janet Woodcock and the Office of New Drugs, will adapt the regulatory pathway to enable drug development at a more moderate cost.
Comparable regulatory milestones need to be built into the new seamless clinical trial process. We need to ensure we provide comparable interactions and oversight.
He also noted that as developers move toward faster, seamless studies — dropping the traditional Phase I through Phase III development plan — regulators also need to update patients’ awareness of the risks involved in providing their consent for participating in these studies.
Watch Gottlieb’s speech and Q&A
Here are some excerpts from the speech, starting with an outline of the trend toward a single development program for new drugs.
Owing in part to these leadership efforts, we’ve seen more sponsors develop oncology drugs that forgo the conventional three sequential phases of drug development. They opt instead for seamless approaches. Under these trial designs, they’ll typically add cohorts to a first-in-human trial to investigate doses and activity in a variety of cancers.
We’ve seen examples where this approach has allowed the rapid development of drugs in multiple different tumor types. If we had to stop and start formal Phase II trials in each different organ system where a cancer arose, it could have been a protracted process. This approach is well suited to the kinds of drugs that are being developed now, where drugs intervene on common elements found across multiple kinds of disease states. At FDA, we’ve identified more than 40 active commercial investigational new drug applications for large first-in-human oncology trials alone that use these seamless strategies.
Gottlieb also talked about using broad protocols that allow developers to tackle multiple targets at once.
We’re also advancing the use of ‘Master Protocols’ 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.
This approach is particularly relevant when it comes to targeted drugs. These are drugs that may intervene on markers that are relevant across many different disease subtypes. We may, for example, want to evaluate these different targets simultaneously, as part of one large study. This could give us a better way to understand the comparative benefits of a drug across different settings. To enable these master protocols, it’s often important to do molecular patient screening. This can lead to the development of a diagnostic that can also be used to guide patient care.
Gottlieb outlined plans to invest more of the FDA’s money in new technology that can assist this faster/better/cheaper approach to drug development.
On the second point that I wanted to highlight today, we’re also taking new steps to modernize how sponsors can evaluate clinical information, and how FDA reviews this data as part of our regulatory process.
This starts with better use of more advanced computing tools, and more sophisticated statistical and computational methodologies, as part of the drug development and the drug review process. This includes more widespread use of modeling and simulation, and high performance computing clusters inside FDA.
FDA already has high performance computing clusters. These tools help us develop more sophisticated methods for evaluating the data that’s submitted to us from clinical trials. The computing tools also enable us to properly evaluate the more sophisticated components that are submitted to us as part of product review applications.
Gottlieb also talked about shaking up the R&D approach to some specific diseases that have proved particularly hard to deal with.
Additionally, to better delineate how we’re going to approach the overall development and evaluation of drugs targeted to certain unmet medical needs, we plan to begin work on at least ten new disease-specific guidance documents over the next year. Some of these documents are already underway. Among the diseases we’re targeting are areas of significant unmet need like Amyotrophic Lateral Sclerosis (ALS).
Revamping the economics of drug R&D is no simple task.
The oncology field has been able to move farther and faster than other disease fields due to its ability to test new drugs on patients with advanced disease and dwindling hope of survival. So it won’t be easy to translate that same approach to mass market diseases like diabetes and cardio, where millions are treated for years for chronic disease.
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.
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