We're just scratching the surface of what is possible, but early advances verge on the miraculous. Last year, for instance, the U.S. Food and Drug Administration approved the drug Kalydeco for a small group of cystic fibrosis patients with a particular gene abnormality. Kalydeco is the first drug to actually treat the root causes of CF -- and for the patients it benefits, it's a functional cure.
The next challenge will be developing treatments for diseases with complex genetic or molecular drivers that don't respond to any single drug but require complex therapies. Unleashing the full potential of molecular medicine won't be realized without important changes in the federal agency that evaluates and approves new drugs: the FDA.
For all the good it does in protecting the public's safety, the FDA has been hesitant to embrace new approaches to drug regulation. The agency has long known that medicines that work miraculously for some don't work for others, and may even cause harm in some. But the FDA's current clinical trials requirements prevent companies and researchers from unraveling the molecular code that underpins variation in patient response to new medicines.
In a recently released paper, "The Digital Future Of Molecular Medicine: Rethinking FDA Regulation," the Manhattan Institute's Peter Huber argues that traditional FDA clinical trial protocols are impeding innovation. Faced with a broad array of new technologies, the FDA is effectively "frozen in the headlights," of the 1960s.
With few exceptions, the FDA continues to evaluate new drugs based on how they affect the "average patient." But this ignores what we now know about the molecular diversity inherent in diseases and patients and may result in good drugs being tossed on the scrap heap.
As Huber writes, it doesn't have to be this way. The FDA has a separate track -- "accelerated approval" -- that allows companies to avoid the FDA's standard process by proving that the drug can affect "surrogate endpoints" -- molecular targets like HIV viral load for AIDS -- rather than "clinical endpoints," which can span the gamut from improved walking ability to not dying.
By using molecular targets that can "likely" predict future clinical response, or identify specific subpopulations of patients, companies can receive FDA licensing much faster than in traditional trials. Companies also agree to conduct confirmatory trials later; in the interim, doctors can use the drugs in combinations that the drug's maker or the FDA never predicted.
Formalizing this virtuous cycle of patient-centered innovation for every disease can accelerate innovation and allow doctors (and powerful computers, like IBM's EuResist database) to stay one step ahead of the complex diseases that would otherwise kill us.
Unfortunately, the FDA has been slow to utilize the accelerated approval framework outside of HIV, cancer and some rare diseases. Indeed, only four out of 39 drugs approved in 2012 received accelerated approval designation. As Huber noted in a recent Intelligence Squared debate, the incredible success of the accelerated approval track by itself indicates that there is something fundamentally wrong with the regular approval track.
The FDA's reluctance to broadly adopt new standards is untenable given what we now know about the root causes of diseases.
Reforming the FDA should start, as President Obama's own science advisers recommended, with extending the accelerated approval track to all diseases with serious unmet medical needs.
This would unleash tremendous innovation in drug development and in patient access to new therapies. It is an "accelerated" change that is long overdue.