Sunday, January 22, 2011, the New York Times reports a new federal research center will help “develop medicines”. This billion-dollar effort is prompted by concerns “about the slowing pace of new drugs coming out of the pharmaceutical industry.”
No one can dispute that truly new pharmaceuticals are becoming ever more rare. Most “new” drugs approved these days are actually chemically-tweaked variants on pre-existing molecules, not entirely new classes of medicine. Big, scary, important diseases, such as Alzheimer’s, multiple sclerosis, and many cancers lack adequate therapy.
But is this Big Pharma’s fault? Are private-sector innovation and research really in decline? And can the federal government do better?
I’m not a scathing critic of all things federal, but I do think this new effort is misguided. Redirecting NIH dollars into translational research–research to bridge the gap between basic scientific discoveries and practical solutions to medical problems–will weaken one of our nation’s most important basic science institutions, and I doubt the new center will achieve its goals.
Why? Because the reason for the increasing scarcity of novel therapies isn’t a lack of effort by the pharmaceutical industry. They spend enormous sums on research and development, and their scientists are as skilled and motivated as those in academia. There are two main obstacles to drug development, and neither will be solved by a new federal center.
One: As the saying goes, the low-hanging fruit has been plucked. The easy stuff, pharmaceutically speaking, has been done. We have vaccines for virtually all the common, deadly childhood diseases; we have antibiotics that despite increasing resistance still cure the majority of infections; we’ve got blood thinners and cholesterol-lowering drugs and stomach acid-blockers that act on clearly understood biochemical pathways. But schizophrenia? Metabolic syndrome? These kinds of diseases are swamps of scientific ignorance: complicated, multifactorial, impenetrable. Throwing money at “translational research” isn’t going to magically create the basic knowledge needed to tackle these.
Two: Americans have become 100% risk-averse. This is a problem because life–and pharmaceuticals–are never 100% risk-free. Arguably the greatest drug in history–penicillin–can kill (by anaphylactic shock, if untreated) somewhere between 1 in 10,000 and 1 in 100,000 patients who take it. No drug is absolutely safe. Yet the regulatory system in the U.S. is heavily slanted toward the impossible goal of zero risk.
In short, it’s so hard to meet the FDA standards for approval of new drugs and devices that the cost becomes prohibitive, and the investment risk (if the new technology sputters or is never approved) unacceptably high. Substituting government money for private sector money in translational research will not bring down the cost, nor will it change the risk/benefit calculation that currently gives so much more weight to risk than relative benefit.
If Americans want more new drugs faster, they’ll have to accept something less than a gold-plated guarantee that their medicines will never cause them harm.