For many years, the FDA was not unlike the Federal Reserve in its ability to only primarily exert a single weapon.  In the Fed’s case, it was its ability to modify the interest rate that banks charge each other.  In the FDA’s case, it is the ability to approve or reject a new drug application.  While this is clearly an oversimplification of both the Fed and the FDA, it has been the general public’s perception of both.  Other than Vioxx (which I would highlight as the harbinger of change), few drugs have been taken off of the market.  Few required Phase IV trials have been conducted and pharma advertising campaigns have advanced largely unchecked, with actions only after they have largely run their course.  Some might say that the Vioxx issue only exacerbated FDA’s use of its major weapon, slowing and further tightening its approval process for new drugs.

Recent changes at the FDA seem to suggest that it is developing both a bark and a bite as it becomes increasingly visible to the general public.  Key changes include the FDA Amendments Act of 2007, which provided the FDA with a stick to encourage drug companies to conduct promised Phase IV trials or face fines.  The Sentinel Initiative seeks to find better ways to more quickly, routinely, and effectively identify drugs with significant safety issues.  Errant drug advertising and promotion is incurring billion dollar fines.  And finally, comparative effectiveness lies on the horizon.  The shape of things to come?

Emerging reports provide a mixed message regarding the industry’s conduct of required Phase IV clinical trials.  In September, it was reported that around 80% of Phase IV studies were either on schedule or finished on time.  An subsequent October GAO report indicated that many studies are years late and, in addition, provoked no action regardless of study outcome.  With heightened cost consciousness and comparative effectiveness at the front of the FDA’s collective minds, the potential for dramatic action (i.e. drugs taken off the market or new clinical trial requirements) is growing.

The ability to more quickly and routinely identify drug safety issues remains a major priority at the FDA, supported by diminishing consumer tolerance for significant adverse events of any kind.  Two year delays after market approval (i.e. Vioxx) in identifying problems is unacceptable and larger clinical trials with enhanced post-marketing surveillance is becoming the norm.  Expect tighter time requirements in drug adverse event reporting during clinical trials and more efforts like the Sentinel Initiative to better manage drug safety issues both before and after drug approval with FDA action to match.

While Pfizer’s recent $2.3 billion fine for off-label promotion of approved drugs may seem like a large penalty, it dwarfs potential revenues that drugmakers are gaining from such improper efforts.  Until fines or other punitive actions act as effective deterrents to illegal activities, companies will perceive them as acceptable costs of doing business.  Expect increasing actions by the FDA, combined with parallel legislation reflecting growing consumer discontent.

With healthcare costs continuing to grow and current drug company profits being eyed as obvious targets for savings, government agencies, including FDA and CMS (the Centers for Medicare and Medicaid Services), are being given increased leverage to act, in response to consumer demands for safer drugs and more active regulation of the industry.  Healthcare cost pressures are likely to keep these issues on the front page for the foreseeable future.  An FDA with both bark and bite is here to stay.

As always, I welcome comments and discussion based on your own insights and experience.