We face a 'critical path' gap

April 1, 2006

After more than a year of almost nonstop claptrap about there being "too many" new drugs comes a sobering fact ? even as pharmaceutical companies poured a record amount of money into drug development in 2005, statistics from the Food and Drug Administration (FDA) show that the agency approved only 20 new drugs, down from 36 in 2004. Only once in the last 10 years has the number of newly approved drugs been lower than last year's figure.

Which begs the question - are the 21st century drug development tools that would make possible a swifter path from bench to bedside withering? More importantly, where's the outrage? Where's the outcry from the halls of Congress about "who lost the critical path"? Where are the scathing articles about the critical path gap?

Success of failing

Currently, 50 percent of drugs that undergo large-scale phase 3 trials turn out to be too unsafe or not effective enough for marketing. That is not a sustainable model for the 21st century.

The FDA has the technical expertise that can draw together stakeholders and help prioritize research that is most needed, and to partner with others to conduct this research. Obviously, solutions will have to come from sources with the greatest expertise. This could entail contracting with academic organizations, private industry and other global translational research groups.

The FDA's critical path initiative will enable innovative growth companies to better and more efficiently attack the steep hurdles facing them, allowing them to compete more effectively against the bigger, better-funded players in the market on a more level playing field. That means a real change in the risk/benefit equation for both emerging growth companies and the public health.

But more needs to be done.

A family has been described as "a parliament of habits." The FDA is a 10,000-person family. And while its habits may seem arcane from the outside, there is an internal logic that moves things forward. That logic is its mission - to protect the public health. But as the agency enters its second hundred years, that mission must change to both protecting and advancing the public health. And in order to do that, the agency that regulates upwards of 25 percent of the U.S. economy needs new tools, new expertise and more funding.

Internal bickering

As with any large family there is internal bickering - but when the family is attacked, the wagons circle. That is the circumstance today.

The current unpleasantness has created an FDA that, instead of searching for ways to be more relevant in the 21st century, is scrambling for quick-fix solutions, responding to ad hoc attacks from politicians and pundits with clumsy measures that, in many cases, are based on the precautionary principle - the very antithesis of a science-driven FDA. And that certainly is change - dangerous change. And unless we want it to become a permanent sea of change, we must replace it with something else, something more positive, more future-oriented and science-based, change that allows the agency to move down the critical path more directly and rapidly and both protect and advance the public health.