When it comes to FDA reform, if you don't ask the right questions, you won't get the right answers.
It’s been widely reported this week that the FDA approved more “first-of-a-kind” drugs in 2008 (21) than it did in 2007 (18). During that same period fewer “black box” warnings (both new and updated) were awarded in 2008 (46) than in 2007 (62). What do these numbers mean?
To some (and you know who you are) they signal an agency that isn’t as concerned with safety as it was only twelve months earlier. More drugs approved? Fewer black boxes? And why, these same FDA watchers wonder, isn’t DDMAC sending out more warning and untitled letters to curb the abuses of pharmaceutical marketing – particularly of the off-label variety?
These are some of the questions they would ask. The media-friendly questions. The staff-prepared hearing questions. The trial lawyer questions. The politics before public health questions. The "tabloid medicine" questions. And they are the wrong questions.
For those who actually understand what’s going on there is a different set of queries altogether: Why did the FDA miss review deadlines for at least 15 drugs? Why are a growing number of complete response letters sounding more and more like the old-style not-approvable variety? And why are more and more complete response letters requesting information that was never discussed during the review process or at advisory committee meetings? Savvy pharmacenti also want to know what in Hell’s Bells is going on with “early safety” communications and signal-to-noise ratio issues.
But the big question those who know are asking is, Why is ambiguity trumping predictability in the regulatory process? This is the key issue that must be addressed by the new FDA Commissioner. That’s the question. The fear is Precautionary Principle creep. If the FDA adopts the position of doing nothing until it knows everything -- that will send a chilling message to the pharmaceutical industry to dial back R&D unless the program looks like a sure thing.
And there are no “sure things” in pharmaceutical discovery and development.
Industry seeks clarity. They want bright lines. They want to know the rules. They want predictability. This may sound simple and fair, but inside the FDA it has proven to be a fractious bureaucratic kulturkampf. “Change is not required,” as management guru W. Edwards Deming once said. “Survival is not mandatory.” And that doesn’t mean change for show, for politics – it means thoughtful, timely, strategic change that enhances the public health. And that kind of change requires not walking on egg shells – but breaking them. Andy von Eschenbach learned that the hard way.
Changing the minds of regulators to embrace bright lines rather than broad definitions is a challenging proposition -- because changed minds must begin with change agents. The new Commissioner must seek out and work with those career officials within the FDA who are smart, confident and gutsy enough to embrace new ways of doing business, who support bright lines over draft guidances, pragmatism over dogmatic doctrine. And those people are there and are excited about the possibilities of an FDA that will lead rather than an agency that buffets from side-to-side based on gusting political winds.
FDA’s Critical Path initiative is a promising example of the agency’s desire to embrace change. Going forward, the agency’s stakeholders will be looking for other “surrogate markers” to gauge FDA’s willingness to continue the McClellan era’s aggressive determination to both protect and advance the public health.
Despite new draft guidances that attempt to draw bright lines, what is and is not “in compliance” remains more art than science. Industry is confused, and the public health is not served. Predictability is power in pursuit of the public health. Predictability is the result of creative, forward-thinking leadership that rises above bureaucratic ambiguity. Driving this philosophy won’t be easy for the new Commissioner -- because swimming against the tide of an entrenched bureaucracy never is. But if the Commissioner communicates this philosophy, leads by doing and empowers change agents within the FDA career staff, the tide will turn.
As Winston Churchill said, “Ease is relative to the experience of the doer.”
It’s been widely reported this week that the FDA approved more “first-of-a-kind” drugs in 2008 (21) than it did in 2007 (18). During that same period fewer “black box” warnings (both new and updated) were awarded in 2008 (46) than in 2007 (62). What do these numbers mean?
To some (and you know who you are) they signal an agency that isn’t as concerned with safety as it was only twelve months earlier. More drugs approved? Fewer black boxes? And why, these same FDA watchers wonder, isn’t DDMAC sending out more warning and untitled letters to curb the abuses of pharmaceutical marketing – particularly of the off-label variety?
These are some of the questions they would ask. The media-friendly questions. The staff-prepared hearing questions. The trial lawyer questions. The politics before public health questions. The "tabloid medicine" questions. And they are the wrong questions.
For those who actually understand what’s going on there is a different set of queries altogether: Why did the FDA miss review deadlines for at least 15 drugs? Why are a growing number of complete response letters sounding more and more like the old-style not-approvable variety? And why are more and more complete response letters requesting information that was never discussed during the review process or at advisory committee meetings? Savvy pharmacenti also want to know what in Hell’s Bells is going on with “early safety” communications and signal-to-noise ratio issues.
But the big question those who know are asking is, Why is ambiguity trumping predictability in the regulatory process? This is the key issue that must be addressed by the new FDA Commissioner. That’s the question. The fear is Precautionary Principle creep. If the FDA adopts the position of doing nothing until it knows everything -- that will send a chilling message to the pharmaceutical industry to dial back R&D unless the program looks like a sure thing.
And there are no “sure things” in pharmaceutical discovery and development.
Industry seeks clarity. They want bright lines. They want to know the rules. They want predictability. This may sound simple and fair, but inside the FDA it has proven to be a fractious bureaucratic kulturkampf. “Change is not required,” as management guru W. Edwards Deming once said. “Survival is not mandatory.” And that doesn’t mean change for show, for politics – it means thoughtful, timely, strategic change that enhances the public health. And that kind of change requires not walking on egg shells – but breaking them. Andy von Eschenbach learned that the hard way.
Changing the minds of regulators to embrace bright lines rather than broad definitions is a challenging proposition -- because changed minds must begin with change agents. The new Commissioner must seek out and work with those career officials within the FDA who are smart, confident and gutsy enough to embrace new ways of doing business, who support bright lines over draft guidances, pragmatism over dogmatic doctrine. And those people are there and are excited about the possibilities of an FDA that will lead rather than an agency that buffets from side-to-side based on gusting political winds.
FDA’s Critical Path initiative is a promising example of the agency’s desire to embrace change. Going forward, the agency’s stakeholders will be looking for other “surrogate markers” to gauge FDA’s willingness to continue the McClellan era’s aggressive determination to both protect and advance the public health.
Despite new draft guidances that attempt to draw bright lines, what is and is not “in compliance” remains more art than science. Industry is confused, and the public health is not served. Predictability is power in pursuit of the public health. Predictability is the result of creative, forward-thinking leadership that rises above bureaucratic ambiguity. Driving this philosophy won’t be easy for the new Commissioner -- because swimming against the tide of an entrenched bureaucracy never is. But if the Commissioner communicates this philosophy, leads by doing and empowers change agents within the FDA career staff, the tide will turn.
As Winston Churchill said, “Ease is relative to the experience of the doer.”
