In STEM CELLS IN THE NEWS on April 15, 2012 at 11:06 pm


When I was commissioner of the Food and Drug Administration (FDA) from 2005 to 2009, I saw firsthand how regenerative medicine offered a cure for kidney and heart failure and other chronic conditions like diabetes. Researchers used stem cells to grow cells and tissues to replace failing organs, eliminating the need for expensive supportive treatments like dialysis and organ transplants.

But the beneficiaries were laboratory animals. Breakthroughs for humans were and still are a long way off. They have been stalled by regulatory uncertainty, because the FDA doesn’t have the scientific tools and resources to review complex innovations more expeditiously and pioneer regulatory pathways for state-of-the-art therapies that defy current agency conventions. Fortunately, Congress may have an opportunity as soon as this week to begin changing that.

The FDA isn’t obstructing progress because its employees are mean-spirited or foolish. But for decades, Congress has starved the agency of critical funding, limiting its scientists’ ability to keep up with peers in private industry and academia. The result is an agency in which science-based regulation often lags far behind scientific discovery. This forces the FDA to slow the approval of new treatments—and at times
creates acrimonious litigation between the FDA and innovators, not to mention disillusionment among desperate patients.

For example, in August 2010, the FDA filed suit against a company called Regenerative Sciences. Three years earlier, the company had begun marketing a process it called Regenexx to repair damaged joints by injecting them with a patient’s own stem cells. The FDA alleged that the cells the firm used had been manipulated to the point that they should be regulated as drugs. A resulting court injunction halting use of the technique has cast a pall over the future of regenerative medicine.

From the agency’s perspective, it had only called a “time out” until it could apply its regulatory process designed to analyze the therapy’s effectiveness and potential risks. For the industry, however, government had intervened in a way that seemed to bar the established clinical practice of using an individual’s own cells to advance the healing process.

Lawyers—many lawyers—are now trying to resolve this dispute. But at a time when science and technology are creating marvelous medical breakthroughs, the FDA should be leading and guiding the development of state-of-the-art therapies like regenerative medicine. Instead, the agency’s process for regulating complex new technologies often starts too late, after companies and researchers have sunk millions of dollars and thousands of hours into painstaking research.

It makes far better sense for the FDA to work collaboratively with physicians, patients, companies and academic researchers to craft standards for evaluating new technologies while they are still being developed, not years later when a company makes a marketing application for a breakthrough product.

Until that time, FDA scientists typically have little contact with the scientists who know the most about these innovative technologies.

This is not because they don’t want to. But consumer groups distrustful of industry have led Congress to erect ever greater barriers between regulators and those they regulate. The FDA can convene advisory committees of outside experts, but these experts weigh in only at the end of the regulatory process.

Worse, congressionally mandated conflict-of-interest rules keep many of the most knowledgeable academic and industry scientists off advisory committees out of fear that industry ties might bias their judgment.

Meanwhile, budget constraints have eroded the agency’s scientific foundations. When I moved from director of the National Cancer Institute at the National Institutes of Health (NIH) to become FDA commissioner in 2005, I was surprised to learn there are no provisions for continuous education to acquire new skills in emerging fields such as stem-cell biology, nanotechnology or computational biology. Even sending agency staff to academic conferences provoked a congressional outcry over meeting and travel costs.

If we want the FDA to lead innovation, and not lag behind it, Congress must give the agency the resources to be the world’s foremost science-based regulatory agency. It should endorse formal career development programs and encourage more collaboration with scientists in academia, industry, NIH and other federal agencies.

Congress should also make conflict-of-interest restrictions more rational, to ensure that agency staff can get early access to external scientific expertise to evaluate emerging technologies. The FDA should be able to make greater use of scientific consultants as “Special Government Employees” to broaden the pool of qualified and approved advisers for the agency.

FDA scientists I have encountered do care deeply about patients and want to say “yes” to safe and effective new therapies. Regulatory approval is the only bridge between miracles in the laboratory and lifesaving treatments. Yet until FDA reviewers can be scientifically confident of the benefits and risks of a new technology, their duty is to stop it—and stop it they will.

Congress has an opportunity to improve all this as it considers renewing FDA user-fee legislation this year. The temptation will be to reauthorize the fees as quickly as possible and move on as usual for another five years. This would be a grave mistake. Reforms that allow the FDA to say “yes” to innovative therapies serve all the agency’s stakeholders, including current and future patients.

Otherwise we had better get used to the agency saying no by calling “time out” or, worse, “game over” for American companies developing new, vital technologies like regenerative medicine.

Dr. von Eschenbach, a former director of the National Cancer Institute and commissioner of the Food and Drug Administration from 2006 to 2009, is chairman of the Manhattan Institute’s Project FDA.

  1. That was one of the most rational and insightful writings on this subject I have seen in a long, long time. Thank you from the bottom of my heart.

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