More muscle needed for regulatory science …

September 17, 2009 – 4:10 pm by Michael Christel

Or all the billions of dollars poured into biomedical research and development in the U.S. won’t mean a thing. That was the prevailing message delivered yesterday by new FDA commissioner Margaret Hamburg during a candid keynote address at the Regulator Affairs Professionals Society’s annual conference in Philadelphia.

“In order to strengthen the FDA’s ability to make wise regulatory decisions, in order to see the progress of biomedical research translate into new therapies patients can really use, in order to prevent harm from contaminating food and drugs, rather than reacting until the harm is done, we need to develop a comprehensive and robust discipline of regulatory science,� Dr. Hamburg said. “We cannot afford to have a muscular investment in fundamental research and discovery but only a scrawny counterpart in regulatory capacity. Our efforts will be seriously compromised if we don’t convincingly increase the sophistication of our regulatory science soon.�

According to Dr. Hamburg, who has spent the majority of her career in public health, including positions at the NIH and HHS (see her background here), the challenge of improving regulatory science transcends any one agency or sector, and must be fully embraced in academic, industry, and government circles. She said the goal should be to place emerging areas of science and technology, such as genomics, personalized medicine, stem cell therapies, and nanotechnology, fully in the service of public health.

“We need to harness the best minds and the best science,� Dr. Hamburg said. “We must streamline and strengthen the science.�

Dr. Hamburg cited a few areas where this is being accomplished, including FDA’s partnership with the Intercontinental Conference on Harmonization around quality by design guidance for drugmakers. More on the development end, she said FDA reviewers are commonly exploring and learning about the potential of alternative methods, such as adaptive design.

Dr. Hamburg also stressed the importance of continued investment from government and industry in the orphan drug program. She noted that between 1973 and 1983, the FDA approved only 10 drugs for so-called rare diseases – those that affect fewer than 200,000 patients in the United States – but that the number has jumped significantly since Congress passed the Orphan Drug Act in 1983, providing economic incentives to drug developers active in rare disease markets. According to Dr. Hamburg, FDA has approved 334 orphan drugs to date.

The new commissioner didn’t shy away from addressing the longtime perception of the agency, one defined by a poor reputation and general disconnect from the American public.  She pointed out that FDA ranked second to last – even below the IRS – in a recent gallop poll rating federal agencies.

“Clearly my first priority as commissioner is to get us above the IRS,� Dr. Hamburg joked, eliciting laughter from the crowd assembled at the Philadelphia Marriott Downtown Grand Ballroom.

Dr. Hamburg quickly turned serious, however, when discussing the need for FDA to repair its tarnished image. She said the agency must raise its visibility level significantly, and be much more accountable and forthright in sharing information with the public, defining its expectations, and explaining the decision-making behind the approval of certain drugs, biologics, and medical devices.

“The agency must show industry and consumers that we’re on the job,� Dr. Hamburg said. “We must publicize our enforcement actions and the rationale of those actions. This will increase public confidence, encourage compliance, and educate patients and consumers about potential risks.�

In efforts to do just that, FDA turned some heads back in June, just a month after Dr. Hamburg was confirmed, when it established a transparency task force, including the creation of the FDA Transparency Blog.

“It’s incumbent on me as FDA commissioner to make sure that we get out there more, that I speak on behalf of the FDA on these important issues,� Dr. Hamburg said. “It’s something I’m committed to doing and part of the reason I took this job, because I felt that the FDA has a story that hadn’t been adequately told.�

She attributes the distorted message to the turmoil surrounding the agency in recent years, triggered in part by a slew of prominent drug recalls. Nevertheless, the reality is the decisions made by FDA affect the lives of every American every day, Dr. Hamburg stressed. She noted that a quarter of every consumer dollar is spent on products regulated by FDA.

“The average American has no idea what the FDA does, no appreciation of the impact that FDA activity has on their daily life, whether it’s the toothpaste they use, the food they eat, the drugs they take, the vaccines that they have their pediatricians give their kids, the cosmetics that they use,� Dr. Hamburg said.

While quick to defend the integrity of the FDA and its people, Dr. Hamburg hinted strongly that a new mindset needs to be fostered within the agency.

“We are clearly understood worldwide as the gold standard for consumer protection, yet within our own country we’ve allowed our name and our people to be battered because of crises that can occur, that will occur.� Dr. Hamburg said. “We can’t allow ourselves to be characterized only by when things go wrong. The truth is that things go right every single day. It’s extraordinary the work that happens within the FDA and the very important work that happens in partnerships between the FDA and other entities.�

In a questions and answer session following Dr. Hamburg’s speech, the issue of FDA funding, or lack there of, was brought up. The agency’ new leader, while acknowledging that FDA has to do a better job regardless, didn’t hold back when addressing the problem.

“It’s stunning to me how underfunded we have been and how stretched all of our staff and programs are,” Dr. Hamburg said. “But we are getting some additional resources. Sometimes a crisis results in some action.â€�

Dr. Hamburg noted that President Obama is proposing historic increases to the FDA budget in 2010.

“I’m hoping that we will see not just one or two increases in our budget but sustained increases,” she said. “Because we do have to really build our scientific and IT infrastructure in order to really do our job and do it well.”

Tags: FDA, Margaret Hamburg, regulatory