The FDA: Balancing innovation and safety

If you talk about the need to speed up the approval process for new drugs and devices, the U.S. Food and Drug Administration is often the first criticized for the time lag to new treatments. But, as the Partnering for Cures session, “What You Probably Don’t Know about Innovation at FDA” made clear, the agency is trying to become more innovative and approve new treatments more quickly and efficiently, but there are obstacles.

The biggest danger, said Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, is “where public criticism and controversy outweigh science.” Deliberations should be made based on the latest science, she said, but controversy affects how the agency works and creates what she called a “circling of the wagons,” a “let’s not be too innovative” mindset.

She noted that the agency approved 10 cancer drugs in the last fiscal year and is on track to do so again this year, but it is work the FDA should not be doing alone. “It is the job of the larger biomedical community in concert with FDA” to get new drugs on the market, she said.

Some drug makers are wary of using innovative approaches with the FDA. “Once we get to the clinical study, if we want to be innovative, we have to do the traditional approach alongside, sort of carrying two tracks to get the product to market,” said Joseph DeGeorge of Merck Research Laboratories.

Many challenges to new drug approval start well before a drug finds its way to the FDA. Jonathan Leff of Warburg Pincus noted that venture capital investment in life sciences has been decreasing dramatically, and in the first nine months of this year, the number of first-time companies receiving venture capital investment was the lowest it has been since 1995. Those venture capital firms that are investing, he added, are shifting their investments to other countries or in less innovative products. “We have tremendous opportunity to translate science that is happening into cures, but the system isn’t working as well as it needs to,” he said. “We are not getting the throughput. We need to address the issues of translating scientific discoveries to new therapies.”

Some stakeholders are finding ways to assist the FDA in the process. Richard Insel of JDRF said his organization decided it had a role in educating the agency in novel treatments and their development. And when JDRF wanted to work on an artificial pancreas, there was no regulatory guidance. So with the FDA’s knowledge, the group convened opinion leaders and created draft guidance that was submitted to the FDA. This helped the FDA draft its own regulatory guidance for the artificial pancreas, which just came out in final form.

Frank Douglas with Austen BioInnovation Institute said his organization decided to bring the FDA in earlier in the process and found that things went smoother. “We spent a lot of time trying to figure out how to approach the FDA to make them feel they were in a safe harbor for collaboration,” he said.

The panelists agreed that bringing patient foundations, as well as the patients and families, into the regulatory process to discuss risks and benefits of individual drugs is vital. “We need to hear from affected communities about what tradeoffs they think are acceptable,” said Woodcock. “That should be our guiding light.”

So what would help the FDA improve the process, moderator Margaret Anderson of FasterCures asked the group.

The innovative spirit that is evident at the highest levels of the FDA need to be transitioned to the practitioners of the review process, said Insel.

Douglas noted that as the FDA collaborates more, it should identify other bodies that can be brought in to help with reviews.

Woodcock noted that the workload at the FDA continues to be a problem. It is hard to feel innovative when you are facing a mountain of paperwork, she said.

Anderson noted that the Alliance for a Stronger FDA, which she urged attendees to support, is focused on increasing the agency’s budget.

One panelist answered the question with praise. “The FDA environment, in terms of helping to advance and stimulate innovation, is better today than it has been in a while, and that is a credit to the leadership,” said Leff.