For the second time in as many weeks, Food and Drug Administration commissioner Margaret Hamburg hit the lecture circuit to lay out the agency's vision on personalized medicine, though "vision" is perhaps too generous. Much of the speech, which left an unscientific sampling of attendees sitting near us hungry for actual content, was an apology for the agency's slow adaptation to the dawning era of more targeted therapies.
It wasn't all mea culpas and mushiness, to be sure. Speaking to a conference in San Francisco Monday, Hamburg said that her agency was on track to deliver draft guidance on biomarker qualification by the end of the year, an early step in creating a pathway for drug and diagnostic makers to bring more tailored therapies to market.
Introduced as "our friend Peggy" by conference host Steven Burrill, a San Francisco-based merchant banker and venture capitalist, Hamburg also acknowledged the difficulty in creating such a pathway, which will involve merging responsibilities that are now divided between two FDA divisions. "Personalized medicine in the treatment of disease equals the integration of drugs and diagnostics," she said. "It's clear we need to develop a consistent, integrated approach in the evaluation and regulation of products that comprise personalized medicine."
Close FDA watchers might have noticed the speech was at times verbatim to the talk Hamburg gave Oct. 26 during a personalized-medicine gathering in Washington ("The Pink Sheet" DAILY, Oct. 26, 2009.)
She made several conciliatory gestures to the conferees mainly gathered to discuss ways to make money from diagnostics. More than once she included investors among those who need to see "the promise of new therapies," and conceded that their "ability to realize their investments depends on what they see when they look at the FDA."
"Regulatory agencies are not known for openness, and FDA in particular has gained a reputation for being a bureaucracy-bound black box," she said. "And regulatory agencies are not known for being clear, but we need to be. Few runners would show up for a race without knowing the distance to the finish line."
(Not so business friendly, though, was the lunch menu just before Hamburg's speech that featured a build-it-yourself taco bar. For at least one lunch hour, slippery hand-held fajitas were a graver threat to business attire than an opaque regulatory process.)
After Hamburg's speech, she sat with conference host Burrill for a short Q&A session, which, far from illuminating the details of FDA's personalized medicine pathway, showed off Hamburg's question-avoidance skills. At one point Burrill asked whether industry taxes in the health care reform bills could diminish FDA's ability to raise future funding through its own fees. Hamburg's answer was brilliantly non-committal: "I think industry understands the importance of having a strong FDA. It's clear in the world we live in that success will have to be a partnership. It makes sense to me that we should have a funding stream that reflects industry understanding of the benefits of a strong FDA."
But at the end of the Q&A Hamburg seemed to shine a little light on comparative effectiveness. Burrill asked if comparative effectiveness was "a friend" to FDA (he likes to describe the world along friend/enemy lines, which makes In Vivo Blog wonder what he thought of the tacos at lunch). Would comparative effectiveness become a way for FDA to regulate pharmacoecnomics, Burrill asked?
Here's what Hamburg said: "In our work to date we have a very clear legal mandate to examine safety and effectiveness of products, it's not compared to this or that. But the truth is, some comparative effectiveness knowledge enters into some of what we do" in labeling, recommendations for first line or second line, risk/benefit acceptance, and the number of drugs available in the space in question. She cited interest in new models the EMEA is considering to look at what she called "economic and value issues."
Hamburg also made a pitch for tighter coupling of FDA and the Center for Medicare Services, or CMS. "It really makes sense for us to create a system where there's more coordination and sharing of information and thinking earlier in the process... To have CMS more involved with FDA in a more collaborative way, to talk about products in the pipeline is something people have advocated for." - Alex Lash
Photo courtesy of the FDA.