FDA, After Taking Heat, Offers Up Reforms to Support Pharma, Biotech & Device Innovation

Luke Timmerman10/5/11Follow @ldtimmerman

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The FDA has spent decades talking about how its job is to make sure all kinds of U.S. medical products are safe to use, and effective. Now it’s coming out with a new strategic outline on how it intends to do all that same stuff, while also spurring the development of more innovative drugs and devices that will help the U.S. hold onto its competitive advantage in the life sciences.

The FDA commissioner, Margaret Hamburg, today is releasing the 40-page overview with a decidedly non-adversarial title of “Driving Biomedical Innovation: Initiatives for Improving Products for Patients.”

While the full report has some defensive notes in it about how the FDA has already been trying to stir innovation for years through its 2004 Critical Path Initiative and other vehicles, it acknowledged a number of complaints that entrepreneurs, big companies, and investors have leveled against the agency for years. It cites the common beefs about how the agency can be unpredictable; not so good at helping small businesses navigate its processes; not exactly on the technological cutting edge; and inefficient. The FDA now says, after hearing input from various outside groups for months, that it wants to reform by improving its outreach to small businesses; creating a fast approval pathway for certain targeted therapies; improved data sharing; scientific training; and streamlining of processes and regulations.

It cites the familiar refrain to all industry observers about how the government and industry is pouring $95 billion a year into biomedical R&D, and getting fewer returns in the form of novel new products to help patients.

There’s a political backdrop to all of this, of course, as the President and the Congress are deeply worried about the stubbornly high jobless rate. Pharmaceuticals, biotech, and medical devices are three industries in which the U.S. is the undisputed world leader—yet there’s almost universal scorn for an FDA that has become so cautious and bureaucratic that it is stifling those industries and sending much of the U.S. competitive advantage overseas. Some prominent venture capitalists, and industry trade groups, have been pressuring people in the White House and in Congress for months to shake up the FDA, which I wrote about here back in April.

FDA commissioner Margaret Hamburg

The FDA talks like it has been listening. Hamburg wrote in the report that the agency “is working to position itself not only as a positive driving force in the ecosystem as a regulator, but also to facilitate medical product innovation.”

And Kathleen Sebelius, the U.S. Secretary of Health and Services of which the FDA is a part, added in a statement that: “The Obama Administration is committed to encouraging the entrepreneurs and businesses that are modernizing and strengthening our health care system. The innovation blueprint is another part of our effort to help businesses grow and keep Americans healthy.”

The blueprint is fairly vague in parts, and Hamburg notes in her introduction that it is an ongoing initiative and that it will be expanded.

One venture capitalist, Bob Nelsen of Arch Venture Partners, said he’ll need more assurance the FDA reforms are meaningful.

“It is a positive step that the FDA in recognizing that innovation matters. The verdict is out as to whether they will really change, so I will look for data. The FDA is under bipartisan pressure to re-think their policies that are leading the US to lose its huge advantage in medical devices to Europe, and [prompt] biotech investors like me to start investing in China,” Nelsen said in an e-mail.

“We no longer can justify investing in huge public health priorities like diabetes and Alzheimers, that cry for innovation investment, as a direct result of FDA policies that are muddled, unclear and don’t take patients into account. There is a dialog, and that is certainly a good thing, but as recently as two weeks ago Margaret Hamburg blamed the slow pace of drug approval on the lack of good science, not the FDA, which was a fall out of your chair moment, so we have a long way to go,” Nelsen said.

Still, the FDA document states repeatedly that the agency wants to be more supportive, and cites some of President Obama’s more business-friendly initiatives, such as the “Startup America” program which seeks to encourage high tech entrepreneurship as well as the “Strategy for American Innovation.”

Here’s the quick rundown on some of the programs the FDA has sketched out in its report, which it says should help spur more biomedical innovation:

—The FDA plans to establish a Small Business Liaison program staffed with people who have experience starting and running biomedical companies that have been able to navigate the FDA regulatory process to develop new products. These same people will help train the FDA on how to better understand and work with the small business people who are seeking various FDA approvals.

—Young entrepreneurs from university business schools will be recruited into fellowship programs at the FDA. That part of the program will help provide “first-hand access to information about regulatory review and what it takes to move a product through the FDA approval process. This knowledge base will enhance their effectiveness as future business partners, founders of companies, or investors,” the FDA said.

—The agency is working to find ways to incorporate new technologies like whole genome sequencing and biomarkers that are supposed to help pave the way for more personalized medicine, rather than prescribing drugs through trial and error to see what works for an individual—which is usually how it works today. Much of the science to support personalized medicine is “underdeveloped” and falls across multiple centers within the FDA, the agency said. To improve the scientific understanding and encourage more co-development of diagnostics to help properly select patients for personalized medicines, the FDA says it plans to increase its investment in “regulatory science”. It defines this field as “the knowledge, tools, standards, and approaches necessary to assess the safety, efficacy, quality, and performance of FDA-regulated products, will play an important part in addressing the challenges presented by personalized medicine.”

(Incidentally, UC San Francisco Chancellor Susan Desmond-Hellmann, the former president of product development at Genentech, recently told me in an interview that while some people don’t appreciate it on campus, she wants to put greater emphasis on “regulatory science” at UCSF as a way to help basic science become translated into new products for patients. The FDA said it plans to support regulatory science through collaboration with other government agencies, academic institutions, and public/private partnerships.)

—To improve coordination among the various divisions within FDA, the commissioner said she appointed a deputy commissioner for medical products to oversee drugs, biologic drugs, and devices.

—The FDA cites some willingness in the report to move more quickly on reviewing treatments with what it calls “exceptional promise.” It cited the history from the early 1990s, when AIDS activists pushed the FDA to make experimental treatments available more quickly for people who had a life-threatening illness at the time. One of the ideas FDA is working on is in regard to helping select the patients most likely to benefit in clinical trials, as a way of making the trials much more efficient than in the past.

—For medical devices, the FDA is talking about a new regulatory pathway besides the PMA path for groundbreaking new devices (which many entrepreneurs consider too time-consuming and costly) and the 510k pathway for relatively modest iterations on existing technologies. The new pathway will be the “Innovation Pathway” and will be faster than the PMA route, the FDA said. The agency will commit time and resources for better understanding of these innovative products early in the development process so “”so scientific issues and regulatory hurdles can be identified early on and unnecessary delays can be avoided.” It is also developing a “Network of Experts” to help the agency better understand the new technology.

Hamburg plans to discuss the report later today in Washington DC at the Washington Ideas Forum. I’m curious to hear your reaction to the FDA’s Innovation Initiative—what you like or dislike about it; what’s missing; and what effect you think this might have on company formation and investment in biomedical R&D. As always, you can leave a comment in the space at the bottom of this story.

Luke Timmerman is the National Biotech Editor of Xconomy. E-mail him at ltimmerman@xconomy.com Follow @ldtimmerman