Wednesday, April 28, 2010

Accelerating Cures by Building a More Effective and Efficient FDA

Kristin Schneeman, Program Director, FasterCures
A distinguished panel of observers from patient organizations and industry declared themselves to be in “violent agreement” on the urgent need to increase resources and build scientific capacity at the FDA, with one panelist calling it of “strategic importance to the country.” Among the points of agreement were:
  • FDA suffers from an almost scandalous lack of resources. The agency that regulates 25 cents of every dollar U.S. consumers spend has a budget that is half what Americans spend every year on potato chips.
  • Scientific capacity at the agency is very weak and is inhibiting the agency’s ability to evaluate new therapies moving through the pipeline.
  • The agency badly needs to be able to collaborate meaningfully with other sectors – what some refer to as the “triple helix” of government, academia and industry – without being constantly “demonized” for conflict of interest.
Gail Cassell, Vice President of Scientific Affairs at Eli Lilly & Co., shared the results of an important review of FDA’s science capacity that she chaired in 2007 as a member of FDA’s Science Board, which put a bright spotlight on the resource constraints under which FDA operates and their serious consequences. “Chronic underfunding of FDA puts American lives at risk.”

Garry Neil, Corporate Vice President at Johnson & Johnson’s Corporate Office of Science and Technology, detailed the risky, unpredictable, and costly pathway to new drug approval, a process that hasn’t changed substantially in 50 years. He reiterated some oft-cited facts – that it costs well over $1 billion to develop an approved drug, and that despite exponential increases in investment by government and industry the number of new therapies approved every year has declined. “There is nothing black and white here. The whole process is about the progressive removal of uncertainty. And there will never be complete certainty.”

Ellen Sigal, Chair and Founder of Friends of Cancer Research and an advisor to many government cancer research initiatives, provided the view of many patient advocates who find FDA impenetrable, with no mechanism in place for patient input, a confusing structure, and a lack of expertise in the places where it’s needed. She said the patient voice is not being heard on Capitol Hill, that concerns (many of them legitimate) about safety were all but drowning out patients’ appetite for innovation, willingness to risk-share, and need for better drugs.

Jonathan Simons, President and CEO, Prostate Cancer Foundation, offered up some interesting models for addressing some of these challenges at FDA. One was for some kind of “honest broker knowledge exchange” within FDA that facilitates collaboration among stakeholders in the system, evaluates all data presented by stakeholders, and creates value through transmission of knowledge. He also proposed that FDA have a sort of “amicus curiae” process, as they do in the court system, to allow meaningful input by stakeholders other than regulated industry.

Cassell held up as a success story the development of anti-retrovirals to treat HIV – 29 new therapies approved in relatively short time, achieved when stakeholders worked together across sectors with optimal speed. Simons noted that a significant reason for this success was leadership – that Anthony Fauci at the National Institute of Allergy and Infectious Diseases had responsibility for addressing the crisis and invited in the patient community to provide guidance. Can this success be replicated? “It happened with HIV, it can happen again,” said Neil.

The panel ended on a surprisingly hopeful note, with most panelists echoing Sigal’s optimism that “there is a growing recognition that the agency needs more resources, needs collaboration, needs science, and that its science is different from NIH’s science.” All agreed that the public and Congress need to be better educated about the importance and impact of FDA – not only on health but on our economy and national security.

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