ARTICLE IN BRIEF
✓ A new IOM report recommends 25 action steps that Congress should take to enable the FDA to ensure that new therapies are safe for the publication.
The FDA needs more money, manpower, and muscle - in the form of regulatory authority - to address the growing perception of crisis that compromises the agency's credibility and the pharmaceutical industry as a whole. That's the conclusion of The Future of Drug Safety: Promoting and Protecting the Health of the Public, the Institute of Medicine's (IOM) September report on the FDA and the US drug safety system. It's one that leading neurologists wholeheartedly endorse - although they're not holding their breath that the needed funds will be forthcoming.
With recent issues about drug toxicity and adverse events, and increasing concerns about consumer pitches of drugs that can be somewhat distorted, the role of the FDA becomes extremely important, said Patricia Coyle, MD, Professor of Neurology at the State University of New York at Stony Brook School of Medicine and Director of the Stony Brook Multiple Sclerosis Comprehensive Care Center.
The FDA is overworked and underfunded. There's too much to do for the level of support and the personnel they have, and there's a concern that things can fall through the cracks. They also don't seem to have had much in the way of teeth, of enforcement power.
IOM RECOMMENDS 25 ACTION STEPS
The IOM report aims to correct that, by laying out a menu of 25 recommended action steps for Congress. Among them, it recommends clarifying the FDA's regulatory authority, giving it the ability to require needed post-marketing surveillance, risk assessment and risk management; mandating clinicaltrials.gov registration of, at a minimum, all phase 2 through 4 studies; stabilizing the FDA leadership by giving the FDA commissioner a six-year term; and substantially increasing the FDA budget to accomplish these goals.
Congress should act on the recommendations swiftly, urged Lily Jung, MD, Interim Medical Director of the Multiple Sclerosis Program at the Swedish Neurosciences Institute, and Neurology Division Chief at Swedish Hospital Medical Center in Seattle. Dr. Jung serves on the FDA's Peripheral and Central Nervous System Drugs Advisory Committee.
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NEED FOR POST-MARKETING ASSESSMENT
She pointed to post-marketing risk assessment and risk management as particularly urgent. There really isn't a process in place to monitor drug safety once a drug has been released. It's catch as catch can, and we're pretty lucky that nothing too bad has happened with neurology drugs because we don't have a plan in place.
It's a wake-up call: waiting for something to happen, or hoping to catch something as opposed to systematic monitoring, is crazy. With natalizumab, we were lucky that there was as much close attention paid to those patients and that Biogen was able to identify them as quickly as they did, she said.
Ralph Sacco, MD, Professor of Neurology and Epidemiology at the Neurological Institute of Columbia University College of Physicians and Surgeons, who also serves on the FDA Peripheral and Central Nervous System Drugs Advisory Committee, agrees with Dr. Jung's assessment. The FDA recommends that we should monitor for X, Y, and Z in this drug, post-approval, but they aren't the ones doing the monitoring. Usually, that's all done by the pharmaceutical company, and the data at that point would be reviewed by the FDA. But the FDA hasn't had the people-power, financial resources, or authority to expand their post-marketing surveillance.
Indeed, said Dr. Coyle, the TOUCH prescribing program (Tysabri Outreach: Unified Commitment to Health, www.fda.gov/cder/drug/infopage/natalizumab/RiskMAP.pdf) being used to monitor natalizumab since its reintroduction to market could serve as a good model for an expanded FDA role. That surveillance is being done because of specific concerns about a serious infection, but overall it's going to be a good surveillance system for following up on the drug and possible adverse effects. These are important and valuable concepts.
SIX-YEAR FDA COMMISSIONER TERM
Appointing an FDA commissioner with the independence of a six-year post, and a background with the requisite scientific expertise - another IOM report recommendation - is also essential, say neurologists.
The commissioner cannot be a political appointee with an agenda. It has to be someone who's committed to the public health responsibility of the FDA, said Dr. Jung.
There was widespread agreement with the requirement that all phase 2 to 4 trials be registered with clinicaltrials.gov as well. Although many leading medical journals now mandate that trials must have been registered in a public database from inception for publication of results, a legal requirement would provide additional teeth.
If we're going to be clear about tracking all of the studies out there, some of which won't make it into journals, we need to be able to capture all that data, and unless it's mandated, we're not going to be able to do it, says Dr. Jung. I'm always amazed at how the pharmaceutical companies, who have a lot of vested interest in pulling out each other's dirty laundry, so to speak, are able to use the resources they have to dig up information from unpublished studies that the FDA or the government doesn't have the staff or resources to find.
IS INCREASED FDA FUNDING LIKELY?
And there's the rub. All 25 recommendations hinge on just one of them: the one that calls for increased FDA funding. The FDA's current budget is woefully lacking, all agree; its resources barely sustain the mandate it currently has, much less any expanded authority.
I have a huge amount of respect for the FDA staff, said Dr. Jung. They are excellent scientists, they take their jobs to heart, and it's frustrating to see good people have their hands tied because they're not adequately funded to do the work they want to be doing and should be doing. You can't fault an agency for not doing its job when you don't adequately fund the work.
But in a time of a growing federal deficits and agency budget-cutting, when the NIH has just seen its first real-dollar budget cut in decades, there is skepticism that Congress will appropriate the needed funds. Given the current financial climate, it's difficult to see the US Congress appointing more funds for the FDA at this point in time, Dr. Sacco said. But we can hope that will change.
But without funding, the rest of the report will fall by the wayside, said Dr. Coyle. This pushes it back on Congress. If they endorse the recommendations but don't appropriate adequate funding, it's really just a paper tiger. There are lots of priorities for governmental funding, but hopefully government is going to see that this needs to be a priority for the country. Without that support, it will undermine all the other recommendations, which would be a shame because these recommendations are timely and important.
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