There is a growing recognition that the U.S. Food and Drug Administration is more of a threat to human safety than a protector of it. Maybe it needs to come with its own warning: This agency may be hazardous to your health!
Stephanie Ross might have appreciated just such a warning. The Drexel University (Philadelphia) student died unexpectedly on March 13 after being hit with a rare bacterial strain known as meningitis B.
The University of California Santa Barbara recently had its own outbreak of the disease, as did Princeton University last year. Both schools took a very unusual step.
They imported thousands of doses of the meningitis B (MenB) vaccine Bexsero that is widely available in Europe, Canada and Australia — but not in the U.S. — so students who wanted it could be vaccinated. The reason why the schools had to go outside the U.S. is that the FDA hasn't approved the vaccine.
Turning the notion of preventive medicine on its head, the FDA gave a limited approval of the vaccine only after the outbreaks occurred and only at the two universities.
Deadly Pattern
MenB can act very quickly, taking a life or leaving a victim severely disabled in less than 24 hours. So the post-outbreak strategy is effectively a death or disability sentence on many of those unfortunate enough to contract the disease.
The FDA is now working — albeit reactively rather than proactively — with Bexsero's manufacturer, Novartis, toward getting the vaccine approved. The question remains whether the agency will act quickly or slow-walk the process.
If this fatal tragedy were the only case, lawmakers might look past it. But delaying or outright denying approval of promising drugs is becoming a pattern at the agency — one that must change.
As the New York Times quoted Dr. Vance G. Fowler Jr., a Duke University infectious-disease expert, last year about the FDA approval process, "It has been progressively more difficult to usher a new anti-infective to market."
For example, the FDA recently rejected the pathbreaking drug Lemtrada that is being used to treat multiple sclerosis in 30 countries, including Canada, Australia and European Union nations. In this case, the FDA claimed that the studies did not conform to its requirement for being double-blind and placebo-controlled.
But placebo-controlled studies are not practical in some instances. Doctors can't compare a new anesthesia against a placebo in surgery. In those cases, they have to compare the new drug against an existing one.
The FDA has also refused to approve the drug pirfenidone, which has been shown to slow the decline in lung function for people with black lung disease. Yet the drug has been available in the EU since 2011 and Canada since 2012.
An Incentive Problem
The growing number of FDA critics worried the White House so much that the President's Council of Advisors on Science and Technology has urged the FDA to expand its use of fast-track approvals for drugs that target a narrow but high-risk population. Meningitis B, multiple sclerosis and black lung disease certainly seem to fall in that category.
There are good and dedicated people at the FDA. But the pressures on bureaucracies such as the FDA are the opposite of those faced by innovator companies and patients. Companies have huge incentives, both humane and financial, to get their drugs to their target populations as quickly and as safely as possible. Patients have similar interests.
But for bureaucracies, the downsides of acting quickly far outweigh the upside. FDA officials get very little notice when they approve a drug that works well; but they get tremendous criticism when they approve a drug and problems emerge.
Thus, the incentive is: If there's even a little doubt, then don't — an approach that can leave patients, and potential patients in the case of MenB, in the lurch.
When there is compelling evidence from several other countries that share essentially the same standards and concerns as the U.S. that an important new vaccine or drug is safe and effective, the FDA should move quickly to ensure that U.S. citizens have access to it.
Being proactive rather than reactive may be a challenge for bureaucracies, but it's what patients deserve.