The FDA Should Be In Ebola-Mode All the Time
The Food and Drug Administration is encouraging companies to move fast on treatments for Ebola, the disease that is rapidly spreading through West Africa. FDA is fast-tracking GlaxoSmithKline's promising Ebola vaccine, and supporting the efforts of Mapp Biopharmaceutical to develop more supplies of ZMapp, an experimental treatment for Ebola patients.
If FDA applied the same sense of urgency to Ebola vaccines in the past or to other drugs in its pipeline, millions of people would be better off. Instead, it stalls until a crisis arises, resulting in more deaths and untold suffering.
Take the case of Sanofi's Lemtrada, a drug to treat multiple sclerosis that has been approved all over the world. Taken once a year, it alleviates the symptoms of the disease and delays the onset of paralysis. Although the drug has already been approved to treat leukemia-so it is safe-FDA did not approve the design of its Phase III study and refused to approve it last December. Sanofi resubmitted the drug with additional data in May, but no word yet from the FDA.
Another drug that made headlines is Bexsero, made by Novartis, a vaccine against Meningitis B. Following outbreaks of Meningitis B across college campuses, last April Bexsero received Breakthrough Therapy designation, which expedites treatments for life-threatening diseases if the new treatment is better than existing alternatives. The vaccine has been used against the bacterial infection over half a million times worldwide, including around 30,000 times at Princeton and the University of California at Santa Barbara after the FDA granted special permission. More than 30 other countries allow Bexsero, including the EU, Australia, and Canada. There are FDA-approved vaccines for the other strains of Meningitis, but none for serogroup B-a particularly deadly strain. Some people can now get the vaccine, but not others. The system leaves people at risk of deadly infection when a safe alternative exists.
Eteplirsen, made by Sarepta Therapeutics, has been awaiting FDA approval as a treatment for Duchenne muscular dystrophy. This disease affects about 15,000 boys in the United States and usually leads to death by the time patients reach their early 20s. There is currently no FDA-approved treatment. The drug has been held up since December 2012 without guidance from the FDA on how the company should proceed. Some patients are too debilitated by their disease to participate in clinical trials, so they are left hopeless. Patient advocacy groups are pushing for accelerated approval while the drug continues to undergo testing. Parents have been vocal opponents of FDA's skepticism over Eteplirsen.
Tresiba, made by Novo Nordisk, is a long-acting insulin drug that has been shown through late-stage trials to be safe and effective for long-term use in children and young adults with type 1 diabetes. The drug has also been found to be safe for type 2 diabetes. Diabetes affects about 29 million Americans and 382 million worldwide, about 10 percent of whom suffer from type 1 (or juvenile) diabetes. Over concerns about risks to the heart, which were not found to be statistically significant but could warrant further study, FDA has refused to approve Tresiba. FDA could have approved the drug and ordered a long-term trial on its heart effects, but it chose to delay improved treatment for millions of patients instead. The drug is approved for use in Europe and Japan. Before the unanticipated rejection of Tresiba, an FDA advisory panel voted 8-4 in favor of approving the drug.
Why is the FDA slow-walking Lemtrada, Bexsero, Eteplirsen, and Tresiba, as well as many other drugs? The public façade is that the FDA wants to reduce risk, to avoid disasters such as Thalidomide, a drug given to pregnant women in the 1950s that caused birth defects. Ostensibly, FDA wants to protect patients from dangerous drugs.
In reality, FDA's bureaucratic power is enhanced by slowing down the approval process. A slow process gives the agency greater discretion in extracting concessions from these companies. This gives their congressional and executive branch overseers more power. The pharmaceutical industry lobbies and gives campaign contributions to key members of Congress, who then lean on the FDA to move forward with important drugs.
According to the Center for Responsive Politics, "The primary goal of much of the money that flows through U.S. politics is this: Influence. Corporations and industry groups, labor unions, single-issue organizations - together, they spend billions of dollars each year to gain access to decision-makers in government, all in an attempt to influence their thinking."
Pharmaceutical companies are not allowed to lobby the FDA, so they lobby politicians, who then hold hearings, or call the agency. This gives the FDA power in Washington, and enables government bureaucrats to get well-paying jobs when they leave. The pharmaceutical industry is one of the biggest lobbyists in town. In 2013, according to the Center for Responsive Politics, the industry spent $226 million on lobbying. The top 3 companies in spending were Eli Lilly ($9.9 million), Amgen ($9.1 million), and Pfizer ($9.0 million).
The Ebola outbreak should be teaching us that the FDA review process needs reform. Companies should not have to spend millions on lobbying to get their drugs approved. We need to be pushing out the envelope of medical innovation, and the FDA should be a partner, rather than a stumbling block. Although the absence of an Ebola vaccine and treatment is a tragedy today, it was not a tragedy a year ago. Let us not wait for the next unknown epidemics to expedite the cures.
