Think back to the Ebola chaos of last year. Nobody except the caregivers came away looking good—not the White House, not the Department of Health and Human Services, not the World Health Organization, not Congress, not most of the media.
No organization failed more miserably than the Food and Drug Administration. As I pointed out in The Weekly Standard last year (“Failing to Rise to the Challenge,” December 1, 2014), the FDA refused to use its discretion under a 2007 law to provide vouchers for an accelerated review to any sponsor who successfully developed a drug for a designated “neglected tropical disease.” The FDA never added Ebola—or any tropical disease—to the list included in the statute. It also never gave the pharmaceutical industry clear guidance on the requirements for Ebola clinical trials—no small matter when huts and rudimentary equipment replace posh teaching hospitals and state-of-the-art devices.
In the midst of the Ebola scare, the FDA did issue a July 2014 tropical disease “guidance,” but that hastily prepared attempt at damage control offered nothing except a generic litany of clinical trial approaches that the FDA may or may not approve in any particular case. This FDA intransigence backfired—it stifled Ebola drug development and created a backlash that forced the FDA to allow the use of drug candidates untested in human subjects. If the agency had embraced the 2007 neglected tropical disease legislation instead of ignoring it, multiple drug candidates tested in humans would almost certainly have been available; lives might have been saved and hysteria might have been avoided.
The FDA culture has always resented outsiders—whether senior executive branch officials or Congress—“interfering” with their increasingly barnacled drug approval process. In the eighties the agency fought Commissioner Frank Young when he first created “fast tracks” for HIV drug approvals. A few years later the FDA unsuccessfully fought extension of those “fast track” principles to drug approvals for other fatal diseases. Despite the fact that the “accelerated approval” regulations of 1992 helped millions of Americans with cancer, fatal protein deficiencies, multiple sclerosis, cystic fibrosis, and other conditions, two decades later the FDA did not support innovative legislation to award “priority review” vouchers to sponsors of approved drugs for neglected rare and devastating diseases of children.
After failed attempts to preserve its unnecessarily sluggish bureaucracy, the FDA resorted to abusing its administrative discretion to undo accelerated approval reforms on a case-by-case basis during the secrecy of the drug approval process. Some reviewers gamed the system by threatening sponsors who did not “voluntarily” request more time for their reviews.
After Congress passed the “neglected tropical diseases” law in 2007, the FDA treated it as dead on arrival. The agency apparently felt that it could finally draw a line against accelerated approvals because the targeted diseases lacked the patient advocacy groups and media attention that drove earlier reforms.
That shameful strategy worked until Ebola. Shortly after my Weekly Standard piece last year, momentum rapidly built for a bill to add Ebola to the list of neglected tropical diseases that merited incentives for approvals. Despite the fact that FDA officials circumvented the White House and “unofficially” went to Congress to try to thwart this legislation, Congress unanimously passed the bill in the lame duck session; it became law on December 16, 2014.
In the wake of continuing criticism of its handling of Ebola, on August 20, 2015, the FDA decided to head off additional criticism by adding Chagas disease and neurocysticercosis to the statutory list of neglected tropical diseases. With these two diseases, however, the FDA apparently decided to try once more to draw the line against additional accelerated approvals—a decision that will cost thousands of lives.
One example of a disease for which the FDA should be encouraging drug development is Chikungunya, a disease first identified in Tanzania in 1952 that spread to the Americas in 2013 (Lindsay Lohan suffered from it last year). Now present in more than 60 countries, it is caused by a virus transmitted by mosquitos, and its symptoms are similar to—but far more painful than—those of Lyme disease. It is fatal in the short run in about one case in one thousand, but the lingering inflammation it causes eventually kills more patients by damaging their hearts and other organs. The virus also tends to mutate as it adapts to new environments.
Owing to its recent arrival and diffuse symptoms, Chikungunya is often misdiagnosed in the United States, but there are already more than a million people in this hemisphere and hundreds in this country with the disease, and the history of this epidemic strongly suggests that those numbers will explode in the next few years. In other words, now is the exact time the FDA should be responding aggressively instead of merely noting that it “considered” a proposal in 2008 to add Chikungunya to the neglected tropical diseases list.
To be fair, the FDA’s Center for Biologics Evaluation and Research, the component within the FDA that regulates the blood supply, has reacted very differently from the FDA’s drug approval component. Officials there grew concerned about this risk to the blood supply years ago and used their scarce research funds to search for ways to identify and neutralize the Chikungunya virus in blood banks. Perhaps these two parts of the FDA should actually talk to each other.
As is traditional with new epidemics, the FDA prefers to protect its institutional prerogatives rather than protect people suffering from this horrid—and spreading—disease; change will have to come from outside. HHS secretary Sylvia Burwell should clear a day to bring in experts from the National Institutes of Health, the Centers for Disease Control, the World Health Organization, the Agency for International Development, academic leaders, and foreign health ministers to develop a plan. Burwell’s first step should be easy: Direct the FDA to add Chikungunya to the neglected tropical diseases list.
Her second step should be to direct the FDA to convene an advisory committee to help the agency craft guidelines for Chikungunya clinical trials within six months. Such a directive would be timely because there are promising, but underfunded, efforts at Vanderbilt University to develop a treatment for Chikungunya. The work at Vanderbilt is doubly important because the technology being studied for use as a treatment might also help create vaccines.
Burwell’s third step should be to scour the executive branch to find funds to help Vanderbilt and researchers at other institutions who are struggling to develop treatments. If Hillary Clinton can divert $27 million from the State Department to one of her husband’s pet health care projects in Africa, the Obama administration should be able to come up with a similar amount to assist in the development of potentially lifesaving treatments.
The Senate should also raise the FDA’s inaction over Chikungunya and the broader issue of its resistance to accelerated approvals with President Obama’s nominee for FDA commissioner, Dr. Robert Califf. Given Dr. Califf’s broad experience working on clinical trials for pharmaceutical companies, he should understand the urgency of action, distance himself from failed policies and cultural norms, and embrace the accelerated approvals that have saved the lives of millions of Americans and desperate people all over the world.
Michael J. Astrue is a former commissioner of Social Security and a former general counsel of the Department of Health and Human Services.
