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.