Is the FDA's glass half-empty or half-full?

Next week the Senate HELP committee is expected to approve Dr. Robert Califf to lead the Food and Drug Administration (FDA). A well-respected medical researcher who is already working at the FDA as a senior-level political appointee, his credentials are impeccable. But before lawmakers cast their votes, there are a few questions that need to be raised.

FDA recently issued a 48-page report detailing its performance for the year. While preliminary, the data boasted that almost two-thirds of new drugs world-wide were first approved in the U.S. and that the agency has increased the share of drug applications it approves on the first try, which, according to the FDA, is a dramatic improvement over the past couple of decades.

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But these statistics only tell part of the story.

These numbers conceal the actual amount of time patients wait for drug approval, and for someone who is terminally ill and in a personal race against time to save his own life, the current process to access investigational drugs is so bureaucratic that approval often comes too late.

Take, for example, Esbriet, which the FDA approved last October. This breakthrough drug treats idiopathic pulmonary fibrosis (IPF), which is one of the nation's leading killers, taking about 40,000 lives annually (roughly equivalent to the number of breast cancer deaths each year). The disease is terminal for most patients within three years.

Esbriet has been available in Japan since 2008 and in Europe since 2011. An FDA advisory committee actually recommended approval back in 2010.

Not satisfied with the available data, the FDA required additional studies, resulting in years of additional delays. Between the time that the FDA advisory committee recommended approval and the time that the FDA finally gave it, another 150,000 Americans died of the disease.
The FDA’s “compassionate-use” program is supposed to allow terminally patients access to potentially life-saving treatments before they receive final FDA approval. But the reality is that the application process is so complex it can take a physician about 100 hours to complete.
As a result of public awareness and pressure, the FDA released a new draft application in February that it estimates will require only 45 minutes to complete. Yet more than ten months later, this new application is still not available.

Given these bureaucratically imposed obstacles, it should come as no surprise that the Goldwater Institute's "Right to Try" legislation has passed in 24 states. These laws provide an important avenue for terminal patients to access potentially life-saving treatments. Terminally ill patients who have exhausted all treatment options and do not qualify for a clinical trial can, under the care of their physician, seek treatments that have successfully completed basic FDA safety testing and are being safely used in clinical trials.

Right to Try provides terminally ill patients an additional treatment path that does not require the patient to beg the federal government for permission to try to save his own life. While state Right to Try laws are an important first step, federal lawmakers should be looking at more life-saving pathways for terminally ill patients.

For example, the U.S. government's own National Institutes of Health, is actively involved in ongoing clinical trials of the Ebola drug ZMapp in West Africa where both safety and efficacy are being tested simultaneously. Under this approach, once a drug is shown to improve outcomes, it is quickly integrated in the control group’s treatment. Under the current FDA system, even when a treatment appears successful in clinical trials, the control group still gets placebo and final approval still takes years. Why aren’t we using adaptive trials for all terminal illnesses?

Just last month, legislation was introduced in the U.S. Senate that would allow for reciprocal approval of drug treatments approved in other advanced nations. Let’s hope this speeds through Congress; after all, there are dozens of drugs already approved in Europe, Japan, and other First World countries that could save American lives.

For those Americans with terminal illnesses who are forced to rely on treatments that were developed decades ago, promising new treatments – some already available in other countries --  are being stifled by an outdated approach that is failing to keep pace with 21st century medicine. Will one of Dr. Califf's first goals be to help patients who are needlessly suffering, blocked by their own government from using potentially life-saving treatments?

Lopez-Bauman is the director of healthcare policy at the Goldwater Institute.

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