- - Wednesday, March 2, 2016

ANALYSIS/OPINION:

Last week, the Senate overwhelmingly approved a new leader for the U.S. Food and Drug Administration (FDA), Robert Califf, a well-respected medical researcher. Dr. Califf now has a unique opportunity to ensure that terminally ill patients gain the benefits of research in time to save their lives — benefits that are usually kept out of reach under the FDA’s current policies.

Before Americans can use any medicine, the FDA must deem it both safe and effective. But that process — which requires several rounds of testing and clinical trials — is long and expensive. Even after a drug is deemed safe, it’s still off-limits until the agency is satisfied it works, and those tests can take years and cost billions of dollars. In the meantime, countless Americans suffer and die of diseases those drugs could treat, or even cure.

The FDA has a program called “compassionate use,” which allows terminal patients to use potentially lifesaving treatments that are undergoing safety and efficacy testing, but have not yet received final FDA approval. But the reality is that fewer than 1 percent of terminally ill patients are ever able to take advantage of the compassionate use exception.

More than half a million patients have died of cancer alone in the past year — most without access to the more than 700 new treatments already under consideration. Countless more suffer and die from terminal illnesses with no FDA-approved cure, such as amyotrophic lateral sclerosis, or ALS, (commonly known as Lou Gehrig’s disease), Duchenne muscular dystrophy, and Alzheimer’s. But there are promising treatments for them already in the FDA’s pipeline.

The FDA claims it approves almost every compassionate use application it receives. That’s true. But a new report by the Goldwater Institute found that most patients and doctors either don’t know about the option or find the process so complex that treatment comes too late.

Something is clearly amiss in a system where more than 99 percent of terminal patients cannot take advantage of the compassionate use exception. Compassionate use should be the rule, not the exception.

That is why states are adopting a new pathway for terminally ill patients to access investigational treatments. Under Right To Try, terminal patients may, with the approval of their physicians, seek treatments that are being safely used in clinical trials, but are still awaiting final approval by the FDA. Right to Try, which is law in 24 states, is now extending and saving lives.

But while state Right to Try laws represent significant progress, federal lawmakers still must work to remove the many disincentives that make the current federal rules “a system of all risk and no reward,” as the Goldwater Institute report puts it.

Dr. Califf could do so today, allowing doctors to prescribe — and manufacturers to sell — drugs to terminal patients after they have passed Phase 1 safety testing on a provisional basis. This would put treatments and medicines in the hands of dying patients today and allow companies to begin recovering costs to finance subsequent clinical trials and create an early income stream for investors.

Additionally, the FDA should allow for reciprocal approval of treatments approved in advanced nations. An estimated 30 percent of the newest advances in medicine are first available overseas. Drugs that have already received the green light in countries such as Germany, Japan and Switzerland should be made available to patients in the United States. That would bring proven, lifesaving treatments to patients in America now.

Dr. Califf’s first goal should be to address the patients who are needlessly suffering, blocked by their own government from using potentially lifesaving treatments. This is wrong and immoral. It’s easily fixable and wouldn’t require any new spending.

Terminally ill patients shouldn’t have to beg the federal government for permission to save their own lives. If there’s a drug out there that could save you, you should have access to it now, not years from now.

Naomi Lopez Bauman is the director of health care policy at the Goldwater Institute.

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