Winning the Right to Save Your Own Life As the FDA dawdles, 24 states pass ‘right-to-try’ laws giving terminally ill patients access to drugs. By Darcy Olsen
http://www.wsj.com/articles/winning-the-right-to-save-your-own-life-1448574113
How far would you go to get a drug that could save your child’s life? Across an ocean? That is exactly what the federal government is forcing some American families with dying children to do.
In 2012, when Diego Morris was 11 years old, he was diagnosed with a deadly cancer in his leg called osteosarcoma. Doctors at St. Jude Children’s Research Hospital in Memphis, Tenn., removed the tumor, but the prognosis was poor. There was a significant risk that even extensive chemotherapy after surgery would not prevent the cancer from returning.
Fortunately, a team of doctors at MD Anderson Cancer Center in Houston and Memorial Sloan Kettering Cancer Center in New York City had developed a revolutionary new drug, mifamurtide (MTP), that can prevent osteosarcoma from coming back. A study by Dr. Eugenie Kleinerman of MD Anderson and Dr. Paul Meyers of Sloan Kettering showed the drug resulted in a 30% reduction in the osteosarcoma mortality rate at eight years after diagnosis.
The drug was approved in 2009 by the European Medicines Agency and is currently the standard of care in Europe, Israel and many other countries. In 2012 it received the prestigious Prix Galien Award, the gold medal for pharmaceutical research and development in the United Kingdom.
MPT was exactly what Diego needed. But there was one problem: The drug was not available in America because the Food and Drug Administration had rejected it, demanding additional studies. That meant that Diego had to travel from Phoenix to London to get the drug he needed to save his life—a drug that was available in almost every industrialized nation and should have been available in the U.S.
According to the Boston-based Center for Information and Study on Clinical Research Participation, 40% of cancer patients in the U.S. attempt to join a clinical trial to get access to potentially lifesaving treatments, but only 3% succeed. Hundreds of thousands of patients who want access to promising new medicines cannot get them—and are forced to deteriorate or die while the drugs wind their way through the FDA.
The lack of access to lifesaving drugs is not limited to cancer. Idiopathic pulmonary fibrosis (IPF) is a fatal lung disease that kills about 40,000 Americans each year. There were no effective treatments until InterMune, a U.S. company, developed a breakthrough therapy called Esbriet. The FDA finally approved it for use in the U.S. in October 2014—nearly five years after an FDA advisory committee had recommended approval. Meanwhile, Esbriet was available in Japan seven years earlier, in Europe four years earlier, and in Canada two years earlier.
According to President Obama’s Council of Advisors on Science and Technology, it now takes, on average, 14 years to bring a new drug to market in the U. S.—up from eight years in the 1960s. As a result, Americans with terminal illnesses typically are forced to rely on treatments that were developed 10 or 15 years ago, with cutting-edge innovations and cures locked behind the bars of archaic federal policy.
Americans with terminal illnesses are supposed to have access to investigational drugs through the FDA’s “compassionate-use” program, but that’s not happening either. The FDA claims that it approves nearly all applications for compassionate use, roughly 1,800 last year. What the agency neglects to mention is that 99% of patients never complete the application process. Why? In part, because the application process is so complex it takes an average of about 100 hours to complete. Another obstacle is that, under the rules of the compassionate-use program, companies can’t charge for treatments above cost, so there is little financial incentive to participate.
This is why the “right-to-try” movement at the state level is so important. Right-to-try legislation, once passed by state lawmakers and signed into law, allows patients with terminal illnesses access to investigational drugs that show promise in clinical trials but still may be years away from receiving FDA approval.
To qualify, a treatment must have cleared the FDA’s Phase I safety trials and be in ongoing Phase II or III trials where dosing and efficacy are established. Once Phase I is complete and the manufacturer and a physician agree to provide it, treatment can begin. No approval from Washington necessary.
In just over a year, 24 states have passed right-to-try laws, including Texas, Florida and Virginia. In February, after five states adopted right-to-try laws in rapid succession, the FDA pledged to “simplify and accelerate” the application process. The agency promised a new application process that would take less than an hour to complete. Sadly, the agency hasn’t fulfilled that promise.
California nearly became the 25th state with such a law. But on Oct. 11, less than a week after signing a right-to-die bill into law that allows terminally-ill patients to be prescribed drugs to end their lives, Gov. Jerry Brown vetoed the right-to-try bill.
Gov. Brown’s reasoning? A perfect Catch 22: “Patients with life threatening conditions should be able to try experimental drugs, and the United States Food and Drug Administration’s compassionate use program allows this to happen.”
No, it does not. Each year hundreds of thousands of Americans die without getting access to promising treatments that could save or extend their lives. This is unnecessary, immoral and the worst kind of bureaucratic overreach. No one should have to beg the federal government for the right to save his own life.
Ms. Olsen, president of the Goldwater Institute, is the author of “The Right to Try,” out this month from HarperCollins.
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