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Duchenne muscular dystrophy and the Right To Try

September 21, 2016

On September 19, 2016, the FDA approved a drug that had been safely and effectively treating children with Duchenne muscular dystrophy in clinical trials for years. The approval means children, mostly boys, born with this disease that leaves its victims in wheelchairs by 13 and dead by 20, have hope for the future.

This approval is a win for patients and their families, and a win for advocates who have been pushing the FDA to approve treatments more quickly for rare diseases.

But the win came too late for many families. Three years ago when data first emerged from clinical trials about the effectiveness of this drug, patients and their families literally begged the FDA to expand the tiny trial to more children so they too could have a shot at the benefits they were hearing about. For many patients who could not qualify for the clinical trial because they were too sick—or not sick enough—there would be no help from the FDA. There was simply waiting. During that time, countless children died, and many have permanently lost the ability to walk and even use their arms to hug their moms.

Everyone agrees that there’s a role for the FDA in determining the safety of drugs that end up on our pharmacy shelves. But, there also needs to be recognition that not all treatments are the same—and some should be available much sooner than the current process allows.

That’s where Right To Try laws come in. Right To Try allows doctors to prescribe to terminally ill patients medicines that are being safely used in clinical trials, but that are not yet fully approved. These laws have been adopted by 31 states in two years and there is legislation pending in Washington to protect these state-passed laws from federal interference.

Right To Try laws are in place in: Alabama, Arizona, Arkansas, Colorado, Connecticut, Florida, Georgia, Idaho, Illinois, Indiana, Louisiana, Maine, Michigan, Minnesota, Mississippi, Missouri, Montana, Nevada, New Hampshire, North Carolina, North Dakota, Oklahoma, Oregon, South Carolina, South Dakota, Tennessee, Texas, Utah, Virginia, West Virginia, and Wyoming. The law has been introduced in an additional 18 states. The national bipartisan effort to give terminally ill Americans access to investigational medications is being led by the Goldwater Institute.

As the Duchenne example illustrates, millions of Americans die each year from terminal illnesses for which there are treatments and cures. About 40,000 women with breast cancer will hear from their doctor this year that they have no treatment options left. But there are 22 pioneering breast cancer treatments waiting for the FDA’s green light; some of them are already available and safely treating women in Europe. Right To Try gives women access to those treatments today.

Right To Try is changing the game for terminal patients. You can learn more about the law and how it’s working today at the Liberty International healthcare conference in Austin, Texas, on October 22.

I hope to see you there!

You can also follow progress of the national Right To Try movement on Facebook or at And you can read more about the bureaucratic structure of the FDA that keeps promising treatments out of the hands of terminal patients in this Goldwater Institute report, Dead on Arrival.



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