After baby Eli was born, his family received the devastating news that he had a fatal type of spinal muscular atrophy.
With a disease that damages the motor nerve cells in the spinal cord, ultimately taking away the ability to eat and breathe, Eli was not expected to live long enough to celebrate his first birthday.
Eli, however, is now 2 years old. He talks and sings and, with the help of a walker, can walk.
He was among a fortunate group of 15 children who gained access to a clinical trial. While Eli received a partial dose of the investigational treatment used on the group, the infants who received full-dose treatments are not showing any disease symptoms today.
Terminal patients like Eli don’t have time to wait for the bureaucratic sanctioning of promising new treatments, which take, on average, more than a decade to obtain Food and Drug Administration approval.
That is why a federal version of what’s called a right-to-try law is needed. Under these laws, terminally ill patients under the care of a doctor who has exhausted other treatment options, are permitted to seek treatments that have successfully completed basic safety testing on humans in Phase 1 FDA trials and are continuing on the path to full FDA approval.
Such laws are now on the books in 33 states and under consideration in the other 17. Terminal patients understand that these laws don’t offer guarantees; but they do offer hope.
A Texas oncologist told a U.S. Senate committee last year he had already treated 78 patients under Texas’ right-to-try law. Some had only three months to live when they began treatment. Today, months later, most are doing very well.
Unfortunately, there is fear in the medical and manufacturing communities that the FDA could retaliate against those using these laws.
That is why a federal right-to-try law is needed. A federal law would, simply, require the federal government to abide by the state right-to-try laws.
Some opponents fear such laws are too risky. But these laws work in tandem with the human safety requirement used by the FDA. If the treatment is safe enough to use on others in clinical trials, why isn’t it safe enough for a terminal patient under their own doctor’s care?
Others fear that manufacturers could use such laws to sell their treatments. But federal law already makes it illegal to provide investigational treatments for more than the actual documented cost.
In reality, most manufacturers that participate elect to provide the treatment for free because they are reluctant to make their cost information public.
Other critics point out that the FDA’s compassionate use program, which is supposed to give patients access to investigational treatments, already provides such access to terminal patients. But the FDA granted fewer than 1,300 such requests in 2015.
That same year, more than 1.3 million died from the three leading disease killers: heart disease, cancer and chronic obstructive pulmonary disease. That does not include other devastating diseases, such as Alzheimer’s, Lou Gehrig’s disease and Parkinson’s.
Something is clearly amiss in a system that is so bureaucratic and time-consuming that fewer than one-tenth of 1 percent of terminal patients can take advantage of the FDA’s compassionate use exception.
Federal right-to-try legislation would make compassionate use the rule not the exception in all 50 states — not just the 33 that already have passed it.
No one should be forced to beg the federal government for permission to try to save their own lives. Families like Eli’s or maybe even yours are why we need a federal right-to-try law.
Naomi Lopez Bauman is director of health care policy at the Goldwater Institute, a conservative nonprofit group that works to defend and strengthen constitutional guarantees at the federal and state level. Readers may write her at Goldwater Institute, 500 E. Coronado Road, Phoenix, AZ, 85004.