“So, the question families are asking is fair: Whatever happened to Right to Try?” Right to Try was a promise. It’s time for the FDA to keep it.

Across this country, mothers of children with rare diseases watch their children lose the milestone moments of childhood: tying their shoes, lifting a fork, climbing a single step. They aren’t asking for miracles. They are asking for more time.

For thousands of families, rare diseases are a daily reality that progressively and irreversibly steal abilities from children one by one, and don’t give them back. What these diseases share is particularly cruel: few or no approved treatments, and a risk that some of these therapies could be taken away.

Families continue to fight.

Earlier this year, more than 100 families carried a coffin to the Food and Drug Administration’s doorstep in a symbolic funeral march, protesting regulatory delays affecting children with Sanfilippo, Hunter and Hurler syndromes. Three therapies developed for Hunter syndrome have been denied or delayed, with families arguing that the FDA’s resistance to accelerated approval pathways is costing children time they cannot get back. Their message is simple: When time is measured in lost function, delays have consequences.

In the country of Georgia, at the intersection of Europe and Asia, families of boys with Duchenne muscular dystrophy have spent 500 days and nights protesting in the streets, demanding access to these drugs. We are watching, and we want the FDA to know: if it comes to it, American moms will show up too.

Boys with Duchenne muscular dystrophy are missing a section of DNA, and without it, the body can’t make the protein it needs. Exon-skipping therapies teach the body to work around the damaged region, like skipping a broken step on a staircase, so it can begin producing dystrophin again. Not all of it, but some. This slows the damage and buys these boys time. Time to play, grow, learn, and be with their families.

These Duchenne muscular dystrophy drugs are available today, and the FDA has an opportunity to consider the full body of evidence supporting their continued use. Several have been used for more than a decade. One has data from more than 1,800 children. One drug pushed back the need for a breathing machine by seven and a half years. Another slowed the loss of lung function. These are not small things.

Yet the FDA is holding back. One clinical trial didn’t meet a specific endpoint – how fast kids could climb four stairs. We understand the need for standards. We also know what these families have lived. We’re asking the people making this decision to look at the whole picture, not just one number on one page.

President Trump understood this reality when he championed Right to Try, and Congress backed him up. The Right to Try Act, signed in May 2018, made a clear promise: the federal government would not stand between a terminally ill patient and an investigational therapy when all other options were exhausted. Under the law, eligible patients can request access to investigational drugs directly from manufacturers. Those manufacturers are shielded from liability when acting in good faith. The FDA is prohibited from using Right to Try data to delay or deny approval. And insurers cannot be penalized for covering a patient who pursues it.

These provisions were a direct rebuke to bureaucratic delay. They recognized that for a child losing function by the day, waiting for more data is not a neutral act. It is a decision with consequences.

So, the question families are asking is fair: Whatever happened to Right to Try?

That law’s promise; that patients deserve access, that families deserve agency, that government should not be the last obstacle between a sick child and a treatment that might help, should be animating every FDA decision on rare and progressive disease. Yet here we are, watching families march with coffins while children deteriorate and regulators debate procedural thresholds.

A mother is not thinking about a four-stair speed test. A mother is thinking about whether her son will be able to feed himself next year, or whether he will still be walking at his next birthday.

Mothers make decisions based on what their children need. A child who gets one more year of walking, one more year of independence, does not benefit from something statistically insignificant. He got more out of his life. And to his mother, there is nothing more significant than that.

Right to Try was a promise. It’s time for the FDA to keep it.

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Article by Raquel Mitchell
Deputy Director of Moms for America Action.
Article original written for InsideSources.com and published by DC Journal

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