OPINION:
Last month, Republicans in Congress did something that does not happen often enough in Washington: They acted with urgency on behalf of someone else.
Rep. Diana Harshbarger of Tennessee, Rep. Andy Biggs of Arizona and Sen. Ron Johnson of Wisconsin introduced the Right to Try for Individualized Treatments Act.
This bill would give rare disease patients who have life-threatening or severely debilitating illnesses a chance to access treatments developed for their unique medical needs through a new statutory pathway outside the standard FDA approval process.
Current frameworks are built on larger patient populations with longer lifespans, leaving rare disease treatments running into unnecessary bureaucratic walls.
I was in the White House when President Trump signed the original Right to Try Act in May 2018. That law gave terminal patients access to investigational treatments that had cleared Phase 1 trials but had not run the full FDA gauntlet.
Lives that would have ended were extended, and families got time they would not otherwise have had. It worked because it put patients first and got government bureaucracy out of their way.
Right to Try 2.0 does that for the next generation of medicine. The FDA’s accelerated approval process was designed for the roughly 1 in 10 Americans living with rare diseases, though the program extends beyond rare disease patients alone.
Advocates say all 50 states have movements to codify the right to try. So far, 17 states have enacted similar legislation. That effort has been driven largely by Republican legislators in red and red-leaning states, though expanding options for patients with rare and life-threatening diseases should attract bipartisan support.
Despite state progress, the FDA has dragged its feet over the past 18 months or so, delaying or rejecting a number of treatments for rare diseases, many of which are irreversible once decline begins.
For example, a child with Duchenne muscular dystrophy loses the ability to walk by his early teens. Soon, he loses his ability to breathe. The disease usually claims its victims when they are in their early 20s.
For a child with Sanfilippo syndrome, a few years of apparent normalcy give way to dementia, loss of speech and movement, then death, usually in his teens.
Adults with Huntington’s disease progressively decline physically, emotionally and cognitively, until death around 15 years after symptoms begin.
Innovative treatments could slow or reverse these terrible declines, but over the past year, the FDA has betrayed the principle of right to try. Families watched the agency reject or stall treatments for diseases that have no approved alternatives, reverse prior agreements with drug developers without explanation and apply one-size-fits-all standards to diseases affecting a small population of patients.
Former FDA Commissioner Marty Makary and his former colleague Vinay Prasad, who served as director of the FDA’s Center for Biologics Evaluation and Research,;are no longer with the agency. This provides the Trump administration and Congress with an opportunity to install new leaders who understand that rare disease patients have loving family members and advocacy groups — but no time.
Through Right to Try 2.0, Republicans can turn back the clock on disease progression. The FDA needs codified patient-first processes that include patient voices as a standard.
When releasing Right to Try 2.0, Ms. Harshbarger noted, “When someone is fighting for their life, the last thing they need is the government standing in their way.” Nothing could be more right, nor more universally accepted.
Americans love children — everybody’s children. We also care deeply about human suffering. As we celebrate America’s 250th birthday, we are reminded that “life, liberty, and the pursuit of happiness” applies to everyone, large and small.
Winning the war against rare diseases takes political courage — the kind of leadership we see with state and federal Right to Try 2.0 legislation.
America’s 15 million children with rare diseases do not vote, but their families and caregivers do — millions of them. They will remember who stood up for them. Republicans who get behind Right to Try 2.0 will find those families to be fierce and loyal allies in November.
To be clear, this is not just about politics. Saving children is a good policy and the right thing to do. Voters need to remember who is doing it.
Congress should pass Right to Try 2.0 and show the country that the Republican Party is the party that fights for children.
• Paul Teller is president of Teller Strategies. He served in the White House for all four years of the Trump-Pence administration.
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