Few leaders have built an “Action Jackson” reputation like Trump. His governing style hinges on identifying a problem, cutting through bureaucracy, and acting decisively. But even the most results-driven leaders have a blind spot — no one can fix a problem he hasn’t clearly seen or fully heard.

Before 2018, no president had taken such decisive action to accelerate treatments for people facing devastating diseases until Trump. By championing and signing the Right to Try Act, which I helped advance from my perch at the White House Office of Legislative Affairs, he made clear his willingness to push for faster access to potentially lifesaving therapies for patients with limited options. 

The gap between need and action is painfully clear in the world of rare diseases. Families across the country are still waiting as the FDA moves slowly on approving treatments for children facing life-threatening conditions. For these families, time is not an abstract concept — it is everything. 

The FDA’s regulatory delays are not merely procedural frustrations for rare disease patients and families. In many homes across America, they are measured in suffering and, in some cases, lost lives.

It was encouraging to see the FDA approve Regeneron’s gene therapy that can restore hearing in children born profoundly deaf. We have also seen movement on psychedelic treatment options for veterans, prompted by high-profile voices like Joe Rogan. In both cases, the right voices reached the right ear.

Now imagine that same attention directed toward rare diseases like Sanfilippo syndrome, Duchenne muscular dystrophy, or Barth syndrome — all of which have had treatments denied or delayed over the last year. The average life expectancy for children with these diseases ranges from age 10 to a maximum of early 20s.

These families are not asking for shortcuts — they are asking to be heard. They bring data, lived experience, and urgency that cannot be replicated in bureaucratic paperwork alone. If those voices reached the executive branch, meaningful change could follow.

Results require awareness. And awareness begins with listening. Is there really anyone who heard the full details about a rare disease and its possible treatments — and saw the children and their families who are suffering while they await bureaucratic movement in Washington — who would still be opposed to quick action on behalf of these families?

Many in the rare disease community have spoken about their appreciation for the Right to Try Act while asking for a personal “right to try,” as the 2018 act excludes rare disease treatments (because it does not require manufacturers to provide or develop personalized therapies for a smaller population of patients).

FDA accelerated approval pathways were created for exactly these short-lifetime disease situations — allowing potentially lifesaving therapies to reach patients faster when urgent medical need outweighed the limitations of traditional approval timelines. Regulators understood that for some diseases, waiting years for perfect datasets could effectively mean denying treatment altogether.

Rare childhood diseases present especially difficult challenges. Patient populations are often extremely small, disease progression can be rapid and irreversible, and conventional clinical end points may fail to capture what meaningful success actually looks like for affected families.

As Dr. Rod Wong explained: “Regulatory flexibility is much easier to come by for drugs where patients improve from baseline, but that is biased against certain disease biologies. [For] some diseases a cure is stopping progression.”

RETURNING PASSENGERS FROM CRUISE SHIP BEING MONITORED FOR HANTAVIRUS IN US

Rare disease patients and families are today’s version of that old Verizon ad, “Can you hear me now?” Let’s hope the president does, as he has an opportunity to extend his unique brand of urgency and leadership to children suffering from rare diseases, where time is critical and the need for action is great.

I know from experience that Trump has the heart and the hand to get this done. 

Paul Teller is president of Teller Strategies. He served four years in the White House during the Trump-Pence administration.