In the months of his second term, President Donald Trump’s top health officials were clear.
They promise to focus on “treatment” and “meaningful treatment.”
They wanted to develop revolutionary medicine in the United States.
They did not want the drugs of the future to come from China.
But about a new ideological shift at the Food and Drug Administration threatens to undermine these worthy goals — from within.
Patients are frustrated, and wait a long time for basic treatment.
Biotechnology companies are confused.
The spirit of the “right to try” philosophy of Trump’s first term has hit a wall of consistency for the second time.
A “rare” disease is one that affects fewer than 200,000 people in the United States – but you may know someone who does.
An estimated 30 million Americans have one or more rare diseases, 95% of which have no FDA-approved treatment.
I run the non-profit Alliance for Regenerative Medicine.
We represent biotech companies, research institutions and patients developing advanced therapies called cell and gene therapies, or CGTs.
This is Rare Disease Week, an international celebration aimed at celebrating the patients we help and the amazing science we can now use to attack diseases at their source – often with a single dose.
Over the past decade, the FDA has approved a dozen CGTs that have saved or changed the lives of thousands of people living with rare diseases who have faced death or serious disability.
At least 900 ongoing clinical trials in the United States are testing some of these powerful drugs: Each one represents great hope for millions of other patients and their families – not only for those suffering from rare diseases, but also common ones such as heart disease, Parkinson’s disease and diabetes.
Turning this revolutionary science into an FDA-approved drug takes years and hundreds of millions of dollars.
China is racing to overtake the US as the world leader in this revolutionary area of biotechnology.
Let’s go back to Trump’s vision: Last year, in an unprecedented show of support, his top health officials announced that they want America to remain a global leader in biotech.
Senior management leaders convened managers, scientists and patient groups to plan a positive vision to accelerate the development of these important medicines.
But in the past four months, the FDA has surprisingly rejected several promising CGT drugs.
Yes, obstacles do happen in drug development.
The FDA’s new and unusual behavior is harmful – both to patients and to Trump’s opinion.
In one of these rejections, the FDA changed the approval requirements seemingly at the last minute, reversing commitments it had made months earlier to bring these drugs forward.
The parallels with the agency’s initial rejection of Moderna’s flu vaccine this month are striking.
The FDA told Moderna it was okay with its clinical trial design.
However, it initially refused to even consider the company’s application, saying the design was not suitable.
Fortunately for Moderna, and those looking to protect themselves from the flu next winter, the agency has withdrawn its studies and will now review Moderna’s evidence.
But the thousands of patients, many of them children, waiting for CGTs from biotech firms such as Atara Biotherapeutics, uniQure, Ultragenyx and REGENXBIO still have no clear way forward.
They don’t have seven months until the next flu season – because for these patients with rare diseases, time is of the essence.
For them, time is life. . . or death.
We urgently need common sense training at the FDA that puts patients first.
First, the agency must honor the commitments it made to companies and patients earlier about what evidence would be acceptable for approval.
Second, it can further confirm strong indications that these drugs are working by collecting more data from patients after authorizing treatments.
Finally, it can assemble a panel of outside experts, known as an FDA Advisory Committee, to openly review these drugs and listen to patient testimony.
These practical steps put patients first, and radical ideas come a distant second.
The FDA has routinely used these methods in approving dozens of CGTs over the past decade, including during the first Trump administration.
The president needs an FDA that lives up to its responsibilities and uses his vision for the future of medicine.
We can help thousands of rare disease patients right now – and millions more in the future – with the FDA following his call.
Tim Hunt is the CEO of the Alliance for Regenerative Medicine and is the father of a young man with a rare disease.
