Patients Race the Clock: Where is the FDA? 👀

Janson Silvers  0:00  
Welcome to Health Care Policy Pop. I'm Janson Silvers. It's Tuesday October 31, 2023. Today's pop topics: a bipartisan bill introduced in Congress this summer is gaining some traction. The Promising Pathway Act was introduced by bipartisan groups in both the Senate and the House, and will create a provisional approval pathway for Promising treatments for fatal diseases that progress rapidly and have few or no treatment options. Pam Andrews has two children living with a deadly and ultra rare progressive disease called Niemann-Pick disease type C1. Andrews' oldest child enrolled in a clinical trial in 2016. She was placed on the placebo.

Pam Andrews  0:44  
We can tell pretty early on that Our older daughter was not getting the medicine. My older daughter lost the ability to walk, and was rescued thankfully off of the placebo, and began taking the drug in January of 2017. She is now stabilized and my younger daughter, who is now almost 10 years old, has no symptoms of the disease. They both have been receiving this medicine for nearly eight years.

Janson Silvers  1:13  
Even though the drug has showed remarkable success for this family, it hasn't been FDA approved.

Pam Andrews  1:20  
Rhere were 51 patients that were in the trial, the trial ended up not, I guess the data didn't give the results that the FDA was looking for. And so we have sort of remained in this limbo, where we still get the medicine, but it has not been approved by the FDA.

Janson Silvers  1:42  
Andrew says the FDA approval process is baffling because she sees the success every day.

Pam Andrews  1:48  
For some reason for a fatal neurodegenerative disease that affects children, they want more data and won't approve a drug that clearly is preventing any sort of disease progression in my younger daughter and has stabilized my older daughter, and we face the possibility of losing access.

Janson Silvers  2:07  
Andrews' children have access to the experimental treatment due to an expanded access program also known as Compassionate Use. But two years ago that access was threatened when the manufacturer went bankrupt, and the FDA wanted to terminate the expanded access program. And what would it mean for Andrews and her family to lose access?

Pam Andrews  2:27  
If the medicine is taken away from my children, they will die. That's what it means. And yeah, they will die. They will go downhill rapidly. The disease is progressive, it's neurodegenerative, you know, so just like cancer is progressive. Niemann-Pick type C1 is also progressive, it is always moving, the disease is always progressing.

Janson Silvers  2:53  
Due to patient advocacy efforts and financing from a new manufacturer, the expanded access program remains open, but patients aren't in the clear until there is an FDA approved therapy. Andrew says this disease and many other rare diseases move faster than the FDA. So in her view, it comes down to one question.

Pam Andrews  3:13  
The question is how important are children who have rare diseases? I mean, that's the question. Are they important? And if they are important, and they matter, then changes at the FDA are critical, because without changes at the FDA, then we are not prioritizing and giving these children a chance.

Janson Silvers  3:37  
Andrews and her husband run a nonprofit called the Firefly fund. You can learn more about it in the show notes.

On Thursday, Senator Mike Braun delivered remarks about the Promising Pathway Act to the Special Committee on Aging. He said there are more than 7000 rare progressive and serious diseases known to man, and about 95% of them are without a treatment. Braun said quote, the Promising Pathway Act would create a rolling, real time drug approval pathway to speed access for individuals with rare, progressive and serious diseases. Patients Rising Now, an organization that fights for access, affordability, and transparency of health care, on behalf of the 133 million Americans with chronic disease fully supports the Promising Pathway Act. That's all for today. We're back on Thursday for another Health Care policy Pop, a resource of Patients Rising Now. I'm Janson Silvers, have a great day.