This petition request hit my email this morning, for a rare disease I'd never heard of - Niemann Pick Type C (NPC). We need a re-tooled FDA.
----------------------------------
(boldface mine)
We need your help to save our kids fighting fatal NPC.
We are patients and parents of children and adults living with an ultra-rare and fatal disease called Niemann Pick Type C (NPC). We are in a fight for our lives to get new therapies approved by the US FDA, and we need your help. The clock is ticking, and every day that goes by NPC is damaging us and our children.
NPC is like Alzheimers, Parkinson’s and ALS rolled into one, except even more rare, causing extreme declines in how we move, talk, think, and even eat - pretty much everything. This disease occurs across all ages - and, horrifically, will frequently first show up in young children, even babies.
There is no cure or approved therapy here in the United States...but there are several medicines that have been in development and used experimentally by patients for years and years. Patients AND our treating doctors see the tremendous benefits of these medicines in helping us keep the ability to walk, talk, play and swallow. This is a BIG deal. It’s life saving.
Heartbreakingly, however, the FDA has become a barrier to helping these therapies cross the finish line - requiring multiple placebo control trials, not listening to our expert clinicians, discounting patient benefits, and not understanding our ultra rare variable and fatal disease. And we have had enough. It is time for regulators to:
Think differently about treatments for NPC and the multiple clinical trials that have demonstrated how these therapies have benefits that outweigh their risks;
Listen to patients and families about their needs; and
Act on expert guidance from doctors and researchers across the country who have studied this disease and treated patients for decades and recommend approval of investigational therapies.
Our community has met many times with the decision makers at FDA over the years to discuss the desperate need for approved therapies - including as recently as August 3, 2021. And while government officials say they “hear us,” there is no sign of movement or action to advance our cause. In fact, FDA has tried to keep new patients from accessing these experimental medications by erecting hurdles to early access programs and denying requests for approvals.
We need the following changes to occur immediately:
The FDA must lift all barriers that have blocked new patients from participating in programs that allow access to experimental medications, or have created uncertainties for patients currently receiving these medications. This includes removing any barriers to receiving drugs known as adrabetadex and arimoclomol.
The FDA must evaluate medications to treat NPC with the knowledge that NPC is ultra-rare, degenerative and fatal, and use regulatory flexibility when reviewing clinical trial data that shows meaningful improvements, particularly given the known natural history of this fatal disease.
The FDA must work with the scientific and patient community to design trials that are feasible and meaningful and that the trials do not put patients in harm’s way.
Add your signature to tell the FDA that it must address the needs of those with NPC by approving medicines and increasing access to existing medicines. Please tell FDA, don’t give up on NPC - for the patients of tomorrow AND those alive today. Please help save our lives. Thank you!!
