By IGT Patient Advocacy Advisory Council Member and Huntington's Disease Patient Advocate Seth Rotberg
As we enter the third year of the COVID-19 pandemic, the tried-and-true Food and Drug Administration (FDA) review and approval process remains in the spotlight - having helped bring us critical vaccines and treatments for this evolving virus. The public now more than ever understands the need for safe and effective treatments – as well as the urgency. As a patient with a rare disease, this has been top-of-mind for me for years.
And yet, some continue to challenge one critical FDA tool for making new therapies available to patients who don’t have treatment options. It’s called the accelerated approval pathway. Not only does accelerated approval provide a potential opportunity for the patient, but it can help our overall healthcare system save on medical expenses over time. For example, the Partnership To Fight Chronic Disease recently highlighted that neurodegenerative diseases costs are over $655 billion a year in medical expenses and economic losses. By getting new and potential treatment options to patients who need them most, we could cut costs for care in the long run.
One of those neurodegenerative diseases is Huntington’s Disease (HD) which slowly deteriorates a person’s physical and cognitive abilities over 10-20 years. Some describe HD as having ALS, Alzheimer’s, and Parkinson’s all at once. I witnessed my mom’s digression of HD for more than 17 years before she lost her battle in March 2015. But the battle isn’t over yet for our family – I tested positive 11 years ago when I was only 20 years old, and I am guaranteed to have HD one day.
There are no effective treatment options to slow down or halt HD in its tracks. Even worse, I do not qualify for any clinical trials since I am still considered pre-symptomatic. Based on therapies available today, it will be too late to treat me by the time I get sick. This is why it’s critical that we support research into innovative ways to treat diseases like HD.
One promising area of research is looking at different biomarkers that can help measure whether a potential treatment is working. We have a ton of data through studies such as PREDICT-HD, Track-HD, and Enroll-HD that demonstrate changes in the brain occur years before clinical onset. Knowing and applying this data to trials can offer new hope to the community. Additionally, it could help us treat HD earlier on, which would bring down the medical expenses and economic losses that HD families are currently facing.
The accelerated approval pathway is an essential option for making progress where there currently are no treatment options. By considering surrogate endpoints – like changes in the brain in the case of HD – the FDA has a better opportunity to help patients with rare diseases. Studies can be conducted faster (crucial for the many patients that don’t have the luxury of time), which, in turn, allows for FDA approval sooner while maintaining its strict scientific standards.
Even more exciting for many more rare disease patients, is the promise of gene therapies. These treatments target the cause of genetic diseases directly to create a potentially long-lasting change in the body. The possibilities are incredible. The FDA has indicated that the accelerated approval pathway may be an appropriate avenue for gene therapies. This is something we all must advocate for.
I’ve been a member of the HD community for 16 years without an effective treatment option. Advancing science and the accelerated approval pathway give patients, like myself, hope that we can change that reality.
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