Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) are a group of rare conditions that impair the body’s metabolism from breaking down certain fats from food into energy. Although there are an estimated 2,000-3,5000 people living with LC-FAOD in the U.S., researchers have limited data on how LC-FAOD progresses overtime and how it is managed in the real world.
This is why Ultragenyx Pharmaceutical and PicnicHealth are partnering on LC-FAOD Odyssey, a new type of patient-first research that uses the anonymous data from medical records to better understand the day-to-day challenges of living with LC-FAOD. Understanding how a disease is managed and treated and the effects of these approaches in the real-world, combined with understanding individual experiences based on patient-reported surveys, can help address some important questions that could improve the future quality of care.
Ultragenyx and PicnicHealth recently presented preliminary data at the International Network for Fatty Acid Oxidation Research and Management (INFORM) LC-FAOD conference (October 27 - 28, 2021). From August 2020 - August 2021, thirty three (33) patients enrolled into the study. Some of the data is summarized below, and the full poster can be found here https://informnetwork.org/inform-families/:
These preliminary results from the LC-FAOD Odyssey demonstrate the power of linking patient medical records and patient reported outcomes. The data helps researchers understand how complex LC-FAOD care is, with multiple providers, care sites, and management strategies.
"I founded PicnicHealth after managing my own Crohn's Disease diagnosis," said Noga Leviner, co-founder and CEO of PicnicHealth. "We quickly realized we were actually solving a much bigger problem by turning each patient's messy, nonuniform medical records into structured data. The result not only helps patients directly but it also really moves the needle for research - which is demonstrated by these early results from LC-FAOD Odyssey."
“We’re excited to see these early results from LC-FAOD Odyssey that showcase the power of taking a patient-centric approach to LC-FAOD research,” said Nina Thomas, Vice President of Global HEOR & Epidemiology at Ultragenyx Pharmaceutical.
Moving forward, the LC-FAOD Odyssey team is looking for additional volunteers living with LC-FAOD to join the study and contribute their anonymized data to research. By working together, patients, Ultragenyx, and PicnicHealth can help change the future of LC-FAOD research.