Managing Director, Policy, Patient Support and Industry Liaison, The Isaac Foundation
Talk title – Early Access Programs: collaborating, listening and educating effectively with patient groups
Bio: Alix’s work at The Isaac Foundation and Equal Access for Rare Disorders focuses on advocating for patients’ safe and ethical access to treatment, and managing patient education and rare disease awareness programs. She oversees the Patient Support Program, providing individualized support to patients and their families. She has a background in education, community engagement and outreach.
Find out more about The Isaac Foundation by visiting their website.
Q. This will be CamRARE’s 5th summit. What is the best/most impactful/most promising change you’ve seen happen in rare disease progress in the last 5 years?
The rate at which potentially curative treatments are being developed for rare diseases is incredible. Two gene therapy projects that we have been funding over the years are either in, or heading into, clinical trial. Likewise, the fact that Expanded Access Programs are slowly becoming more commonplace is tremendously important to patients currently experiencing an unmet need for treatment. We hope these programs continue to become the norm, rather than the exception.
Q. How has your work impacted rare disease progress over the last 5 years?
In addition, to support research into potential cures for rare diseases, including MPS II and MPS VI, our education and support programs for patients are core to the work we do.
Q. What next? What do you want to see happen or change in the next 5 years that will have the most impact on diagnostics, care or treatment for those affected by rare conditions?
The COVID-19 pandemic has been proof that it is possible to think outside of the box to meet the needs of patients. From remote clinical trials, to virtual appointments, we must continue to improve accessibility – at every level – for patients.