Advocacy
As the only patient advocacy group in the world focusing on LBSL, Cure LBSL has been advocating for the patient voice to be heard with industry and government regulators. We will continue to lead or support engagement with the FDA, including:
Patient Listening Sessions
Cure LBSL has been advocating for the patient voice to be heard with the US Food and Drug Administration (FDA).
Our first engagement with the FDA was a Patient Listening Session on Tuesday, May 30, 2023. This private meeting was not recorded, but we are in the process of creating a detailed report highlighting the important information presented to the FDA. Please check back to view the meeting report.
On February 18, 2023, we held an LBSL Community Meeting to share information about the upcoming FDA Patient Listening Session.
For more information about FDA Patient Listening Sessions, please click below:
PFDD
We are in the process of researching our next engagement with the FDA. This may come in the form of a Patient Focused Drug Development Meeting (PFDD). As with the Patient Listening Session, Cure LBSL will take the lead in this meeting. Unlike the Patient Listening Session, a PFDD is a long public meeting that will be recorded and shared with any interested party.
To learn more about the PFDD, please visit the FDA’s website: https://www.fda.gov/drugs/development-approval-process-drugs/cder-patient-focused-drug-development
CPIM
As our researchers get closer to having treatment ready for clinical trials, they may engage with FDA in a meeting called a CPIM. These Critical Path Innovation Meetings help researcher and FDA plan in advance to ensure that they are on the right track for future clinical trial development. Cure LBSL would offer support to any researchers engaging the FDA in a CPIM or related meeting.
To learn more about the CPIM process, please visit the FDA’s website: https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/critical-path-innovation-meetings-cpim