For those trying to better understand gene therapy, read Maria Kefalas’ post below. A Lenmeldy/Libmeldy patient: “Giovanni is now 15 years old. His friends and teachers have no idea he has MLD.” And for employers, especially self-funded employers opting not to cover via Stoploss/Reinsurance, consider the costs of not covering (besides the obvious moral ones): “Cal [patient who did not have access to Lenmeldy/Libmeldy owing to timing] had 1712 confirmed provider contacts over a ten year period: 194 blood tests, 42 x-rays, 22 ER visits, 16 admissions (including 2 PICU stays ), 11 ultrasounds, and two MRIs.” This, all pales in comparison to the mental anguish of losing a child. Not covering gene therapy, as Mosaic ‘Life Care’ attempted to do in St. Louis, Missouri last month, could lead to a two class system where employees’ children will pass because employers opted to employ excuses not to cover vs. tactics to ensure risk mitigation. #mosaiclifecare #zolgensma #genetherapy #orchard #lenmeldy #libmeldy #kirowakiran
Professor at Saint Joseph's University, Executive Director @TheCalliopeJoyFoundation and @Cure_MLD. Author @BeaconPress
It was an honor to represent the MLD community at the recent in-person ACHDNC hearings at the Department of Health and Human Services to advocate for the inclusion of MLD on the US Recommended Uniform Screening Panel. Thanks to Joan Keutzer for this photo! Here is my public comment: My name is Maria Kefalas and I am the Executive Director of Cure MLD. As a parent advocate since 2013, I am here to state that MLD has met the requirements for inclusion in RUSP: an effective assay, an FDA approved gene therapy, and a standard of care that has already been successfully used in the EU and the UK. Lenmeldy is widely viewed to be one of the most transformative gene therapies in the world. The oldest US patient received gene therapy in the original clinical trial back in 2009, Giovanni is now 15 years old. His friends and teachers have no idea he has MLD. When Giovanni provided his testimony to the FDA, he stated, “I guess gene therapy works, because I would be dead.” MLD is a devastating disease that causes tremendous suffering for the child and taxes the health care system. According to my late daughter's doctors, Cal had 1712 confirmed provider contacts over a ten year period: 194 blood tests, 42 x-rays, 22 ER visits, 16 admissions (including 2 PICU stays ), 11 ultrasounds, and two MRIs. I entreat the ACHDNC to move as quickly as possible to include MLD on the RUSP. Of the dozens of condolences I received after Cal died, a message from Michael H Gelb, the scientist who invented the MLD newborn screening assay rises above the rest. His message read “I am sorry we could not save Cal.” It is too late for Cal and so many others, and it is in your power to give our children a different ending to their stories. It is intolerable to tell newly diagnosed families that we have an FDA approved therapy that we can’t use because there is no newborn screening.
Thank you for sharing this powerful insight, Courtney. The impact of gene therapy on individuals and families is truly profound, and your perspective on the potential consequences of not covering gene therapy is thought-provoking. It's a reminder of the far-reaching implications of healthcare decisions.
Health Policy Regulatory and Legislative Expertise; Market Innovator
6moI like how you said tactics for risk mitigation. Working out the reinsurance angle seems key. Even perhaps the creation of a private fund to pay reins premiums for small employers who offer insurance. At one time the Ryan White centers were paying premiums so there is precedent. Just thinking out loud.