Powerful Patient Stories Make Sparks Fly for FDA Advisory Committee
On October 12, World Sight Day, a Food and Drug Administration (FDA) advisory committee voted unanimously 16-0 in favor of recommending the approval of Luxturna, a gene therapy developed by Spark Therapeutics for the treatment of a rare (and currently untreatable) form of inherited blindness.
FDA advisory committees are conducted to provide independent advice to the FDA on decisions related to the approval of new medications. The meetings typically include a public comment period in which the patient community and other stakeholders address the committee.
The voice of patients can have extreme sway when it comes to severe and untreatable conditions
This vote for Spark was significant – not only because Luxturna may become the first gene therapy approved in the U.S. for an inheritable disease, but because it reinforced the power of the public comment period. During this time, the advocacy community came out in force to move the committee toward a favorable vote, telling emotional stories of finally living in vibrant color, after a life of black and white film. This, coupled with compelling visual evidence of the treatment’s effects, made a strong case for the committee.
The voice of patients can have extreme sway when it comes to severe and untreatable conditions, and Spark made the most of this.
Notably, Spark’s meeting followed the FDA’s inaugural Patient Engagement Advisory Committee. This is the first FDA committee consisting only of patients, caregivers and patient advocates. While patients have always had a voice at the FDA, this marks a formal effort to further integrate patient perspectives into the drug development process.
Patients continue to have a powerful platform to speak and share, and as health communicators, we have tremendous opportunity to help them tell their stories effectively. These stories not only have the potential to inform, but to influence and inspire – helping push forward a new era of modern medicine.
Megan Mileusnic is an Account Coordinator at JPA Health.