Rare Disease Advocacy

Specialty Pharmacy Quality Survey

GCA undertook a survey of its community to evaluate patient satisfaction with specialty pharmacies. The valuable insights garnered from the survey offer opportunities for enhancing patient satisfaction and provide input for decision-making for specialty pharmacies. Gaucher patients have many daily struggles and GCA believes that working with specialty pharmacies to improve services can alleviate frustration and stress related to receiving treatment. GCA hopes that our specialty pharmacies will be interested in listening to patients and improving their care. Please read the complete survey results here.

FDA nGD Patient Listening Session

On September 9th, the Gaucher Community Alliance held an FDA Patient Listening Session for the nGD patient community. It was a powerful 90 minutes of testimony from three patient families with neuronopathic Gaucher disease, Dr. Ozlem Goker-Alpan, Tanya Collin-Histed of the International Gaucher Alliance, and Cyndi Frank and Aviva Rosenberg of the Gaucher Community Alliance. FDA representatives from many departments listened to the testimony and had the opportunity to ask questions of the presenters. A summary can be found here.

Gene Therapy 

As gene therapy becomes more of a reality for our community, GCA is working with professional organizations to ensure that accessible, accurate, and responsible information and resources about gene and cell therapy are provided to patients and caregivers. Please review this Patient Education Resource Kit designed by The American Society of Gene and Cell Therapy for Gaucher disease.

Other Advocacy Opportunities