The most recent illustration of the strength of patient advocacy is the FDA’s approval of new ALS medicine, which was developed with $2 million in support from an “Ice Bucket Challenge.” But there is still work to be done. In a new edition of the I am BIO Podcast released today, two patient activists discuss the impact of their determined efforts to support the development of medications for rare diseases.
“The minute my daughter was born, I looked at my mom and said, ‘That’s it. She’s not going to suffer the way that you and I have,’” Susan Ruediger, Chief Mission Officer of the CMT Research Foundation, tells the podcast. She established the foundation to combat Charcot-Marie-Tooth (CMT) disease, a hereditary neurological condition that affects 3 million people worldwide, including 18 members of her family right now.
The CMT Research Foundation is collaborating with a small firm and made an initial $125,000 investment during the 2019 BIO International Convention, thanks to connections made through BIO’s One-on-One Partnering™ platform. Since then, the firm has raised $100 million, and the CMT Research Foundation has given money to 15 development projects over four years, Two of those projects are in the clinical development stage, and one of them is undergoing a Phase 3 trial.
Along with limited drug development, another obstacle to dealing with rare diseases is general knowledge about the diseases, according to Nasha Fitter, co-founder, and CEO of the FOXG1 Research Foundation and mother of a child who suffers from FOXG1, a crippling hereditary neurological illness. There is “a lack of overall understanding of the natural progression of this disease,” says Fitter.
Wondering “how can we use tech to solve this problem,” Fitter says she used her experience in ed tech to create natural history research with 100 patients and more than 10 years of data in only one year, after attempts to hold studies of the disease failed to attract the required enrolment. Patients are now being enrolled in the first clinical study that is specifically for FOXG1, at NYU.
“As patients, especially with a progressive disease, we don’t have time to waste, we don’t have money to waste,” says Ruediger. “Our job is to make it easy for the drug developers, the drug hunters, to be attracted to our disease.”
Listen to the full episode The Surprising Role of Patient Advocates in Drug Development
Join patient advocates in Washington, D.C., next week! The BIO Patient and Health Advocacy Summit returns to an in-person event from October 27-28 in Washington, D.C.—learn more and register now!
