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Rare Disease Foundation Launches #Ask100Patients Initiative
By: Saving Case & Friends, Inc.
Sponsor of the #Ask100Patients initiative, Saving Case & Friends is a nonprofit dedicated to supporting a cure for the ultra rare disease Hunter Syndrome. The announcement comes as the organization’
“How do you explain why some clinical trials enroll quickly and others sit stagnant for months, or worse, years?” she asked the audience of pharmaceutical and biotech executives, think tank representatives, and patient advocates. She then went on to detail how her journey over the last five years in a clinical trial with her son had changed her perspective on drug development and her role in the process.
Hogan's 8-year-old son Case suffers from Hunter Syndrome or MPS II, which affects approximately 2,000 children worldwide, almost exclusively boys. Case inspired her, along with the parents of several other children with Hunter Syndrome, to form the nonprofit Saving Case & Friends. Hunter Syndrome typically causes progressive loss of all skills, including speech, cognition, walking, and eating, with an average life span of 12-15 years old.
Although Case enrolled in a clinical trial in 2010, most children with the disease did not qualify for the trial or even for another one that opened up more recently. The frustration from that experience inspired Hogan to become involved as a Patient Representative with the Food and Drug Administration, which oversees clinical trials, as well as advocating to the drug companies themselves.
Hogan hopes that the #Ask100Patients initiative will grow, not only with rare disease communities like Hunter Syndrome, but also in broader disease communities like breast cancer that may have more sway in encouraging industry to engage with patients sooner in the drug development process. Social media images encourage companies to ask 100 patients how their clinical trial could be better, what endpoints impact patients’ daily lives, and how their trial could enroll more patients.
Not to leave it to chance, Saving Case & Friends also funds its own research in an effort to further drug development in Hunter Syndrome. Earlier this year, they launched a social media campaign called Project Alive (http://projectalive.org) to raise funds for research. It features typical children sharing what they want to be when they grow up, contrasted with boys with Hunter Syndrome, including Hogan’s son Case, who just want to be alive when they grow up.
Saving Case & Friends, Inc.
Melissa Hogan, Preside