Opinion: California bill would stop insurers from blocking r...

OPINION — A patient spends years looking for a diagnosis, searching from specialist to specialist, hoping someone will finally recognize what they're seeing. For someone with a rare disease, that search alone takes an average of seven to eight years. When the diagnosis finally comes, so does the hope of treatment: A doctor can assess the options, prescribe one and begin care. That should be the end of the story. For too many California families, it's only the beginning of a new fight: a prior authorization request, a denial, an appeal, sometimes a forced trial with a drug the specialist already knows won't work. For rare disease patients, especially children with rapidly progressing diseases, those delays aren't paperwork. They can mean the difference between a child who walks, breathes independently or reaches school age, and one who doesn't. I've spent decades in the California Capitol advocating for other people's priorities. When my daughter Jordan was born with an ultra-rare genetic disorder, my priority became advocating for her. Doctors traced the disorder to a mutation on a single gene, PPP2R5D. We were told she was the first, but we had nothing more — no name, no specialist who had seen it before, no support group, no protocol, no chapter in any book, no other parent anywhere on Earth I could call to ask how to care for our daughter. After Jordan's diagnosis, my wife, Cynthia, and I started a foundation, Jordan's Guardian Angels, and built a research program from nothing. With the support of legislators who understood that rare disease research can teach us about far more common conditions too, doctors at 10 universities and hospitals were able to collaborate instead of competing for scraps of grant funding.