California bill would stop insurers from blocking rare disease care

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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.

Jordan is 20 now. She is a joyful child who loves going on outings with her service dog, Angus. Somewhere in a lab right now, people are working on a treatment that may help her and the roughly 500 other children who share her diagnosis. We believe it is coming. And we refuse to accept that when it arrives, the thing standing between our daughter and it will be a queue.

My work has brought me together with other rare disease families who’ve faced many of the same roadblocks. It’s time for one of the worst to be eliminated: insurance companies requiring prior authorization before covering a rare disease treatment.

Rare disease touches an estimated one in 10 Americans. For children, the stakes of delay are stark: an estimated 30% of children with a rare disease won’t live to see their 5th birthday for lack of timely access to treatment.

When it comes to rare diseases, prior authorization is an inhumane cost-control tool. For these patients, a specialist’s prescription is often the only effective option available. Forcing a patient to “try and fail” on a treatment the specialist already knows won’t work isn’t caution, it’s a dangerous detour. 

Insurers and third-party reviewers who evaluate these requests frequently lack expertise in the rare conditions leading to reversed denials, sometimes only after a hospitalization, a permanent disability or worse. The American Medical Association’s own physician survey has documented the harm of this practice for years.

I’ve spent recent months working alongside legislators, patient advocates, and researchers who’ve had enough. Assembly Bill 1887, authored by the chair of the Rare Disease Caucus, Assemblymember Rick Zbur, would eliminate prior authorization and step therapy requirements for FDA-approved rare disease treatments when a specialist has determined the treatment is medically necessary. It’s a meaningful solution for patients with the fewest alternatives and the least time to spare, and it will undoubtedly save lives.

The Assembly passed the bill unanimously. The Senate followed suit in August.

California has built an unparalleled biotech research ecosystem. Now patients need access to what our researchers discover. I’ve rarely asked lawmakers for something as straightforward as this: When a specialist prescribes an FDA-approved treatment for the rare disease it was built for, let the patient have it. That’s why I’m urging Gov. Gavin Newsom to sign AB 1887 into law.

I still hope, every day, that better treatment options will improve Jordan’s life. Until then, no rare disease patient should have to wait on an insurance company’s paperwork for the treatment their own doctor already prescribed.

Joe and Cynthia Lang are the founders of Jordan’s Guardian Angels, a Sacramento-based foundation funding research into rare genetic conditions.