(This is an op-ed that I am hoping will be published in a newspaper soon, but it feels too important to continue to wait so I wanted to publish it here in the meantime.)

Imagine this: You and your child are in a cold, sterile examination room where motivational posters on the wall preach empty platitudes of encouragement. You’re anxious about getting bad news – and then you do. Your child has a rare genetic disease. Disability is unavoidable. Early death is likely. The doctor delivers the diagnosis with genuine empathy, but still, your world is crumbling.

But the doctor has more to say. There’s a new, FDA-approved treatment that can help limit or reverse the disease’s progression. It can help preserve precious time and quality of life. Do you want to try?

Yes. Of course. Please. Immediately.

Only then to learn that your health insurance company will require you to wait at least a year and a half to start.

That’s the harrowing reality facing Pennsylvanians on Independence Blue Cross health insurance plans. The company recently enacted a policy that automatically denies coverage for 18 months of new treatments outside oncology that the FDA authorizes under its accelerated approval pathway. What a backward, twisted, life-altering, and potentially life-threatening approach from an entity supposed to help people receive necessary health care. The most gutting, ironic part is the FDA uses a program called accelerated approval to deliver therapies as quick as possible to patients who otherwise have no options.

Most people in this scenario don’t have eighteen months to spare. Bureaucracy should never decide their fate. I’ve already lived this reality because we lost our daughter to Krabbe disease at 20 months old. 

Pennsylvanians deserve better. This state is home to many top science and medical experts and several of the country’s best medical facilities. While supporting the development and manufacturing of many incredible treatments, Pennsylvania institutions like the Children’s Hospital of Philadelphia serve local patients and welcome others who seek their expertise from across the country. At CHOP, in particular, specialty care and knowledge about cutting-edge treatments for rare diseases is unrivaled.  

Why should Pennsylvania residents not get to pursue what these expert doctors prescribe and what may be their only option simply because of the specific company that provides their health insurance?

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There’s a phrase often used in rare disease advocacy: ZIP codes determine life or death. A patient’s location tends to dictate their access to timely diagnoses, treatment, and ultimately, hope. And now, an insurer is driving the disparities even further, overstepping the FDA’s permissions to arbitrarily deny Pennsylvanians in need.

Maybe Independence Blue Cross doesn’t understand the rigorous, gold-standard process behind the FDA’s accelerated approvals. Maybe the company doesn’t recognize the value the pathway has provided patients for more than three decades. Or maybe Independence Blue Cross is just oblivious to the desperation and urgency of rare diseases.

Parents are supposed to protect their children, not bury them. I’d be willing to try anything to have been able to save my child’s life. Instead, we were robbed of that opportunity and our daughter died at just 20 months old. 

With over 10,000 rare diseases identified to date, patients and their families who face one of the 5% with an approved treatment can feel oddly fortunate – even more so when a diagnosis happens early enough for intervention. Imagine having hope dangled before you — only for an insurer to sap it. 

Patients or their families should be the sole decision-makers about treatment. It’s our child’s life at stake, not an insurance company’s. If the scientific community confirms a treatment is safe and beneficial, that should be enough for insurers to provide support. The work that I and so many other rare disease advocates do to encourage drug development, to ensure access to approved treatments, and to pursue timely diagnoses is fruitless if insurers like Independence Blue Cross enact blanket policies without room for appeal. 

Rare disease families don’t expect perfection in treatment. We simply want options – no matter where we live. 

Lesa Brackbill is the Associate Director of Advocacy for Patient Advocacy Strategies.