Missouri, give sickle cell disease patients a better chance with new gene therapies | Opinion

As a mother to a sickle cell disease patient, I’ve seen it all when it comes to this devastating — not to mention historically overlooked and underfunded — disease. My daughter Ronicia, whom we lovingly called “Ro,” passed away living with sickle cell disease four years ago while being treated for a pain episode in the hospital. She was only 29.

Last December, two gene therapies were approved that are giving sickle cell disease patients renewed hope. These new options represent a previously unthinkable chance at a healthy life for those living with this disease.

However, last week, Missouri’s Advisory Council on Rare Diseases and Personalized Medicine made a grave mistake to recommend that the state’s Medicaid agency add only one of these new Food and Drug Administration-approved gene therapies to the state’s preferred drug list. As a mother who lost their child to sickle cell disease, it’s heartbreaking that the committee failed to consider taking the decision to a vote. The unfavorable outcome could have detrimental repercussions for a patient community that has long awaited meaningful new treatment options.

The final decision around Medicaid’s coverage of these new treatments will come when Missouri’s Drug Utilization Review Board meets this week. The physicians and pharmacists on this board need to do right by the approximately 2,000 Missourians living with sickle cell disease and ensure patients can access both treatment options without undue burden.

While the committee expressed concerns about the costs of these treatments, I know too well the cost of not treating the disease. For Ro, living with sickle cell disease was an unpredictable roller coaster with unimaginable hurdles. She frequently experienced pain episodes so intense that she was hospitalized for days or sometimes weeks and missed out on school and important life events.

Beyond the impact to one’s health, the lifetime cost of treating a patient with sickle cell disease can be $4 to $6 million — despite a median age of death of only 45 years old. And that doesn’t even include the out-of-pocket costs a patient incurs or the financial impact on caregivers.

As executive director of Sickle Cell Association, I’ve also heard countless stories of individuals in Missouri living with sickle cell disease, their families and caregivers forgoing their hopes and dreams because they don’t have the time, freedom or financial means to pursue them fully.

At this critical juncture, I strongly urge Missouri’s Medicaid agency and Drug Utilization Review Board to step up for the sickle cell disease community by keeping clinical decision-making in the hands of people living with this disease and their health care providers.

These treatments could have saved Ro’s life. I hope they can save someone else’s.

Rosemary Britts is the executive director of Sickle Cell Association, a St. Louis-based 501(c)(3) nonprofit, which she founded in November 2011. It does not lobby. She has a master’s of business administration from the University of Phoenix.