Access barriers loom in Minnesota for blockbuster sickle cell therapy

A fight over insurance coverage is looming for a blockbuster gene therapy that could bring hope and health to Minnesotans with sickle cell disease.

Federal regulators are expected to approve the therapy, called exa-cel, Friday. Insurers will set coverage requirements for its estimated $2 million cost, but University of Minnesota Medical Center leaders are worried that they will decline or skimp on payments for the underlying hospital stays to provide it.

Without funding, the U might be unable to provide the therapy, considering it often comes with a four- to six-week hospital stay, said Dr. Taj Mustapha, chief equity strategy officer for M Health Fairview, which operates the academic medical center. The U is one of the only centers in the Midwest that can provide this gene therapy, and is one of the largest recruiters for a nationwide clinical trial of a competing sickle cell therapy.

"I am so worried that we're just not going to be able to offer this to the vast majority of patients who could benefit," she said.

Exa-cel is set to become the first U.S. therapy based on CRISPR technology that enables laboratories to remove, add or realign human DNA. Treatment involves extraction of stem cells from blood, which are edited and infused back into patients. Hospitals have kept patients admitted during clinical trials of the therapy to monitor for infections or complications.

Minnesota health officials have long been looking for ways to confront the rising toll of sickle cell disease, including a doubling of emergency room visits since 2010. The state screens newborns and finds about 20 to 30 per year with predispositions for the hereditary disease. Black people are more likely to carry the sickle cell gene.

About 1,500 Minnesotans have sickle cell disease, which turns healthy red blood cells into hardened C shapes and reduces their distribution of oxygen throughout the body. The oxygen depletion can lead to early death, organ failure and other problems, and it causes crushing episodes of pain.

"It feels like you got in a bad car accident or someone is literally hitting you with a baseball bat over and over. It's a violent type of pain," said Elizabeth Otunuga, 26, a U student whose lifelong battle with sickle cell disease worsened in her young adult years.

Otunuga took four years off college because of the pain, which she had to manage with opioids. She returned and is studying to become a nurse after being one of the first recipients of a rival gene therapy, called lovo-cel, as part of the U's clinical trial. FDA approval of that therapy is anticipated soon.

"Day by day, the pain would go away" after the transplant, she said. "I would wake up sometimes and be like 'whoa!' "

Spokespeople for Minnesota's three largest insurers said in statements that they will start setting coverage policies for exa-cel after approval by the Food and Drug Administration.

"The goal ... is always to make sure members can access the most effective drugs while keeping healthcare affordable," said David Martinson, a HealthPartners spokesperson.

Coverage by Minnesota's Medical Assistance program will be vital, Mustapha said, because the health care program for the poor and disabled includes a disproportionate share of Black people. Pain from sickle cell disease also drives people out of work, putting them into poverty and qualifying them for public benefits, she added.

M Health Fairview leaders are concerned, in advance of exa-cel's approval, because of belabored negotiations with insurers over other gene therapies. Zynteglo received federal approval in August 2022 for treatment of a rarer blood condition, but the health system is still negotiating a year later with insurers on its coverage, said Dr. Ashish Gupta, a U specialist in pediatric blood and marrow transplants.

"Right now, nobody gets it," he said.

Only a handful of medications exist for sickle cell disease, including hydroxyurea, which can reduce painful episodes. Otunuga said it didn't help her, but it worked for her brother before he died after contracting COVID-19.

Bone marrow transplants also offer a cure for sickle cell disease and have increased the average lifespan for patients if they find donors with matching blood types. The potential advantage with gene therapy, Gupta said, is it uses patients' own cells that are less likely to be rejected by their bodies.

Rising prescription drug costs fueled a 7% increase in per person health care spending last year in Minnesota, according to a report last month by MN Community Measurement. A new class of weight loss drugs fueled much of the increase, and must be taken for life.

The new gene therapies present a different challenge with their million-dollar pricetags. They are considered "curative" after a single treatment, so they are among the most expensive therapeutics in medical history but also can eliminate a lifetime of other medical expenses and increase productivity of people who have sickle cell disease, Gupta said.

"I do see a lot of value in there and I hope insurance companies do see value as well," said Gupta, who predicts the price of the gene therapy will come down as others are approved.

Mustapha said the gene therapies also could address racial inequities, such as higher overall rates of premature death and disability, caused by the lack of treatment options for sickle cell patients, who are predominantly Black.