Maisie Forrest’s mother wouldn’t take no for an answer, and that’s basically the story of how the Grand Junction baby got a $2.1 million drug to save her life.

Maisie wasn’t expected to make it to her second birthday when the miracle drug, called Zolgensma, was approved last May. It was a one-time injection, a drug developed at high cost that would work only on a few thousand people, one that had the power to stop the rapid degeneration of spinal muscular atrophy that was destroying Maisie’s muscles and her ability to breathe.

The girl’s insurance company initially said no. But Maisie’s mother, Ciji Green, took to social media and created a persistent and loud group of 700 friends and strangers who call themselves Maisie’s Army. She also looked the insurance company executives in the eyes and told them the only way they would end up spending less than $2.1 million on Maisie is if they were planning on her dying. She won on appeal.

But in Maisie’s story, her mom says, the drug company is the hero.

In the era of specialized medicine, when researchers are using gene sequencing to create drugs that work only for a rare few — or just one person, as in the case of another Colorado girl named Mila Makovec — that version of the story is becoming more familiar. For families who’ve been waiting on life-saving treatments, the target for reform is not Big Pharma, but the insurance industry.

Ciji Green holds her daughter, Maisie, in a hospital bed. The girl received a life-saving, $2.1 million drug treatment in July after her mother appealed to their insurance company. (Provided by Ciji Green)

Green praised AveXis, the company that developed Zolgensma, for spending the money and time to create a drug that saved Maisie’s life. It makes sense to her that the cost is high because the market is tiny.

“I can’t tell you how grateful our community is for these drug companies,” she said. “These geneticists and doctors, they are creating things for these families that give us all hope. They have literally rewritten my life.”

What happened with Maisie’s family is common in the world of speciality drug development. The insurance company’s first response was no — the drug was not on their approved list and was not deemed “medically necessary.” Green appealed, a process that can last for months.

Green and the network of parents she has built online blame insurance companies for refusing to cover, or at least for delaying coverage, of life-saving, speciality drugs. In Maisie’s case, every day without Zolgensma was a day that her condition further deteriorated. The effects of spinal muscular atrophy are not reversible, so the earlier it is treated, the better the lifelong outcomes.

Green believes insurance companies can easily deny coverage because of the pervasive, anti-Big Pharma sentiment throughout the country and beyond. Her mission since winning approval for Maisie’s drug is to change the public perception of pharmaceutical companies, particularly those developing expensive drugs to treat rare, previously untreatable diseases, sometimes called “orphan diseases.” She finds it painfully ridiculous that insurance companies will pay for a comparably expensive, life-saving heart surgery that would save a baby’s life, but could easily refuse Maisie a drug that would fix the protein in her cells and keep her alive.

“The populace looks at it as, ‘It’s a huge number, why would we pay for that?” she said. “What do insurance companies do? They follow the populace.”

Maisie Forrest was diagnosed at six months old with the deadliest type of spinal muscular atrophy, and doctors warned her parents she wasn’t likely to live past 2. She received a new specialty drug, for $2.1 million, in July after her mother battled with their insurance company. (Photo provided by Ciji Green)

The insurance industry, however, says drug companies need to reform their business model and that setting the price of a drug at $2 million when the company has a monopoly over that drug makes price negotiation nearly impossible. While insurance companies want to encourage development of new drugs, they also must “recognize the need to balance innovation, accessibility and affordability,” said Amanda Massey, executive director of the Colorado Association of Health Plans.

Either way, it’s a fact that speciality drugs are straining the state Medicaid program’s budget, where many of the most expensive drugs are newly-developed speciality drugs. And private insurance companies are struggling to figure out how to cover expensive medications without passing the cost on to employers and patients.

The relationship between insurance and drug companies shows signs of evolving, though.

For example, some insurance companies are pursuing agreements with drug companies that are “outcome-based,” meaning payment is linked to how well the drug actually works on a patient, Massey said. “This may help to make care, treatments and technology more accessible to patients as more and more million-dollar drugs come to market.”

Medicaid’s most expensive drugs

The most expensive doctor-administered dose of medicine covered by the Colorado Medicaid program last year was a speciality drug called Spinraza, a 3-year-old treatment that can mean the difference between a newborn baby dying as a toddler or surviving to adulthood.

Infants with the most serious type of spinal muscular atrophy, which destroys muscle function, including the ability to breathe, live only until about age 2. But Spinraza can slow the disease, to the point that a child who receives it at birth may be running around as a preschooler instead of using a wheelchair and a breathing tube.

Medicaid paid for the drug seven times in Colorado in 2019, at a cost of $53,351 each. It’s given in hospitals, where a physician injects it into a child’s spine.

The most expensive Medicaid-covered drug dispensed by a pharmacy last year was Ruconest, developed in 2014 to control swelling and breathing attacks in people with a rare and deadly disorder called hereditary angioedema. The medicine is a manmade protein derived from the milk of genetically altered rabbits.

Per dose, it cost Medicaid $159,303, and the government insurance program for low-income patients and those with disabilities paid for it 11 times last year in Colorado.

The state’s Drug Utilization Review Board determines whether the state Medicaid program will cover new drugs and establishes criteria about which patients will qualify. The process is typically long — meaning a new speciality drug can take months or years to get added to the list.

The Medicaid program didn’t have to pay for Maisie’s drug only because of a partnership with Rocky Mountain Health Plans on the Western Slope, where Maisie lives. The girl is a Medicaid patient, but Medicaid contracts with RMHP to provide coverage for Medicaid patients in a six-county area.

RMHP agreed to cover Maisie’s $2.1 million dose of Zolgensma in July, after an in-person meeting with her mother. Spokeswoman Leanne Hart said she could not discuss the specific case, because of confidentiality laws, but confirmed that the insurance company now covers Zolgensma.

In general, when a doctor asks the insurance company to cover a high-cost drug, RMHP reviews the patient’s medical records and consults with their doctors, and if necessary, independent experts, Hart said.

Personalized medicine can save money long-term

Speciality drug development — by research institutions and pharmaceutical companies — has expanded the disconnect between manufacturers and payers, said experts in speciality medicine. Colorado is adding more rare diseases, including spinal muscular atrophy, to the newborn screening list, but some advocates question what good that does if treatment is unaffordable.

“For personalized medicine to truly be successful in the United States, we need to have payers on board,” said Dr. Kathleen Barnes, director of the Colorado Center for Personalized Medicine on the University of Colorado Anschutz Medical Campus in Aurora.

“There is no question that we have a crisis in the United States with more commonly prescribed drugs often costing more than they should. But for rare diseases, one can understand why it’s so costly for a pharmaceutical company to make a drug that they are not going to be able to market to very many patients.”

The Center for Personalized Medicine, founded in 2014, is a sort of health-data warehouse, where researchers study the genetics of 112,000 patients who have so far agreed to provide blood samples to the biobank. The center is helping doctors determine which drugs would work best for patients’ health conditions, based on their specific genetic mutations.

If a doctor is about to prescribe a certain heart medication, for example, they can check a patient’s medical record to see whether that medicine is a good choice based on the patient’s DNA. So far, the center has completed genetic sampling of about 30,000 people, with a goal of completing all 112,000 within the next five years.

In the future, researchers hope to be able to inform patients — if they want to know — whether they are predisposed to diseases including sickle cell anemia, cystic fibrosis, cardiomyopathy or cancer.

The future of personalized medicine is brighter, though, if insurance companies see the value in paying for genome sequencing and preemptive medicine for all, Barnes said. In many cases, it would cost less to treat an infant who is predicted to develop a devastating disease than to spend a lifetime treating that disease, she said.

“Personalized can save money by eliminating the trial and error approach,” Barnes said.

Research institutions, including the University of Colorado, are beginning to play a larger role in drug development because of speciality drugs, said Dan LaBarbera, an associate professor of drug discovery at CU’s Skaggs School of Pharmacy. It costs billions of dollars and anywhere from five to 20 years to develop a drug, and creating one to cure an orphan disease is not typically a sensible business model, leaving discovery to universities, said LaBarbera, who is working to develop new cancer drugs.

Still, when a university develops a drug, spending federal and state research dollars, who gets to take it?

“As technology improves, how are we going to use this technology to treat patients regardless of economic background?” LaBarbera asked.

Julia Vitarello visits her daughter Mila, who is fighting Batten disease, a rare fatal condition with no cure, in her room at Childrens Hospital. Mila is the first person in the world to receive a drug customized for just one person. It was named Milasen. (John Leyba, Special to The Colorado Sun)

Doctors at Children’s Hospital Colorado, on the same medical campus, are treating a 9-year-old girl with a drug that was developed only for her. The drug was created by researchers at Boston Children’s Hospital and includes a 22-letter DNA sequence that matches the one in Mila Makovec’s cells that is broken.

Mila’s mother, Julia Vitarello, created Mila’s Miracle Foundation and raised $3 million in one year to save her daughter. She declined to say how much the drug cost to design and manufacture, but researchers and a drug company donated some of their time.

Maisie’s drug worked in 36 hours

Since winning Maisie’s case, Ciji Green is helping other families across the country fight denials from insurance companies. Maisie’s Army recently helped win approval for Zolgensma for a baby diagnosed at birth with spinal muscular atrophy. But it took four months, Green said.

“Early detection is great,” Green said. “But insurance companies don’t want to pay the price.”

At 6 months old, Maisie was diagnosed with the deadliest type of spinal muscular atrophy, and doctors warned her parents she wasn’t likely to live past age 2. She started doses of Spinraza, but by the time Maisie was 18 months old, her health was fading anyway.

Spinraza was keeping her alive, but Maisie’s respiratory system was declining to the point she was using a ventilator 22 hours out of every day. “I was certain we were not going to have her when she was 2,” Green said. “Nobody goes into parenthood thinking, ‘I’m going to bury my baby.’”

Maisie Forrest was diagnosed at six months old with the deadliest type of spinal muscular atrophy, and doctors warned her parents she wasn’t likely to live past 2. She received a new specialty drug, for $2.1 million, in July after her mother battled with their insurance company. (Photo provided by Ciji Green)

Zolgensma was approved just in time, Green said. “From Day One, I knew I wanted it,” she said. “Whatever muscles are still firing will get stronger over time. When you’re dealing with a disease that is going to take your baby’s life, you want to stop that.”

Within 36 hours of Maisie’s dose, she could control her head and neck. She was soon sitting on her own, then laughing. The 2-year-old has enough strength now to pull her legs up to her face — and to pull the pulse-oxygen monitor off her toe, which makes it beep.

Most babies with Type 1 spinal muscular atrophy, the type Maisie has, are like a “bag of water,” Green said. They seem normal at birth, but they don’t reach the typical milestones — they don’t sit up or roll over.

Maisie, who got Zolgensma just before her second birthday, will likely need a feeding tube for the rest of her life. But she won’t spend her life lying down and “looking at the sky or the ceiling,” her mom said.

“Now she gets to see the world as we do.”

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