When the family of 20-month-old Maisie Green heard late last month that their insurance company in Grand Junction, Colo., had agreed to cover a new gene therapy for her spinal muscular atrophy, they were elated. They also knew it was no accident.

For two months, the family and a group of more than 700 volunteers, calling themselves “Maisie’s Army,” ran a social media campaign to convince the Greens’ insurer to overturn its decision to deny Maisie access to Zolgensma — the world’s most expensive drug at $2.1 million. The army contacted local news outlets to raise awareness of Maisie’s case. And her mother, Ciji Green, arranged an in-person meeting with Maisie and representatives of the insurance company.

“I want you to look her in the eye and tell her no,” Green recalled thinking about the meeting.

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After the approval from Rocky Mountain Health Plans came through, one of the volunteers posted a call to arms on Facebook: “New slogan!!! Not stopping at one!! Other families are gonna need us army!!!”

The army has stayed true to that call. Fueled by the success of Maisie’s story, the group said it has already helped several other kids with spinal muscular atrophy, or SMA, win access to Zolgensma through their insurers, and is in the process of assisting at least two more. The families are all across the country — from Massachusetts and Minnesota to Missouri and California.

The group communicates nearly every day on Facebook (FB). Volunteers provide phone numbers and email addresses for people at a child’s insurance company, as well as Twitter handles for reporters and news outlets. There’s a night crew and a day crew providing updates on each child’s case. There is no script the volunteers are asked to follow when contacting companies, but they are encouraged to make it courteous and plead on behalf of the kids. Amid updates on the Facebook page on how specific cases are going, encouraging GIFs and memes are sprinkled in.

Their success is a reflection of not only the effectiveness of public pressure campaigns but also of the stakes for families. Most children who are diagnosed with SMA as infants do not survive past early childhood due to respiratory failure. And for families trying to prevent that fate, the financial cost of doing so can be enormous.

Maisie & Ciji Green
Maisie and Ciji Green at Children’s Hospital Colorado during Zolgensma treatment earlier this month. Annique Green

Green had set up the Maisie’s Army Facebook group more than a year ago, initially to raise awareness about the rare disease and help the family cope with the cost of getting Maisie to and from Denver four hours away to get another SMA treatment, Biogen’s Spinraza.

“We ended up losing our house that year,” Green said, and she created the group because “we knew someday we would need a voice.”

The army’s work ramped up when the Food and Drug Administration approved Zolgensma in late May. Marketed by Novartis (NVS) and developed by its AveXis subsidiary, Green said the drug is an attractive option because it’s delivered in a one-time infusion.

“Of course we wanted it,” she said.

Like Maisie, many of the kids who are now under the army’s wing have also received Spinraza, but there are big differences with Zolgensma: After the initial doses, kids have to get Spinraza every four months for life. Each injection also includes a spinal tap. And at $125,000 per dose, it’s an annual cost of $375,000, though many insurers have thus far covered the treatment.

Zolgensma’s one-time infusion seemed like a clear choice, said the families STAT spoke with. And those who have already been helped by Maisie’s Army say the group’s support was invaluable.

Estelle Lemieux of Walpole, Mass., is scheduled to be dosed with Zolgensma this week. Her family was left homeless after Hurricane Irma hit Florida in 2017, forcing them to Massachusetts to live with Estelle’s grandparents. At the time, Estelle’s mom, Amy Lemieux, was eight months pregnant with her now 21-month-old daughter.

When Amy Lemieux joined the Facebook group and shared that her insurer was Aetna, Maisie’s Army went to work adding Estelle’s name to the calls and emails it had already been making for a child in Missouri whose family was also covered by the insurer.

Three days after the Lemieuxs received their initial denial, Aetna changed course and said it would change its policy for kids with SMA to also cover gene therapy.

“I started crying on the phone, I couldn’t believe it,” Lemieux said.

Lemieux wasn’t even aware of the extent of the army’s work until contacted by STAT. But just knowing that the group existed was helpful, she said. As far as the support from Green, “she really made me feel like they were going to go to battle for Estelle,” Lemieux said.

For all the kids the group has tried to help so far, Green said she’s made over 200 calls, which she prefers to writing letters and emails.

“I think my desperation and pleas come across better in a phone call. It makes it more human,” Green said.

When reached for comment, an AveXis spokesperson said, “While we can’t discuss the specifics of any particular patient, our goal is to support access for patients who need this one-time gene therapy and we’re pleased each time a child is approved for treatment with Zolgensma. To facilitate this, we are actively partnering with insurers to accelerate coverage decisions that will support access for eligible patients.”

Maisie’s Army is still making appeals for Maddy Bruce of Blaine, Minn. She turns 2 — the age limit in the FDA’s Zolgensma approval — in less than a month.

“We’ve already been denied several times,” Maddy’s mother, Angie Bruce, said of their insurer, Minnesota’s Medicaid system. “The letters that I’ve gotten have stated that it’s not medically necessary.”

Maddy Bruce
Maddy Bruce and her mother, Angie Bruce, during a Spinraza treatment at the University of Minnesota Masonic Children’s Hospital this month. Courtesy Cristine Davidson

Zolgensma is covered by the state’s Medicaid, but it needs to be approved by a physician and shown, among other things, that other drugs haven’t worked. (The financial costs related to drugs like Zolgensma are also raising tough questions for state Medicaid programs, which are grappling with how to afford them.)

Maddy, who has been receiving Spinraza since she was 2 days old, has seen benefits from the drug. “She is pulling herself in a standing position quite a bit,” Bruce said. “She’s not walking just yet, but I think she’s going to soon.”

Still, “Spinraza is super hard on her,” Bruce said. “She’s such a fighter and so strong and doesn’t like being held down by doctors.”

The Minnesota Department of Human Services said in a statement that the state’s Drug Formulary Committee earlier this month began reviewing how Zolgensma could be covered without needing the prior authorization, “but tabled the discussion pending the drug maker’s response to Congress about data manipulation during its clinical trials.”

For Bruce, like many of the other parents of children with SMA, the potential that getting the one-time treatment holds is huge. “I’m just hopeful that in a year from now she’ll be running in the yard with her brother,” she said.

The Bruce family belongs to the Ojibwe tribe, and Bruce hopes that one day her daughter will be able to take part in the tribe’s traditional dances.

In California, 15-month-old Lincoln DellaMaggiora’s family is similarly hoping that their insurance company, Kaiser Permanente, will change course. Lincoln’s older brother, Landry, died at 47 days old in 2017. At the time, the cause was explained to the family as possibly being sudden infant death syndrome, and that loss is fueling the family’s fight for Lincoln.

“I have already lost one son. I’m not about to lose another,” mother Katee DellaMaggiora said.

“We have tremendous empathy for this family and want their child to have the support and care he needs,” Kaiser Permanente said in a statement.

“It’s been a lot of waiting and dealing with medical egos,” DellaMaggiora said, fighting back tears. She said Lincoln’s physician deemed the new drug unlikely to produce many benefits, since the data to support Zolgensma’s FDA approval is largely from babies 6 months and younger. The physician’s decision in turn led the insurance company to deny coverage. An appeal to a California agency that reviews insurance decisions also resulted in a denial late last week.

DellaMaggiora found Maisie’s Army through other SMA support groups on Facebook about a week ago, and said action has been swift.

“Boom — they’ve been helping with everything,” DellaMaggiora said, including making calls to Kaiser Permanente, connecting her with news outlets (including STAT), and sharing Lincoln’s story on social media. “The community is amazing.”

While Maddy and Lincoln’s families await further word on Zolgensma coverage, those who have gotten the drug have already started to see its impact. For Maisie, who received the infusion two weeks ago, her mother said there have been marked changes.

“Before she could maybe sit up with help for a maximum of nine seconds,” Green said. “Last week, it was 53 seconds on her own. It was amazing.”

Correction: An earlier version of this story misstated Estelle Lemieux’s age.

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  • I am the mother of two children with cancer. One has an ultra rare Clear Cell Sarcoma that has no protocol or standard of care. I am so happy for this group and cheer them on for paving the way for the hundreds of other groups like us. I used to blame Big Pharma until I attended a talk for the CEO’s of those companies and realized they WANT to get patients the needed drugs. They have to negotiate with the payers (Government and private insurance) to get them to pay. One way they do this is to argue that if insurance is willing to pay one million for a heart transplant that extends life by a 5 year average then they should pay $250,000 per year for a drug that is likely to extend life by the same amount. Its easy to assume its just the pharma companies but the insurance (lack of competition is key) in my opinion is the real issue. We have to find ways to save our children’s lives by manipulating and shaming the insurance just like this family did. Assuming pharma is behind this group seems odd to me. We WISH pharma helped us with our fight. I know from my experience, and that of the other parents like me, we would never push for a drug that was not the absolute best option for our child. We beg the pharma companies to help us and most that I have seen give the drug for free. The hospital then becomes the issue because they won’t get paid for administering it. Most of us have become the experts in our child’s disease. Our organization has worked tirelessly to connect the scientists with each other to share information and hopefully educate the clinicians. If we (parents of a child with a rare disease) are fighting for a drug you can bet your life it is the best option for the quickest life saving result. I just wanted to make this statement because you only understand this when you become part of it and it’s a realization no parent or family should have to face. Thank you for reporting on this. There have been only about 6 new drugs for pediatric rare disease since the 70’s. Hopefully social media will keep changing that. sarascure.org

  • Has STAT checked to see if any of the members of Maisie’s Army are connected to PHARMA? I have read in other articles that a number of “support groups” that push for approval/access to medications that have little or no track record receive backing from thinly disguised pharmaceutical groups.

    • Parents live are are concerned that ICER is recommending denial of drugs for rare diseases. No connections but check the multiple connections between ICER and insurance and PBMs. Denial is the connection with reviews with advisory boards made up of greedy insurance providers at the expense of a child’s life.
      It’s in our face.

  • Politicians should become more humane and treat ALL with Genetic Diseases and STOP Greedy Big Pharma from charging Atrocious Prices for these Drugs.

    • Follow the greedy insurance/PBMs who use ICER which makes this child and will
      Make Cystic Fibrosis children “life’s value not worthy of the cost of the drugs to bring them a healthy life.

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