'Bubble Boy' Legislation to Create Home Treatment Program

'Bubble Boy' bill to create home treatment program awaits Obama's signature.

December 27, 2012, 10:32 AM

Dec. 27, 2012, — -- If there's one thing Jenny Gardner is afraid of, it's hospitals. She has a primary immunodeficiency disease, also known as the "bubble boy" disease, which renders her body unable to combat the everyday viruses and germs that could lurk in hospital corridors.

And, as fate would have it, Gardner has to go to a hospital every three or four weeks to get the treatment she needs to stay alive.

"That's the last place in the whole world we need to be. That's where the germs are. That's where the sick people are," Gardner said. "There are some winters where I have pneumonia two or three times a year."

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People with primary immunodeficiency diseases, or PIDDs, need intravenous infusions of immunoglobulin, a serum extracted from the plasma of between 1,000 and 15,000 blood donors to give them the antibodies they lack. Like Gardner, most patients receive these infusions at a hospital, but many would prefer to have them administered by a nurse administer at home, but neither Medicare nor Medicaid covers this home treatment.

But that could all change. A new bill called the Medicare IVIG Access Act passed the Senate Friday night after passing the House last Wednesday. If President Obama signs it, it would allow for a three-year pilot program to pay for home infusions.

"It's very important for these individuals," said Dr. Michael Blaese, medical director of the Immune Deficiency Foundation. "If they don't get it, they get infected, and they die of pneumonia, meningitis, septicemia."

Even if they survive, the respiratory infections result in irreversible lung damage, he said.

Of the 250,000 people in the United States with one of the 185 primary immunodeficiency diseases, many live in rural areas, far away from hospitals, or they have other ailments that prevent them from traveling to get their infusion treatments, Blaese said. So they skip it and hope for the best.

"It used to drive me nuts," Blaese said. "There's nothing more disconcerting for a physician than to have a treatment that's effective for a patient, and then we can't get it to them."

The existing law had what the Immune Deficiency Foundation called a "quirk": It paid for the infusion drugs at home but didn't pay for the associated costs, including nurses. Blaese said the drug's occasional reactions, including headaches, back pain, nausea and a rare "shock-like" reaction necessitates a trained nurse during treatment.

Rep. Kevin Brady, R-Texas, said the Medicare IVIG Access Act was inspired by the late David Vetter, the original "boy in the bubble." Brady and Rep. Doris Matsui, D-Calif., introduced the bill in the House. Sens. John Kerry, D-Mass., and Lamar Alexander, R-Tenn., introduced the bill in the upper chamber.

"I've been inspired by the courage of David Vetter and his mother, Carol Ann Demaret, who, as a leader in the Immune Deficiency Foundation, helped me understand how dangerous it is to send PIDD patients to hospitals and doctors' offices each month," Brady said in a statement. "For those most at risk, it's important that we continue to make health care more affordable and accessible."

Gardner said has been calling congressional aides for years, hoping to convince them that people with primary immunodeficiency diseases need home treatment, but they often tell her the same thing: "You don't look sick."

"We hate to hear that," Gardner said. "I've been sick my whole life, and I'm 62 years old. … I missed a lot of school. My parents were actually investigated I missed so much school one year."

Gardner wasn't diagnosed until she was in her late 30s, but she worries most about young families struggling to protect children with primary immunodeficiency diseases.

"I grieve for them, because not only do they have to deal with a very rare disease for their children, but then on the other hand, they have to fight the financial issue of what is covered and not covered," she said. "I don't think a young family with children should have to fight on both sides of the coin. Their fight should be for the child's well-being."

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