July 21, 2007
A rare and painful disease, known as nephrogenic systemic fibrosis, has caused her legs and right arm to become frozen in a bent position.She fears that the condition, known to affect 200 to 250 people worldwide, soon will take hold of her left arm, leaving her an invalid.”It’s completely stopped my life,” the Dickson woman said, choking back sobs. “My mother comes over here to take care of me. She’s 77 years old. I should be taking care of her.”
A Vanderbilt University Medical Center doctor says there is a therapy that may improve Deason’s condition. However, TennCare, the state’s insurance plan for the poor and disabled, will not pay for it because it’s considered to be experimental.
Getting treatment for rare diseases is a widespread problem, said Mary Dunkle, spokeswoman for the National Organization for Rare Disorders. The organization estimates that there are 6,000 to 7,000 rare diseases affecting more than 25 million Americans.
“There simply aren’t treatments for many of these diseases,” Dunkle said. “When there are experimental treatments, people often have difficulty getting access because the treatments tend to be very expensive and insurance companies won’t cover them.”
Marilyn Wilson, TennCare’s spokeswoman, said TennCare had never covered experimental treatments. The agency does not track the number of enrollees who request such treatments each year.
“The state has the responsibility to make sure that our limited health-care dollars are used to treat medical conditions with evidence-based treatments that are proven safe and effective,” Wilson said.
Patients with rare diseases are often left in a precarious predicament: Insurance companies typically won’t cover a treatment unless the FDA has approved it for that disease, but for the majority of rare diseases there are no FDA-approved treatments.
Abbey Meyers, president of the National Organization for Rare Disorders, says it’s a matter of economics. Investigators have difficulty securing research funding because the diseases affect such a small number of people.
“Drug companies won’t put money toward researching rare diseases because they won’t make their money back,” Meyers said.
When insurance companies deny treatment coverage, patients are left with few options.
“In a perfect world, the federal government would regulate the insurance industry and no one would be left out,” Meyers said. “We feel that every person should have access to treatment as long as it’s safe and likely effective.”
That’s improbable when even government-funded in surance programs refuse experimental treatments.
“Experimental treatments have never been covered in Tennessee’s Medicaid program, or any other government-funded insurance program that we are aware of, including Medicare,” Wilson said. “This is an area of coverage where TennCare is closely in line with commercial industry standards rather than providing a benefit not available to any other Tennessean in a private commercial insurance industry.”
Wilson said patients denied experimental treatments may consider participating in clinical trials or turning to an academic medical center for help.
Dr. John Zic, Deason’s physician and an assistant professor of dermatology at Vanderbilt, said that for nephrogenic systemic fibrosis and some other rare diseases, there are no clinical trials. He said Vanderbilt was trying to help Deason “by providing information to her insurance carrier, so she can get approval for treatment.”
Deason, 47, is appealing TennCare’s decision. An administrative law judge ruled in her favor in April, but that decision was overturned by a state finance representative. Her case will be reviewed for the last time by a judge in Davidson County Chancery Court. The date has not been set.
Kevin Fowler, an attorney with the Legal Aid Society of Middle Tennessee and the Cumberlands who is representing Deason, is arguing that her illness is so rare that all treatments for it are by definition experimental.
“This treatment has been shown to work,” Fowler said. “When only 250 people in the world have a disease, how could you statistically prove what treatment is most effective?”
Discount Pharmacy - Buy Pharmacy at discount prices including free shipping.Discount Pharmacy provides confortable and easy way to order discount pharmacy online.In addition, Fowler says covering the treatment could save TennCare money in the long run. “If the disease progresses, she could end up needing nursing home care, with the state paying for it,” he said. “Treatment could prolong her independence at a more reasonable cost to the people.”
TennCare’s spokeswoman said she could not speak specifically about Deason’s situation because of the pending litigation.
Deason’s disease is newly recognized and not well understood by doctors.
The first case was identified in 1997. It has occurred only in people with kidney disease, according to The International Center for Nephrogenic Fibrosing Dermopathy Research.
Deason, who was born with kidney disease, said doctors were baffled by her new health problems. She spent months typing her symptoms into Google: swelling and tightening of the skin, difficulty straightening the arms and legs, and pain in the affected areas.
“It’s the worst pain I’ve ever experienced,” Deason said. “It feels like my legs are being electrocuted.”
Eventually, Deason stumbled into an online chat room of similar sufferers.
“A lady was talking about how she was on dialysis and what her symptoms were, and I thought that sounds like me,” she said. “I had a biopsy to test for it. And it came back positive.”
The cause of nephrogenic systemic fibrosis has not been determined. However, a solution that’s injected into a patient’s veins to enhance the quality of an MRI may be the culprit.
In May, the Food and Drug Administration requested that the manufacturers of this contrasting agent include a warning on the product, stating that patients with kidney disease who are exposed to the solution are at risk of developing a “debilitating, and a potentially fatal disease.”
“FDA has been carefully monitoring potential safety signals related to these contrast agents after receiving reports about the risk of this potentially life-threatening disease,” Dr. Steven Galson, director of the FDA’s Center for Drug Evaluation and Research, said in a prepared statement.
Deason underwent a magnetic resonance imaging scan in October and says her symptoms began a few weeks later. A law firm in Ohio has taken Deason’s caseagainst the manufacturers.
“Up until this disease you wouldn’t have even known I was sick,” she said. Now, Deason uses a wheelchair, which she has trouble maneuvering because she has use of only her left hand.
The therapy that Deason is seeking costs several thousand dollars per month.
Deason would need to undergo two treatments a month for one to two years, according to Zic. It’s something Deason could never afford with the $679 in Social Security she collects each month.
The longer she goes without treatment, the worse her condition becomes.
“During all these appeals, I’ve lost (the use of) my legs,” she said. “I can’t do anything. I was a very independent person. Now I can’t even fix a grilled cheese.”
Zic says a procedure called extracorporeal photopheresis offers Deason real hope for improvement. Photopheresis is FDA-approved to treat other diseases and there are few side effects, he said.
The treatment involves removing blood from a patient’s body, exposing it to ultraviolet light and then reinfusing the blood back into the body. It’s believed that the treated blood alters the body’s immune system so it can better fight the disease.
“This treatment has the potential to soften the skin to the point where patients have full range of motion,” Zic said. “I treated one patient, who initially was unable to close her hand. After three months, the patient was able to use a pen to sign documents. Another patient began with significant joint stiffness and had difficulty walking. After six to eight months, the patient was walking with no significant limp.”
Out of the 200 to 250 patients around the world with Deason’s illness, Zic has treated four with photopheresis. Three improved and one died because of issues with dialysis treatment, not because photopheresis was ineffective, he said.
Without therapy, Zic said, Deason’s condition will probably continue to deteriorate.
“As the skin becomes thicker and thicker, patients become more immobile and more prone to respiratory and skin infections,” he said. “These conditions can lead to death.”
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