THE WASHINGTON TIMES published June 9, 2003
Twenty years ago, when President Ronald Reagan signed the Orphan Drug Act into law, he said, "I only wish with a stroke of this pen I could also decree that pain and heartache of people who suffer from these diseases would cease."
This is a promise that has been kept, with remarkable success. In the decade before the Orphan Drug Act was enacted, only 10 new treatments for orphan diseases were developed. In the 20 years since it was passed, more the 240 new treatments for rare disorders have been approved by the Food and Drug Administration. An additional 900 potential new treatments are in various stages of development. People once dying from rare diseases are now living with them. People who were incapacitated by the pain that disease inflicts on the human body are now leading productive, happy and pain-free lives. Unfortunately, an important part of the on going legacy of the Orphan Drug Act is now in jeopardy. The federal Centers for Medicare and Medicaid Services (CMS), responsible for the Medicare and Medicaid, has recently made most injectable and intravenous orphan drugs less available for Medicare patients. While the CMS regulation is arcane and complex, the effect is likely to be tangible and devastating for those suffering from rare diseases.
Last January, CMS made drastic cuts - an a verage of 35 percent - to Medicare reimbursement rates for drugs covered by the Hospital Outpatient Prospective Payment System (HOPPS). Hospitals must choose between losing money on every injection or living up to their to alleviate pain and suffering.
They should not have to make these choices.
Those with rare diseases are particularly at risk when these decisions are being made. Orphan drugs sustained cuts at least as deep as the average. Hospitals are less likely to stock orphan drugs because they are associated with small numbers of patients. A number of orphan drugs are bundled with high-volume, lower cost drugs, assuring a wide gap between the cost of the orphan drug and the Medicare reimbursement.
This didn't need to happen.
Within the HOPPS rogram, CMS has the authority to exclude all drugs designated as orphans by the Food and Drug Administration. Of the 85 to 90 orphan drugs used by Medicare HOPPS program, CMS excluded only four.
People with rare diseases are re-experiencing pain - acute and immediate, as well as pain from knowing that a bureaucratic decision is keeping them from cutting-edge drug therapies. CMS is backtracking on the covenant that Congress and the federal government has had with the orphan disease community to encourage development of new "orphan" treatments and make them accessible to patients.
Dean Cole, 52, of Little Rock, is living proof of the medical progress CMS is reversing through this rule. Suffering from dystonia, a rare neurologic movement disorder, Mr. Cole experiences recurring severe pain all day, every day, from the cramping the disease causes in his arm and shoulder muscles. Three shots every three month, and much of Mr. Cole's pain from spastic muscle cramping was alleviated. In January, because of the Medicare reimbursement cuts in the HOPPS rule, the hospital told Mr. Cole that it would no longer provide his orphan drug treatments. Spastic muscle contractions returned, along with horrific pain. Mr. Cole has called CMS, dystonia support groups, and legislators for answers.
Recently, he told the Arkansas Democrat-Gazette that he doesn't care about Medicare reimbursements or orphan drug legislation. "All I want," he said, "is for the pain to stop."
So what has to happen for the pain to stop for Mr. Cole and thousands like him?
Further action by CMS is unlikely. Intervention by Congress is, therefore, necessary so that Medicare will pay for injectable and intravenous orphan drugs in outpatient settings. The upcoming package of Medicare enhancements and reforms present an ideal vehicle.
Congress must quickly legislate this fix so that patient access isn't denied; patient pain and suffering is alleviated and the progress that began in 1983 with the stroke of President Reagan's pen reversed.
Abby Meyers is president of the National Organization for Rare Disorders.
Diane E. Dorman, Vice President for Public Policy
National Organization for Rare Disorders, DC Office
1050 17th St. NW, Suite 600
Washington, DC 20036
202-496-1296 ext, 3014
