NORD: Proposed CMS Rule Jeopardizes Care for Seniors with Rare Diseases; Congressional Action Needed to Remedy Problem 8/12/2003
From: Diane Dorman of the National Organization for Rare Disorders, 202-496-1296, or 202-258-6457 (cell) DANBURY, Conn., Aug. 12 -- The National Organization for Rare Disorders (NORD) today expressed grave concerns about a proposed Medicare regulation that will restrict access to critically needed medicines for beneficiaries with rare diseases. At stake is how the Centers for Medicare and Medicaid Services (CMS) will pay for the roughly 90 orphan drugs that are administered in hospital outpatient departments. "We are starting to see serious access problems for rare disease patients based on last year's rule," said Abbey Meyers, NORD founder and president. "Unless CMS revises the new proposal, or Congress overrides it, the problem will be far worse six months from now." As an example, Meyers pointed to a recent incident in Arkansas, where Medicare payment policy was identified as the reason for eliminating patient access to a therapy for dystonia, a rare neurological movement disorder. "Similar access-to-care issues for rare disease patients around the country are imminent," she said. "If the agency is waiting for evidence of more access problems before it adopts an appropriate orphan policy, it is playing Russian roulette with the rare disease community." NORD's concerns with the Proposed Rule include: -- CMS has invented its own definition of "orphan drugs" that covers only 11 of the 90 orphan drugs administered in the outpatient setting. This definition is arbitrary and without precedent, and it is a dramatic departure from the definition of "orphan drugs" under the Orphan Drug Act. CMS should respect, rather than undermine, the orphan designations of FDA's Office of Orphan Product Development. -- While the Proposed Rule would exempt most orphan drugs from the OPPS "bundling" payment system, 24 orphan drugs administered to patients in the outpatient setting would be bundled into a procedural payment. -- Payment levels for all orphan drugs would be set using historical claims data that are widely understood to be so flawed that no valid conclusions can be drawn from them. Thus, the "special consideration" given to orphan drugs under the Proposed Rule will not result in adequate payments to hospitals. Preliminary analysis indicates that the payment for 48 orphan drugs would be below the payment levels for 2002, which were already reduced substantially as a result of an across-the-board "pro rata reduction" imposed that year. "For the second year in a row, CMS has ignored the needs of beneficiaries with rare diseases," said Diane Dorman, vice president for public policy at NORD. "Orphan drugs present unique access-to-care issues. Hospitals cannot discontinue stocking drugs for more common conditions, even if reimbursement for the drugs is inadequate. This is not the case for orphan drugs, which often are quite expensive and may serve only a handful of rare disease patients in any facility." "We are troubled by CMS's continued failure to respond to the needs of the rare disease community," added Meyers. "This Proposed Rule is just the latest in a series of moves by the agency that evidence a complete and utter lack of regard for the importance of orphan drugs to the millions of Americans suffering from rare disorders." NORD will attempt to obtain a more appropriate orphan drug policy in the final OPPS rule, but Meyers said CMS's latest action demonstrates the need for quick passage of the Medicare Patient Access to Drugs for Rare Diseases Act of 2003 (H.R. 2700), which was introduced last month by Congressman Chris Cox (R-CA) to ensure adequate payment for rare disease under OPPS. "While it is our hope that CMS will be responsive to our concerns this time, we are increasingly of the belief that only a legislative solution will ensure that the needs of the rare disease community are met," said Dorman. "We are immensely grateful for Congressman Cox's leadership on this critical issue, and we urge other Members of Congress to join the effort." ------ NORD is a federation of approximately 125 voluntary health organizations and over 60,000 patients, healthcare providers, and individuals dedicated to helping people with rare "orphan" diseases. NORD is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service. |