NORD Urges Congress to Ensure Access to Rare Disease Therapies; Goal is Either: Protections in Medicare Drug Bill or Passage of HR 2700

7/14/2003

From: Diane Dorman of the National Organization for Rare Disorders, 202-496-1296 or 202-258-6457 (cell)

DANBURY, Conn., July 14 -- The National Organization for Rare Disorders (NORD) today hailed the introduction of the Medicare Patient Access to Drugs for Rare Diseases Act of 2003 (H.R. 2700). NORD urged Congress to either pass the legislation or put similar protections into the Medicare drug legislation being considered by House and Senate conferees.

"Twenty years ago, Congress enacted the Orphan Drug Act to ensure that patients suffering from rare diseases have access to the therapies they need," said Abbey Meyers, Founder and President of NORD. "The Orphan Drug Act is a covenant between the federal government and the rare disease community, founded on the principle that rare disease therapies must be available for patients who need them, regardless of whether these products are economically viable for patients or manufacturers."

"Regrettably, the tremendous success of the Orphan Drug Act is now being undermined by Medicare payment policy," Ms. Meyers said. She added, "H.R. 2700 is a promising first step toward remedying serious problems with the payment for orphan drugs under the Medicare Hospital Outpatient Prospective Payment System (HOPPS)."

"The current payment policy for rare disease therapies neglects the needs of the rare disease community by failing to adequately compensate hospitals for making these critical therapies available," said Representative Christopher Cox (R-CA), who introduced H.R. 2700. "The 1999 HOPPS Law was intended to include all orphan drugs, and my bill will ensure that hospitals continue to be reimbursed for these important drugs so that Americans with rare diseases do not suffer needlessly."

"The rare disease community is immensely grateful to Congressman Cox and to the growing list of cosponsors from both parties for their continuing commitment to this critical issue," said Diane Dorman, Vice President for Public Policy at NORD. "Without this bill, or similar protections added to the Medicare drug bill, beneficiaries who suffer from rare diseases may be denied treatment because healthcare providers refuse to stock orphan drugs. Congress cannot allow this to happen."

Before January 1, 2003, all orphan drugs administered in hospital outpatient facilities were exempted by statute from the HOPPS payment methodology. Beginning this year, the Centers for Medicare and Medicaid Services (CMS) combined the payment for many orphan drugs with the payment for the procedure with which the drugs are associated, significantly reducing total payments. Most other orphan drugs are paid separately under HOPPS but at payment rates inadequate to ensure patient access.

In addition to Congressman Cox, those co-sponsoring the legislation when it was introduced on July 11, 2003 included Representatives Norwood (R-GA), Issa (R-CA), Engel (D-NY), Boucher (D-VA), Berman (D-CA), and Pomeroy (D-ND).

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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.