NORD Applauds New US Legislation for Rare Disease Funding

But patient advocacy group says package is 'deeply' disappointing

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by Mary Chapman |

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This illustration shows the U.S. Capitol building, home to the government's legislative branch.

While unhappy with shortcomings in the legislation, the National Organization for Rare Disorders (NORD) nonetheless is applauding a year-end U.S. spending package that included several programs and reforms crucial to those with rare diseases, including Lambert-Eaton myasthenic syndrome (LEMS).

As part of an expansive $1.7 trillion package, signed by President Joe Biden to fund government operations through September, U.S. lawmakers voted to:

  • fortify the U.S. Food and Drug Administration’s (FDA) accelerated treatment approval pathway to protect and ensure patient access to treatments.
  • re-authorize through 2027 several programs, including Orphan Products Grants, Pediatric Device Consortia Grants, and the National Institutes of Health’s Best Pharmaceuticals for Children Act.
  • ensure greater representation of underserved and diverse populations in clinical studies.

As the chief advocacy group for the more than 25 million Americans living with a rare disorder, NORD had led a robust effort to get lawmakers to prioritize such programs and reforms. Each was considered key, by groups representing or treating people with rare diseases, to assuring patient access to diagnostics and essential therapies.

“The National Organization for Rare Disorders (NORD) applauds Congress’ decision to address unfinished business and ensure these measures were approved before the end of the year,” Peter Saltonstall, NORD’s president and CEO, said in a press release, adding, “Each of these actions will better support efforts for rare disease patients to obtain safe and effective therapies.”

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The package also included plans that extend coverage, through 2024, of certain telehealth services covered by Medicare. According to NORD, expanded access to remote care has been invaluable to the rare disease community since the COVID-19 pandemic because it mitigates the need to travel for in-person care.

The U.S.-based organization also is pleased that Congress extended 12 months of continuous eligibility to low-income children covered by Medicaid and the Children’s Health Insurance Program, known as CHIP. These are the main sources of insurance coverage for children with special healthcare needs, including those with rare diseases, NORD said.

Still, the group said it is “deeply disappointed” by a failure to clarify how FDA-approved treatments are awarded orphan drug status under the Orphan Drug Act. Such clarification is necessary for continued investment in rare disease treatment development, particularly for children and other vulnerable demographics, according to NORD.

There also was no advancement on other measures important to the rare disease community. Such legislation includes:

  • the Accelerating Kids Access to Care Act. It aims to establish a better and easier enrollment process for children covered by Medicaid and CHIP who need out-of-state care.
  • the Medical Nutrition Equity Act. Action on this program would require insurance coverage of medical nutrition products for patients who rely upon medically formulated foods.
  • the Newborn Screening Saves Lives Reauthorization Act. Funding for improvements in state programs that provide screening for infants shortly following birth would have been provided under this act. Such programs allow screening for treatable conditions that aren’t clinically manifest in the newborn period. However, the year-end spending package did include funding for the National Academy of Medicine to ultimately recommend ways to modernize and improve such screening.

“NORD and our partners will continue to advocate for these and other priorities in the 118th Congress that will help advance safe and effective therapies for those with rare diseases and expand access to affordable and comprehensive care,” the organization stated in the press release.

Last year, NORD had lobbied Congress to include provisions omitted in its temporary funding package, passed in October. That legislation, which kept the government funded through last month, included five-year extensions for several FDA user fee programs, considered crucial to timely patient access to diagnostics and medicines.