NORD Urges Strengthening of Rare Disease Protections in Final Budget

Orphan drug incentive among issues in US spending bill, facing Friday deadline

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by Mary Chapman |

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AADC deficiency | AADC News | Illustration of a lone rare person in a crowd

As U.S. lawmakers prepare to head home for the holidays, the National Organization for Rare Disorders (NORD) held out hope they would still act to ensure that people with rare diseases such as aromatic l-amino acid decarboxylase (AADC) deficiency get timely access to treatments and diagnostic tools.

On Tuesday, the Senate put forth an expansive spending package that would keep the government open until next fall, and it’s expected to debate and vote on the bill Wednesday night, after the chance for further amendments. The year-end spending bill, put at about $1.7 trillion, includes more than $772 billion for domestic programs.

NORD wants the budget bill to include provisions left out of temporary funding, passed in October as part of legislation funding the government through this month. Specifically, the organization wants legislators to:

  • Reform the U.S. Food and Drug Administration’s (FDA) accelerated treatment approval pathway to protect patient access to therapies. The pathway was created to facilitate treatment development in small and diverse patient populations with unmet needs.
  • Bolster an incentive created by the Orphan Drug Act for continued investment in rare-disease treatment development.
  • Expand clinical study diversity requirements for therapy manufacturers to increase representation of diverse and dispersed populations affected by rare disorders.
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“Together, we are asking Congress to do the right thing and ensure the 1 in 10 Americans living with rare diseases can continue to have timely access to safe and effective therapies,” Peter Saltonstall, NORD president and CEO, said in a letter to Congress released to the public.

If passed by the Senate, the funding bill will return to the House of Representatives and, if passed there, go to the president to be signed. The package must be passed by midnight Friday to avoid a government shutdown.

Affected programs

The FDA-administered programs that would fortify rare disease research were only authorized in October through Dec. 16, rendering their futures uncertain.

Those programs include the Best Pharmaceuticals for Children program, which encourages clinical study in pediatric patients with AADC deficiency and other rare diseases, and the Orphan Products Grants program, which backs clinical researchers in developing safe and effective treatments.

“Each of these programs has a long and proven record of spurring safe and innovative products for rare disease patients and other vulnerable populations and has a history of bipartisan support regardless of whether Democrats or Republicans are in charge,” Saltonstall said.

There are thought to be more than 7,000 rare diseases, affecting some 25 million U.S. residents. More than 90% of such diseases lack a federally approved therapy.

Last month, NORD led an effort by groups representing or treating those with rare diseases that sought to get legislators to include key FDA programs and reforms in the year-end spending bill.

In noting the legislation’s shortcomings, NORD still welcomed passage of the budget, which now includes a five-year reauthorization of a number of FDA programs, including user fees, considered important to assuring that patients can access diagnostics and essential treatments.

User fees are payments collected by the FDA from companies that sell certain medical products in the country or are applying for a product’s approval.

NORD is the leading advocacy organization for U.S. residents living with a rare disease.