NORD welcomes advance in US Congress of key rare disease legislation

4 acts considered in House subcommittee support therapy work, access to care

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by Mary Chapman |

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The National Organization for Rare Disorders (NORD) welcomes the advancement, by the U.S. House of Representative’s Energy and Commerce Health Subcommittee, of “critical” bills that encourage rare disease therapy development and improve patient access to care.

The nonprofit organization, which supports rare disease patients through advocacy, research funding, education, and service providers, urges continued bipartisan support to resolve remaining differences regarding the legislation. It estimates that 30 million people live with a rare disease, such as atypical hemolytic uremic syndrome (aHUS), a disorder believed to affect fewer than 625 U.S. residents.

Favorable moves for the bills came during a subcommittee markup meeting. These legislative meetings are held to debate amendments to measures being reviewed, and to consider on how to “report” the proposed bills, including moving them forward from an assigned subcommittee or committee.

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‘Technical issues’ surrounding the legislation still need be resolved

“Today’s markup was a step in the right direction as we work to improve health outcomes for the millions of Americans living with rare diseases,” Heidi Ross, vice president of policy and regulatory affairs at NORD, said in a press release from the group.

“There are more than 10,000 known rare diseases … and too few treatments or knowledgeable health care specialists available to meet patients’ daily needs,” Ross said. “NORD urges leaders of the Energy and Commerce Committee to resolve some of the remaining technical issues discussed during the markup today to maintain the strong, bipartisan support for these bills.”

The following bills, which NORD and its Rare Action Network advocates urge the full Congress to pass, were considered on May 16:

  • Creating Hope Reauthorization Act (HR 7384). Pharmaceutical companies developing treatments for rare diseases that mainly affect children are eligible for rare pediatric disease priority review vouchers supporting their work. Children represent about half of all people living with a rare condition. Unless it’s reauthorized, the voucher program is due to expire on Sept. 30.
  • RARE Act (HR 7383). This legislation ensures that incentives for rare disease research and development are preserved and adequately supported, protecting the U.S. Food and Drug Administration’s long-standing interpretation of the Orphan Drug Act. That act, which came into law in 1983, facilitates the development of rare disease treatments.
  • Accelerating Kids’ Access to Care Act (HR 4758). This legislation aims to make it easier for children with rare diseases whose health care coverage comes through Medicaid or the Children’s Health Insurance Program to access quality, specialized care. Because care for rare diseases often requires distant travel, the bill targets current bureaucratic barriers to out-of-state care.
  • Telehealth Modernization Act (HR 7623). This measure extends for two more years flexibilities in the use of telehealth set up by Medicare during the COVID-19 pandemic, which are due to expire at year’s end. Telehealth refers to remote care given via video conferencing or other technologies, considered helpful to rare disease patients with limited ability to  travel for specialized care or needing to travel long distances for such care.

Under the committee markup, the texts of two bills — Creating Hope and the RARE Act — were incorporated into the Give Kids a Chance Act (HR 3433), NORD reported.

NORD strongly favors reauthorizing the Creating Hope act for four years, “given the significant time it can take to develop a safe and effective rare disease therapy,” it stated in its release.

“Negotiations around the Creating Hope Reauthorization Act must be based on current data,” Ross said. “NORD’s recent analysis found more than half of all vouchers redeemed, awarded or designated through the rare disease pediatric priority review voucher program have happened since 2019.

“The vast majority of rare diseases helped by this program had no FDA-approved treatment prior to the rare pediatric disease priority review voucher program’s establishment,” she added. “Rare disease patients are just starting to fully realize the benefits of the program and we urge Congressional leaders not to let it lapse.” 

This voucher program was enacted in 2012.