Rare disease legislation in US for research, access to care, advances
Congressional subcommittee votes called 'a step in the right direction'
The Energy and Commerce Health subcommittee of the U.S. House of Representatives has voted to advance proposed legislation — several bipartisan bills — to support research into rare disease, including conditions such as aromatic l-amino acid decarboxylase (AADC) deficiency.
Among the bills moving forward are two that will encourage the development of treatments for rare diseases, while another two will make it easier for people with rare conditions to access necessary care.
“[This] 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 the National Organization for Rare Disorders (NORD), said in a press release.
“There are more than 10,000 known rare diseases, many of which are severe and life-limiting, and too few treatments or knowledgeable health care specialists available to meet patients’ daily needs,” Ross said.
NORD calls for bipartisan support of US rare disease legislation
The markup includes the Creating Hope Reauthorization Act (H.R. 7384). Beginning in 2012, the U.S. Food and Drug Administration (FDA) awarded companies that develop new therapies for children with rare diseases with priority review vouchers (PRVs) to help accelerate innovation. The new legislation would reauthorize the voucher program for four more years. However, it must pass before the Sept. 30 deadline to ensure this program continues.
Also voted for by the subcommittee was the RARE Act (H.R. 7383), a bill that will ensure incentives to drive rare disease research and development are correctly awarded. The Orphan Drug Act (ODA), now more than 40 years old, is designed to provide incentives for companies to develop drugs for rare diseases by offering market exclusivity and tax credits. This bill would secure the FDA’s long-standing interpretation of the ODA, maintaining these incentives for continued orphan drug research.
NORD urges leaders of the Energy and Commerce Committee to resolve some of the remaining technical issues discussed during the markup … to maintain the strong, bipartisan support for these bills that will foster innovation and address some of the challenges felt by our patients and their families.
The Accelerating Kids’ Access to Care Act (H.R. 4758), also known as the AKACA, aims to streamline the process for medical providers to see patients enrolled in Medicaid and in a Children’s Health Insurance Program (CHIP) in another state. This bill will reduce red tape and make it easier for children in these programs to access care across state lines.
The Telehealth Modernization Act (H.R. 7623) will extend Medicare telehealth, granted during the pandemic and set to expire at the end of this year, for two more years.
“NORD urges leaders of the Energy and Commerce Committee to resolve some of the remaining technical issues discussed during the markup … to maintain the strong, bipartisan support for these bills that will foster innovation and address some of the challenges felt by our patients and their families,” Ross said.
As part of the markup, the text of the Creating Hope Reauthorization Act and the RARE Act were incorporated into the Give Kids a Chance Act (H.R. 3433). While these bills have strong bipartisan support, some differences regarding the proposed legislation must still be resolved, mainly related to reauthorizing the rare pediatric disease PRV program and its duration. NORD urged the committee to reauthorize it for at least four years, given how long it can take to develop new rare disease therapies.
“Negotiations around the Creating Hope Reauthorization Act must be based on current data,” Ross said, noting that “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.”
Ross noted that Congresswoman Cathy McMorris Rodgers, a Republican from Washington and the chair of the Energy and Commerce Committee, cited that data in opening remarks at the markup.
“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,” Ross said. “Rare disease patients are just starting to fully realize the benefits of the program and we urge Congressional leaders not to let it lapse.”
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