{"id":273522,"date":"2024-09-18T17:24:32","date_gmt":"2024-09-18T21:24:32","guid":{"rendered":"https:\/\/rarediseases.org\/?p=273522"},"modified":"2025-12-22T14:15:23","modified_gmt":"2025-12-22T19:15:23","slug":"house-energy-and-commerce-committee-09182024","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/house-energy-and-commerce-committee-09182024\/","title":{"rendered":"NORD Applauds House Energy and Commerce Committee Advancement of Bipartisan Legislation to Support Rare Disease Patient Access to Care and Treatment"},"content":{"rendered":"<p><b><span data-contrast=\"auto\">WASHINGTON, D.C., September 18, 2024 \u2014 <\/span><\/b><span data-contrast=\"none\">Today, members of the House Energy &amp; Commerce Committee unanimously voted to advance out of Committee the <\/span><i><span data-contrast=\"none\">Give Kids a Chance Act<\/span><\/i> <span data-contrast=\"none\">(H.R. 3433), <\/span><span data-contrast=\"none\">bipartisan legislation to spur more pediatric rare disease drug development, and the <\/span><i><span data-contrast=\"none\">Telehealth Modernization Act<\/span><\/i><span data-contrast=\"none\"> (H.R. 7623), which would extend for another two years flexibilities that have enabled broader use of telehealth services.\u00a0<\/span><span data-ccp-props=\"{&quot;201341983&quot;:0,&quot;335551550&quot;:6,&quot;335551620&quot;:6,&quot;335557856&quot;:16777215,&quot;335559739&quot;:150,&quot;335559740&quot;:240}\">\u00a0<\/span><\/p>\n<p><span data-contrast=\"none\">The <\/span><i><span data-contrast=\"none\">Give Kids a Chance Act<\/span><\/i><span data-contrast=\"none\"> includes provisions to reauthorize the Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) program through <\/span><span data-contrast=\"none\">2029<\/span><span data-contrast=\"none\">. The RPD PRV program offers a vital incentive encouraging drug developers to invest in rare pediatric diseases, which often face significant challenges in research and development. \u00a0<\/span><a href=\"https:\/\/rarediseases.org\/wp-content\/uploads\/2024\/05\/NORD_PRV-white-paper_FINAL.pdf\"><span data-contrast=\"none\">According to a recently released NORD report<\/span><\/a><span data-contrast=\"none\">, <\/span><span data-contrast=\"none\">as of April 30, 2024, the program has successfully supported the development of <\/span><span data-contrast=\"auto\">53<\/span><span data-contrast=\"none\"> life-enhancing treatments, benefiting 39 <\/span><span data-contrast=\"auto\">rare disease patient communities, nearly all of which had no FDA-approved treatment prior to the program\u2019s establishment in 2012. This program has tremendous support within our patient community, with close to 200 patient organizations joining NORD in sending a letter of support to <\/span><a href=\"https:\/\/rarediseases.org\/wp-content\/uploads\/2024\/09\/PRV-Final-with-Logos_09.16.24_House-2.pdf\"><span data-contrast=\"none\">House<\/span><\/a><span data-contrast=\"auto\"> and <\/span><a href=\"https:\/\/rarediseases.org\/wp-content\/uploads\/2024\/09\/PRV-Final-with-Logos_09.16.24_Senate-1.pdf\"><span data-contrast=\"none\">Senate<\/span><\/a><span data-contrast=\"auto\"> leaders earlier this week, urging its swift reauthorization.<\/span><span data-ccp-props=\"{&quot;134233117&quot;:false,&quot;134233118&quot;:false,&quot;201341983&quot;:0,&quot;335551550&quot;:6,&quot;335551620&quot;:6,&quot;335559738&quot;:0,&quot;335559739&quot;:0,&quot;335559740&quot;:240}\">\u00a0<\/span><\/p>\n<p><span data-contrast=\"none\">The <\/span><i><span data-contrast=\"none\">Give Kids a Chance Act<\/span><\/i><span data-contrast=\"none\"> also incorporates additional provisions from bipartisan legislation to ensure rare diseases are studied in children, including:<\/span><span data-ccp-props=\"{&quot;134233117&quot;:false,&quot;134233118&quot;:false,&quot;201341983&quot;:0,&quot;335551550&quot;:6,&quot;335551620&quot;:6,&quot;335559738&quot;:0,&quot;335559739&quot;:0,&quot;335559740&quot;:240}\">\u00a0<\/span><\/p>\n<ul>\n<li data-leveltext=\"-\" data-font=\"Calibri\" data-listid=\"9\" data-list-defn-props=\"{&quot;335551500&quot;:0,&quot;335551671&quot;:0,&quot;335552541&quot;:1,&quot;335559685&quot;:720,&quot;335559991&quot;:360,&quot;469769226&quot;:&quot;Calibri&quot;,&quot;469769242&quot;:[8226],&quot;469777803&quot;:&quot;left&quot;,&quot;469777804&quot;:&quot;-&quot;,&quot;469777815&quot;:&quot;hybridMultilevel&quot;}\" aria-setsize=\"-1\" data-aria-posinset=\"0\" data-aria-level=\"1\"><span data-contrast=\"none\">The <\/span><i><span data-contrast=\"none\">Innovation for Pediatric Drugs Act<\/span><\/i><span data-contrast=\"none\"> (H.R. 6664), which updates how research is currently funded as part of the Best Pharmaceuticals for Children Act (BPCA), which helps close data gaps around pediatric uses for approved drugs, and strengthens FDA\u2019s ability to enforce post-market commitments around pediatric studies; and<\/span><span data-ccp-props=\"{&quot;134233117&quot;:false,&quot;134233118&quot;:false,&quot;201341983&quot;:0,&quot;335551550&quot;:6,&quot;335551620&quot;:6,&quot;335559738&quot;:0,&quot;335559739&quot;:0,&quot;335559740&quot;:240}\">\u00a0<\/span><\/li>\n<\/ul>\n<ul>\n<li data-leveltext=\"-\" data-font=\"Calibri\" data-listid=\"9\" data-list-defn-props=\"{&quot;335551500&quot;:0,&quot;335551671&quot;:0,&quot;335552541&quot;:1,&quot;335559685&quot;:720,&quot;335559991&quot;:360,&quot;469769226&quot;:&quot;Calibri&quot;,&quot;469769242&quot;:[8226],&quot;469777803&quot;:&quot;left&quot;,&quot;469777804&quot;:&quot;-&quot;,&quot;469777815&quot;:&quot;hybridMultilevel&quot;}\" aria-setsize=\"-1\" data-aria-posinset=\"1\" data-aria-level=\"1\"><span data-contrast=\"auto\">The <\/span><i><span data-contrast=\"auto\">RARE Act<\/span><\/i> <span data-contrast=\"auto\">(H.R. 7384), which would <\/span><span data-contrast=\"none\">cement the FDA\u2019s long-standing interpretation of how to properly award orphan drug exclusivity, a key drug development incentive established by the life-changing Orphan Drug Act.<\/span><span data-ccp-props=\"{&quot;134233117&quot;:false,&quot;134233118&quot;:false,&quot;201341983&quot;:0,&quot;335551550&quot;:6,&quot;335551620&quot;:6,&quot;335559738&quot;:0,&quot;335559739&quot;:0,&quot;335559740&quot;:240}\">\u00a0<\/span><\/li>\n<\/ul>\n<p><span data-contrast=\"none\">\u201cIn 2024, half of the estimated 30 million Americans living with rare diseases are children and many see their lives cut short by these devastating and progressive conditions,\u201d said <strong>Heidi Ross, Vice President of Policy &amp; Regulatory Affairs at the National Organization for Rare Disorders (NORD)<\/strong>. \u201c<\/span><span data-contrast=\"auto\">We are pleased to see the House Energy and Commerce Committee take this important step toward addressing the urgent and often life-threatening needs in rare disease. <\/span><span data-contrast=\"none\">NORD applauds lawmakers on both sides of the aisle for working together to help <\/span><span data-contrast=\"auto\">millions of children who need treatment options.<\/span><span data-contrast=\"none\"> However, if Congress does not pass legislation to reauthorize the RPD PRV program, it will lapse on September 30, 2024, which would be devastating to pediatric rare disease patient communities desperate for new and innovative therapies.\u201d<\/span><span data-ccp-props=\"{&quot;201341983&quot;:0,&quot;335551550&quot;:6,&quot;335551620&quot;:6,&quot;335557856&quot;:16777215,&quot;335559739&quot;:150,&quot;335559740&quot;:240}\">\u00a0<\/span><\/p>\n<p><span data-contrast=\"none\">Additionally, the Energy and Commerce Committee also took an important step forward to protect continued access to telehealth services for millions of Americans by voting to advance the <\/span><i><span data-contrast=\"none\">Telehealth Modernization Act<\/span><\/i><span data-contrast=\"none\"> out of Committee.\u00a0 Telehealth has been critical for rare disease patients, where there are often only a few providers in the country with the specialized knowledge necessary to treat their condition. This results in significant geographic barriers to access, longer wait times, and worse outcomes. A <\/span><a href=\"https:\/\/rarediseases.org\/wp-content\/uploads\/2020\/11\/NRD-2088-Barriers-30-Yr-Survey-Report_FNL-2.pdf\"><span data-contrast=\"none\">2019 NORD survey<\/span><\/a><span data-contrast=\"none\"> of rare disease patients and caregivers found that nearly 40% of respondents traveled more than 60 miles to obtain medical care.<\/span><span data-ccp-props=\"{&quot;201341983&quot;:0,&quot;335551550&quot;:6,&quot;335551620&quot;:6,&quot;335557856&quot;:16777215,&quot;335559739&quot;:150,&quot;335559740&quot;:240}\">\u00a0<\/span><\/p>\n<p><span data-contrast=\"none\">The <\/span><i><span data-contrast=\"none\">Telehealth Modernization Act<\/span><\/i><span data-contrast=\"none\"> would extend flexibilities given to Medicare providers during the COVID-19 pandemic, which include waiving unnecessary paperwork requirements, permitting reimbursement for audio-only telehealth, expanding the types of providers eligible to provide care via telehealth, and permitting rural health centers and federally qualified health centers to receive reimbursement for telehealth for an additional two years.<\/span><\/p>\n<p><span data-contrast=\"none\">NORD thanks the Energy and Commerce Committee members for their bipartisan, unanimous support to advance these important bills as a critical step towards ensuring rare disease patients of all ages have access to necessary care and treatment. \u00a0Next, these bills head to the full House of Representatives for a vote.\u00a0<\/span><\/p>\n<h6><span data-contrast=\"none\">The time for action is now, since the Rare Pediatric Disease PRV program is set to lapse at the end of this month, and telehealth flexibilities will expire at the end of this year<\/span><span data-contrast=\"none\">,<\/span><span data-contrast=\"none\"> without Congressional action. <\/span><span data-contrast=\"auto\">Your voice can make a difference! <\/span><a href=\"https:\/\/rarediseases.org\/driving-policy\/take-action\/#\/232\"><span data-contrast=\"none\">Contact your lawmakers NOW<\/span><\/a><span data-contrast=\"auto\"> to share why the Rare Pediatric Disease Priority Review Voucher program must continue and how telehealth has benefited you!<\/span><span data-ccp-props=\"{&quot;134233117&quot;:false,&quot;134233118&quot;:false,&quot;201341983&quot;:0,&quot;335551550&quot;:6,&quot;335551620&quot;:6,&quot;335559738&quot;:0,&quot;335559739&quot;:0,&quot;335559740&quot;:240}\">\u00a0<\/span><\/h6>\n<h4><b><span data-contrast=\"auto\">Related resources:<\/span><\/b><span data-contrast=\"auto\">\u202f<\/span><span data-ccp-props=\"{&quot;134233117&quot;:false,&quot;134233118&quot;:false,&quot;201341983&quot;:0,&quot;335559738&quot;:0,&quot;335559739&quot;:0,&quot;335559740&quot;:240}\">\u00a0<\/span><\/h4>\n<p><b><span data-contrast=\"auto\">NORD Research: <\/span><\/b><span data-contrast=\"auto\">New analysis by NORD, <\/span><i><span data-contrast=\"auto\">Reauthorize the Rare Pediatric Disease Priority Review Voucher Program: Support H.R. 7384, the Creating Hope Reauthorization Act<\/span><\/i><span data-contrast=\"auto\">, can be <\/span><a href=\"https:\/\/rarediseases.org\/rare-pediatric-disease-prv-program\/\" target=\"_blank\" rel=\"noopener\"><span data-contrast=\"none\">read here.<\/span><\/a><span data-contrast=\"auto\">\u202f<\/span><span data-ccp-props=\"{&quot;134233117&quot;:false,&quot;134233118&quot;:false,&quot;201341983&quot;:0,&quot;335559738&quot;:0,&quot;335559739&quot;:0,&quot;335559740&quot;:240}\">\u00a0<\/span><\/p>\n<p><b><span data-contrast=\"auto\">Advocate with NORD<\/span><\/b><span data-contrast=\"auto\">: Individuals looking to make a difference by getting involved in rare disease policy and advocacy are encouraged to <\/span><a href=\"https:\/\/rarediseases.org\/driving-policy\/take-action\/\"><span data-contrast=\"none\">join our action alerts here<\/span><\/a><span data-contrast=\"auto\">.\u202f\u202f<\/span><span data-ccp-props=\"{&quot;134233117&quot;:false,&quot;134233118&quot;:false,&quot;201341983&quot;:0,&quot;335559738&quot;:0,&quot;335559739&quot;:0,&quot;335559740&quot;:240}\">\u00a0<\/span><\/p>\n<p><b><span data-contrast=\"auto\">NORD Policy Statements:<\/span><\/b><span data-contrast=\"auto\"> Learn about NORD\u2019s public policy work and our efforts to advance new and better therapies, shorten the diagnostic odyssey, and improve access to affordable health care and treatment, <\/span><a href=\"https:\/\/rarediseases.org\/driving-policy\/public-policy-positions\/\"><span data-contrast=\"none\">here<\/span><\/a><span data-contrast=\"auto\">.<\/span><\/p>\n<p><span data-contrast=\"auto\">Media interviews available upon request by contacting <\/span><a href=\"mailto:media@rarediseases.org\"><span data-contrast=\"none\">media@rarediseases.org<\/span><\/a><span data-contrast=\"auto\">.\u202f\u202f<\/span><span data-ccp-props=\"{&quot;134233117&quot;:false,&quot;134233118&quot;:false,&quot;201341983&quot;:0,&quot;335559738&quot;:0,&quot;335559739&quot;:0,&quot;335559740&quot;:240}\">\u00a0<\/span><\/p>\n","protected":false},"excerpt":{"rendered":"<p>WASHINGTON, D.C., September 18, 2024 \u2014 Today, members of the House Energy &amp; Commerce Committee unanimously voted to advance out of Committee the Give Kids a Chance Act (H.R. 3433), &hellip; <\/p>\n<p class=\"link-more\"><a href=\"https:\/\/rarediseases.org\/es\/house-energy-and-commerce-committee-09182024\/\" class=\"more-link\">Continue reading<span class=\"screen-reader-text\"> &#8220;NORD Applauds House Energy and Commerce Committee Advancement of Bipartisan Legislation to Support Rare Disease Patient Access to Care and Treatment&#8221;<\/span><\/a><\/p>\n","protected":false},"author":13,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_relevanssi_hide_post":"","_relevanssi_hide_content":"","_relevanssi_pin_for_all":"","_relevanssi_pin_keywords":"","_relevanssi_unpin_keywords":"","_relevanssi_related_keywords":"","_relevanssi_related_include_ids":"","_relevanssi_related_exclude_ids":"","_relevanssi_related_no_append":"","_relevanssi_related_not_related":"","_relevanssi_related_posts":"","_relevanssi_noindex_reason":"","_price":"","_stock":"","_tribe_ticket_header":"","_tribe_default_ticket_provider":"","_tribe_ticket_capacity":"","_ticket_start_date":"","_ticket_end_date":"","_tribe_ticket_show_description":"","_tribe_ticket_show_not_going":false,"_tribe_ticket_use_global_stock":"","_tribe_ticket_global_stock_level":"","_global_stock_mode":"","_global_stock_cap":"","_tribe_rsvp_for_event":"","_tribe_ticket_going_count":"","_tribe_ticket_not_going_count":"","_tribe_tickets_list":"[]","_tribe_ticket_has_attendee_info_fields":false,"footnotes":""},"categories":[232,4066,4364],"tags":[],"class_list":["post-273522","post","type-post","status-publish","format-standard","hentry","category-advocacy","category-press-releases-advocacy","category-press-releases"],"acf":[],"_links":{"self":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/273522","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/users\/13"}],"replies":[{"embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/comments?post=273522"}],"version-history":[{"count":0,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/273522\/revisions"}],"wp:attachment":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/media?parent=273522"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/categories?post=273522"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/tags?post=273522"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}