{"id":59347,"date":"2021-11-18T13:15:21","date_gmt":"2021-11-18T18:15:21","guid":{"rendered":"https:\/\/rarediseases.org\/with-the-build-back-better-act-congress-threatens-progress-for-americans-with-rare-diseases\/"},"modified":"2021-11-18T13:15:21","modified_gmt":"2021-11-18T18:15:21","slug":"with-the-build-back-better-act-congress-threatens-progress-for-americans-with-rare-diseases","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/with-the-build-back-better-act-congress-threatens-progress-for-americans-with-rare-diseases\/","title":{"rendered":"With the Build Back Better Act, Congress Threatens Progress for Americans with Rare Diseases"},"content":{"rendered":"

As Congress continues to debate the policies to include in the final version of the Build Back Better Act, the House of Representatives has inexplicably chosen to take aim at one of the most vulnerable populations in our nation: the rare disease community. The Build Back Better Act, H.R. 5673, currently includes a harmful provision that threaten the decades-long success of the Orphan Drug Tax Credit (ODTC) and drastically reduce the therapeutic options available for the 25-30 million rare disease patients in America.<\/span>\u00a0<\/span><\/p>\n

Rare diseases\u00a0have\u00a0small patient populations<\/span>,\u00a0<\/span>which\u00a0makes developing drugs\u00a0to treat these conditions\u00a0inherently more complicated and costly than for common medical conditions.\u00a0Prior to 1983, there were only\u00a030\u00a0drugs approved by the FDA for rare diseases.\u00a0Simply put<\/span>,<\/span>\u00a0the pharmaceutical industry was just not interested in making the investment necessary to\u00a0treat<\/span>\u00a0<\/span>rare diseases, only to find a small market\u00a0once<\/span>\u00a0<\/span>a\u00a0drug was FDA approved<\/span>.\u00a0<\/span>\u00a0<\/span><\/p>\n

Recognizing\u00a0this market failure,<\/span>\u00a0<\/span>Congress<\/span>\u00a0<\/span>passed the\u00a0landmark Orphan Drug Act, which\u00a0established a\u00a0clever\u00a0mix of\u00a0policies<\/span>\u00a0<\/span>designed to make small\u00a0patient\u00a0populations a more attractive prospect for the pharmaceutical industry.\u00a0One of the key\u00a0incentives<\/span>\u00a0<\/span>was\u00a0the\u00a0ODTC, which<\/span>\u00a0<\/span>allows\u00a0drug developers to write off significant portions of the clinical trial costs associated with rare disease drug development.\u00a0<\/span>The ODTC has been instrumental in encouraging drug companies\u00a0to\u00a0pursue clinical trials to prove that a drug is safe and effective for a rare disease, or sometimes multiple rare diseases.<\/span>\u00a0<\/span>By most accounts,\u00a0both\u00a0the Orphan Drug Act\u00a0and<\/span>\u00a0<\/span>ODTC\u00a0are working as intended. In stark contrast to\u00a0before the passage of the Orphan Drug Act,\u00a0today there are 652 drugs approved for 1,006 rare disease conditions.<\/span>\u00a0<\/span>\u00a0<\/span><\/p>\n

Still, the need for the package of incentives that became law in 1983\u00a0is\u00a0as unmistakable today as it was 38 years ago<\/span>.\u00a0<\/span>1-in-10 people in the United States are living with one of the approximately 7,000 known rare diseases, many of which are life-limiting or fatal. More than 90% of these diseases have no treatment or cure that has been approved by the FDA.<\/span>\u00a0<\/span><\/p>\n

Yet, the latest version of the Build Back Better Act includes a provision that would severely diminish the ODTC by limiting its availability to only the first orphan use of a drug.\u00a0<\/span>It is not unusual for a single drug to be determined to be safe and effective for the treatment of multiple rare diseases. For rare disease patients, every time a treatment for a rare disease (or \u201corphan indication\u201d) is approved by FDA, it is often life-saving progress. It helps patients and their health care providers have confidence that the drug they are utilizing is safe and effective for their condition. FDA approval can also make it easier to get coverage for the treatment and to<\/span>\u00a0<\/span>lower patient out of pocket costs.\u00a0 History has proven that there must be appropriate incentives, like the ODTC, to see the continued development drugs to treat rare diseases.\u00a0<\/span>\u00a0<\/span><\/p>\n

While negotiations to include drug pricing provisions\u00a0in the Build Back Better Act continue,\u00a0tying\u00a0the ODTC to the on-going drug pricing conversation is misguided and limited.\u00a0To be clear, NORD shares concerns over the high cost of drugs in America<\/span>,<\/span>\u00a0but<\/span>\u00a0<\/span>attacking rare disease drug development, particularly\u00a0an incentive that helps offset the\u00a0high costs associated with clinical testing\u00a0in rare disease patients, isn\u2019t drug pricing reform<\/span>.\u00a0<\/span>In its efforts to alleviate the burden of high prescription drug costs,\u00a0Congress should focus on the true drivers of high drug costs, and not attack the incentives necessary for developing products for those who need them most: rare disease patients.<\/span>\u00a0<\/span>\u00a0<\/span><\/p>\n

On behalf of the\u00a0nearly\u00a030\u00a0million Americans with rare diseases and their families,\u00a0NORD urges Congress to recognize the success of\u00a0the Orphan Drug\u00a0Tax Credit\u00a0and preserve it in the name of protecting these individuals\u00a0and their hope for a better tomorrow<\/span>.\u00a0<\/span>There is still more work to be done, and\u00a0rare disease\u00a0patients cannot wait.\u00a0<\/span>\u00a0<\/span><\/p>\n

Peter L. Saltonstall is president and CEO of the National Organization for Rare Disorders (NORD).<\/em><\/p>\n","protected":false},"excerpt":{"rendered":"

As Congress continues to debate the policies to include in the final version of the Build Back Better Act, the House of Representatives has inexplicably chosen to take aim at … <\/p>\n

Continue reading “With the Build Back Better Act, Congress Threatens Progress for Americans with Rare Diseases”<\/span><\/a><\/p>\n","protected":false},"author":1,"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":[232,662],"tags":[2877,2878,2837,2838,236,906,1272,262,347,360],"class_list":["post-59347","post","type-post","status-publish","format-standard","hentry","category-advocacy","category-get-involved","tag-bbb","tag-bbba","tag-build-back-better","tag-build-back-better-act","tag-congress","tag-house","tag-house-of-representatives","tag-orphan-drug-act","tag-orphan-drug-tax-credit","tag-peter-saltonstall"],"acf":[],"_links":{"self":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/59347","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\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/comments?post=59347"}],"version-history":[{"count":0,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/59347\/revisions"}],"wp:attachment":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/media?parent=59347"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/categories?post=59347"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/tags?post=59347"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}