{"id":59269,"date":"2021-03-25T13:29:19","date_gmt":"2021-03-25T17:29:19","guid":{"rendered":"https:\/\/rarediseases.org\/new-study-investigates-the-number-of-available-orphan-products-generics-and-biosimilars\/"},"modified":"2021-03-25T13:29:19","modified_gmt":"2021-03-25T17:29:19","slug":"new-study-investigates-the-number-of-available-orphan-products-generics-and-biosimilars","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/new-study-investigates-the-number-of-available-orphan-products-generics-and-biosimilars\/","title":{"rendered":"New Study Investigates the Number of Available Orphan Products, Generics and Biosimilars"},"content":{"rendered":"

Washington, DC, March\u00a0<\/span><\/b>25<\/span><\/b>, 2021\u2014<\/span><\/b>The National Organization for Rare Disorders (NORD<\/span>\u00ae<\/span>)<\/span>\u00a0<\/span>today announced the\u00a0<\/span>findings of a new study that details<\/span>\u00a0the\u00a0<\/span>number<\/span>\u00a0of<\/span>\u00a0orphan products,\u00a0<\/span>generics and biosimilars<\/span>\u00a0available to treat rare diseases<\/span>.<\/span>\u00a0<\/span>NORD\u00a0<\/span>commissioned\u00a0<\/span>Avalere to conduct\u00a0<\/span>the\u00a0<\/span>analysis<\/span>\u00a0to examine if\u00a0<\/span>laws and regulations are\u00a0<\/span>helping to<\/span>\u00a0bring new treatments<\/span>\u00a0<\/span><\/i>to market<\/span>\u00a0for rare disease patients<\/span>.<\/span><\/i>\u00a0<\/span><\/p>\n

According to the\u00a0<\/span>report<\/span>,<\/span>\u00a0<\/span>the US Food and Drug Administration (FDA) approved\u00a0<\/span>5<\/span>99<\/span>\u00a0<\/span>orphan products<\/span>\u00a0to treat rare diseases<\/span>\u00a0<\/span>between 1983 and July 2020<\/span>,<\/span>\u00a0552<\/span>\u00a0of which<\/span>\u00a0<\/span>were<\/span>\u00a0<\/span>on the market at the time of the study<\/span>.\u00a0<\/span>Before the Orphan Drug Act became law in 1983, only 38 orphan products existed. T<\/span>h<\/span>e majority (75%)\u00a0<\/span>of<\/span>\u00a0<\/span>FDA-approved orphan products treat one<\/span>\u00a0rare\u00a0<\/span>disease and have no other use<\/span>. The\u00a0increase in number of\u00a0FDA\u00a0orphan\u00a0approvals\u00a0since 1983,\u00a0and the limited use of most orphan products\u00a0for treating rare diseases only,\u00a0underscore\u00a0the significance\u00a0of the Orphan Drug Act in helping to bring\u00a0novel\u00a0rare disease treatments\u00a0to market.<\/span><\/p>\n

In recent years,\u00a0<\/span>an increasing number of<\/span>\u00a0orphan products<\/span>\u00a0have<\/span>\u00a0<\/span>become available that are<\/span>\u00a0FDA<\/span>-approved<\/span>\u00a0to treat\u00a0<\/span>more than one<\/span>\u00a0medical condition (or \u201cindication\u201d). Avalere\u2019s\u00a0<\/span>analysis<\/span>\u00a0show<\/span>s<\/span>\u00a0that\u00a0<\/span>154 orphan products were\u00a0<\/span>first<\/span>\u00a0<\/span>FDA-approved<\/span>\u00a0to treat\u00a0<\/span>a single<\/span>\u00a0rare disease and, after additional research, earned one or more orphan indications<\/span>. In addition, a small number (37<\/span>)<\/span>\u00a0of\u00a0<\/span>orphan products were initially FDA-approved to treat a common medical condition and, upon further clinical study, earned orphan indication<\/span>(s)<\/span>. Still,\u00a0<\/span>just 10% of<\/span>\u00a0all<\/span>\u00a0orphan products have t<\/span>hree or more orphan indications, demonstrating that the majority of orphan products treat very few rare diseases<\/span>\u00a0and, in turn, small numbers of rare disease patients.<\/span><\/p>\n

In the US, a rare disease is one that affects fewer than 200,000 people. Approximately 7,000 rare diseases have been identified<\/span>.\u00a0<\/span>Whil<\/span>e<\/span>\u00a0each rare disease affects a small<\/span>\u00a0number of people, together\u00a0<\/span>rare diseases affect more than 25 million Americans. More than 90% of rare diseases have no FDA-approved treatment.<\/span><\/p>\n

While most rare diseases\u00a0<\/span>do not yet have a<\/span>n FDA-approved<\/span>\u00a0treatment<\/span>, some have multiple\u00a0<\/span>treatments<\/span>\u00a0available due to the presence of generics and biosimilars.\u00a0<\/span>At the time of the study,\u00a0<\/span>158<\/span>\u00a0orphan products<\/span>\u00a0were eligible for generic<\/span>\/<\/span>biosimilar competition due to expired patent<\/span>s<\/span>\u00a0and orphan drug exclusivity<\/span>; g<\/span>enerics<\/span>\/<\/span>biosimilars were available for 81 (51%) of these<\/span>\u00a0products<\/span>.<\/span>\u00a0<\/span><\/p>\n

The\u00a0<\/span>researchers<\/span>\u00a0<\/span>examined why the\u00a0<\/span>majority (394) of orphan products were\u00a0<\/span>not eligible for g<\/span>eneric or biosimilar competition<\/span>,\u00a0<\/span>and found that<\/span>\u00a080% of orphan products<\/span>\u00a0were<\/span>\u00a0protected\u00a0<\/span>from competition due to<\/span>\u00a0the<\/span>\u00a0patent life of the product, and 20%<\/span>\u00a0were<\/span>\u00a0protected because of orphan drug exclusivity.\u00a0<\/span>Notably, 22% of<\/span>\u00a0all<\/span>\u00a0orphan products ha<\/span>d<\/span>\u00a0patent protect<\/span>ions las<\/span>ting more than 20 years<\/span>.<\/span>\u00a0<\/span><\/p>\n

\u201c<\/span>More than 9 out of 10 orphan products on the market today would n<\/span>ever have been developed without the\u00a0<\/span>Orphan Drug Act,\u201d said Peter L. Saltonstall, President and CEO of NORD. \u201c<\/span>The vast majority of p<\/span>eople with rare diseases still\u00a0<\/span>have no treatment<\/span>, and we need government to provide a framework that<\/span>\u00a0helps patients by<\/span>\u00a0encourag<\/span>ing<\/span>\u00a0the development of\u00a0<\/span>innovative\u00a0<\/span>therapies<\/span>, and<\/span>\u00a0spurs competition after a reasonable amount of time.\u201d<\/span>\u00a0<\/span><\/p>\n

The\u00a0<\/span>report<\/span>, \u201c<\/span>Orphan Drugs in the United States: An Examination of Patents and Orphan Drug Exclusivity,\u201d<\/span>\u00a0is available at\u202f<\/span>rarediseases.org\/rareinsights<\/span><\/a>.<\/span><\/p>\n","protected":false},"excerpt":{"rendered":"

Washington, DC, March\u00a025, 2021\u2014The National Organization for Rare Disorders (NORD\u00ae)\u00a0today announced the\u00a0findings of a new study that details\u00a0the\u00a0number\u00a0of\u00a0orphan products,\u00a0generics and biosimilars\u00a0available to treat rare diseases.\u00a0NORD\u00a0commissioned\u00a0Avalere to conduct\u00a0the\u00a0analysis\u00a0to examine if\u00a0laws and … <\/p>\n

Continue reading “New Study Investigates the Number of Available Orphan Products, Generics and Biosimilars”<\/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":[190,210,504,193],"tags":[2726,398,270,244,2720,262,263,199,292,1623,2032],"class_list":["post-59269","post","type-post","status-publish","format-standard","hentry","category-featured-news","category-industry","category-press-releases","category-research","tag-avalere","tag-biosimilars","tag-drug-development","tag-fda","tag-generics","tag-orphan-drug-act","tag-orphan-drugs","tag-orphan-products","tag-patent","tag-rareinsights","tag-us-food-and-drug-administration"],"acf":[],"_links":{"self":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/59269","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=59269"}],"version-history":[{"count":0,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/59269\/revisions"}],"wp:attachment":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/media?parent=59269"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/categories?post=59269"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/tags?post=59269"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}