{"id":2901,"date":"2019-05-02T09:21:58","date_gmt":"2019-05-02T13:21:58","guid":{"rendered":"https:\/\/colpittsclinical.com\/?p=2901"},"modified":"2022-08-29T01:58:04","modified_gmt":"2022-08-29T05:58:04","slug":"improving-access-and-affordability-of-new-orphan-drugs-while-ensuring-continuous-investment","status":"publish","type":"post","link":"https:\/\/colpittsclinical.com\/ro\/improving-access-and-affordability-of-new-orphan-drugs-while-ensuring-continuous-investment\/","title":{"rendered":"\u00cembun\u0103t\u0103\u021birea accesului \u0219i a accesibilit\u0103\u021bii noilor medicamente orfane, asigur\u00e2nd \u00een acela\u0219i timp investi\u021bii continue"},"content":{"rendered":"<p>\u0218tim cu to\u021bii c\u0103, \u00een \u00eentreaga lume, exist\u0103 o nevoie complex\u0103 \u0219i continu\u0103 de medicamente noi pentru tratarea cancerului, a bolilor imunologice \u0219i a bolilor rare \u0219i orfane.  Provocarea de a aduce noi medicamente pe pia\u021b\u0103 pentru a trata aceste tipuri de indica\u021bii se confrunt\u0103 cu cre\u0219terea costurilor de dezvoltare, produc\u021bie \u0219i comercializare a acestor noi medicamente.  Ca urmare a acestei probleme continue, Congresul mondial al medicamentelor orfane 2019 a reunit un grup de lideri realiza\u021bi din lumea dezvolt\u0103rii bolilor rare \u0219i orfane pentru a discuta despre inova\u021biile din cadrul industriei \u0219i, \u00een unele cazuri, despre propriile lor companii, pentru a ajuta la men\u021binerea echilibrului \u00eentre acces \u0219i accesibilitate.<\/p>\n<p>Dan Faga, CEO al Spark Therapeutics, a oferit o discu\u021bie conving\u0103toare \u00een cadrul c\u0103reia a vorbit despre crearea Spark Path, care \u201cofer\u0103 o<em>rabaturi bazate pe rezultate \u0219i un model de contractare inovator care sprijin\u0103 accesul pacien\u021bilor \u00een SUA, urm\u0103rind \u00een acela\u0219i timp reducerea riscului \u0219i a sarcinii financiare pentru pl\u0103titori \u0219i centrele de tratament.\u201d  \u00cen Spark Path este inclus un program de reduceri pentru pacien\u021bi, prin care ace\u0219tia primesc o reducere \u00een anul 1 \u0219i \u00een anul 3 dac\u0103 produsul pe care \u00eel iau nu func\u021bioneaz\u0103.\u00a0 <\/em><\/p>\n<p><em>De pe site-ul Spark: \u201c<\/em>LUXTURNA \u0219i alte poten\u021biale terapii unice se confrunt\u0103 cu provoc\u0103ri unice \u00een materie de asigur\u0103ri de s\u0103n\u0103tate, av\u00e2nd \u00een vedere practicile \u0219i reglement\u0103rile actuale din sistemul de s\u0103n\u0103tate din SUA. Printre obstacolele \u00een calea ofertei de modele alternative se num\u0103r\u0103 accentul pus de sistem pe valoarea pe termen scurt (\u00een mare parte deoarece majoritatea pacien\u021bilor schimb\u0103 companiile de asigur\u0103ri de s\u0103n\u0103tate \u00een medie la fiecare trei ani), cerin\u021bele guvernamentale de raportare a pre\u021burilor care nu sunt concepute pentru a reflecta anumite acorduri bazate pe rezultate, limit\u00e2nd astfel capacitatea unui produc\u0103tor de a oferi reduceri semnificative bazate pe performan\u021b\u0103, \u00een special pentru bolile cu popula\u021bii mici de pacien\u021bi \u0219i modele de distribu\u021bie complicate, care adaug\u0103 costuri \u0219i riscuri financiare p\u0103r\u021bilor implicate \u00een furnizarea \u0219i rambursarea medicamentelor de specialitate \u0219i a asisten\u021bei medicale specializate.\u201d<\/p>\n<p>Dl Faga a continuat s\u0103 explice c\u0103, ca urmare a Spark Path, Spark Therapeutics a tratat mai mul\u021bi pacien\u021bi \u00eentr-un an dup\u0103 aprobare dec\u00e2t \u00een 10 ani \u00een timpul dezvolt\u0103rii.<\/p>\n<p>\u00cen plus, Emil Kakkis, CEO al Ultragenyx, a declarat c\u0103 \u201cfiecare pacient care ar beneficia ar trebui s\u0103 aib\u0103 acces\u201d.  Ultragenyx a propus un model de stabilire a pre\u021burilor mai moderat, cu promisiunea pl\u0103titorilor de a sprijini \u00een mod egal pacien\u021bii adul\u021bi \u0219i pediatrici.  Kakkis a continuat s\u0103 explice c\u0103, din punct de vedere istoric, preferin\u021ba pl\u0103titorilor a fost s\u0103 se concentreze pe pediatrie, deoarece ace\u0219tia au o nevoie nesatisf\u0103cut\u0103 perceput\u0103 mai mare.  Cu toate acestea, Kakkis este ferm convins c\u0103 este imperativ ca to\u021bi pacien\u021bii s\u0103 aib\u0103 acces dac\u0103 ar putea beneficia de medicament, nu doar cei cu o nevoie neacoperit\u0103 perceput\u0103 mai mare.  \u00cen concluzie, el a citat un exemplu de inovare din partea dezvoltatorului de medicamente Vertex din Irlanda, care a convenit cu sistemul de s\u0103n\u0103tate irlandez s\u0103 trateze to\u021bi pacien\u021bii la un pre\u021b stabilit - un exemplu conving\u0103tor de inovare care, \u00een opinia sa, ar putea fi benefic pentru sistemul american.<\/p>\n<p>\u00cen mod clar, nu exist\u0103 o solu\u021bie simpl\u0103 la aceast\u0103 problem\u0103 complex\u0103.  Cu toate acestea, atunci c\u00e2nd liderii industriei noastre aleg s\u0103 colaboreze \u0219i s\u0103 inoveze \u00eempreun\u0103 pentru a impulsiona schimbarea \u00een vederea asigur\u0103rii unui acces \u0219i a unei accesibilit\u0103\u021bi mai mari, toat\u0103 lumea beneficiaz\u0103 - inclusiv cel mai important membru al acestei discu\u021bii, pacientul.<\/p>\n<p>&nbsp;<\/p>","protected":false},"excerpt":{"rendered":"<p>We all know that there is a complex and continued unmet need for new medicines across the Globe to treat cancer, immunological and rare and orphan diseases.\u00a0 The challenge to bring new drugs to market to treat these types of indications is met with the rising cost of developing, producing and marketing these new drugs.\u00a0 [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":2904,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_exactmetrics_skip_tracking":false,"_exactmetrics_sitenote_active":false,"_exactmetrics_sitenote_note":"","_exactmetrics_sitenote_category":0,"_jetpack_memberships_contains_paid_content":false,"footnotes":""},"categories":[6,9],"tags":[],"class_list":["post-2901","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-clinical-trials","category-events"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>improving-access-of-new-orphan-drugs<\/title>\n<meta name=\"description\" content=\"It is a challenge to bring new drugs to market. 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