Indlela Uphando noPhuhliso lweeNyango zeNtsholongwane zikhuthazwa ngayo
Yintoni yonyango lenkedama kunye naluphi uMthetho weNtsholongwane? Kutheni uphando kunye nophuhliso lwalezi ziyobisi zibalulekile kwaye zeziphi imiqobo?
Iyintoni i-Orph? - Inkcazo
Iyeza lenkedama liyeza (imichiza) ehlala lingakhuselekanga ngenxa yokungabi nenkampani yokufumana inzuzo yonyango. Ngokuqhelekileyo isizathu sokuba isilwanyana esingenakuncedo kukuba kukho abantu abambalwa abaza kuthengisa iziyobisi xa belinganiswa nophando kunye nophuhliso olufunekayo ukuvelisa izidakamizwa.
Ngamazwi alula, iziyobisi zezintandane zizo ezo nkampani ezingalindelekanga ukuba zenze imali eninzi, kwaye kunoko zichaze imizamo yazo kwiziyobisi eziza kuzisa imali.
Kutheni ezinye iziyobisi ziyi "Orphan drugs"
Iinkampani zamachiza (iziyobisi) kunye neenkampani ze-biotech zihlala ziphanda kwaye ziphuhlise amayeza amachiza ukunyanga iimeko zonyango, kwaye amatsha amachiza aya kwiimarike rhoqo. Abantu abahluphekileyo kwizifo ezingaqhelekiyo okanye ukuphazamiseka, ngakolunye uhlangothi, ababonanga ingqwalaselo efanayo yophando lweziyobisi kwizifo zabo. Oku kubangelwa ukuba amanani abo amancinci kwaye ngoko ke i-market inokusetyenziswa kwamachiza amasha ukuphathwa kwezi zifo ezingaqhelekanga (ezibizwa ngokuthi "izidakamizwa zezintandane") zincinci.
Isifo esinqabileyo sisenzeka kubantu abangaphantsi kwama-200,000 e-United States okanye ngaphantsi kwama-5 ngabanye abantu abayi-10 000 kwi-European Union. Amaziko olawulo karhulumente e-United States kunye neYurophu yaseYurophu baye bathatha amanyathelo okunciphisa ukungafani nokuphuhliswa kweziyobisi
Izikhuthazo zokunyusa ukuPhuculo lweeNyununu
Ukuqaphela ukuba izidakamizwa ezaneleyo ezinokungabonakali kweziphazamiso azizange zenziwe kwi-US kwaye iinkampani zonyango ziza kubakho ukulahleka kwezemali ekuphuhliseni iziyobisi kwiimeko ezingaqhelekanga, i-US Congress yadlulisela uMthetho we-Orphan Drug Act ngo-1983.
I-Ofisi ye-US yeNtetho yeNkqubela
I-US Food Management and Drug Administration (i-FDA) inoxanduva lokuqinisekisa ukhuseleko nokusebenza kwamachiza kwiimarike e-United States.
I-FDA yasungula i-Ofisi yeNtuthuko yeMveliso yeNtsapho (OOPD) ukukunceda ekuphuhlisweni kweziyobisi zezintandane (kunye nezinye iimveliso zonyango ngenxa yezifo ezingavamile), kuquka ukunikela ngemali yocwaningo.
Iidakamizwa zeengcongolo, njengamanye amayeza, zifunyenwe zikhuselekile kwaye ziphumelele ngophando kunye nezilingo zekliniki phambi kokuba i-FDA iza kuvuma ukuthengisa.
Umthetho we-Ork Drug Act ka-1983
Umthetho we-Orphan Drug Act ukhuthaza abantu ukuba bavelise iziyobisi (kunye nezinye iimveliso zonyango) kwiimarike ezincinci zabantu ngabanye abaneengxaki ezingavamile (e-US, 47 ekhulwini lweengxaki ezingaqhelekanga zithintela abantu abangaphantsi kwama-25,000). Ezi khuthazo ziquka:
- Izikripthi zerhafu zaseburhulumenteni zenzelwe uphando (ukuya kuma-50 ekhulwini weendleko) ukuphuhlisa isicatshulwa sezintandane.
- Unyaka oneminyaka engama-7 wokuzithengisa ngokuthengisa iziyobisi kwinkampani yokuqala ukufumana imvume yokuthengiswa kwe-FDA yezilwanyana ezithile. Oku kusebenza kuphela ekusebenziseni okuvunyiweyo kweziyobisi. Esinye isicelo sokusetyenziswa okuhlukileyo singagunyazwa yi-FDA, kwaye inkampani iya kuba namalungelo okuthengisa okukhethekileyo kwilisi ekusebenziseni.
- Imirhumo yemirhumo yemvume yokuvunywa kwezidakamizwa kunye neentlawulo zeemveliso ze-FDA zonyaka.
Ngaphambi kokugqithiswa komthetho we-Orphan Drug Act, izidakamizwa ezimbalwa zezintandane zifumaneke ukuphatha izifo ezingaqhelekanga.
Ukususela kuMthetho, amayeza angama-200 angama-orless avunywe yi-FDA yokuthengisa eMelika
Umphumo we-Orphan Drug Act kwi-US Pharmaceuticals
Ukususela ngoMthetho we-Orphan Drug Act waba ngowama-1983, kuye kwaba noxanduva lokuphuhliswa kwamachiza amaninzi. Ngo-2012 kukho ubuncinane izidakamizwa ezingama-378 ezivunyiwe ngale nkqubo, kwaye inamba iyaqhubeka ukunyuka.
Umzekelo WeeDrugs ezikhoyo Ngenxa yoMthetho weeNtsholongwane
Phakathi kweziyobisi ezivumile zibandakanya ezo zinto:
- I-adrenocorticotropic hormone (ACTH) yokunyanga i- spasms infasms
- I-Tetrabenzine yokunyanga i-chorea eyenzeka kubantu abanesifo sikaHuntington
- I-enzyme yonyango yokutshintshwa kulabo abane-glycogen disorder disease, isifo sikaPompe
Uphando lwaMazwe ngamazwe kunye noPhuhliso lweeNyununu
Njenge-US Congress, urhulumente we-European Union (EU) uye waqonda isidingo sokwandisa uphando nophuhliso lweziyobisi zeengcongolo.
IKomiti yeeNtsholongwane zoNyango
Eyilwe ngo-1995, i-European Medicine Agency (EMEA) inoxanduva lokuqinisekisa ukhuseleko nokusebenza kwamachiza kwiimarike kwi-EU. Idibanisa imithombo yenzululwazi ye-25 Ilungu lamaLungu ase-EU. Ngo-2000, iKomiti yeeNtsholongwane zeNtsholongwane (COMP) yasungulwa ukuba ijongelwe ukuphuhliswa kweziyobisi zeengcongolo kwi-EU.
Ummiselo kwiiNtsholongwane zeeNtsana
Ummiselo weeMpilo zoNyango, ogqitywe yiBhunga leYurophu, unika izikhuthazo ekuphuhlisweni kweziyobisi zezintandane (kunye nezinye iimveliso zonyango kwiingxaki ezingavamile) kwi-EU, kuquka:
- Imivuzo yeemirhumo eziphathelele kwinkqubo yokumaketha yokuthengisa.
- Unyaka oneminyaka eli-10 othembekileyo ekuthengiseni iziyobisi kwinkampani yokuqala ukufumana imvume yokuthengisa i-EMEA. Oku kusebenza kuphela ekusebenziseni okuvunyiweyo kweziyobisi.
- Ugunyaziso lokuthengiswa koluntu - inkqubo ephambili yokuthengiswa kwegunya lokudlulisela kuzo zonke iilungu ze-EU.
- Uncedo lweProtocol, olubhekiselele kunikezelo lweengcebiso zenzululwazi kwiinkampani zonyango malunga neemvavanyo ezahlukahlukeneyo kunye nezilingo zamachiza eziyimfuneko ezifunekayo ukuba isilwanyana siphuhliswe.
Ummiselo weeNtsholongwane zeNtsholongwane uye waba nempembelelo efanayo kwi-EU yokuba uMthetho we-Orphan Drug Act wayenabo e-US, ukwandisa kakhulu ukuphuhliswa nokuthengiswa kweziyobisi zeenkedama kwiintsholongwane ezingavamile.
Ngaphantsi koMthetho weeNtsholongwane
Ngelo xesha kukho impikiswano enkulu kwi-Orphan Drug Act, eneemfuneko zokonyango kwizifo ezingaqhelekanga kwicala elinye, kunye nemibuzo malunga nokuzinza kwenye. Ngombulelo, ezi zenzo, e-United States naseYurophu ziye zaphakamisa ukuqwashisa ngezifo ezininzi ezingabonakaliyo, eziya kuhlanganiswa ndawonye, akuzona zonke izinto ezingaqhelekanga.
Imithombo:
I-Herder, M. Iyintoni injongo yoMthetho weeNtsholongwane? . PLoS Medicine . 2017. 14 (1): e1002191.
Murphy, S., Puwanant, A., no R. Griggs. Iimpawu ezingalindelekanga zeNkcazo yeMveliso yeNkathazo yeeNtsholongwane zeNtsholongwane. Annals of Neurology . 2012. 72 (4): 481-490.
US Food and Drug Administration. Ukutyunjwa kweNkcazo yeMveliso: Iziyobisi kunye neMveliso yeBilo. Ukuhlaziywa 05/02/16. https://www.fda.gov/forindustry/productionsproductionsproductsforrarediseasesconditions/howtoapplyfororphanproductdesignation/default.htm