Nzira yekutsvakurudza nekugadziriswa kwemishonga inherera inobatanidzwa
Chii chiri chirwere chemherera uye chii chiri Orphan Drug Act? Sei kutsvakurudza nekuvandudzwa kwemishonga iyi zvichikosha uye ndezvipi zvipingamupinyi?
Chii chinonzi Orphan Drug? - Tsanangudzo
Imwe nherera mushonga mishonga (mishonga) iyo inoramba ichisimbiswa nekuda kwekushaya kwekambani yekuwana mishonga inobatsira. Kakawanda chikonzero chokuti mushonga uyu haubatsiri ndeyekuti kune vanhu vashomanana vanozotenga mishonga kana vakaremerwa nekutsvakurudza uye kubudirira kunoda kugadzira mishonga.
Mumashoko akajeka, mishonga yemherera ndeye iyo makambani asingatarisiri kuita mari yakawanda kubva, uye panzvimbo payo anongedzera kuedza kwavo pamishonga inodhaka ichaunza mari.
Chikonzero Nei Mishonga Inodhaka "Inherera Dhiragi"
Mishonga inodhaka (zvinodhaka) uye yekodhikiki inogara iri kutsvakurudza nekuvandudza mishonga mitsva yekurapa mishonga, uye mishonga mitsva inouya pamusika kazhinji. Vanhu vanotambura nezvirwere zvisingawanzoitiki kana kushungurudzwa, kune rumwe rutivi, havaoni mutsvaga wekutsvakurudza mishonga yekucherechedza zvirwere zvavo. Izvi ndezvokuti nhamba yavo iduku uye saka misika inogona kuwanikwa yemishonga mitsva yekurapa zvirwere zvisingawanzozivikanwa (zvinowanzonzi "nherera dzinodhaka") zvakare shoma.
Chirwere chisinganzwisisiki chinowanikwa pasi pevanhu vanosvika 200 000 muUnited States kana pasi pe5 kusvika kune vanhu 10 000 muEuropean Union. Hurumende dzehurumende dzekutonga muUnited States neEuropean Union dzakatora matanho ekuderedza kupesana uku mukukudzwa kwemishonga
Zvinokurudzira Kuwedzera Kuvandudzwa kweAraphan Drugs
Kuziva kuti mishonga yakakwana yezvinetso zvisingawanzove yakasimbiswa muU.S. uye iyo makambani emishonga aizopinza mari yekurasikirwa mumari mukudzidzira mishonga zvisingawanzoitiki, US Congress yakapa Orphan Drug Act muna 1983.
US Office Of Orphan Product Development
IU.S. Food and Drug Administration (FDA) inofanirwa kuchengetedza kuchengetedza uye kubudirira kwemishonga pamusika muUnited States.
Iyo FDA yakasimbisa Hhodhi yeOrphan Product Development (OOPD) kuti ibatsire pakuchengetwa kwemishonga yeherera (nezvimwe zvipatara zvemishonga nokuda kwezvinetso zvisingawanzoitiki), kusanganisira kupa zvipo zvekutsvakurudza.
Mishonga yemherera, semimwe mishonga, inofanira kuwanikwa yakachengeteka uye inobudirira kuburikidza nekutsvakurudza uye kuongororwa kwemakiriniki asati FDA avabvumira kuti vatengese.
I 1983 US Orphan Drug Act
The Orphan Drug Act inopa zvikurudzira kuita makambani kukura mishonga (uye mamwe mishonga yezvokurapa) pamakwenzi maduku evanhu vane zvinetso zvisingawaniki (muU.S., 47 muzana yezvinetso zvisingawanzoitika zvinokanganisa vashoma kupfuura 25 000 vanhu). Izvi zvinokurudzira zvinosanganisira:
- Nhepfenyuro yemitero yehurumende yekutsvakurudza kwakaitwa (kusvika ku50 muzana yemari) kukura nherera yemishonga.
- Imwe yemakore manomwe yakagadziriswa yekutengeswa kwezvinodhaka kwekambani yekutanga kugamuchira FDA kugamuchirwa kwekutengeswa kweimwe mishonga. Izvi zvinoshanda chete kune kushandiswa kunobvumidzwa kwechirwere. Chimwe chekushandiswa kwekushandiswa kwakasiyana kunogona kugamuchirwa neDFA, uye kambani inenge iine kodzero dzekutengeserana chete dzechirwere chekushandisa iko zvakare.
- Waivers yemishonga yemvumo yekubhadharwa mari yekushandiswa uye yegore reDFA michina yemari.
Isati yasvika yeAralphan Drug Act, mashoma emishonga emherera aivepo kuti apore zvirwere zvisingawanzoitiki.
Sezvo Mutemo, mishonga inopfuura 200 yemherera yakagamuchirwa neDDA yekutsvaga muUnited States
Migumisiro yeOrphan Drug Act muUnited States Pharmaceuticals
Sezvo Orphan Drug Act yakatanga kuva muna 1983, yakave ichiita zvekuvandudzwa kwemishonga yakawanda. Pakazosvika gore ra2012 pakanga paine madzimai makumi matatu nemazana matatu nemazana matatu (378) akave agamuchirwa nenzira iyi, uye nhamba ichi inoramba ichikwira.
Muenzaniso weMishonga Inowanikwa Pamusoro peOrphan Drug Act
Pakati pezvinodhaka izvo zvakagamuchirwa zvinosanganisira izvo zvakadai se:
- Adrenocorticotropic hormone (ACTH) yekurapa vana vaduku
- Tetrabenzine yekurapa chorea inowanikwa kuvanhu vane chirwere chaHuntington
- Enzyme replacement therapy kune avo vane chirwere chokuchengetedza glycogen, chirwere chePompe
International Research and Development for Orphan Drugs
Kufanana neU.S. Congress, hurumende yeEuropean Union (EU) yakagamuchira kudiwa kwekuwedzera kutsvakurudza uye kukura kwemishonga yemherera.
Komiti yeAraphan Mishonga
Yakasimbiswa muna 1995, European Medicines Agency (EMEA) inotarisira kuchengetedza nekubudirira kwemishonga pamusika muEU. Inounza pamwe chete masayenzi ezvesayenzi 25 EU Union States. Muna 2000, Komiti yeAraphan Mishonga (COMP) yakagadzirirwa kutarisira kusimbiswa kwemishonga yemherera muEU.
Mutemo weNherera Mishonga
Murairo weNherera Mishonga, yakapfuudzwa neEuropean Council, inopa zvikurudzira zvekugadzirwa kwemishonga yeherera (nezvimwe zvezvokurapa zvinokonzerwa nezvinetso zvisingawanzo) muEU, kusanganisira:
- Waivers yemari inofanirwa nekugadzirwa kwemvumo yekutsvaga.
- Imwe yemakore gumi yakavimbiswa yekutengeswa kwezvinodhaka kwekambani yekutanga kuwana mvumo yekudhinda EMEA yemishonga. Izvi zvinoshanda chete kune kushandiswa kunobvumidzwa kwechirwere.
- Mutemo wekutengeserana kwemagariro - chirongwa chepakati pekutengeserana kunoendesa kune dzimwe nhengo dzenhengo dzeEU.
- Kubatsirwa kwePurogiramu, zvinoreva kupiwa kwezano rezvesayenzi kumakambani emishonga nezvekuedza kwakasiyana-siyana uye kuongororwa kwekliniki kunodiwa kuti mishonga ichigadziridzwe.
Murairo weNherera Mishonga yakave nemigumisiro yakafanana muUnited States iyo Orphan Drug Act yaive muU.S., ichiwedzera zvikuru kukura nekushambadzira kwemishonga yenherera kune zvinetso zvisingawanzoitiki.
Pasi pepamusoro paOrphan Drug Act
Panguva iyo pane kukakavara kukuru pamusoro peOrphan Drug Act, pamwe nekuda kwekurapa kwezvirwere zvisingaoneki kune rumwe rutivi rwechechi, uye mibvunzo pamusoro pekusimudzira pane imwe. Ndinofara, zviito izvi, muUnited States neEurope zvakasimudzira ruzivo rwezvirwere zvakawanda zvisingadikanwi, iyo, kana yakawedzerwa pamwe chete, hazvisi zvese zvisingawanzoitiki.
Sources:
Herder, M. Chinangwa chei Orphan Drug Act? . PLoS Medicine . 2017. 14 (1): e1002191.
Murphy, S., Puwanant, A., naRo Griggs. Migumisiro isingadikanwi yeNherera Mutengo Mukurumbira weNorth Neurological Disorders. Annals of Neurology . 2012. 72 (4): 481-490.
US Food and Drug Administration. Kusarudzwa Nherera Mutengo: Zvinodhaka uye Zvigadzirwa zveMagetsi. Updated 05/02/16. https://www.fda.gov/forindustry/developingproductsforrarediseasesconditions/howtoapplyfororphanproductdesignation/default.htm