Momwe Kafukufuku ndi Kukula kwa Mankhwala Osamalira Ana Amasiye Amakhudzidwa
Kodi mankhwala osungirako amasiye ndi chiyani ndi Orphan Drug Act? Nchifukwa chiyani kafukufuku ndi chitukuko cha mankhwalawa ndi zofunika ndipo ndizo zotani?
Kodi mankhwala osokoneza bongo ndi chiyani? - Tanthauzo
Mankhwala osamalira ana amasiye ndi mankhwala (mankhwala) omwe amakhalabe opanda ntchito chifukwa cha kusowa kwa kampani kupeza mankhwala opindulitsa. Kawirikawiri chifukwa chomwe mankhwalawo sagwiritsire ntchito ndikuti pali anthu ochepa omwe angagule mankhwalawa poyesedwa ndi kufufuza ndi chitukuko chofunikira kupanga mankhwalawa.
Mmawu osavuta, mankhwala osungirako ana amasiye ndi omwe makampani sakuyembekeza kuti azipeza ndalama zambiri, ndipo m'malo mwake amayendetsa zoyesayesa zawo pa mankhwala omwe amabweretsa ndalama.
Chifukwa Chimene Mankhwala Osokoneza Bongo ali "Mankhwala Osokoneza Bongo"
Makampani opanga mankhwala ndi mankhwala osokoneza bongo akufufuza nthawi zonse ndikupanga mankhwala atsopano kuti athe kuchipatala, ndipo mankhwala atsopano amabwera pamsika nthawi zambiri. Anthu omwe amavutika ndi matenda omwe sawoneka kapena osokonezeka, komano, samawona momwe kafukufuku wamankhwala amachitira chidwi ndi matenda awo. Izi zili choncho chifukwa chiwerengero chawo ndi chaching'ono ndipo msika wogulitsa mankhwala atsopano ochizira matendawa omwe amachititsa kuti azidwala matendawa amakhalanso ochepa.
Nthenda yosaoneka imapezeka pakati pa anthu oposa 200,000 ku United States kapena osachepera 5 pa anthu 10,000 ku European Union. Mabungwe olamulira a boma ku United States ndi European Union atenga njira zothetsera kusiyana kumeneku mu chitukuko cha mankhwala
Zowonjezera Kuonjezera Kukula kwa Mankhwala Osamalira Ana Amasiye
Kuzindikira kuti mankhwala okwanira pa zovuta zosayembekezereka sizinapangidwe ku US ndipo makampani osokoneza bongo angapangitse ndalama kukulitsa mankhwala osokoneza bongo chifukwa cha zovuta, US Congress inagwiritsa ntchito Orphan Drug Act mu 1983.
Ofesi ya US of Orphan Product Development
Dongosolo la US Food and Drug Administration (FDA) liri ndi udindo woonetsetsa kuti chitetezo ndi kupambana kwa mankhwala pamsika ku United States.
A FDA adakhazikitsa Office of Orphan Product Development (OOPD) kuthandiza kuthandizira mankhwala osamalira ana amasiye (ndi mankhwala ena ovuta), kuphatikizapo kupereka zopereka zofufuzira.
Mankhwala osamalira ana amasiye, monga mankhwala ena, adapezekanso otetezeka ndi ogwira ntchito kudzera mu kafukufuku ndi mayesero am'chipatala pamaso pa FDA.
Mchaka cha 1983 cha US Orphan Drug Act
Buku la Orphan Drug Act limalimbikitsanso makampani kukhala ndi mankhwala osokoneza bongo (komanso mankhwala ena) pamisika yaing'ono ya anthu omwe ali ndi vuto lalikulu (ku US, 47 peresenti ya zovuta zomwe zimachitika kawirikawiri zimakhudza anthu oposa 25,000). Izi zimaphatikizapo:
- Zopereka za msonkho za boma zafukufuku (mpaka 50 peresenti ya ndalama) kuti akule mankhwala osungirako amasiye.
- Chigwirizano chokhazikika cha zaka 7 chogulitsa mankhwala osokoneza bongo kwa kampani yoyamba kupeza mgwirizano wa FDA kulandira mankhwala enaake. Izi zikugwiritsidwa ntchito pokhapokha kugwiritsa ntchito mankhwalawa. Ntchito ina yogwiritsiridwa ntchito mosiyana ingagwirizanitsidwe ndi FDA, ndipo kampaniyo ikanakhala ndi ufulu wotsatsa malonda kwa mankhwalawa.
- Zowonjezera ndalama zothandizira mankhwala ndi madola a pachaka a FDA.
Asanayambe kuchoka kwa Orphan Drug Act, mankhwala ochepa amasiye analipo kuti athetse matenda osadziwika.
Kuchokera mu Act, mankhwala oposa ana amasiye oposa 200 avomerezedwa ndi a FDA pofuna kugulitsa ku US
Zotsatira za Mchitidwe Wamasiye wa Orphan ku US Pharmaceuticals
Popeza kuti Act Orrug Drug Act inayamba mu 1983, yakhala ikuyambitsa mankhwala ambiri. Pofika chaka cha 2012 panali mankhwala osachepera 378 omwe avomerezedwa kupyolera mu ndondomekoyi, ndipo nambala ikupitiriza kukwera.
Chitsanzo cha Mankhwala Opezeka Chifukwa cha Orphan Drug Act
Zina mwa mankhwala omwe amavomereza ndi awa:
- Adrenocorticotropic hormone (ACTH) yochizira matenda ochepa
- Tetrabenzine pochiza chorea yomwe imapezeka mwa anthu omwe ali ndi matenda a Huntington
- Matenda opatsirana a enzyme kwa omwe ali ndi matenda osungirako glycogen, matenda a Pompe
Kafukufuku Wadziko lonse ndi Kupititsa patsogolo Mankhwala Osokoneza Bongo
Mofanana ndi US Congress, boma la European Union (EU) lazindikira kufunikira koonjezera kafukufuku ndi chitukuko cha mankhwala osungirako ana amasiye.
Komiti ya Mankhwala Osamalira Ana Amasiye
Yakhazikitsidwa mu 1995, European Medicines Agency (EMEA) ndiyo yowonetsetsa kuti chitetezo ndi kupindulitsa kwa mankhwala pamsika ku EU. Zimabweretsa pamodzi zokhudzana ndi sayansi m'mayiko 25 a EU. Mu 2000, Komiti ya Orphan Medicinal Products (COMP) inakhazikitsidwa kuti iyang'anire chitukuko cha mankhwala osamalira ana amasiye ku EU.
Malamulo pa Mankhwala Osamalira Ana Amasiye
Lamulo la Mankhwala Osamalira Ana Amasiye, loperekedwa ndi European Council, limapereka chisonkhezero chothandizira kusamalira ana amasiye (ndi mankhwala ena chifukwa cha matenda osadziwika) ku EU, kuphatikizapo:
- Zowonetsera ndalama zokhudzana ndi malonda ovomerezeka.
- Mayi wokhala ndi zaka 10 wokhazikika pa malonda a mankhwala osokoneza bongo kwa kampani yoyamba kupeza mgwirizano wa EMEA wovomerezeka wa mankhwala. Izi zikugwiritsidwa ntchito pokhapokha kugwiritsa ntchito mankhwalawa.
- Chilolezo chogulitsa malonda - ndondomeko yapadera ya chilolezo chogulitsa chomwe chikufikira mayiko onse a EU.
- Kuthandizidwa kwa pulojekiti, kutanthauza kupereka kwa uphungu wa sayansi kwa makampani osokoneza bongo za mayesero osiyanasiyana ndi mayesero a chipatala ofunikira mankhwala.
Lamulo la Mankhwala Osamalira Ana Amasiye lakhala ndi zotsatira zabwino zomwezo ku EU kuti Orphan Drug Act inali ku US, ikuchulukitsa kwambiri chitukuko ndi malonda a mankhwala osungirako ana amasiye chifukwa cha matenda osadziwika.
Mfundo Yofunika Kwambiri pa Milandu ya Mankhwala Osamalira Ana Amasiye
Pa nthawiyi pali kutsutsana kwakukulu pa lamulo la Orphan Drug Act, ndi kufunikira kwa chithandizo cha matenda osadziwika kumbali imodzi ya msinkhu, ndi mafunso okhudzana ndi chitukuko. Mwamwayi, ntchito izi, ku United States ndi ku Ulaya zakhala zikudziwitsa za matenda ambiri omwe sali osowa, omwe, pamene awonjezeredwa pamodzi, si onse omwe sali achilendo.
Zotsatira:
Herder, M. Kodi cholinga cha Orphan Drug Act ndi chiyani? . PLoS Medicine . 2017. 14 (1): e1002191.
Murphy, S., Puwanant, A., ndi R. Griggs. Zotsatira zosayembekezereka za Mtundu Wopanda Chiberekero Wopatsa Mavuto Ambiri Okhudza Mitsempha. Annals of Neurology . 2012. 72 (4): 481-490.
US Food and Drug Administration. Kupanga Mankhwala Osamalira Ana Amasiye: Mankhwala Osokoneza Bongo ndi Zamoyo. Kusinthidwa 05/02/16. https://www.fda.gov/forindustry/developingproductsforrarediseasesconditions/howtoapplyfororphanproductdesignation/default.htm