Hindustan Abdul Ahad, Chinthaginjala Haranath, Bhupalam Pradeep Kumar, Debaraj Roy, Bhavani Haribabu Sai Dharani, Momin Umaira Ayisha
Hindustan Abdul Ahad1*, Chinthaginjala Haranath1, Bhupalam Pradeep Kumar2, Debaraj Roy2, Bhavani Haribabu Sai Dharani2, Momin Umaira Ayisha2
1Department of Industrial Pharmacy, Raghavendra Institute of Pharmaceutical Education and Research (RIPER), Ananthapuramu – 515721 Andhra Pradesh, India.
2Department of Pharmacology, Raghavendra Institute of Pharmaceutical Education and Research (RIPER), Ananthapuramu – 515721 Andhra Pradesh, India.
Volume - 11,
Issue - 3,
Year - 2021
This article gives a brief knowledge about orphan diseases and drugs. An orphan disease is a rare illness lack market and resource data for the treatment and the drugs used for their treatment are called orphan drugs. The pharmaceutical firms show less interest in the development of drugs to treat them, and lack profit without government assistance. The authors reviewed published scientific literature and grouped a broad list of Orphan diseases and orphan drugs. The article summarizes the entire list of orphan diseases and drugs with their description.
Cite this article:
Hindustan Abdul Ahad, Chinthaginjala Haranath, Bhupalam Pradeep Kumar, Debaraj Roy, Bhavani Haribabu Sai Dharani, Momin Umaira Ayisha. Asian Journal of Research in Pharmaceutical Sciences. 2021; 11(3):247-2. doi: 10.52711/2231-5659.2021.00039
Hindustan Abdul Ahad, Chinthaginjala Haranath, Bhupalam Pradeep Kumar, Debaraj Roy, Bhavani Haribabu Sai Dharani, Momin Umaira Ayisha. Asian Journal of Research in Pharmaceutical Sciences. 2021; 11(3):247-2. doi: 10.52711/2231-5659.2021.00039 Available on: https://ajpsonline.com/AbstractView.aspx?PID=2021-11-3-11
1. Kontoghiorghe CN, Andreou N, Constantinou K, Kontoghiorghes GJJWjom. World health dilemmas: orphan and rare diseases, orphan drugs and orphan patients. 2014;4(3):163.
2. Outterson KJAjol, medicine. Should access to medicines and TRIPS flexibilities be limited to specific diseases? 2008;34(2-3):279-301.
3. Scheindlin SJMi. Rare diseases, orphan drugs, and orphaned patients. 2006;6(4):186.
4. Cook NS, Cave J, Holtorf A-PJFim. Patient preference studies during early drug development: aligning stakeholders to ensure development plans meet patient needs. 2019;6:82.
5. Ferreira CRJAJoMGPA. The burden of rare diseases. 2019;179(6):885-892.
6. Song P, Gao J, Inagaki Y, Kokudo N, Tang WJI, research rd. Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives. 2012;1(1):3-9.
7. Abhi S, Venugopal V, Shastri S. Social Entrepreneurship—Building Sustainability Through Business Models and Measurement of Social Impact. Entrepreneurial Ecosystem: Springer; 2015:295-323.
8. Daniel MG, Pawlik TM, Fader AN, Esnaola NF, Makary MAJAjoco. The Orphan Drug Act: restoring the mission to rare diseases. 2016;39(2):210-213.
9. Shulman SR, Manocchia MJP. The US orphan drug Programme. 1997;12(3):312-326.
10. Zimmerman BJRoPR. Orphan living situations in Malawi: A comparison of orphanages and foster homes. 2005;22(6):881-917.
11. O’Sullivan BP, Orenstein DM, Milla CEJJ. Pricing for orphan drugs: will the market bear what society cannot? 2013;310(13):1343-1344.
12. Shih A, Duong A, Tao C, et al. Clinical and economic evidence thresholds for orphan drugs: Are requirements for favorable health technology assessment and reimbursement on the rise? 2013;16(3):A108.
13. Gibson S, von Tigerstrom BJJoL, Biosciences t. Orphan drug incentives in the pharmacogenomic context: policy responses in the US and Canada. 2015;2(2):263-291.
14. Joppi R, Garattini SJEJocp. Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU. 2013;69(4):1009-1024.
15. Franco PJDDT. Orphan drugs: the regulatory environment. 2013;18(3-4):163-172.
16. Wellman-Labadie O, Zhou YJHP. The US Orphan Drug Act: rare disease research stimulator or commercial opportunity? 2010;95(2-3):216-228.
17. Mechler K, Mountford WK, Hoffmann GF, Ries MJOjord. Pressure for drug development in lysosomal storage disorders–a quantitative analysis thirty years beyond the US orphan drug act. 2015;10(1):1-9.
18. Thomas S, Caplan AJJ. The orphan drug act revisited. 2019;321(9):833-834.
19. Thamer M, Brennan N, Semansky RJJoHP, Policy, Law. A cross-national comparison of orphan drug policies: implications for the US Orphan Drug Act. 1998;23(2):265-290.
20. Côté A, Keating BJViH. What is wrong with orphan drug policies? 2012;15(8):1185-1191.
21. Melnikova IJNRDD. Rare diseases and orphan drugs. 2012;11(4):267.
22. Kesselheim AS, Sinha MS, Avorn JJJim. Determinants of market exclusivity for prescription drugs in the United States. 2017;177(11):1658-1664.
23. Hussar DA, Hussar EFJPT. New therapeutic agents marketed in 2018: Part 4. 2019;25(8):40-53.
24. Richey EA, Lyons EA, Nebeker JR, et al. Accelerated approval of cancer drugs: improved access to therapeutic breakthroughs or early release of unsafe and ineffective drugs? 2009;27(26):4398.
25. Fagnan DE, Gromatzky AA, Stein RM, Fernandez J-M, Lo AWJDdt. Financing drug discovery for orphan diseases. 2014;19(5):533-538.
26. Seoane-Vazquez E, Rodriguez-Monguio R, Szeinbach SL, Visaria JJOjord. Incentives for orphan drug research and development in the United States. 2008;3(1):1-7.
27. Organization WH. The use of stems in the selection of International Nonproprietary Names (INN) for pharmaceutical substances: 2004: World Health Organization; 2004.
28. Kakitani M, Oshima T, Horikoshi K, et al. A novel transgenic chimaeric mouse system for the rapid functional evaluation of genes encoding secreted proteins. 2005;33(9):e85-e85.
29. Sardana D, Zhu C, Zhang M, Gudivada RC, Yang L, Jegga AGJBib. Drug repositioning for orphan diseases. 2011;12(4):346-356.
30. Mullard AJNRDD. 2012 FDA drug approvals. 2013;12(2):87.
31. Xu K, Coté TRJBib. Database identifies FDA-approved drugs with potential to be repurposed for treatment of orphan diseases. 2011;12(4):341-345.