Review on Development of Orphan Drug for Rare Disease
Keywords:
Orphan Drug, Rare Disease, committee on orphan medicinal products
Abstract
Simply receiving a diagnosis of a rare disease often becomes a frustrating quest, since many doctors may have never before heard of or seen the disease. This is, however, a time of great progress and hope. Biopharmaceutical research is entering an exciting new era with a growing understanding of the human genome. Scientific advances have given researchers new tools to explore rare diseases, which are often more complex than common diseases.
“Who else has this rare disease? How many of us are there? What can I expect now? What is known or not known about this disease?” These are among the questions that patients and family members ask as they become, out of necessity, advocates for themselves or others.
References
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25.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm070124
2. Aronson J.K., Rare diseases and orphan drugs. British Journal of Clinical Pharmacology 2006, DOI:10.1111/j.1365-2125.2006.02617.x
3.Mphahlele M.J., Lau G.K., Carman W.F.,HGV the identification, biology and prevalence of an orphan virus. Liver 1998; 18(3): 143–55.
4. Melnick J.L., Application of tissue culture methods to epidemiological studies of poliomyelitis. Am J Public Health 1954; 44(5): 571–80.
5. Lands W.E., A review of alcohol clearance in humans. Alcohol 1998; 15(2): 147–60.
6.Meunier J.C., Mollereau C, Toll L, Suaudeau C, Moisand C, Alvinerie P, Butour J.L., Guillemot J.C., Ferrara P, Monsarrat B,Isolation and structure of the endogenous agonist of opioid receptor-like ORL1 receptor. Nature 1995; 377(6549): 532–5.
7. Reinscheid R.K., Nothacker H.P., Bourson A., Ardati A., Henningsen R.A., Bunzow J.R., Grandy D.K., Langen H, Monsma F.J.Jr, Civelli O, Orphanin F.Q., a neuropeptide that activates an opioidlike G protein-coupled receptor. Science 1995; 270(5237): 792–4.
8. Corbett A.D., Henderson G, McKnight A.T., Paterson S.J., 75 years of opioid research. The exciting but vain quest for the Holy Grail. Br J Pharmacol 2006; 147(Suppl 1): S153–62.
9. Henderson G, McKnight A.T.,The orphan opioid receptor and its endogenous ligand – nociceptin/orphanin FQ. Trends Pharmacol Sci 1997; 18(8): 293–300.
10. Dooley C.T., Spaeth C.G., Berzetei-Gurske I.P., Craymer K, Adapa I.D., Brandt S.R., Houghten R.A., Toll L.,Binding and in vitro activities of peptides with high affinity for the nociceptin/orphanin FQ receptor, ORL1. J Pharmacol Exp Ther 1997; 283(2):735–41.
11. slideshare.net/PhRMA/rare-diseases-a-report-on-orphan-drugs-in-the-pipeline
12. lgdalliance.org/en/aboutGorhamsDisease/Default.aspx.
13. rarediseasesnetwork.epi.usf.edu/vcrc/about/index.htm
14. ssa.gov/compassionateallowances/NORDTestimonyCompassionateAllowances120407.
15. nlm.nih.gov/medlineplus/ency/article/000679.htm.
16. D’Andrea, A.D.,Susceptibility pathways in Fanconi’s anemia and breast cancer. NewEngland Journal of Medicine 2010, 362:1909-1919.
17. Dale D.C., Link D.C., The many causes of severe congenital neutropenia. New England Journal of Medicine 2009, 360(1):3-5.
18. de Siqueira, I.C., Dias J, Ruf H, Ramos E.A.G, Pires Maciel E.A., Rolim A, Jabur L, Chromobacterium violaceum in siblings, Brazil. Emerging Infectious Diseases 2005 (11)9.
18.de Siqueira, I. C., Dias J, Ruf H, Ramos E. A. G., Pires Maciel E. A., Rolim A, Jabur L, et al., Chromobacterium violaceum in siblings, Brazil. Emerging Infectious Diseases 2005 (11)9.
19.Glaser, C, Schuste F, Yagi S, Gavali S, Bollen A, Glastonbury C, Raghavan R, et al., Balamuthia amebic encephalitis—California, 1999-2007. Morbidity and Mortality Weekly Report 57(28):768-771.
20. nlm.nih.gov/medlineplus/ency/article/000339.htm
21. rarediseases.about.com/od/rarediseaseso/a/orphandrug.htm
22. globalgenes.org/rare-disease-drug-development-its-complicated/
23.Mossinghoff G.J., Overview of the Hatch-Waxman Act and its impact on the drug development process. Food and Drug Law Journal 1999, 54(2):187-194.
24. Glover, G. J., The influence of market exclusivity on drug availability and medical inno-vations. AAPS Journal 2007, 9(3):34.
25.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm070124
Published
2014-08-01
How to Cite
[1]
Sharada, K., Bhagwat, P. and Amol, G. 2014. Review on Development of Orphan Drug for Rare Disease. PharmaTutor. 2, 8 (Aug. 2014), 66-72.
Section
Articles
