Main Article Content

Abstract

The World Health Organization defines orphan diseases, as all pathological conditions, affecting 0.65-1 out of every 1000 inhabitants. They are usually not studied for their path physiology or for newer therapeutic options, as these are not economically viable. The Orphan Drug Act was passed on January 28, 1983 by USA to stimulate the research, development, and approval of those products that treat orphan diseases. Till date, 11 drugs (4.87%) for tropical infectious diseases has been designated with orphan drug status, and as many drugs for other infectious diseases. Several drugs with orphan status are used in the treatment of diseases that no longer meet orphan status criteria, such as AIDS and end-stage renal disease. Understanding of the human genome, nuclear cloning, rational drug designing, and application of high throughput screening in drug discovery programs, might lead to new drug discoveries for orphan diseases. Hence, there is hope in the future for patients neglected by for-profit drug discovery efforts.

Keywords

Newer Therapeutic Options Orphan Drug Act Drug Discovery Programs

Article Details

How to Cite
Venkat Paluri, Arun Mathew, Venkateswaramurthy N, & Sambath Kumar R. (2021). A review of orphan drugs and rare diseases. International Journal of Research in Pharmacology & Pharmacotherapeutics, 5(2), 178-183. https://doi.org/10.61096/ijrpp.v5.iss2.2016.178-183

References

  1. [1]. Song P, Gao J, Inagaki Y, Kokudo N, Tang W. Intractable and rare diseases research in Asia. Biosci Trends. 2012; 6: 48-51.
  2. [2]. Cui Y, Han J. A proposed definition of rare diseases for China: From the perspective of return on investment in new orphan drugs. Orphanet J Rare Dis. 2015; 10:28.
  3. [3]. Lavandeira A. Orphan drugs: Legal aspects, current situation. Haemophilia 2002; 8: 194-8.
  4. [4]. Campos-Castello J. Orphan drugs and orphan diseases. Rev Neurol 2001;33:216-20
  5. [5]. Loizzo A, Tebano MT. Orphan diseases. Recenti Prog Med 1993; 84:786-93.
  6. [6]. Henkel J. How TV Launched the Orphan Drug Law. FDA Consumer Magazine. Available from: http://www.fda.gov/fdac/features/1999/399_orph.html#tv
  7. [7]. Trouiller P, Olliaro P, Els Torreele, Orbinski J, Laing R, Ford N. Drug development for neglected diseases: A deficient market and a public-health policy failure. Lancet 2002; 359: 2188-94.
  8. [8]. Swoboda ML. The ethics of pharma-economics: An examination of the limit of corporate responsibility in the pharmaceutical industry. 1999.
  9. [9]. Martinez-Pardo M. Orphan drugs and metabolic disorders. Rev Neurol 2001 1-15; 33:220-5.
  10. [10]. Haffner M. Orphan products: Origins progress and prospects. Annu Rev Pharmacol Toxicol 1991; 31: 603-20.
  11. [11]. Haffner M, Kelsey J. Evaluation of orphan products by the US. Food and Drug Administration. Int J Tech Assess Health Care 1992; 8: 647-57.
  12. [12]. Katzung BG. Basic and clinical pharmacology. 9th ed. New York: McGraw Hill 2004.
  13. [13]. Haffner ME. Developing treatments for inborn errors: Incentives available to the clinician. Mol Genet Metab 2004; 81: 63-6.
  14. [14]. Thamer M, Brennan N, Semansky R. A cross-national comparison of orphan drug policies: implications for the US Orphan Drug Act. J Health Pol Policy Law 1998; 23: 265-90.
  15. [15]. Ariely R. The Rise of Biopharmaceutical Orphan Drug Adoption. [Published: 01 December 2001 Source: Frost & Sullivan]. Available from: http:// www.inpharm.com/intelligence/frost071201.html.
  16. [16]. Trouiller P, Rey JL, Bouscharain P. Pharmaceutical development concerning diseases predominating in tropical regions: the concept of indigent drugs. Ann Pharm Fr 2000; 58:43-6.
  17. [17]. Bennet PN, Brown MJ. Clinical Pharmacology. 9th ed. Edinburgh: Churchill Livingstone; 2003.
  18. [18]. Scharf S. Orphan drugs: the question of product liability. Am J Law Med 1985; 10:491-513.
  19. [19]. Lang J, Wood SC. Development of Orphan Vaccines: An industry perspective. Emerging infectious diseases. Available from: http://www.cdc.gov/ncidod/EID/ vol5no6/lang.htm
  20. [20]. Wilde H. What are today’s orphaned vaccines? Clin Infect Dis 2001; 33: 64850.
  21. [21]. Medical Devices. Humanitarian Use Devices. Final rule federal register, v. 61. 1996.
  22. [22]. Haffner ME. Orphan drug product regulation-US. Int J Clin Pharmacol Ther 2002; 40: 84-8.
  23. [23]. Van Osterom AT, Judson I, Verweji J. Safety and efficacy of imatinib (STI571) in metastatic gastrointestinal stromal tumors: A Phase I study. Lancet 2001; 358:1421-3.
  24. [24]. Food and drug administration. Guidance for industry: Fast track drug development programs — designation, development and application review. [Accessed 2016 April 16]. Available from: http://www.fda. gov/cber/gdlns/fsttrk.pdf
  25. [25]. Kawaguchi Y, Jinnai I, Nagai K. Effect of a selective Abl tyrosine kinase inhibitor, STI571, on in vitro growth of BCR-ABL-positive acute lymphoblastic leukemia cells. Leukemia 2001; 15: 590-4.
  26. [26]. Frasca F, Vigneri P, Vella V. Tyrosine kinase inhibitor STI571 enhances thyroid cancer cell motile response to hepatocyte growth factor. Oncogene 2001; 20: 3845-56.
  27. [27]. Kilic T, Alberta JA, Zdunek PR. Intracranial inhibition of platelet-derived growth factor-mediated glioblastoma cell growth by an orally active kinase inhibitor of the 2-phenylaminopyrimidine class. Cancer Res 2000; 60: 5143-50.
  28. [28]. Krystal GW, Honsawek S, Litz J. The selective tyrosine kinase inhibitor STI571 inhibits small cell lung cancer growth. Clin Cancer Res 2000; 6: 3319-26.
  29. [29]. Motl S, Miller SJ, Burns P. Established by FDA to Expedite Patient Access to Medications. Am J Health-Syst Pharm 2003; 60:339-45.
  30. [30]. Banerji J. New initiative to develop drugs for neglected diseases. Essential Drug Monitor 2003; 33: 28.
  31. [31]. Hochedlinger and Jaenisch. Nuclear transplatation, embryonic stem cells, and the potential for cell therapy. N Engl J Med 2003; 349: 275-86.
  32. [32]. Xu XM, Lei AM, Hua JL, Dou ZY. Nuclear transfer and therapeutic cloning. Yi Chuan 2005; 27: 289-96.
  33. [33]. Derenda N, In Their Lifetime: Outlook for Rare Disease and Orphan Drug Research https://www.medpace.com/PDF/Articles/Rare_Disease_white_paper.pdf
  34. [34]. Emmanuelle L, Development of International Orphan Drug Policies http://www.rarediseases.eu/wpcontent/uploads/2014/05/0401_Emmanuelle_LECOMTE-BRISSET.pdf
  35. [35]. Enrique V, Rosa M, Jay V. Incentives for orphan drug research and development in the United States. Orphanet J Rare Dis, 2008; 3(33): 1-8.
  36. [36]. Peipei S, Jianjun G, Yoshinori I, Norihiro K, Wei T. Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives. Intractable & Rare Diseases Research, 2012; 1(1): 3-9.
  37. [37]. Aarti S, Abraham J, Dushyant K. Orphan drug: Development trends and strategies. J Pharm Bioallied Sci, 2010; 2(4): 290-9.
  38. [38]. Ajay B, Anil B, Mahesh KK, Meena G, Vikas A, Vipin K. An Insight on Regulations Governing Orphan Diseases and Drugs. Research Jour Pharm Bio Chemical Sciences 2011; 2(3):373.
  39. [39]. Arno P, Bonuk K, Davis M. Rare diseases, Drug development and AIDS: the impact of an orphan drug act. Milbank Q 1995; 73(2):231-252.
  40. [40]. Compagni A, Reich MR, Villa S. Orphan drug legislation: lessons for neglected tropical diseases. International Journal of Health Planning and Management 2009; 24(1):27-42.
  41. [41]. Parasher S, Sumanth M. “Rare diseases”. Indian Journal of Pharmaceutical Sciences 2004; 66:587-594.
  42. [42]. Campos-Castello J. Orphan drugs and orphan diseases. Rev Neurol 2001; 33:216-220.
  43. [43]. Haffner ME, Moses M, Whitley J. Two decades of orphan product development. Nat Rev Drug Discov 2002; 1(10):821-825. American foundation for the blind, Retinitis pigmentosa, American academy of ophthalmology, 10/09, 057169.
  44. [44]. Christain H. Retinitis pigmentosa. Orphanet J Rare Dis 2006; 1:40.
  45. [45]. Akira M, Mitsuru N, Nobuhisa N, Shigeki, Shuichi Y, Takeshi S. Topical Isopropyl Unoprostone for Retinitis Pigmentosa: Microperimetric Results of the Phase 2 Clinical Study. opthalmol thr 2012; 1(5):1-16.
  46. [46]. Openshaw A, Branham K, Heckenlively J. Understanding Retinitis Pigmentosa. University of Michigan hospital, kellog eye center, 2008.
  47. [47]. Howell RR, Kishnani PS. Pompe disease in infants and children. J Pediatr 2004; 144:S35–S43.
  48. [48]. Dohna SC, Stehling F, Teschler H, Ragette R, Voit T, Mellies U. Respiratory failure in Pompe disease: Treatment with noninvasive ventilation. Neurology 2005; 64(8):1465-1467.
  49. [49]. Brady RO, Schiffmann R. New prospects for thetreatment of lysosomal storage diseases. Drugs 2002; 62(5):733-742.
  50. [50]. Renu S, Amit K, Manoop M, Yadvinder S, Hari R. Raynaud’s Phenomenon. API, 58, may 2010.