ORPHAN DRUGS: THE CURRENT GLOBAL AND INDIAN SCENARIO
It was not until a few decades ago that orphan drugs, still enjoyedâ€ the status of pharmaceutical touch-me-not entities. However, the past two decades
have witnessed a radical shift in the approach of global pharmaceutical industry toward orphan drugs. This has stemmed from an apparent innovation
crisis in the domain of common diseases, progressively increasing stringency in the regulations, and the decline of the blockbuster business model.
Further, the success stories of a few orphan drugs, for instance, eculizumab has gone a long way in breaking the myth of non-profitability associated
with orphan drug development endeavor. This combined with the high degree of incentivization attached with orphan drug development makes it a
very lucrative avenue for further investment by the pharmaceutical industry. Sadly, the Indian scenario with respect to orphan drugs is a throwback
to the dark ages.â€ The progress seen across the developed nations, for instance, the United States of America has not permeated into the Indian
market. India, with its huge population base, stands to provide a hugely lucrative market for orphan drug development. However, this point seems
to have escaped the notice of the Indian authorities and the pharmaceutical sector in India. Thus, with the various patient advocacy groups and nongovernment
championing the cause of orphan diseased patients
in India, the time is ripe for
the concerned authorities
and the pharma
in India to
cognizance of this gaping
lacuna in the health-care
services and undertake
Keywords: Pharmaceutical touch-me-not, Orphan, Indian scenario.
Aronson JK. Rare diseases and orphan drugs. Br J Clin Pharmacol
Griggs RC, Batshaw M, Dunkle M, Gopal-Srivastava R, Kaye E,
Krischer J, et al. Clinical research for rare disease: Opportunities,
challenges, and solutions. Mol Genet Metab 2009;96(1):20-6.
Shirkey H. Therapeutic orphans. J Pediatr 1968;72(1):119-20.
Campos-CastellÃ³ J. Orphan drugs and orphan diseases. Rev Neurol
Rajasimha HK, Shirol PB, Ramamoorthy P, Hegde M,
Barde S, Chandru V, et al. Organization for rare diseases India
(ORDI) - Addressing the challenges and opportunities for the Indian
rare diseasesâ€™ community. Genet Res (Camb) 2014;96:e009.
Thielke D, Thyssen JP, Hansen BJ. Orphan drugs â€“ medications for
patients with rare diseases. Ugeskr Laeger 2006;168(23):2236-8.
Sharma A, Jacob A, Tandon M, Kumar D. Orphan drug: Development
trends and strategies. J Pharm Bioallied Sci 2010;2(4):290-9.
Fagnan DE, Gromatzky AA, Stein RM, Fernandez JM, Lo AW.
Financing drug discovery for orphan diseases. Drug Discov Today
Maher PD. Pharmacogenomics of rare and monogenic disorders. In:
Cohen N, editor. Pharmacogenomics and Personalized Medicine. 1
New York: Humana Press; 2008. p. 479-97.
Hay M, Thomas DW, Craighead JL, Economides C, Rosenthal J.
Clinical development success rates for investigational drugs. Nat
Available from: http://www.thomsonreuters.com/products/ipscience/04_013/1001450.pdf.
Asian J Pharm Clin Res, Vol 9, Issue 4, 2016, 46-50
Agarwal et al.
Parker C, Omine M, Richards S, Nishimura J, Bessler M, Ware R, et al.
Diagnosis and management of paroxysmal nocturnal hemoglobinuria.
Zimmerhackl LB, Besbas N, Jungraithmayr T, van de Kar N,
Karch H, Karpman D, et al. Epidemiology, clinical presentation, and
pathophysiology of atypical and recurrent hemolytic uremic syndrome.
Semin Thromb Hemost 2006;32(2):113-20.
Garnier JP. Rebuilding the R&D engine in big pharma. Harv Bus Rev
;86(5):68-70, 72-6, 128.
Light DW, Lexchin JR. Pharmaceutical research and development:
What do we get for all that money? BMJ 2012;345(1):e4348.
DiMasi JA, Grabowski HG. The cost of biopharmaceutical R&D: Is
biotech different? Manage Decis Econ 2007;28(4-5):469-79.
Hait WN. Forty years of translational cancer research. Cancer Discov
Avery M. Personalized medicine and rescuing unsafeâ€ drugs with
pharmacogenomics: A regulatory perspective. Food Drug Law J
Morgan SG, Cunningham CM, Law MR. Drug development: Innovation
or imitation deficit? BMJ 2012;345:e5880.
Gibson S, Raziee HR, Lemmens T. Why the shift? taking a closer look
at the growing interest in niche markets and personalized medicine.
World Med Health Policy 2015;7(1):3-27.
Boon W, Moors E. Exploring emerging technologies using metaphors â€“
A study of orphan drugs and pharmacogenomics. Soc Sci Med
CotÃ© TR, Xu K, Pariser AR. Accelerating orphan drug development.
Nat Rev Drug Discov 2010;9(12):901-2.
Schieppati A, Henter JI, Daina E, Aperia A. Why rare diseases are an
important medical and social issue. Lancet 2008;371(9629):2039-41.
Available from: http://www.fda.gov/fdac/features/1999/399_orph.
Kataria MK, Garg M, Anand V, Bilandi A, Kukkar V, Bhandari A.
An insight on regulations governing orphan diseases and drugs. Res J
Pharm Biol Chem Sci 2011;2(3):373-84.
Bigoniya P. Implementation of orphan drug regulation in India: The
need of the time. Pharm Rev 2010:83-7. Available from: http://www.
Reddy DS, Pramodkumar TM, Reddy Y, Sirisha K. Orphan regulations
for orphan drug development in India. Asian J Pharm 2014;8:130-2.
How to Cite
The publication is licensed under CC By and is open access. Copyright is with author and allowed to retain publishing rights without restrictions.