Orphan drugs: A medicinal product developed specifically to treat arare medical condition, or the “orphan disease.” These are not developed by the pharmaceuticalindustry for economic reasons but which respond topublic health need. The indications of a drug may also be “orphan”.
Orphan diseases:• Diseases that manifest in patient populations representing at themaximum 6–8% of the world population are defined as rarediseases or orphan diseases.Orphan disease Per 100,000 peopleGuillain-Barre syndrome 50Melanoma, familial 50Autism, genetic types 45Marfan syndrome 30Non-Hodgkin malignantlymphoma30
The Orphan Drug Act (ODA) was passed onJanuary 28, 1983. USA was the first nation to propose a legal frameworkto encourage development and availability of orphandrugs. A group of pharmacologists requested the Indiangovernment to institute ODA at the conference held bythe Indian Drug Manufacturing Association inNovember 2001, but nothing concrete has materializedso far.
Incentives forODSTax benefits7 yearsexclusivityExemption fromapplicationfiling feesGrant for phaseI & II clinicaltrials
ODA exists in various countries like USA,Japan, Singapore, Australia, Canada, France,Sweden, and United Kingdom.Country Exclusivity time (In years)USA 7Japan 10Australia 5Europe 10
The orphan oncology drug Rituxan (generic:Rituximab) is the world’s second most profitable drug,just behind mainstream blockbuster Lipitor ( as ofNovember 2011). While orphan drugs target much smaller populationsthan traditional mainstream drugs, the high cost oftherapy and various developmental drivers, make themattractive for pharmaceutical companies.
The orphan drug market was worth just over $50billion globally at the end of 2011. The compound annual growth rate (CAGR) ofthe orphan drug market between 2001 and 2010was an impressive 25.8%. While the CAGR of non orphan drugs in thesame time period was found to be 20.1%.
• BioMarin Pharmaceuticals• The company has generated about $500 milliondollars from its following orphan drugs• Aldurazyme,• Naglazyme, and• Kuvan.
• Vertex pharmaceuticals:• Drug Kalydeco for cystic fibrosis• Net product revenues for kalydeco $171.6 million.• Alexion Pharmaceuticals:• Soliris, for two indications:• In 2007 for paroxysmal nocturnal hemoglobinuria (PNH)• In 2011 for (HUS) Hemolytic Uremic Syndrome• Net product sales for its products are expected to be between$1.49 billion to $1.5 billion.
The orphan drugs are awarded a seven yearOrphan Drug Exclusivity (ODE). Non-orphan new chemical entities areawarded five years of exclusivity uponapproval. This means that orphan drugs receive twoyears of additional protection if the orphanindication is awarded upon approval.
Limited public awareness Invisible patient population Disease is poorly understood; no natural history Delay in diagnosis Small patient population Limited treatment availability
The Hyderabad based NATCO Pharma’s novelanti-cancer drug (NRC-AN-019) has received“Orphan Drug Designation” from theUSFDA for three indications- Glioma (brain tumor), Pancreatic cancer, and Chronic Myelogenous Leukemia.
The Cipla’s Mumbai-headquartered firm ismanufacturing the fixed dose combination drug ofArtesunate and Mefloquine (ASMQ FDC) undera technology transfer agreement with NeglectedDiseases initiative (DNDi). The combination of AS and MQ is one of fiveArtemisinin Combination Therapies (ACTs)currently recommended by WHO for the treatmentof uncomplicated P.falciparum malaria, and is thefirst-line treatment in a number of South EastAsian countries.
Cipla, the Indian generic drug maker, has enteredthe biosimilar market, as they just launched thefirst biosimilar of Pfizer and Amgen’s rheumaticdisorder blockbuster Enbrel (Etarnecept) inIndia, under the brand name Etacept. Being a generic, it will sell for cheaper than Enbrelin India, at 6,150 rupees, 30% less than theinnovator product, which “will enable access ofthis drug to a greater number of patients in India”,
Enbrel was 2012′s second-best-selling drug in theworld, with estimated worldwide sales of $8.37billion
• Seven years of exclusivity for their drugs, taxbenefits in certain situations.• Limited competition.• More leniency by regulatory agencies.
Orphan drugs may help pharma companies to reducethe impact of revenue loss caused by patent expiries ofblockbuster drugs. The new business model of orphan drugs could offeran integrated healthcare solution that enables pharmacompanies to develop newer areas of Therapeutics, Diagnosis, Treatment, Monitoring, and Patient support.
Food and Drug Administration. The Orphan Drug Act (as amended).Availablefrom: http://www.fda.gov/orphan/oda.html Villa S, Compagni A, Reich MR. Orphan drug legislation: Lessons for neglectedtropical diseases. International Journal of Health Planning; 2009;24:27–42. Garg meena; Kataria Mahesh kumar; Anand vikas;research journal ofpharmaceutical, biological, chemical sciences, an insight on regulations governingorphan diseases and drugs, july- September 2011;vol. 2 issue 3; P.no.373. www.orphanet.com/about orphan drugs.html Thamer M, Brennan N, Semansky R. A cross-national comparison of orphan drugpolicies: implications for the US Orphan Drug Act. J Health Pol Policy Law1998;23:265-90. Ariely R. The Rise of Biopharmaceutical Orphan Drug Adoption. [Published: 01December 2001 Source: Frost & Sullivan]. Available from: http://www.inpharm.com/intelligence/frost071201.html www.ip-science.thomsonreuters.com. Scharf S. Orphan drugs: the question of product liability. Am J Law Med 1985;10:491-513. http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/orphan_search.jsp&murl=menus/medicines/medicines.jsp&mid=WC0b01ac058001d12b www.natcopharmaltd.com