Implemented in 1983, the basic intent of the US Orphan Drug Act (ODA) was to stimulate research, development, and approval of products that treat rare diseases. “In the US, these diseases are defined as those which affect less than 2,00,000 patients or those for which cost of development is unlikely to be covered through commercialisation. ‘Orphan drugs’ are thus used for rare diseases for which there is an unmet medical need,” says Dr Chandrashekhar Potkar, Director-Medical and Regulatory Affairs, Pfizer. Examples of these diseases include severe combined immunodeficiency syndrome, cystic fibrosis, hairy cell leukaemia etc. Malaria and tuberculosis also are orphan indications in the US. The concept of orphan drug in the US, apart from covering pharmaceutical or biological products, also covers medical devices and dietary or diet products.
“There is limited commercial opportunity for such drugs. However, research based pharma companies are able to apply their R&D expertise in partnership with government and other medical institutes for orphan indications with unmet medical needs,” Potkar adds. Government provides incentives for sponsors undertaking research on orphan indications. These incentives can take form of tax credits for clinical research expenditure, grants in aid for clinical research, design assistance for investigating orphan indications (eg. open protocols for enrolling patients) and seven year market exclusivity. But the definition of orphan drugs and diseases and the incentives attached to them may vary in different countries depending on the prevalence of diseases there and the respective population strengths.
It is the enforcement of this very Act that encouraged other countries, including European Union (EU), Australia, Japan and Singapore to follow suit. So what is stopping India from adopting a legislation similar to the Act? Potkar says that this may be due to the fact that our current legislation is focused on strengthening R&D in India.
Dr Syamala Ariyanchira, author of BCC Research‘s Global Markets for Orphan Drugs report, opines, “Considering that there are next to none pharma companies in India manufacturing or researching orphan drugs to my knowledge, there will be no incentives for them. However, in 2001, a group of pharmacologists at an Indian Drug Manufacturing Association (IDMA) conference had appealed to the Indian Government to form something akin to the Orphan Drug Act. But I have not heard of any kind of development in this context since then.”¹
¹Aashruti Kak; Express Pharma