Will rare diseases remain orphan?

Currently, 70 million people in India are battling a rare disease. Though most of these diseases are treatable, lack of a policy to support and foster development of orphan drugs is discouraging pharma companies from addressing this unmet medical need .


Despite medical advancements, rare diseases are still a common occurence in India. Rare Diseases India, an organization that focuses on providing scientific data concerning rare diseases and disorders, estimates that around 72,611,605 people in India are suffering from a rare disease. About 6,000 to 8,000 rare diseases, mostly genetic in nature have been identified across the world and only around 500 of them have known therapies. India is the second highly populated nation in the world and hence even the rarest of diseases will have many patients seeking treatment.

Considering the fact that the present government's priority is to address demanding health issues, its attention to the nebulous rare diseases area, barring a handful of rare diseases, has been non-existent. The need for orphan treatments in India is huge and with little in place so far, India represents a lucrative market. Most of these disorders are genetic in nature and timely treatment can assure the victims of a healthy life. The disorders, once rare, are now affecting larger populations, with one in 5,000 suffering from a rare genetic disorder. Currently, drug developers in India are receiving no formal incentives from the government. Hence, orphan drugs need to be imported, which in turn makes them highly unaffordable. Lack of a policy or a legal framework in these countries is deterring the development of these drugs and thus many rare disease patients die. The increasing number of people falling prey to these diseases has reiterated the need for a sound legal policy and framework to support drug development for diseases.

In an exclusive interview with BioSpectrum, Mr Sandeep Sahney, head-intercontinental, Genzyme said, "A major challenge in bringing new orphan drugs to the market is the ability to garner a share of voice at a societal level for the ‘few' patients. The other challenge is creating support from governments and lack of an appropriate policy to create a platform to address these diseases."

For long, the Orphan Drug Act (ODA) has been considered a key game changer that can alter the dynamics of orphan drugs and rare diseases in any country. The ODA, formulated by the US in 1983 can be rightly termed as the catalyst that fostered the development of these drugs. Under this Act, orphan drugs enjoy many regulatory and commercial incentives such as tax credits, grants, waived FDA fees, reduced timelines for clinical development and higher probability of regulatory approval, coupled with commercial drivers such as premium pricing, faster uptake, lower marketing costs, and longer market exclusivity. Only five Asian countries have implemented this Act -Australia, Japan, Korea, Singapore, and Korea. However, countries with a huge population like China and India do not have a policy that can attract global players to show corporate interest in this area. If the governments in these countries do not formulate a policy to address these rare disorders, orphan diseases will unfortunately remain orphan.

"In the case of Orphan Drug Development," explained Dr AKS Bhujanga Rao, president, technical/R&D and director, Natco Pharmaceuticals, "more than the medical advancements, it is the government's initiative, the concerted effort by the medical and pharmaceutical companies, government, academia, and research organizations that can create a difference. The government act can trigger tremendous response for orphan drugs given their high demand and market value."

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