Updated on 15 December 2014
Despite medical advancements, rare diseases are still a unique concept in Asia. An analysis estimates that around 6 to 10 percent of the world's population will be afflicted by a rare disease at some point of time. 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. Nearly two-thirds of the world's population reside in Asia and hence even the rarest disease will have many patients seeking treatment. The need for orphan treatments in Asia is huge and with little in place so far, Asia represents a lucrative market for orphan operations.
Some Asian countries have a well-defined system for orphan drugs and provide health insurance coverage, expedited registration, exclusive marketing periods, and many such incentives to woo pharma companies. However, other Asian countries are only now beginning to look at rare diseases, and what they can do to help citizens afflicted with them.
Taking the lower limit of global prevalence estimate, populous nations like India and China should have more than 70 million rare disease cases each. Most of these disorders are genetic in nature and timely treatment can assure healthy life for the victims. The disorders, once rare, are now affecting larger populations, with one in every 5,000 people suffering from a rare genetic disorder. Currently, drug developers in these countries are receiving no formal incentives from the government. Hence orphan drugs need to be imported which 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 diseases patients die of the disease. According to Rare Diseases India estimates, currently 72,611,605 people in India are suffering from a rare disease. Increasing number of people falling prey to these diseases have reiterated the need for a sound legal policy and framework to support drug development for diseases.
Speaking to BioSpectrum, Mr Martin Andrews, senior vice president, Global Rare Diseases, GSK, said, "Given that there are over 7000 rare diseases, small numbers of patients, unique regulatory hurdles and modest commercial returns, progress in this area requires specialist medicine development. Unfortunately, access to treatment in the majority of Asian countries beyond Japan, South Korea, Singapore, and Taiwan is still limited."
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--Taiwan, Korea, Australia, Japan, and Singapore. 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.