Rare diseases affect more than 55 millions patients in US and EU alone, highlighting the huge societal impact of these diseases. The various clinical, regulatory and commercial challenges to develop treatment for these diseases call for actions from various parties involved.
One of the successful initiatives is the orphan drug legislation by FDA in US. Prior to the Orphan Drug Act, there were minimal drugs that were aimed to treat rare diseases. With increasing success case studies observed in this industry, many pharmaceutical companies are looking into developing drugs for rare diseases. This in turns bring in huge amount of investments and biotechs focusing on rare diseases programmes.
Despite the promising opportunities in this industry, effective therapies are still not available to more than 95% of the patients suffering from these diseases. Furthermore, the annual approvals for orphan drugs are growing slowly even though the legislations have increased. It is clear that the R&D paradigm to develop drugs for rare diseases has very different challenges from those associated with more prevalent diseases. Hence, there is a need for stakeholders to align their ways to address these challenges.
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