Orphan drugs: The current global and Indian scenario

It was not until a few decades ago that orphan drugs, still “enjoyed” the status of pharmaceutical touch-me-not entities. However, the past two decades have witnessed a radical shift in the approach of global pharmaceutical industry toward orphan drugs. This has stemmed from an apparent innovation crisis in the domain of common diseases, progressively increasing stringency in the regulations, and the decline of the blockbuster business model. Further, the success stories of a few orphan drugs, for instance, eculizumab has gone a long way in breaking the myth of non-profitability associated with orphan drug development endeavor. This combined with the high degree of incentivization attached with orphan drug development makes it a very lucrative avenue for further investment by the pharmaceutical industry. Sadly, the Indian scenario with respect to orphan drugs is a throwback to the “dark ages.” The progress seen across the developed nations, for instance, the United States of America has not permeated into the Indian market. India, with its huge population base, stands to provide a hugely lucrative market for orphan drug development. However, this point seems to have escaped the notice of the Indian authorities and the pharmaceutical sector in India. Thus, with the various patient advocacy groups and non-government organizations championing the cause of orphan diseased patients in India, the time is ripe for the concerned authorities and the pharma sector in India to take cognizance of this gaping lacuna in the health-care services and undertake measures to address this situation.