Recently, Chief Justice of India (CJI) Justice D.Y. Chandrachud has stressed on intersection of law, ethics and medical innovation in gene therapy for treatment of rare diseases. Speaking after inaugurating a conference on ‘Gene Therapy and Precision Medicine’ organised by Narayana Nethralaya Foundation, the CJI said societal awareness of genetic disorders is essential for the socio-cultural support base for such patients, and for developing cost-effective gene therapy, the industry must step up its support through Corporate Social Responsibility (CSR) initiatives or direct investment in startups to further innovation in gene therapy and rare diseases. To encourage more industry participation, there should be proposals for tax incentives and benefits to encourage industry participation in research and development for rare disease treatments, especially since the market for rare disease treatments is relatively small. Certainly, such tax incentives would make it easier for companies to invest in research while ensuring that patients and society as a whole benefit from these efforts. Justice Chandrachud also said that factors outside the healthcare system such as class, caste, gender and regional location often play a significant role in determining an individual’s health status and asserted that access to essential treatments is part of the right to health guaranteed under right to life. While appreciating the government efforts to provide insurance cover for such treatments up to Rs. 50 lakhs, the CJI said effective addressing of the situation could only be through development of indigenous technologies tailored for our patient population. More significantly, Justice Chandrachud has rightly said that the quest to treat rare diseases is futile if access to these treatments remains a challenge, especially outside major urban centres.
Indeed, availability of drugs for rare diseases or orphan diseases at reasonably affordable prices has been a major issue in the country, for that matter the entire world, has been facing for a long time now. The field of rare diseases is very complex and heterogeneous; and prevention, treatment and management of rare diseases have multiple challenges. Early diagnosis of rare diseases is a major challenge owing to a variety of factors that include lack of awareness among primary care physicians, lack of adequate screening and diagnostic facilities, etc. There are also fundamental challenges in the research and development for the majority of rare diseases as relatively little is known about the pathophysiology or the natural history of these diseases particularly in the Indian context. Rare diseases are also difficult to research upon as the patient pool is very small and it often results in inadequate clinical experience. Availability and access to medicines are important to reduce morbidity and mortality associated with rare diseases. At present, very few companies are manufacturing drugs for rare diseases. As the number of persons suffering from particular rare disease is small, they do not constitute a significant market for drug manufacturers to develop and bring to market these drugs for them. For the drug manufacturers, such investments may not be financially viable. Some time back, the Union Health Ministry had taken some initiative to allow Indian pharmaceutical companies to manufacture medicines for four rare diseases at a significantly lower price. It was praiseworthy as it will provide relief to the patients with rare diseases across the country. It will provide access to affordable drugs as most of these medicines otherwise need to be imported and are priced exorbitantly which deterred treatment access. The Ministry should take more such initiative to manufacture drugs for rare diseases indigenously as effective addressing of the situation could only be through development of indigenous technologies tailored for our patient population. As Justice Chandrachud rightly said, the quest to treat rare diseases is futile if access to these treatments remains a challenge.
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