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Availability of drugs for rare diseases or orphan diseases at reasonably affordable prices has been a major issue the country, for that matter the entire world, has been facing for a long time now. It is true that for most of the rare disease patients, no treatment or very limited treatment options are available as the treatment cost is prohibitively high as most of the drugs are imported. At present, very few companies are manufacturing drugs for rare diseases. As the number of persons suffering from a 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 cannot be financially viable. So, when they manufacture such drugs, they sell them at prohibitively high costs, arguably to recover the research and development cost. It is a fact that the field of rare diseases is very complex and heterogeneous; and prevention, treatment and management of rare diseases have multiple challenges. A rare disease is a health condition of low prevalence that affects a small number of people compared to other prevalent diseases in the general population. WHO defines a rare disease as an often debilitating lifelong condition or disorder with a prevalence of one or less per 1,000 population. So, availability and access to medicines are important to reduce morbidity and mortality associated with rare diseases. There are over 7,000 rare diseases globally and about 450 of them have been reported in India. The most common rare diseases reported in India include haemophilia, thalassemia, sickle-cell anaemia and primary immuno deficiency in children, auto-immune diseases, lysosomal storage disorders such as pompe disease, hirschsprung disease, Gaucher’s disease, cystic fibrosis, haemangiomas and certain forms of muscular dystrophies.
Under this background, the appeal of the mothers and caregivers of children with rare diseases to the Supreme Court should be taken with the seriousness it deserves. Recently, relatives of rare diseases have sought the intervention of Chief Justice of India, Justice Sanjiv Khanna, to help them access life-saving medicines at affordable cost. They urged the judiciary to reinforce the Central government’s obligation to ensure access to life-saving medicines through compulsory licensing, price control, and local production to allow price-lowering generic competition, as provided under Indian law. In a letter to the Chief Justice, around 220 parents and caregivers of children with rare diseases urged the Supreme Court to take 'suo motu' cognizance of this dire situation and direct the expeditious resolution of pending legal cases concerning rare disease treatments. Five cases are pending with various High Courts and two matters are pending with the Supreme Court itself related to the patents and availability and affordability of rare disease drugs and the parents and caregivers requested immediate intervention of the Chief Justice to prevent further loss of innocent lives. No doubt, the financial assistance of up to Rs. 50 lakh per patient provided under the National Policy for Rare Diseases is commendable, but it falls woefully short in the face of treatments costing several lakhs or even crores. Indian patent law mandates that patents must be worked in a manner that ensures public access to medicines, yet these drugs are either unavailable in India or priced beyond reach. Petitions seeking compulsory licenses, local production, and generic alternatives, as permitted under the Patents Act, 1970 (Sections 83, 84, 92 and 100), have remained unresolved for years. The quest to treat rare diseases is futile if access to these treatments remains a challenge, especially outside major urban centres. So, the voice of this often-neglected community should not fall on deaf ears.
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