A good start: On rare diseases and government support for treatment

It is binding on a welfare state to take care of every single citizen. Securing the wellbeing of every one, particularly those unable to help themselves, irrespective of whether they constitute a critical mass or not, is important. The recent notification of the National Policy for Rare Diseases 2021 after various interventions, including the court, is pegged on this principle of inclusion. A good start, it offers financial support for one-time treatment of up to ₹20 lakh, introduces a crowdfunding mechanism, creates a registry of rare diseases, and provides for early detection. In its final form, however, the policy has left the rare diseases lobby sorely disappointed on a crucial note. Rare diseases are broadly defined as diseases that infrequently occur in a population, and three markers are used — the total number of people with the disease, its prevalence, and the availability/non-availability of treatment options. WHO defines rare disease as having a frequency of less than 6.5-10 per 10,000 people. As per an estimate, there are 7,000 known rare diseases with an estimated 300 million patients in the world; 70 million are in India. According to the Organization for Rare Diseases India, these include inherited cancers, autoimmune disorders, congenital malformations, Hirschsprung’s disease, Gaucher disease, cystic fibrosis, muscular dystrophies and Lysosomal Storage Disorders (LSDs).

Much of the effort in the sector, from the medical side, has been to evolve formal definitions, in the hope that it would support the development of and commercialisation of drugs for treatment, and improve funding for research on rare diseases. Patient support groups have worked towards drumming up funding assistance for the treatment — one time or continual. The notification of the Policy comes as a logical conclusion to a long-fought battle, and yet, stops short of delivering the complete mandate. As per the Policy, diseases such as LSD for which definitive treatment is available, but costs are prohibitive, have been categorised as Group 3. However, no funding has been allocated for the immediate and lifelong treatment needs, for therapies already approved by the Drugs Controller General of India. Experts point out that the costs to help already-diagnosed patients might be in the range of ₹80-₹100 crore annually. If the Centre can extend the cost-sharing agreements that it has worked out with Kerala, Tamil Nadu and Karnataka, with other States too, its share of the annual costs will be halved. The Centre can, however, still set aside a substantial corpus to fund life-saving treatments, even as it rolls out the policy. Doing so will not only complete a job well begun — even if not yet half done — but also cement its commitment towards the welfare of every single citizen in India.