Balm for rare diseases

The National Rare Diseases Policy announced by the government is a step forward in an area that called for much attention from both government and society. It addresses a number of issues and challenges faced by patients of rare diseases and their families and caregivers, but it also has scope for improvement. Rare diseases present unique problems which are not seen in the treatment of common and conventional diseases. There are over 7,000 classified rare diseases in India but less than 5% of them have known and accepted therapies. Even their definitions vary across counties, but it is estimated that 6 out of 10,000 people suffer from some rare disease or another. There is no data about their prevalence in India and so only some known diseases are classified as rare. Most of them are genetic and so treatment is difficult, and even when it is available, it is very costly and beyond the reach of most families.

The policy has recommended the enhancement of government support for the treatment of a rare disease from Rs 15 lakh to Rs 20 lakh. The diseases are classified into three groups and there are conditions attached to the treatment. The government would also set up centres of excellence for treatment and create and upgrade infrastructure for screening, testing and treatment. But there are complaints about every aspect of the policy, including funding and financial support, eligibility for treatment, identification of diseases whose treatment would get support and the various conditions and procedures that need to be followed. While the treatment costs should be shared between the Centre and the states, the performance of the states has been uneven. Karnataka, Tamil Nadu and Kerala have been more supportive and generous in this regard. Organisation for Rare Diseases, an umbrella body representing many organisations in the field, has made many suggestions to improve the policy.

The new policy has occasioned discussions and consultations on rare diseases and issues connected with them. They should lead to greater awareness and the opening of more avenues to help those who are afflicted with such diseases. The policy does not cover all rare diseases, and insurance is not available for most of them. Most diseases need treatment for the long term and sometimes life-long treatment. Private companies are not interested in doing research on such diseases and in developing medicines because there will not be a large market for the medicines. For governments, public health issues that concern large numbers of people are more important. The pandemic has pushed even common diseases into the background. But there is much more that governments can do to facilitate and fund treatment of rare diseases and to bring down the cost of treatment.