While certain southern states in the country have initiatives in place for supporting patients with rare diseases, the Centre needs to take a more serious note of the issue, especially regarding a sustainable funding mechanism for Group 3 rare disease patients in India.
With the Union Ministry of Health & Family Welfare likely to finalise the long pending National Policy on Rare Diseases and notify it by March 31, serious concerns are now being raised by patients and support societies that the revised policy may meet the same fate as the earlier National Policy for Treatment of Rare Diseases 2017 without any provision to support the treatment of patients diagnosed with Group 3 disorders like lysosomal storage disorders (LSDs).
Patient advocacy groups like the Organisation for Rare Diseases India (ORDI) and Lysosomal Storage Disorders Support Society (LSDSS) are concerned that the Union Ministry’s proposed crowdfunding mechanism may be inadequate, just like the provisioning under the umbrella scheme of Rashtriya Arogya Nidhi (RAN), to provide immediate treatment support to patients with these life threatening conditions—categorised as Group 3 disorders—and particularly those for which Drugs Controller General of India (DCGI) approved therapies are available.
According to patients who have been long awaiting support to start treatment, the Union of India should make immediate necessary provisions for all orphan diseases, as has been done by many other countries.
In the absence of a National Policy on Rare Diseases, a few states in the south have sought support from the Union of India to provide a matching grant in order to sustain the ‘proof of concepts’ shown in providing the much-needed funding support to patients diagnosed with life-threatening rare disease conditions, including LSDs.
Group 3 disorders are a category of rare diseases like Pompe disease, Gaucher disease, Fabry disease and MPS I which are serious, chronic, debilitating and fatal disorders, often requiring long-term, specialised treatment and chronic management and often causing severe handicaps and a catastrophic impact on the entire family. This group of rare diseases particularly impact children, causing 35% of deaths before age 1, 10% between the ages of 1-5 years and 12% between 5-15 years.
Karnataka has been engaged with this programme for close to five years now, providing treatment support to such children. Karnataka has reportedly spent around Rs 40 crore on providing treatment and other life support to the patients diagnosed with rare diseases.
Kerala and Tamil Nadu have also allocated funds from the state exchequer to provide life-saving therapies to a few of these children. In September 2020, the Kerala state government started an initiative to enrol two toddlers suffering from Pompe disease for the life-saving Enzyme Replacement Therapy (ERT) at the Government Medical College, Kozhikode. The infusion therapy was started following an order by a division bench of the Kerala High Court hearing a writ petition filed by the patient advocacy group LSDSS, set up to fight the cause of patients suffering from rare diseases across the country. Under the order of the Kerala High Court, the Centre allotted an amount of Rs 1.5 crore while the state sanctioned another Rs 50 lakh for the treatment of the two patients. Interestingly, members of the Kerala High Court Advocates’ Association, following an appeal by the High Court, also mobilised an amount of Rs 5 lakh for the treatment of the rare disease patients.
As for Tamil Nadu, in March 2020, the Madras High Court directed the Centre to pay Rs 4.4 crore for the treatment of patients with rare disease patients in the state. In the same order, the High Court also instructed the Tamil Nadu government to allocate Rs 5 crore for the treatment of the 11 rare disease patients in the state following a public interest litigation filed by the Delhi-based LSDSS in the Madras High Court, seeking financial aid from the government for the treatment of rare disease patients in the state of Tamil Nadu.
The health departments of each of the three states have also written to the Union of India to support the initiative by providing matching grant in order to provide sustenance to the programme besides allowing a scope to expand the treatment support to other eligible patients awaiting funding support.
With Rare Disease Day celebrated worldwide on 28 February each year to raise awareness about the issue, it has been sincerely requested that the government address the urgent need for a sustainable funding mechanism for Group 3 rare disease patients in the country.