Lack of funding support for diagnosed Group 3 patients would sound the death-knell: PAGs

Patient advocacy groups (PAG) members have expressed serious concern at the lack of funding support for patients diagnosed with life-threating rare, genetic disorders in the National Policy for Rare Diseases 2021, explaining that the Union Government has left the patients with Group 3 disorders to fend for themselves.

As per the National Policy, diseases like Lysosomal Storage Disorders (LSDs) for which definitive treatment is available but challenges are to make optimal patient selection for benefit, very high cost and lifelong therapy have been categorised as Group 3.

According to Mr Manjit Singh, National President of Lysosomal Storage Disorders Society of India, the new policy offers no support to the patients, awaiting treatment for a long time since the earlier National Policy for Treatment of Rare Diseases 2017 was kept in abeyance. In the absence of any funding support for the life-saving therapies, close to 130 odd patients are left with no option but to wait for the inevitable. He said several patients — mostly children – have lost their lives in the interim period since the earlier policy was kept in abeyance, explaining that unlike conditions under Group 1 and Group 2, patients with Group 3 disorders require sustainable treatment support.

While the Ministry of Health & Family Welfare cited the need to balance competing priorities of public health in resource constrained settings, health economist Prof Viswanath Pingali from IIM-A said he is surprised to see the lack of any proactive response to address the immediate treatment needs of 130-odd patients with treatable conditions, for which the Drugs Controller General of India approved therapies are already available in the country.

“Looking at the number of Rare Disease patients – diagnosed and considered eligible for treatment by the respective state technical committees, the immediate requirement of funds to support the immediate treatment needs of the diagnosed patients shouldn’t have exceeded INR 80 – 100 crore annually. If one is to look at it holistically, the Centre’s contribution would work out to INR 40 – 50 crore – if it is able to convince the state(s) for a load-sharing model, as a few states like Kerala, Tamil Nadu and Karnataka have already indicated,” he said.

Patients and their support groups had recently written to the health ministry seeking the following before the policy is notified:
Create an immediate seed-funding of INR 80-100 crore while rolling out the National Policy so that the life-saving therapy of all those patients with treatable Group 3 disorders like Lysosomal Storage Disorders, for which DCGI approved treatment is available, can be provided, thereby reducing any further loss of life

Design and execute a 100-day roll-out plan after the National Policy is notified to prioritise treatment of all eligible Rare Disease patients in the country, approximately around 130 of them

Prioritise and encourage states with a matching grant which have demonstrated a proof of concept in providing life-saving therapy to Rare Disease patients in the country. Three states – Karnataka, Kerala and Tamil Nadu – have already made a request to the Centre to provide an immediate matching grant to extend the treatment to more eligible patients. By doing so, the process of diagnosis and treatment would also be decentralised with the states being empowered to take a decision in favour of the Rare Disease patients.