They are disorders amenable to one-time curative treatment, diseases requiring long term or lifelong treatment having relatively lower cost of treatment and diseases for which definitive treatment is available but challenges are to make optimal patient selection for benefit, very high cost and lifelong therapy, like Lysosomal Storage Disorders (LSD) like Gaucher Disease, Hunter Syndrome and Fabry Disease.
Noting that India has close to 50-100 million people affected by rare diseases or disorders, the policy report said almost 80 per cent of these rare condition patients are children and a leading cause for most of them not reaching adulthood is due to the high morbidity and mortality rates of these life-threatening diseases.
The policy proposes establishment of alternate funding of rare diseases through voluntary crowd funding and corporate funding.
It also seeks to provide assistance for patients needing one-time treatment for rare diseases increased to Rs 20 lakh from Rs 15 lakh.
“Assistance to be provided under the Rashtriya Arogaya Yojana,” the policy said.
The policy said the beneficiaries of the assistance are not limited to the BPL families but extended to almost 40 per cent of population eligible under the Pradhan Mantri Jan Arogya Yojana.
The policy also states the formulation of an alternative form of funding to help patients of rare diseases. This includes voluntary crowdfunding treatment by setting up a digital platform for voluntary individual contribution and corporate donors to voluntarily contribute to the treatment cost of patients of rare diseases.
The government has identified eight centres of excellence for aid and support to patients of rare diseases.
These include All India Institute of Medical Sciences, New Delhi, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow, Centre for DNA Fingerprinting and Diagnostics, Hyderabad, King Edward Medical Hospital, Mumbai, Institute of Post-Graduate Medical Education and Research, Kolkata.
Gopal Agrawal, Head of Market Access and Pricing- Takeda India, said over time, rare diseases have been gaining momentum with constant conversations and discussions and the announcement of the Rare Diseases Policy is a comprehensive step forward by the government that will further add impetus to the rare diseases ecosystem, thus ensuring timely access to therapy and treatment.
“With the policy now in place, there is considerable ground work that will be required for successful implementation of the policy. It will be instrumental to build innovative access initiatives and strategic partnerships across the rare disease landscape to bring customized solution for patients with targeted education, awareness, diagnosis and treatment,” he said.