-Mr. Prasanna Shirol, Co-founder and Executive Director, ORDI
With the world’s 2nd largest populous Rare Disease burden of an estimated 70 million patients, ironically, India still does not have a Health care policy in place for Rare Diseases. The benefits that exist are suboptimal, and are applicable only for BPL (Below Poverty Line) Families. While it is essential that the Health Care System caters to its citizens by provision of Excellent Public Health Service, the sorry state in India is that the vulnerable Rare Disease Community is simply overlooked. Patients and Disease support groups are fighting for an inclusive Rare Disease National Policy.
There is an urgent need to facilitate an all-inclusive Rare Disease policy that covers Pre and PostNatal screening, Genetic Counseling, Treatment for Treatable Diseases, and Long Term Supportive care for Non Treatable Diseases. The Policy should enable easy access to International treatment and Clinical Trials, Dedicated Research Funds for Rare Diseases.
The Dept of Health and Family welfare should work on “Rare Disease Policy for All. No Barriers” with funding and clear directives at central and state levels to support rare disease patients. Rare Disease is a National level burden, and having tremendous healthcare inequalities within states is an unreasonable approach.
A meager 5% of Rare Diseases have treatment options available; even those are hard to access as the drugs are available only in international countries and exorbitantly priced. In India, the access to treatment funds for common people impacted by Rare Diseases is Nil; and treatments can run from a few lakhs to crores of rupees depending on the condition. Having said that, today there are many conditions that also cost less, and the burden of treatments such as IEM diets, IVIG, Minor Surgeries, etc. can be managed with the existing policy; but, the benefits are restricted only for BPL(Below Poverty Line) Families, and not the Average Indian.
The only exception being the state of Karnataka, where free treatment is available to everyone in the state, for Lysosomal Storage Disorders (LSD), and PID (Primary Immunodeficiency) based on Judiciary interventions, but, the same can be availed across India only by ESI (Employee State Insurance) beneficiaries. Then we have the RAN- Rashtriya Arogya Nidhi of 15 lakhs, one time assistant for one treatment/intervention for group 1 diseases which include interventions like liver transplantation, stem cell therapy, etc. which is also applicable only for BPL Families.
Treatment must be made available, and accessible for all treatable conditions by providing easy access to all FDA and EMA-approved medicines. Treatment protocols must be standardized for all identified diseases in India through evidence-based treatments and approaches. Finally, there is a need to access International Clinical Trials with the combined support of all stakeholders of the RD Community.
Also, we do not have government infrastructure and facilities for diagnosing and treating Rare Diseases. There are probably only 100 plus well-trained Genetic Consultants available to the disposal of affected Families across India. If anything, people have to seek private hospitals for consultations, treatments, and interventions, which is again very expensive for even a well-to-do family, let alone an average Indian family.
The cost of supportive Therapy and lifelong care for non-treatable conditions is enormous. A Support system for untreatable conditions by means of dietary supplements, providing specialized therapies such as Physiotherapy, Occupational and Speech Therapy, and giving access to medical devices must be implemented. There is also a need for combining different policies and their benefits –The present draft of Rare Disease Policy covers only BPL categories. Rare Disease Policy has to be drafted to be made accessible for all; there should be No Barriers.
We have heard inspiring stories of Crowdfunding recently where nearly 16 crores of funds were raised through the support of the public. The PM making his contribution by waivers towards treatment for one Rare Disease Child was a welcome move. However, considering the actual Rare Disease burden of India, this is not the long-term solution.
The long-pending Rare Disease Policy is expected by 31st March; given the past draft of NPTRD, there isn’t much assurance in finding answers for the current challenges. Recently, the Delhi High Court directed the Central government to finalise and notify the National Health Policy for Rare Diseases on or before March 31, 2021. The Honorable Court has also made recommendations to further optimize the Policy. On behalf of the Indian Rare Disease Community, we are expecting the early implementation of the Rare Disease Policy in a true spirit.
