The government of India has recently submitted the National Policy for Treatment of Rare Diseases to the Delhi High Court. Organization For Rare Diseases India (ORDI) was part of the two-day meeting – National Initiative for Rare Diseases (NIRD) – conducted by AIIMS and ICMR in Delhi. ORDI has contributed to developing the draft policy. Now, we are also waiting for the next court hearing on the matter.
Considering the importance, the policy should be implemented promptly. We seek a clear direction from the central government on implementation. The policy talks about 60:40 ratio of the budget. So, every state should come forward to implement the plan immediately.
My suggestions to properly implement the policy
This policy has to have a short term and long term plan.
1. A department or committee needs to be formed for proper implementation and taking the responsibility of the policy.
2. Immediate Implementation of the policy, i.e., to release funds and ensuring treatment for all the patients who are on the waiting list in the country.
3. Providing treatment for other treatable rare diseases.
4. Developing a system to identify and treat other rare diseases.
5. A system for proper communication and coordination to ensure implementation of the policy in all states of the country.
1. Creating a web-based application for online application process
2. Developing materials to create awareness among the public, patients and their families, and health care providers.
3. Patient registry – a system for reporting and data collections. ICMR has launched the registry, and in the initial stage, only few disease conditions will be covered under the registry. It needs to be improved regularly to help all rare disease patients in India.
4. An epidemiological study should be conducted to estimate prevalence of rare diseases in each state
5. The policy should ensure Research and Development, treatment, diagnostic modalities, care and support, and development of orphan drugs.
Definition of rare diseases
Most importantly, the policy is silent on a definition of rare diseases. Every decision on rare disease depends on the number of patients and prevalence in the country. In the absence of proper data base in India, it is a huge challenge to take any decision to identify patients, prioritize resources, take corrective action, plan and research. Working on the limited data or a few diagnosed cases will not give the desired result.
Secondly, finding prevalence rates of rare diseases is also important. Due to high genetic nature of these conditions, the prevalence of a disease may vary from region to region. Implementing the policy without a definition for rare diseases and in the absence of epidemiology will be difficult. It is like shooting in the dark.
Prasanna Kumar B Shirol
Founder Director- ORDI (Organisation for Rare Diseases India)
Founder & Former President – LSDSS (Lysosomal Storage Disorder Support Society)
President -Pompe Foundation