Dec 26, 2018


1. MOH is unhappy with the court injunctions and does not see it in a positive light. They have decided to revisit the definition of Rare diseases and arrive at a real burden before they suggest a budget to the finance department. They also feel that including only a few diseases may not be fair to other RDs and have therefore requested information from Labs and genetic clinics on existing patients under treatment. The list of diseases for treatment inclusion is likely to broaden beyond LSDs and will include supportive and nutritional therapy.

2. The ministry might consider capping the price of certain medicines, in which case the industry may withdraw the treatments from the country- my inference, so lets be careful.

3. Sanjeeva Kumar has requested me to meet with ICMR and understand the progress for the RD registry. He has also requested help from patient groups to populate the registry

4. It is unclear at present whether the policy if implemented will be through NHM or RAN and until that is clarified the pathways for fund request from states will not be provided through a GO. Certain states like Kerala, Delhi, AP etc have written to the center stating that RDs are not a priority for them and they will not be able to collect a pool for the states’ contribution. The Hemoglobinopathies and PID are through NMH with a dedicated fund allocation (800 crores for Hemoglobinopathies). NHM therefore seems a more feasible option

5. Mr. Jhalani has requested support to draft the guidelines for PIP(even though the court affidavit states that RDs are tertiary care and do not fall under the ambit of NHM)

6. The budget session of parliament is early this year due to elections in April and it appears as if there will be no fund allocation for RDs in the interim budget in February, however it will be considered in the supplementary PIP(if implemented through NHM)

Given the above situation, my suggestion is to stay united and provide a simple but implementable solution to the ministry. At present there are 463 applications for LSD/MPS treatment from 6 states. Can I-ORD, ORDI, LSSDS etc help me arrive at a likely total of all patients that will require treatment. I am trying to collect information for other diseases as well

The CT rules define an orphan drug as treatment for a condition that is in less than 2 lac population. We need to consider whether we will stick to this definition for new drug approvals or align it with the RD policy

A meeting of a few members of the national technical group is likely on 21st Jan and it will be good if we can provide some information by then.

I will be reaching out to ICMR and Dr. Kabra in the first week of Jan as directed by the ministry. Will also meet Dr. Agnani (IAS) who is in charge of the RD policy.

An industry task force on RDs met on 20th December to deliberate on their response to the ministry.

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  • A rare disease is a health condition which has a low prevalence and affects a small percentage of the population. Rare diseases include inherited cancers, auto-immune disorders, congenital malformations, and infectious diseases Some common Rare Diseases are Hemangiomas, Hirschsprung disease, Gaucher disease, Cystic Fibrosis, Muscular Dystrophies and Lysosomal Storage Disorders. In India, as an estimated over 70 million people are believed to be suffering from some kind of rare disorder. These numbers clearly indicate that even though these diseases are rare, the patients suffering from them are not. rare diseases are severe and chronic, and are often life-threatening. They are disabling wherein the quality of life of a patient is compromised dramatically and they are unable to do even simple daily tasks. Rare diseases are difficult to diagnose because in their early stages, symptoms may be absent or masked or misunderstood or confused with other diseases.

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