‘Urgent need for India to implement National Policy for Rare Diseases’
Pharma expert want India implement its National Policy for Rare Diseases effectively
Khammam: There is an urgent need for India to implement its National Policy for Rare Diseases-2021 in an effective way to aid millions of people having rare diseases, suggested Indian Organisation for Rare Diseases (IORD) president and CEO, Dr. Ramaiah Muthyala.
The Ministry of Health formulated National Policy for Treatment of Rare Diseases (NPTRD) in July, 2017, revised it and finalised it in 2021. But it is not backed by epidemiological data on incidence and prevalence of rare diseases, a flaw, admitted by the Centre in its own policy document. How could government implement a policy without proper data, questioned Dr. Muthyala.
The document has not even defined rare diseases but vaguely classified them into three groups mentioning just a few. “There are around 7000 rare diseases, 80 percent of them are genetic. Therapies available for just below five percent diseases, what about the remaining,” he posed.
About 95 percent of rare diseases have no approved treatment; only 1 in 10 patients receive disease specific treatment. Drugs for rare diseases are highly expensive and drug companies shy away from developing and marketing drugs for them as it is not a profitable market.
As many as 450 bulk drugs for rare disease are made in Hyderabad, the drug capital of India. But they are not available locally, but exported to foreign nations to earn profits. India has to import capsules or injections, which is a costly affair, the IORD CEO explained.
Dr. Muthyala told Telangana Today that an ex-councillor of BJP of Vidisha town in MP, Sanjeev Mishra and his entire family died by suicide on Jan 26, 2023. The reason was one of his sons suffered from a genetic disease and he was unable to save him. The incident was taken to the notice of the PM to tell him the gravity of the situation, but in vain, he noted.
“This is the plight of millions in India. What is needed now is data compilation, crucial for early medical intervention. I have written to ICMR many times in this regard but there is no response. The PM’s office and Census Commissioner are approached to add a column in census form for rare diseases to obtain data. The answer was there was no space for a new column” Dr. Muthyala lamented.
Many are not aware of rare diseases including the medical fraternity. Genetics must be taught in schools and colleges. A dedicated office or wing has to be set up at national level to deal with rare diseases, he suggested.
Dr. Muthyala, in association with the district administration, embarked on a project to compile data of persons with rare diseases in November 2022 in 74 habitations in 24 gram panchayats covering 60, 000 people of Kamepalli, besides two lakh population in Khammam city as a sample.
He trained Asha workers and ANMs, prepared a questionnaire on how to identify rare diseases and even personally visited four villages. Data from 17 villages obtained wherein 49 people showed rare medical conditions. But the work stalled later. Many efforts to restore it yielded no results. But when he presented the idea in a WHO meeting in Geneva last year many countries came forward to adopt it.
“India is the only nation to have a system like Asha workers in the world. Data can be compiled by engaging them. No one asked me to do it but I wanted to show that it can be done in a simple, economical and effective way. If Telangana, the only State in the country to recognise importance of IORD’s work, demonstrates it the others States may follow it”, he hoped.
What are rare diseases
India’s NPRD-2021 states that about 450 rare diseases are recorded in the country. The most common rare diseases include Haemophilia, Thalassemia, Sickle-cell Anaemia and Primary Immunodeficiency disorders in children, auto-immune diseases, Lysosomal storage disorders such as Pompe disease, Hirschsprung disease, Gaucher’s disease, Cystic Fibrosis, Hemangiomas and certain forms of muscular dystrophies.