New Delhi: The Union health ministry is working on a Rs 900-crore mega initiative to procure high-value drugs in bulk for some rare diseases, which can be life-threatening and chronic, and medicines for which are prohibitively expensive, ThePrint has learnt.
A rare disease is one that has particularly low prevalence and affects a small number of people — less than one per 1,000 population — according to World Health Organization (WHO) estimates. But rare diseases collectively afflict about 6-8 percent of the population in any country at any given time.
The first-ever plan of its kind has been devised following a nudge by the Delhi High Court to ensure that when patients contract rare diseases for which drugs are available, they are not denied treatment, and they can effectively reap the benefits of the Centre’s National Policy for Rare Diseases, 2021.
The High Court has been hearing several clubbed petitions on making the treatment affordable for rare disease patients.
Later, some intervention applications were also filed to ask the government to negotiate with pharmaceutical companies and provide treatment to all rare disease patients similar to how it is done in China, where the government has negotiated with some drugmakers to procure life-saving drugs at lower rates.
Nearly 80 percent of these diseases are genetic in nature. Out of about 7,000 rare diseases identified, drugs for less than 5 percent are available, many of which are extremely expensive. This often compels people in countries like India to resort to measures like crowdfunding to fund treatment.
According to information shared by the health ministry last year, India could have 8.4 to 19 crore rare disease patients.
The government has categorised 13 rare diseases as priority rare diseases but therapies for all are not available. Efforts are ongoing to develop drugs for some conditions indigenously.
Following recommendations by a high-level committee chaired by Dr Rajiv Bahl, director general of the Indian Council of Medical research (ICMR), the government negotiated with a few multinational drugmakers to procure bulk drugs for some priority rare diseases at lower rates, senior officials in the health ministry told ThePrint.
Last year, the Delhi High Court appointed a five-member committee known as the National Committee for Rare Disease to oversee the implementation of the National Policy for Rare Diseases, 2021.
The pharmaceutical giants which have been part of the committee’s negotiations include Cambridge-based AstraZeneca, Switzerland-based Novartis and Roche, Cambridge-based Sarepta and Paris-based Sanofi, top officials in the health ministry told ThePrint.
Some of the diseases that would be covered under the initiative are: Gaucher disease (genetic disease that results in the buildup of fats to accumulate in organs such as liver and spleen, affecting their ability to function); Duchenne muscular dystrophy (characterised by progressive muscle weakness and degeneration); cystic fibrosis (caused by mutation in a gene and affects lungs, pancreas and other organs); and spinal muscular atrophy (genetic neuromuscular disease that robs people of physical strength by affecting motor nerve cells in the spinal cord).
“The negotiations with drugmakers have been finalised, details of which have also been submitted to the High Court. We are now in the process of working out the modalities of how patients can access the drugs,” a senior health ministry official told ThePrint.
The procurement is estimated to cost the Centre about Rs 900 crore for two years.
Another official in the ministry told ThePrint that the drugs would be made available to patients with rare diseases through designated centres known as centres of excellence (COEs) for rare diseases.
ThePrint reached the health ministry over email for a comment on the proposed initiative. This report will be updated if and when a response is received.
Also Read: Over 60% drug cocktails used for mental illnesses in India unapproved by apex regulator, finds study
Ongoing initiatives falling short
As part of its National Policy for Rare Diseases, the health ministry announced in 2021 a Rs 20 lakh one-time aid for a certain category of rare disease patients.
A year later, this amount was increased to Rs 50 lakh following complaints that Rs 20 lakh was too little. It was also decided that all rare disease patients in India would be eligible for this one-time grant, provided the special committees at the COEs approved their applications.
“The COEs, however, have been struggling to implement the initiative, and have been unable to help a significant number of patients so far,” Prasanna Shirol, co-founder and executive director of Bengaluru-based Organization for Rare Diseases India (ORDI), told ThePrint.
Government statistics support what Shirol says.
In 2022-23, the government supported 203 patients by disbursing medicines worth Rs 35 crore. In 2023-24, this amount rose to Rs 74 crore and in the current fiscal, a budget of Rs 82.4 crore has been allocated for the purpose, of which Rs 34.2 crore has already been disbursed.
The money was disbursed by the government to individual patients following recommendations by the COEs, which include centres such as AIIMS and Maulana Azad Medical College in New Delhi, Sanjay Gandhi Post Graduate Institute of Medical Sciences in Lucknow, King Edward Medical College in Mumbai, and Centre for DNA Fingerprinting and Diagnostics with Nizam’s Institute of Medical Sciences in Hyderabad, among others.
However, Archana Panda, co-founder, and director of patient advocacy for Gurgaon-based patient support organisation CureSMA, told ThePrint that while COEs are disbursing some amount for certain diseases, beneficiaries in most cases are only small children or those who need comparatively less expensive drugs, because COEs prioritise them for treatment.
“There are 12 COEs in India and they are choosing patients based on their own discretion. It is either small children who, in some cases, need lower grants because the cost of the drugs is often linked with weight, or kids for whom the cost of treatment is low. So far, the scheme has benefitted just one spinal muscular atrophy patient — a child less than a year old,” Panda said.
A large number of patients, who are older or need life-saving drugs which are relatively costly and required periodically, are struggling to access the treatment, she added.
The only spinal muscular atrophy patient who was aided under the initiative received a drug called risdiplam from Switzerland-based pharmaceutical firm Roche pharma. Risdiplam is one of the three drugs available to treat the condition, treatment of which costs about Rs 72 lakh annually for the first 2 years and Rs 56 lakh annually from the third year.
Another drug for the condition is Zolgensma, a gene therapy by Novartis. One of the costliest drugs in the world, it works by correcting faulty genes responsible for causing spinal muscular atrophy. For kids under 2, it costs a whopping Rs 17 crore in India.
Senior government functionaries have also acknowledged that it is a worrying fact that few patients, compared to the large number of rare disease patients in need of crucial and innovative therapies, are receiving help.
“…we realise that even this (the financial aid for rare disease patients) is not sufficient as we don’t want to leave any patient behind,” L Swasticharan, additional deputy director general of the directorate general of health services in the health ministry, had said last month during the National Conference on spinal muscular atrophy organised by CureSMA.
A crowdfunding platform initiated by the ministry in 2022 to seek donations for rare disease patients has over 2,800 registered patients but has collected less than Rs 3.5 lakh so far.
“There is a huge unmet need of life-saving drugs for rare disease patients and clearly, existing initiatives have not done much,” Panda said.
Procurement plan: a new hope
Health ministry officials quoted earlier said the plan on bulk procurement of drugs for rare diseases would be rolled out this year after finalising the blueprint on how the drugs will be disbursed to needy patients.
“On several occasions, patients from smaller towns struggle to access the treatment due to difficulty in reaching the designated centres as the number of such institutions is limited,” an official said.
Due to this, he added, some third parties such as non-governmental organisations working in the area could be roped in. “Once this part of the initiative is done, we will be in a position to launch the initiative.”
This may mean good news for people like P.A. Abhinand, a 34-year-old academician and scientist from Chennai and a spinal muscular atrophy patient, who is largely dependent on crowdfunding and support from his relatives and friends to fund his treatment.
“So far, I have not benefited from the government initiative for rare disease patients. On crowdfunding platforms, too, while people feel sympathetic towards kids with life-threatening conditions, there is little support for patients like me,” Abhinand told ThePrint.
(Edited by Radifah Kabir)