Delhi High Court Judgement on Rare Diseases

 

In the first week of October, the Delhi High Court heard several petitions together that were filed by patients afflicted with a rare disease or their parents. The judgement  pronounced by Justice Pratibha Singh offers much hope to us. Here is a summary of key points (emphasis in italics are the aHUS India Foundation’s additions):

• The Govt of India must reconsider the cap of Rs. 50 lakhs for the treatment of rare disease patients under the National Policy for Rare Disease (NPRD). This cap is inadequate for some rare diseases.
• Donations for rare diseases must be added in Schedule VII of the Companies Act to enable Corporate Social Responsibility (CSR) contribution by companies, including Public Sector Undertakings.
• The Govt of India must establish a National Fund for Rare Diseases (NFRD) for which a sum of ₹974 crores must be allocated for the financial years 2024-25 and 2025-26. Either the same or a higher amount must be allocated in the next two financial years.
• The National Rare Diseases’ Cell consisting of one or more Nodal Officers in the health ministry will administer the NFRD and release funds under NPRD as directed by the National Rare Diseases’ Committee (NRDC).
• If the fund is not utilised in a year, it will not lapse (which means it would carry forward to subsequent years)
• A portal to be setup within 3 months that will have a patient registry and information on fund utilisation, nearest centres of excelence etc.
• A crowdfunding platform must be setup in 2 weeks, details of which should be publicised on regular and social media.

This ruling is very far-reaching and impactful if the directions are followed in letter and spirit by the parties concerned. It is important that those directed by the court to do these things do so in a time bound manner. Rare disease patients should not continue to be left to fend for themselves. Health is a basic human right and must not be looked at by the government or anyone as a favour being done on its citizens.

aHUS India needs Volunteers

In India, aHUS is at the cusp of a radical new change in the way the disease is managed. Like you are probably aware, two drugs are under clinical trials in the country and the only drug currently approved elsewhere - Eculizumab - is likely to begin being sold some time next year. 

Until now, things were very different. There was little hope of getting any drugs in India. Hopefully, the coming months will see the beginning of a new dawn for aHUS patients in the country.

This is a time when we patients need to do everything we can to shape the course of drug availability and pricing so that we ensure these events do not remain mere headlines and plans on paper. Of what use is a drug trial if patients do not get access to it after approval? Of what use is drug approval if patients are unable to get it due to the lack of support from the government or pricing being within our reach?

Until now, the aHUS India Foundation did not have any volunteers. This may not be sustainable. We definitely need more hands. If a few of us come forward and pledge our support, we can work together to achieve our goals - treatment for all!

You do not need to commit a whole lot of time. What is needed is a minimal commitment. And everything is completely voluntary.

So, if you would like to be part of this, please send an email to ahusindia@gmail.com and we can discuss the way forward.

Sign up for a newsletter from aHUS India

In recent months, there have been significant developments in the context of aHUS in India such as the approval of Soliris in India and clinical trials for Crovalimab and Iptacopan. This marks an exciting period for aHUS patients, families, and doctors in India. For many years, there was little positive news regarding this disease in our country, but thankfully, that is changing.

The aHUS India Foundation is launching a newsletter to provide important updates on aHUS developments relevant to India. Given the recent advancements in aHUS treatment and research in India, we believe it is an opportune time to start this initiative. Our newsletter will be sent out approximately every 2-3 months, keeping you informed about the latest news and progress in this field.

If you would like to stay updated on these advancements, please sign up for our newsletter. Rest assured, your email and any other details you provide will remain confidential. It is crucial for us to unite, pool our resources, and advocate for ourselves because no one else will do it for us. Sign up here, and we will keep you informed. Thank you.

Soliris recommended for approval for aHUS in India

In some very welcome news for Indian aHUS patients who are not generally used to any such news, the Experts Committee under India's drug regulator, the Central Drug Standard Control Organisation (CDSCO) has recommended to grant approval to Astra Zeneca (the company that owns Alexion Pharmaceuticals) to import and market the drug Soliris (Eculizumab) in India.

Some important takeaways:

1. This approval is subject to Astra Zeneca India conducting Phase IV clinical trials to establish safety and effectiveness in the Indian population. There is no clarity yet on how this will be done and how long it will take.

2. Only "Centres of Excellence" will be authorised to prescribe this drug. This is likely to include reputed institutes such as AIIMS, PGI, Chandigarh etc.

3. There is still no clarity on when the drug will actually become available in India.

4. Most importantly, there is no information on how Astra Zeneca will price the drug. In many developed countries where the drug is available, it is one of the most expensive drugs in the world with the cost of one vial running into several lakhs of rupees and a dose being needed at least every two to four weeks.

aHUS patients and their families should be aware that the Indian government recently expanded their Rare Disease Program to increase the grant provided under this scheme from Rs. 20 lakhs to Rs. 50 lakhs. While this may still not be enough beyond a few months, at least it is a start. aHUS is also included in the list of diseases that are covered by this program. Please see this and this for more information.

After all these years of despondence, there is finally some hope for us. Clearly, we are not out of the woods yet. But there is hope that a path will emerge soon.

Crovalimab trial in India in children and adults

Just a few weeks back, we posted this article where we alerted Indian aHUS patients to a trial on Iptacopan, an oral aHUS drug. And now there is another trial for aHUS patients for the drug Crovalimab from Roche.

Crovalimab is a complement inhibitor which can be administered to patients recently diagnosed with aHUS. The Phase III trial is for both adults and children who have not yet progressed to kidney failure (are dialysis dependent). 

Here are the details of the two trials of this new drug:

Adults and Adolescents:

Information about the trial

Participating sites & Investigators (Currently Active for Enrolment)

1. All India Institute of Medical Sciences, Delhi,  Principal Investigator: Dr Aditi Sinha

2. Medanta, The Medicity, Gurugram | Principal Investigator: Dr Sidharth Sethi

3. Sawai Man Singh (SMS) Hospital, Jaipur, Principal Investigator: Dr Dhananjai Agrawal

Children:

Information about the trial

Participating sites & Investigators (Currently Active for Enrolment)

1. All India Institute of Medical Sciences, Delhi,  Principal Investigator: Dr Aditi Sinha

2. Medanta, The Medicity, Gurugram | Principal Investigator: Dr Sidharth Sethi

This is an earnest request to all nephrologists and other doctors who come across aHUS patients in India. Please take this opportunity to enrol your patients into these trials. Until now, aHUS patients had nowhere to turn to. We no at least have trials happening in the country which should hopefully, pave the way for general availability in due course.