Delays in processing funding applications by CoEs under NPRD

The Government of India has included atypical HUS in its National Policy for Rare Diseases (NPRD). Under this policy, several Centers of Excellence (CoEs) across the country were chosen to handle applications from doctors with patients suffering from these rare diseases for funding treatment to an extent of Rs. 50 lakhs.

Since Soliris became available in India, doctors have been applying for funds allocated for treating aHUS with this drug. Sadly, these applications are not being processed quickly. Atypical HUS is a very serious disease. Because of these delays, patients are either dying or developing kidney failure.

This is unfortunate because the government has provided funds for treating this disease and these funds are not being made available to deserving patients. Due to bureaucratic delays at the CoEs, patients are not getting the treatment they need. This is an appeal to all CoEs to process funding applications for atypical HUS quickly. Delays can endanger patients' lives and lead to death or kidney failure requiring dialysis.

Making available vials of Eculizumab to be used in emergencies

The National Programme for Rare Diseases in India has made drugs like Eculizumab available for patients with Atypical Hemolytic Uremic Syndrome. Centers of Excellence (CoEs) have been chosen to handle applications from doctors who need Eculizumab for their patients.

However, these centers are taking a long time to process applications and provide the drug to patients. Atypical HUS is a serious disease that can quickly lead to death or end-stage kidney disease, so delays are dangerous. 

To solve this problem, each center of excellence could keep a few vials of Eculizumab on hand for emergencies. When a patient shows signs of thrombotic microangiopathy, hemolytic anemia, and renal insufficiency - the triad classically associated with aHUS, they can be given the drug right away. This can help prevent kidney failure and stop the disease from getting worse.

For this plan to work, the government and CoEs need to cooperate and ensure that enough vials are available at each center. This way, patients can receive immediate treatment while waiting for further tests to confirm the diagnosis.

The Atypical HUS India Foundation urges the government and CoEs to work together to make this happen.

How you can get access to Soliris (Eculizumab) in India: Government Funding Route

The Government of India has a National Program for Rare Diseases (NPRD) that funds, up to a certain extent, treatment for rare diseases including aHUS. With Soliris (drug name: Eculizumab) now becoming available in India through the company directly, supply should be more reliable. Until now, the drug was available through importers who imported such drugs through unofficial, non-company based channels.

For those who can afford to pay for the drug out-of-pocket, please refer to this post for more details.

This post will outline the steps to take to apply for assistance in procuring the drug through the Government's NPRD.

• Identify the closest Centre of Excellence (CoE) to you. Here is a complete list of CoEs in India
• Visit the CoE campus and meet a senior doctor in the nephrologiy department. You may need to take an appointment by phone or in-person. At the time of enrolment, you will be given a Hospital ID. Keep this handy
• Carry the following with you when you meet the nephrologist:
• ID proof (Aadhaar / PAN / Voter ID) - original and 2 copies - if patient is a minor, also carry ID proof of parent / guardian
• 2 Passport size photographs
• Your medical file that has past prescriptions
• Any hospitalisation discharge summaries
• Current Prescription from the nephrologist treating you
• Recent lab reports - CBP, LDH, Kidney function tests, Urine Analysis etc.
• Genetic Test Reports, if available
• The nephrologist may require some other tests to be done or additional documents may need to be provided. Arrange these as requested by them
• Once all documents are provided, the nephrologist (or their assistant / adminstrator) will enter the details into the NPRD portal. this will include the number of vials, duration of treatment etc. An enrollment number will be generated and given to you. Keep this number safely
• There is a Rare Disease Committee that has been constituted at each CoE. This committee meets periodically and even on emergency basis to review all applications submitted and decides the merits of each application. The committee meets once every 1-3 months (depending on the hospital and the number of cases to review; they may also meet on emergency basis if needed). The committe decides to approve / reject / recomend to modify the application
• Once a decision has been reached, you will be notified of the decision
• Keep in touch with the nephrologist you met about this and any other queries you may have. Sometimes, the nephrologist might put you in touch with another coordinator who would be able to answer any queries you may have
• The committee may request additional documentation including some investigations. Make sure you get these in a timely manner
• Also, if you undergo any doctor consults / investigations after the initial form is submitted, make sure you give this to the doctor / assistant in the CoE to submit in the portal
• Note that approval by the committee might take up to a few months
• Once the application is approved, then the CoE will raise the funding requisition with the government for which you may be asked to sign some consent forms
• Once the funding request is approved, the CoE will procure the drug from the company through their pharmacy
• Once the drug reaches the pharmacy, speak to the nephrologist to discuss the next steps

Please be aware that this is a relatively new process for the CoEs. Even among the CoEs, there are some that are better equipped than others.  Remember, you need to advocate for yourself or your loved one. You will need to keep following up with the relevant stakeholders. Do not expect things to be handed to you on a platter. You will need to push people to help you. Do drop us a line with your experiences and we will do the best we can to help. All the best!

Changes needed to the National Program for Rare Diseases in India

The Government of India launched the National Program for Rare Diseases (NPRD) a few years back. In its current form, 63 rare diseases (including Atypical HUS) are covered. The program will fund a one time grant of Rs. 50 lakhs to each patient towards treatment. Decisions to fund such treatment will be made by Centres of Excellence (CoE) identified by the government.

Such a policy was unthinkable till a few years back. The debate between spending large sums of money on the treatment of a few people versus spending that money on basic healthcare necessities like preventing infant mortality, basic sanitation and other aspects of primary healthcare which impact several times more people is far from settled. While I may be biased in favour of the former due to my medical history, I do acknowledge that the choice is tricky. I am grateful therefore that the Indian government chose to set up the NPRD which impacts only a few people.

I would still suggest that two changes be made to the NPRD.

One size does not fit all

Currently, irrespective of the disease and the extremely varied types and durations of treatment, the NPRD stipulates the same grant for every patient. While the Rs. 50 lakhs might be more than enough for some, it is woefully inadequate for others. Even within the same rare disease, the requirements could be different. 

Take my disease, aHUS, for instance. Depending on the age of the patient and specific genetic mutation, the dosage and the duration of treatment with Eculizumab (the only approved drug to treat the disease) could vary. Some people would need only two vials twice a month while others could need three to four vials twice a month. Again, some patients may need it lifelong while others could need it only for a few months.

Keeping this reality in mind, the NPRD needs to allocate different funds for different patients. I understand this could make the decision process extremely complex. Who decides, for instance, which patient gets how much money? A group of experts can be constituted for each disease and a framework can be established to guide such decisions. Other solutions can be explored based on the experience of other nations where these problems have been successfully resolved.

Involvement of CoEs

In the current policy, decisions pertaining to applications for assistance under the NPRD are to be taken by CoEs. One can only imagine the delays this would bring about in getting access to the drugs by patients. The process would involve the treating physician getting the history and the clinical summary ready and then getting this information to the appropriate committee in a CoE close by. The authorities who decide on such applications in the CoE would need to then meet and review the dossier submitted. They might have further questions which would need to be communicated to the treating physician and then if everything is clear, they would decide. 

In diseases such aHUS, time is critical. Quick administration of the drug can mean the difference between remission and progression to kidney failure or even death. In such circumstances, does it really make sense to allow so much delay in deciding on the application?

One possible solution could be to not require a committee but one doctor among a panel of doctors in the CoE to decide on the application. The treating physician should be able to request urgent hearing of the case and decisions should be taken in less than 48 hours. Such models have been adopted in other nations already where similar circumstances exist.

The Government of India has shown amazing foresight and consideration for rare disease patients in coming up with the NPRD. If the kinks mentioned above can be ironed out, it would do tremendous benefits to the intended beneficiaries of this policy. 

How India's National Program for Rare Diseases can help aHUS patients

Here are some important facts to know about the Indian Government's National Program for Rare Diseases (NPRD) can help aHUS patients.

National Program for Rare Diseases

This program, instituted by Ministry of Health and Family Welfare, offers financial help and access to care for rare disease patients. As of August 2024, 63 rare diseases were covered under this program. Atypical Hemolytic Uremic Syndrome (aHUS) is part of this list of diseases.

Three groups of rare diseases are covered by this program:

1. Diseases that can be cured with a one-time treatment
2. Diseases that need lifelong treatment but costs are low
3. Diseases that need lifelong treatment and costs are very high

aHUS comes under the third group.

Support offered by the government

The government will fund up to Rs. 50 lakh per patient for treatment. The government may offer extra flexibility for Group 3 cases like aHUS, based on the patient’s situation. No GST or customs duty will be levied on rare disease drugs imported for treatment.

Centres of Excellence

The government has identified a list of Centres of Excellence (CoE) which would be the nodal agencies to facilitate treatment under this program. Here is a list of the CoEs in different cities in India.

Things to do to avail the support from the government

Patients must speak to their doctors about registering with the NPRD at a Centre of Excellence. This would involve medical evaluation to confirm the diagnosis, some documentation which would included some tests, medical history etc. There is an application form that would need to be filled in. Once that is submitted, then patients would need to wait for the approval to come.

List of Centres of Excellence under NPRD

Here is a list of the Centres of Excellence listed under the National Program for Rare Diseases (NPRD). These centres are authorised to approve any treatment for rare disease patients under the NPRD where a fund of up to Rs. 50 lakhs is granted.

• All India Institute of Medical Sciences, New Delhi
• Maulana Azad Medical College, New Delhi
• Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow
• Post Graduate Institute of Medical Education and Research, Chandigarh
• CDFD with Nizam Institute of Medical Sciences, Hyderabad
• King Edward Memorial Hospital, Mumbai
• Institute of Post-Graduate Medical Education and Research, Kolkata
• Center For Human Genetics (CHG) with Indira Gandhi Hospital, Bengaluru
• All India Institute of Medical Sciences, Jodhpur
• Institute Of Child Health and Hospital for Children, Chennai
• Government Medical College, Thiruvananthapuram
• All India Institute of Medical Sciences, Bhopal

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.