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Compassionate use and access programmes for blood cancer | CION Cancer Clinics
Yes, compassionate use exists in India. Your haematologist can ask the national drug regulator and the medicine's maker to release an unapproved medicine for you, if your blood cancer has no approved option left and no trial fits. It is rare, it takes weeks, and the company can refuse. This guide explains the routes, who they suit, and what to ask. At CION Cancer Clinics, our haematology team supports patients and families through treatment, cost questions and life at home.
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Medically reviewed by Dr. Basudev PokhrelConsultant Haematologist · last reviewed September 2026, next review due September 2027
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On this page
- What is a compassionate use programme?
- Which route to an unapproved or costly medicine fits your situation?
- How does a compassionate use request actually work?
- What do these access terms mean in plain language?
- Who is compassionate use likely to suit, and who not?
- What do families often get wrong about compassionate use?
- How is compassionate use different from joining a trial?
- Common questions about compassionate use in India
The short answer
What is a compassionate use programme?
A compassionate use programme lets a doctor request a medicine that is not yet approved in India, for one patient with a serious blood cancer who has run out of approved options. It is possible in India, but it is rare, slow and never automatic.
Why it exists
New blood cancer medicines are often approved abroad before they are approved here. A few patients cannot wait for that. They may have tried the standard treatments, and they may not fit any clinical trial. For them, the law allows a narrow door. India's New Drugs and Clinical Trials Rules let a hospital ask the national drug regulator for permission to bring in an unapproved medicine for a named patient with a life-threatening illness.
Three parties have to say yes
Your haematologist has to believe the medicine is a reasonable next step. The company that makes it has to agree to supply it, and many decline. The regulator, the Central Drugs Standard Control Organisation, has to grant permission. If any one of them says no, the request stops there. You cannot apply on your own as a family.
Compassionate use is not the same as a discount or a free-medicine scheme for an approved drug. Those are separate, and more common.The routes
Which route to an unapproved or costly medicine fits your situation?
Families often use one name for four different things. Knowing which one you mean saves weeks of asking the wrong people.
A clinical trial
The first route to check. The medicine is given inside a study with close monitoring, and the study sponsor usually pays for it.
Does not suit
- People who miss the entry rules
- People too unwell to travel to the site
Compassionate use for one patient
Also called named-patient or expanded access. The hospital applies to the regulator for one person, with the maker's agreement.
Does not suit
- Anyone with an approved option still open
- Situations where days, not weeks, matter
Post-trial access
If you were in a trial and the medicine was helping, the sponsor may continue to supply it after the study closes. Ask about this before you join any trial.
Patient assistance programmes
For medicines already approved in India but hard to afford. Makers sometimes offer free or reduced-price supply against income papers. This is the route most families actually need.
Not sure whether this applies to you?
Ask an oncologistStep by step
How does a compassionate use request actually work?
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Your haematologist reviews every approved option
The first question is whether anything approved, or any open trial, could still help. Compassionate use only makes sense once that answer is no. Bring every report and every past treatment record to this conversation.
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The case goes to a tumour board
A group of specialists looks at the request together. They weigh what is known about the medicine against your health and what else has been tried.
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The maker is contacted
The treating team writes to the company. Many companies run a formal programme. Some supply the medicine free. Some charge. Some refuse, often because supply is limited or the medicine is still early in testing.
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The regulator and the ethics committee are asked
The hospital applies for import permission for a named patient. An ethics committee may also review it. Paperwork and approvals commonly take several weeks.
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You sign consent and start under close watch
You are told that the medicine is unproven for you. Blood tests and visits are usually more frequent than with standard treatment.
Words you will hear
What do these access terms mean in plain language?
- Compassionate use
- Permission to use an unapproved medicine outside a trial, for someone with a serious illness and no approved option left.
- Expanded access
- The term used in the United States for the same idea. You will see it on company websites.
- Named-patient supply
- Medicine released for one specific person, under their name, rather than for general sale.
- Central Licensing Authority
- The office of the Drugs Controller General of India that grants import permission.
- Ethics committee
- An independent panel that checks a request is fair to the patient and that consent is properly taken.
- Relapsed or refractory
- Relapsed means the blood cancer came back after treatment. Refractory means it did not respond. Both words often appear in these requests.
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Being straight with you
Who is compassionate use likely to suit, and who not?
It may suit a person whose leukaemia, lymphoma or myeloma has come back or stopped responding, who has used the approved treatments, and who is well enough to travel for checks. There also needs to be early evidence that the medicine works in that exact type of blood cancer.
When it is unlikely to help
It does not suit someone who still has an approved option or an open trial. It rarely suits someone who is very unwell, because the paperwork may take longer than the illness allows. It does not suit a family hoping for a quicker or cheaper version of an approved medicine. For that, a patient assistance programme is the right door.
What this page cannot tell you
We cannot tell you whether a particular medicine is available through compassionate use. That changes company by company and month by month. We cannot tell you whether it will work for you. An unapproved medicine has, by definition, not finished testing. Side effects may be less well known than with approved treatment. Your haematologist is the right person to weigh your own picture.
Do not stop or change your current treatment while a request is being made. Keep going unless your treating team tells you otherwise.Commonly believed
What do families often get wrong about compassionate use?
"If we find the medicine online abroad, we can just bring it in."
Unapproved medicines need permission to be imported, and a product bought online may be fake or badly stored. Medicines for blood cancers are often damaged by heat. Talk to your haematologist before paying anyone for a medicine from abroad.
"Newer means stronger, so it must work better."
A medicine still in testing has not yet shown how well it works, or for whom. Some promising medicines turn out no better than existing ones. That uncertainty is exactly why the request needs careful review.
"Compassionate use means the medicine is free."
Sometimes it is. Sometimes the company charges. Hospital stays, blood tests and scans usually still cost money, and insurance or government schemes may not pay for an unapproved medicine. Ask about every cost in writing before you agree.
"If the doctor does not offer it, they are hiding something."
More often the medicine is not available for supply, the evidence does not fit your blood cancer, or an approved option still exists. It is fair to ask directly. A good team will explain the reason.
Side by side
How is compassionate use different from joining a trial?
Clinical trial
Compassionate use
Set entry rules decide who can join
Your doctor makes the case for you alone
Medicine usually paid for by the sponsor
Medicine may be free or may be charged
Results add to knowledge that helps others
Aimed at one person, with less data gathered
Tests and visits follow a fixed schedule
Checks are set by your treating team
Usually checked first
Considered only when no trial fits
Questions we are asked
Common questions about compassionate use in India
Can we apply for compassionate use ourselves?
No. The request has to come from a treating doctor through a hospital, because the regulator and the company need a clinical case and a team who will watch you closely. What you can do is ask your haematologist directly whether any route exists, and bring every report so they can judge quickly.
How long does it take?
It varies widely. Contacting the company, gathering documents, ethics review and import permission commonly take several weeks, and sometimes longer. This is why teams try not to leave the question until the very end. Ask your haematologist early whether it is worth starting the conversation alongside current treatment.
Will Aarogyasri or my insurance pay for it?
Usually not for the unapproved medicine itself. Aarogyasri, PM-JAY, CGHS, ECHS, EHS and most insurance policies cover approved treatment, and scheme rules change, so check current terms. The tests, visits and hospital care around it may still be covered. Ask the hospital billing team for a written estimate.
What if the company says no?
It happens often and is rarely personal. Supply may be limited, or the company may only release the medicine through a trial. Ask whether a trial of the same medicine is open in India or nearby. Also ask whether another approved medicine, or supportive care that controls symptoms, is the better next step.
Is an unapproved medicine safe?
Nobody can promise that. It has usually passed early safety testing, but less is known about its side effects than for an approved medicine. That is why checks are more frequent. Report any new fever, bleeding, breathlessness or rash to your team the same day, rather than waiting for the next visit.
Can I get a second opinion before deciding?
Yes, and it is sensible. A haematologist who has not treated you can review your reports and say whether an approved option or a trial has been missed. CION's haematology team, including Dr. Basudev Pokhrel, reviews cases, presents them to a tumour board and helps you understand which route to ask about.
Does CION supply compassionate use medicines?
No, CION does not supply unapproved medicines. Our team can review your case, explain whether a trial, post-trial access or a patient assistance programme is worth exploring, and coordinate with qualified centres. The final decision on supply always rests with the company and the regulator.
What should my family ask the treating doctor?
Ask whether any approved option or open trial is left. Ask what the evidence for this medicine is in this exact blood cancer. Ask who pays for the medicine, tests and admissions. Ask how long approval may take, and what care continues in the meantime. Write the answers down.
Your Haematologist
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Dr. Basudev Pokhrel reviews blood counts, transfusion needs and blood disorders, and works with the CION tumour board on blood cancers.
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Sources
- Central Drugs Standard Control Organisation — New Drugs and Clinical Trials Rules
- National Cancer Institute — Access to Experimental Drugs (Expanded Access)
- Cancer Research UK — Clinical trials and research
- Leukaemia & Lymphoma Society — Clinical trials for blood cancer
This page is general information, not a prescription. Do not change or stop any treatment based on what you read here. If anything is worrying you, contact your own treating team — or call our helpline and we will help you reach the right specialist.
Keep reading
Related pages
Finding a Blood Cancer Trial in IndiaThe route to check before compassionate useCosts Covered and Not Covered in a TrialWhat the sponsor pays and what you may still payClinical Trials in HaematologyThe full guide to trials in blood cancersTargeted therapy guideHow many newer blood cancer medicines work
Talk to us
Wondering whether a new medicine is an option?
Tell us what has been tried so far. Our haematology team will review the reports and help you understand which route is worth asking about. One helpline serves every CION centre.