Compassionate Use and Named Patient Access — The Regulated Route to a Drug India Has Not Yet Approved

Compassionate use is permission for one named patient to receive a medicine that is not yet approved in India, outside a clinical trial. The condition must be serious or life-threatening with no approved alternative left. A treating specialist applies, an ethics committee reviews it, and the regulator decides.

Almost nobody is told this route exists. Families reach the end of approved treatment, hear about a medicine that is available in another country, and assume the only ways in are a clinical trial or a flight abroad. There is a third answer, and it is written into Indian regulation rather than left to goodwill: the New Drugs and Clinical Trials Rules, 2019 allow an unapproved new drug to be permitted for a patient with a serious or life-threatening condition where no approved alternative therapy is available, with the permission of the Central Licencing Authority (CDSCO) and the oversight of a registered ethics committee.

What makes it hard to use is not secrecy. It is that four separate parties each have to say yes, and that the words used for it change depending on who is speaking. “Compassionate use”, “named patient access”, “expanded access” and “managed access” overlap, and a company, a regulator and a hospital may each use a different one for the same thing. The table below separates the routes so you can tell which one is actually on the table.

The rows above describe how these routes are commonly structured in India and are general and indicative, as of August 2026. They are not a decision in your case, and they are not a checklist to act on alone. CDSCO publishes the current application requirements and revises them from time to time — confirm the present position with the regulator or your hospital’s regulatory office rather than relying on any summary, including this one.

Very few people. The usual test is a serious or life-threatening cancer, approved treatment that is genuinely exhausted or unsuitable, no clinical trial you can realistically join, and a specific medicine with a scientific reason to expect benefit. You must also be well enough to receive it safely.

What a request has to be able to show

• The condition is serious or life-threatening — the route exists for situations where waiting for approval is not a realistic option. It is not a way to add a newer medicine to a plan that is working.
• Approved options are genuinely exhausted or unsuitable — this is the question that ends most requests, and it is worth a second opinion. An approved treatment that was never offered is a far better outcome than an unapproved one that may never arrive.
• No suitable trial is open to you — a study is normally the first route, because it is designed for exactly this. Compassionate use is considered where eligibility criteria, distance or timing rule a study out.
• There is a scientific reason to expect benefit in your situation — a biomarker result, an approved indication in another country, or a guideline position from a body such as NCCN, ASCO or ESMO. Hope on its own is not a justification a reviewer can accept.
• You are fit enough for it — performance status, organ function and the ability to be monitored are assessed exactly as they would be before approved treatment. An unapproved medicine is not a lower bar for safety, it is a higher one.
• The manufacturer is willing to supply — the step families least expect. Permission from the regulator is not an agreement from the company, and a company may decline without giving reasons.

No one — not a hospital, not a doctor, not a coordinator, not an agency — can assure you that a request will be granted. Anybody who promises access to an unapproved medicine for a fee should be treated with real suspicion, and reported to your treating hospital. A genuine request travels through your specialist and an ethics committee, and it costs you documentation and time, not a facilitation payment.

No. It is a route around the approval status of a medicine, not around its price. It is also not off-label use, not a place in a trial, and not evidence that the medicine works — investigational means the assessment that approval requires has not been completed.

• Not a discount scheme — affordability is a different conversation, and a more productive one: biosimilars, manufacturer patient assistance programmes and a change of regimen all address price on medicines you can actually obtain today.
• Not off-label use — using a medicine that is already approved in India for an indication it was not approved for is a separate question with its own rules and its own consent discussion.
• Not a trial place — a study provides treatment inside a protocol, with monitoring, and usually with the study drug provided at no cost to the participant. Compassionate use has no protocol behind it and often no such cover.
• Not evidence of benefit — a medicine supplied this way has not finished the assessment that approval requires in India. No benefit is claimed for it on this page, and none should be claimed to you.
• Not fast — ethics committee meetings, company decisions and regulatory permission take time that a rapidly progressing cancer may not allow. Ask about realistic timelines at the very start, and keep an approved plan running in parallel.

Your treating specialist starts it, not you. There is no patient application counter. The doctor writes a clinical justification, takes it to the hospital ethics committee, approaches the manufacturer about supply, and applies to CDSCO for permission. You sign informed consent. Seven steps, and each can end in a no.

1. 1

   Confirm what is still approved for you

   Before anything else, have a medical oncologist review the diagnosis, the biomarker results and the approved options. A large share of access requests end here, because a standard treatment that was never offered turns out to still be available.

2. 2

   Identify the exact medicine and the exact reason

   A request names one molecule, one indication and the evidence behind it. Something new is not a request. This molecule, for this biomarker, on the basis of this guideline position, is a request.

3. 3

   Ask your specialist to check trials first

   A clinical trial is normally the preferred route, and reviewers expect that question to have been asked. Trial registries and the treating hospital are where that check happens, and a study may be open that nobody has mentioned.

4. 4

   Have the treating doctor write the clinical justification

   The justification comes from the doctor responsible for your treatment, on hospital letterhead, setting out the history, the lines of treatment already used, why they are exhausted or unsuitable, and how side effects will be monitored.

5. 5

   Take it to the institutional ethics committee

   A registered ethics committee reviews single-patient access requests. Its job is to check that the risk is understood, that the consent is genuine and that the plan is safe. Build its meeting schedule into your timeline.

6. 6

   Approach the manufacturer about supply

   Permission to use is not the same as supply. The company decides whether to provide the medicine, on what terms and whether it charges. Many companies publish an access policy. The hospital regulatory desk is the right route to that contact.

7. 7

   Apply to CDSCO and sign informed consent

   The application for permission goes to the Central Licencing Authority with the ethics committee approval and the company position attached. You sign a written informed consent stating plainly that the medicine is not approved and that benefit is not established.

The sequence above is general and indicative, as of August 2026, and describes how single-patient access requests are commonly handled in India. It is not a substitute for the current CDSCO requirements, which the regulator publishes and revises from time to time; the exact documents, forms and timelines that apply to your request should be confirmed with CDSCO or your hospital’s regulatory office before you rely on them. No step described here creates an entitlement to a medicine, and no outcome can be assured.

Sometimes, but never assume it. Compassionate use is permission to receive an unapproved medicine, not a promise that anyone will pay for it. Some companies supply at no charge, some supply at a price, and hospital, infusion and monitoring costs remain yours either way.

• Supply terms are the manufacturer’s decision — free supply, reduced-price supply and full-price supply are all possible, and a company’s position can differ by molecule and by country. Get it in writing through your hospital rather than second hand.
• Administration costs stay with you — day-care charges, pre-treatment blood tests, imaging and the management of any side effect are billed as normal whatever the medicine costs. Any figure quoted for these is indicative, as of August 2026.
• Insurance rarely covers an unapproved medicine — a policy generally pays for approved, medically necessary treatment. Ask the insurer for its position in writing before you commit to anything, and do not rely on a verbal assurance from any party.
• Government schemes follow approved indications — Aarogyasri and Ayushman Bharat packages are built around approved treatment, so an unapproved medicine normally sits outside them. Scheme ceilings and package rates change; treat any figure as indicative, as of August 2026.
• If cost is the real problem, say so early — biosimilar versions of some checkpoint inhibitors have changed the affordability picture in India, and manufacturer patient assistance programmes exist for several approved medicines. Both act on treatment you can start now. Any cost figure remains indicative, as of August 2026.

Access questions rarely stand alone. Whether a trial is the better route, what happens to supply when a study ends, whether an already-approved medicine can be used off-label, and how immunotherapy runs outside any study are the four that usually follow. Each is covered below.

• Questions to Ask Before Joining a Trial — the questions that settle the design, the costs and the exit before you agree to anything.
• What Happens When a Trial Ends? — what happens to the medicine, the follow-up and the results when a study closes, and what post-trial access does and does not mean.
• Off-Label Use of Immunotherapy: Is It Allowed? — the separate question of using an approved medicine for an indication it was not approved for, and the rules around it.
• Immunotherapy at CION Cancer Clinics — how eligibility review, day-care treatment and response monitoring run as one pathway on approved treatment.

Access Questions Deserve a Straight Answer

Bring the biopsy report, the last scan and any letter naming the medicine. We will tell you plainly what applies to you and what does not.