Name the access route
Start with the exact product and proposed use, then identify the pathway that could make it available. The name of a pathway tells you how access might work. It does not tell you whether treatment is a good choice.
Before you start: Drug labeling and off-label use separates an approved medicine from an approved use. Clinical trials versus expanded access explains the research and treatment routes for investigational products.
Where this step sits
This is the first lesson in accessing experimental therapy. Next you will check a real trial opportunity, then build the clinical readiness picture.
Ask two opening questions
First: “Is this product approved for any use in the United States?” Second: “Does its current labeling include the proposed use?”
If the answer to both is yes, the discussion concerns a label-matched treatment, along with clinical suitability and practical access. If the product is approved but the use differs, the discussion may concern off-label prescribing. If the product has never been approved, ordinary prescribing does not by itself provide access.
Be specific about the product. A redesigned cell product or a new personalized formulation may not be the same product used in a cited study. Sharing a target, ingredient category or platform name does not establish that its evidence or authorization transfers.
Understand the main routes
| Route | Main purpose or circumstance | Questions that remain |
|---|---|---|
| On-label treatment | An approved drug used within its labeling | Is it suitable, available and covered for this person? |
| Off-label prescribing | An approved drug used outside its labeling | How strong is the evidence for this use, and will the institution and payer support it? |
| Clinical trial | Research under a defined protocol | Is the patient eligible, is the cohort open, and how is treatment assigned? |
| Expanded access | Treatment outside a trial for qualifying circumstances | Will the clinician, supplier and applicable reviews support the plan? |
| Federal Right to Try | A separate pathway for eligible patients and products | Do both qualify, and will a supplier and treating team participate? |
These are categories to clarify with the treating team. They are not interchangeable shortcuts.
A clinical trial follows a study protocol. It can include established drugs as well as investigational products. The purpose is to answer research questions, and the exact treatment may depend on assignment.
Expanded access considers an investigational product outside a trial when the required criteria are met. An Investigational New Drug (IND) application is a regulatory submission that can support that use. A single-patient IND is one form of expanded access; it is not a separate competing pathway. Supplier willingness, clinical risk assessment and applicable reviews still matter.
Federal Right to Try has distinct patient and product requirements. Among them, the patient must have a life-threatening condition, have exhausted approved options, and be unable to participate in a trial of the eligible drug. Product requirements include completed phase 1 testing and active development under specified conditions. The Food and Drug Administration (FDA) does not review individual Right to Try requests, and the law does not require the supplier to provide treatment. Ask the responsible physician and supplier to verify the full current criteria.
Worked example: three fictional proposals
Mara receives three ideas. Drug A is approved for Cancer X but proposed for Cancer Y. Product B has never been approved and is being tested in a trial. Product C is described as “available through a single-patient IND.”
Drug A raises an off-label evidence and prescribing question. Product B raises protocol, eligibility and site questions. Product C raises expanded-access criteria, supplier, treatment-plan and review questions. None of these descriptions establishes that Mara should receive the treatment.
The next useful question is different for each. For Drug A, ask for the evidence matching the proposed use. For Product B, request the current protocol and cohort. For Product C, ask who would supply it and who would take clinical responsibility. Clear categories turn one vague “can we get it?” into an actionable conversation.
What can go wrong at this step
- Availability becomes a benefit claim. Authorization to proceed does not establish effectiveness.
- Off-label and never-approved products are merged. Their access mechanisms differ.
- A supplier's earlier authorization becomes this patient's authorization. Verify the exact product and proposed plan.
- Right to Try becomes a guaranteed supply route. It does not compel a manufacturer to provide a drug.
Try it
A fictional clinician proposes an approved drug at a different dose for an unapproved indication. The family calls it “expanded access.” Is that the right starting category?
Answer: The starting question is off-label prescribing of an approved drug. A clinical trial could also study that use, but expanded access is not the ordinary name for a clinician prescribing an approved medicine outside its label. Evidence, institutional requirements and coverage still need review.
Explain it back
Complete: “This product is ____, and this proposed use is ____. The route we need to clarify is ____.”
One possible answer: “Approved for another disease, outside its label, and the route is off-label prescribing.”
Takeaway
Keep product approval, evidence for the proposed use and the access pathway as three separate questions.
Next: Check a real trial opportunity.
Sources and scope
U.S. source check: October 9, 2026. Mara and all products are fictional. Expert and learner review pending. The Right to Try summary is introductory; use the complete current criteria for an actual request.
- FDA: understanding off-label use — approved drug, unapproved use.
- FDA: expanded-access overview — criteria and submission categories.
- FDA: Right to Try — patient and product criteria, review and supply limitations.