When a patient with a serious or life-threatening condition cannot enroll in a clinical trial, FDA’s expanded access framework is the pathway that can authorize use of an investigational therapy outside a trial. For cell therapies specifically, the single-patient IND is the most commonly used route, and it is also the downstream mechanism that can connect previously banked autologous tissue to authorized use. What follows is a practical walkthrough of the single-patient IND process under 21 CFR Part 312, Subpart I, including where banked tissue fits and the mistakes that most often delay an application.
TLDR: Expanded access (compassionate use) under 21 CFR Part 312, Subpart I lets a physician request an investigational therapy for an individual patient with a serious or life-threatening condition when no comparable alternative exists. The single-patient IND process: identify the product and sponsor, obtain the sponsor’s agreement to supply, submit FDA Form 3926, obtain IRB authorization, obtain written informed consent, then administer, monitor, and report. FDA generally has 30 days to respond to a non-emergency application; emergencies can be authorized verbally. For autologous therapy, banked tissue may be a prerequisite, but the sponsor’s manufacturing and chain-of-custody requirements govern. Banking adipose tissue does not guarantee eligibility, access, or benefit.
Important Disclaimer: Save My Fat does not provide FDA-approved treatments or cures for any disease. Banking adipose tissue today does not guarantee eligibility, access, or clinical benefit from any future therapy, clinical trial, or medical program. No adipose-derived stem cell product currently holds FDA approval for the conditions discussed in this article. All content is for educational purposes only and does not constitute medical or regulatory advice. Patients must consult their own licensed healthcare professionals regarding all medical decisions.
Expanded access is often misunderstood as a fast or informal route. It is neither: it is a structured FDA process with defined steps, a sponsor who must agree to supply the product, and IRB oversight. Understanding the sequence in advance is what keeps an application from stalling at a step the physician did not anticipate, and it is also what lets a physician give a patient an honest account of what the pathway can and cannot do.
What Expanded Access Is and When It Applies
Expanded access, also called compassionate use, allows a physician to use an investigational drug or biologic, including a cell therapy, outside a clinical trial for a patient with a serious or life-threatening condition when no comparable or satisfactory alternative is available. The legal basis is 21 CFR Part 312, Subpart I. There are three categories: individual-patient access (the single-patient IND), intermediate-size population access, and the treatment IND or protocol for larger groups. The focus here is the single-patient IND, the route most often used for an individual patient who cannot enroll in a trial. The broader landscape is covered in the companion overview of expanded access programs.
When Expanded Access Is the Right Pathway
The threshold conditions are specific. The patient has a serious or immediately life-threatening condition; no comparable or satisfactory alternative therapy is available; the potential benefit justifies the potential risk; and providing the product will not interfere with clinical investigations that could support the therapy’s development or approval. The physician must also have identified a specific investigational therapy that may be appropriate for the patient. If those conditions are not met, expanded access is not the right route, and a physician should not represent it to a patient as available before confirming them.
The Single-Patient IND Process, Step by Step
The first step is to identify the specific investigational product and its sponsor. The product must be under an active IND held by a sponsor, whether a company, an academic institution, or the physician acting as sponsor-investigator. The Reagan-Udall Expanded Access Navigator is the standard tool for identifying which sponsors run expanded-access programs for specific products.
The second step is to contact the sponsor and request authorization to use the product. Sponsors are not obligated to provide expanded access, though FDA encourages it where feasible. If the sponsor agrees, it typically supplies a protocol or a letter of authorization, which the physician needs before the product can actually be obtained.
The third step is to submit FDA Form 3926, the Individual Patient Expanded Access IND application. It requires the patient’s diagnosis and condition, a description of the investigational product, the proposed treatment plan, a monitoring plan, and informed-consent documentation. For non-emergency requests, FDA has 30 days to respond, by authorizing, placing the application on clinical hold, or requesting more information. For emergencies, FDA can grant verbal authorization within hours, with written confirmation following.
The fourth step is IRB authorization, which must be obtained before treatment unless a documented waiver applies. For a single-patient expanded-access IND, an IRB can grant authorization through an expedited route, and a physician not affiliated with an institutional IRB must obtain review from an independent IRB. The IRB and IND interaction is discussed further in the FDA regulations overview for adipose tissue.
The fifth step is written informed consent, obtained before treatment, describing the investigational nature of the therapy, the known risks and benefits, the alternatives, and the patient’s right to withdraw.
The sixth step is reporting: the physician submits progress reports to FDA for as long as the patient receives the therapy, and serious adverse events must be reported promptly.
| Step | Action | Timing | Key document |
|---|---|---|---|
| 1 | Identify product and sponsor | Before applying | Reagan-Udall Navigator |
| 2 | Obtain sponsor authorization | Varies | Sponsor letter of authorization |
| 3 | Submit FDA Form 3926 | Before treatment | FDA Form 3926 |
| 4 | Obtain IRB authorization | Before treatment | IRB approval letter |
| 5 | Obtain informed consent | Before treatment | Signed consent form |
| 6 | Administer and monitor | Ongoing | Progress notes, adverse-event reports |
| 7 | Submit reports to FDA | Ongoing/annual | IND progress report |
How Banked Tissue Fits
For an autologous cell therapy, where the investigational product is derived from the patient’s own tissue, the existence of banked autologous adipose tissue may be a prerequisite for pursuing this pathway at all, since the tissue has to exist before it can be processed. A physician who has already banked the patient’s tissue has completed that upstream step, and the expanded-access IND is the downstream authorization mechanism. The essential caveat: the physician should confirm with the sponsor whether banked tissue from a third-party facility meets the product’s specific manufacturing and chain-of-custody requirements before submitting Form 3926, because banked tissue is not automatically interchangeable across manufacturing processes. Banking keeps the pathway open; it does not by itself satisfy a sponsor’s product requirements.
Common Mistakes
The recurring errors are procedural. Submitting Form 3926 before contacting the sponsor means FDA may process the application, but without sponsor cooperation the product cannot be obtained. Failing to secure IRB authorization before treatment is a clear violation of the framework. An insufficient monitoring plan invites an FDA request for more information, since the agency expects a concrete plan for patient safety monitoring. And inadequate informed consent, one that does not specifically address the investigational nature of the therapy, undermines the entire application. Each is avoidable by following the sequence in order.
Expanded Access IND Checklist
- Patient meets the serious or life-threatening condition standard.
- No comparable alternative therapy is available.
- Investigational product identified and sponsor contacted.
- Sponsor has agreed to provide the product.
- FDA Form 3926 completed and submitted.
- IRB authorization obtained.
- Patient informed consent obtained and documented.
- Monitoring plan established.
- Adverse-event reporting plan established.
- Reporting schedule set up.
Frequently Asked Questions
Can I submit Form 3926 before the sponsor agrees to supply the product?
You can, and FDA may process it, but it accomplishes little. Without the sponsor’s agreement to provide the product, there is nothing to administer. Contact the sponsor first.
Does banked autologous tissue automatically qualify for an autologous expanded-access therapy?
No. Banking is often a prerequisite for an autologous pathway, but the sponsor’s manufacturing and chain-of-custody requirements govern whether specific banked tissue can be used. Confirm with the sponsor before applying.
How fast can expanded access move?
For non-emergency applications, FDA generally has 30 days to respond. In a genuine emergency, FDA can authorize use verbally within hours, with written confirmation to follow. Sponsor agreement and IRB authorization still apply.
Key Takeaways
The single-patient IND under 21 CFR Part 312, Subpart I is a structured pathway, not a shortcut: identify the product and sponsor, secure the sponsor’s agreement to supply, submit Form 3926, obtain IRB authorization and written informed consent, then administer, monitor, and report. The threshold conditions, a serious condition, no comparable alternative, and a favorable benefit-risk judgment, have to be genuinely met. For autologous cell therapy, banking is frequently the upstream prerequisite that makes the pathway possible, but the sponsor’s manufacturing and chain-of-custody requirements determine whether specific banked tissue qualifies, so that has to be confirmed before applying. Banking keeps this option open; it remains a preservation decision separate from therapy, with no guarantee of access or benefit.
Save My Fat operates as a tissue preservation service, not a medical practice or treatment provider. Stem cell and regenerative medicine regulations vary by state, including specific informed-consent and disclosure requirements in Florida, Utah, and Nevada governing tissue and stem cell services. Banking adipose tissue does not connect patients to any treatment pathway, and any future use depends on FDA regulatory status, physician guidance, and the availability of approved or investigational pathways at that time.
Physicians weighing an expanded-access pathway for a patient with banked tissue can contact Save My Fat to discuss the preservation side.
Save My Fat partners with L2 Bio for laboratory processing and storage.
This article is for educational purposes only and does not constitute medical or legal advice. Legal and medical review including neurology and neurosurgery input is required before publication. Please consult your neurologist or neurosurgeon before making any decisions about neurologic treatment or research participation.
