Expanded access, also called compassionate use, is the FDA pathway that allows patients with serious or life-threatening conditions to access investigational drugs, biologics, or medical devices outside of a clinical trial when no comparable alternative therapy is available. The process requires the treating physician to submit a request to the FDA and obtain agreement from the drug or biologic sponsor, but it is a formal, documented, FDA-recognized pathway, not a workaround. For patients researching whether banking their own stem cells could support an expanded access application, and for physicians who want to understand how to navigate this process for a qualifying patient, this post explains the full framework.
TLDR: Expanded access (compassionate use) is a formal FDA pathway allowing patients with serious conditions to access investigational therapies outside of clinical trials when no alternatives exist. The physician submits a request to the FDA, and the sponsor must also agree. There are three types: individual patient, intermediate-size group, and widespread treatment use. The Reagan-Udall Foundation’s Expanded Access Navigator is the best starting resource for patients and physicians evaluating this pathway.
Important Disclaimer: Save My Fat does not provide FDA-approved treatments or cures for any disease and does not administer expanded access programs. Banking adipose tissue does not guarantee eligibility for any expanded access program or clinical benefit from any future therapy. The information in this post is educational and based on publicly available FDA guidance. It does not constitute medical or legal advice. Physicians and patients considering expanded access should consult directly with the FDA and with qualified legal and medical counsel.
If you or someone you care for has a serious condition and you have heard that stem cell therapies might help, but those therapies are not yet FDA-approved, expanded access may be the pathway worth understanding. It is not a guarantee of access, and it is not simple. But it is a real, formal FDA mechanism that has provided patients with investigational therapies in serious clinical situations for decades. Understanding how it works is the first step in evaluating whether it is relevant to your situation.
For physicians, expanded access is the pathway you need to understand when a patient asks whether there is any way to access an investigational therapy before it is approved. The answer is sometimes yes, when the patient meets the qualifying criteria, no comparable alternatives exist, the FDA grants the request, and the sponsor agrees to provide the investigational product. Knowing how to navigate this process is part of practicing at the frontier of regenerative medicine.
This post covers the full expanded access framework: what it is, the three types, the eligibility criteria, the process from application to access, the physician’s role, how banking relates to expanded access for adipose-derived stem cell therapies, and the tools available to navigate the process.
What Expanded Access Is and Is Not
The Official Definition
The FDA defines expanded access as the use of an investigational drug, biologic, or medical device outside of a clinical trial to treat a patient with a serious or life-threatening disease or condition when there is no comparable or satisfactory alternative therapy available. The agency’s expanded access overview is the primary source for the framework described throughout this post.
The definition has four operative elements. Investigational means the product has not received FDA approval for the relevant indication. Serious or life-threatening means the patient’s condition must meet this threshold, and patients with less serious conditions do not qualify regardless of their preference for an investigational product. No comparable alternative means the patient cannot be adequately treated with available approved therapies, and the existence of any adequate approved alternative disqualifies the request. Outside a clinical trial means expanded access is distinct from trial enrollment, and the pathway is specifically for patients who cannot access or do not qualify for existing trials rather than for patients who simply prefer not to enroll in one.
What Expanded Access Is Not
Expanded access is not a workaround for patients who simply prefer investigational therapy over approved options. It is not a guarantee that the FDA or the sponsor will provide access, because both must independently agree. It is not an approval of the investigational therapy for the patient’s condition, and nothing about an expanded access grant signals that the FDA considers the product ready for approval. It is not the same as Right to Try, which is a separate federal pathway with different criteria and oversight, and Save My Fat’s overview of what Right to Try is and how it differs draws the distinction in more depth. Finally, it is not available for any investigational product. The sponsor of the investigational therapy must agree to provide it, and sponsors decline to participate in expanded access requests for a variety of legitimate reasons. The FDA’s physician-facing expanded access information covers the process from the treating clinician’s perspective in more detail.
The Three Types of Expanded Access
Individual Patient Expanded Access
This is the most common type. A physician submits a request to the FDA on behalf of a single patient who has a serious or immediately life-threatening condition and no satisfactory alternatives. Two subtypes exist within this category. Emergency use applies to immediately life-threatening situations, where the FDA can authorize access by phone within hours rather than through the standard submission process. Non-emergency individual expanded access follows the standard submission timeline, typically reviewed within thirty days of a complete application. The physician submits FDA Form 3926 for individual patients, or uses the streamlined electronic submission process the agency has developed for this purpose, and the sponsor must independently agree to provide the investigational product before access can proceed.
Intermediate-Size Patient Population
When more than one patient needs access but the number does not justify a widespread treatment protocol, a sponsor can submit an expanded access protocol for an intermediate-size group. This type is typically initiated by the sponsor rather than by an individual physician, because the protocol covers multiple patients across multiple sites and requires the sponsor’s administrative infrastructure to support it. The FDA’s expanded access framework covers the criteria for intermediate-size protocols.
Widespread Treatment Use
For investigational products that are being developed and are likely to be approved, sponsors can establish widespread expanded access programs for patient populations not enrolled in clinical trials. This is the broadest form and is typically seen late in the development cycle when Phase 3 data is available but full approval has not yet been granted. Widespread treatment protocols often serve as a bridge between late-stage trial completion and commercial launch, allowing patients with qualifying conditions to access the therapy during the FDA review period.
Eligibility Criteria
For a patient to qualify for individual expanded access, six conditions must be met. The patient must have a serious or life-threatening disease or condition. No comparable or satisfactory alternative therapy can exist. The patient must be unable to participate in a clinical trial, either because no trial is available for their condition or because they do not qualify for existing trials. The potential benefit of the investigational therapy must justify the potential risks in the physician’s clinical judgment. The sponsor must agree to provide the investigational product. And the FDA must grant the expanded access request after reviewing the submitted application. The FDA’s patient-facing expanded access information covers the criteria in patient-accessible language, and the physician-facing expanded access information covers the submission process in detail.
The Physician’s Role
Expanded access is physician-initiated and physician-managed. The treating physician is the one who determines whether the patient meets the qualifying criteria, identifies the investigational product being requested and the sponsor, contacts the sponsor to obtain agreement to provide the product, submits the FDA expanded access request using Form 3926 for individual patients or an IND application for larger protocols, obtains IRB review or a waiver in emergency situations, obtains and documents informed consent from the patient, administers the investigational product and monitors the patient throughout treatment, and reports adverse events to the FDA as required by the regulatory framework.
The informed consent component deserves specific attention, because expanded access patients are consenting to use of an investigational product outside of a clinical trial, which is a different informed consent posture than either standard clinical care or trial enrollment. Save My Fat’s overview of what informed consent means in regenerative medicine covers the consent framework that applies in related adipose-tissue contexts, and the FDA regulations for adipose tissue resource covers the broader regulatory structure.
Expanded access is not a service physicians typically offer on a routine basis. It is a significant administrative and regulatory undertaking for each patient, and it requires the physician to operate in regulatory territory most clinicians do not occupy in day-to-day practice. The Reagan-Udall Foundation’s Expanded Access Navigator was built specifically to help physicians and patients navigate this process more efficiently, and it is the single most useful external resource for anyone seriously evaluating an expanded access request.
The Reagan-Udall Foundation Navigator
The Reagan-Udall Foundation, an independent nonprofit organization established by Congress to support the FDA’s mission, operates the Expanded Access Navigator, a free online tool that guides patients and physicians through the expanded access process step by step.
The Navigator provides a structured questionnaire to determine whether expanded access may be appropriate for a patient’s situation. It offers guidance on identifying the relevant investigational product and sponsor, which is often the most difficult part of the process for patients and caregivers who do not know where to start. It provides step-by-step instructions for submitting an FDA expanded access request, with annotated guidance for each field on the application. It includes contact information for FDA staff who can answer questions directly, which shortens the path from question to answer for patients and physicians new to the process. And it maintains resources for patients and caregivers navigating the process, including plain-language explanations of the eligibility criteria and the decision points along the way.
For any physician or patient seriously evaluating expanded access, the Navigator is the recommended first resource. Save My Fat’s companion overview of expanded access programs for patients points patients to the Navigator and complements it with adipose-tissue-specific context that the Navigator’s general-purpose framing does not cover.
How Banking Relates to Expanded Access
Autologous Cells and Expanded Access
Some investigational adipose-derived stem cell therapies under development use autologous cells, meaning cells from the patient’s own body rather than from a donor. In the context of an expanded access application for such a therapy, a patient who has already banked their own adipose-derived cells may be better positioned to pursue access than a patient who has not. The banked cells provide the autologous starting material that the investigational protocol requires, and a protocol that calls for autologous cells cannot be executed for a patient who has no autologous cells available. The published adipose-derived cell biology literature and the underlying regulatory framework at 21 CFR Part 1271 together establish the biological and regulatory basis for why autologous starting material matters in this context.
This is not a guarantee of eligibility or access. It is a positioning consideration. Banking preserves the option, including the option of pursuing expanded access if a qualifying adipose-derived stem cell therapy and a qualifying condition align in the future. Save My Fat’s complete guide to banking covers the banking value proposition in patient-facing depth.
The Sequencing Logic
The patient who wants to preserve the option of pursuing expanded access for an autologous adipose-derived stem cell therapy needs to have banked their cells before they need them, not after the qualifying condition develops. Cell quality declines with age and with the biological stress of serious disease. Banking at a point of relative health preserves the best available autologous starting material for whatever pathway eventually becomes available. Save My Fat’s overview of how banking works describes the banking process from consultation through cryostorage in operational detail.
What Banking Does Not Do
Banking does not create an expanded access program for the patient. It does not guarantee that any investigational adipose-derived stem cell therapy will be available through expanded access. It does not guarantee that the patient will qualify for any expanded access request, because qualification depends on the condition, the available alternatives, and the specific investigational product. It does not substitute for clinical trial enrollment, and a patient who can participate in an appropriate active trial is generally better served by that pathway than by expanded access. What banking does is preserve the autologous biological asset that would be required if an expanded access pathway for an autologous adipose-derived stem cell therapy becomes available for a condition the patient develops, which is a narrower claim than marketing language sometimes makes for banking services. The FDA’s patient-facing expanded access information is the canonical reference for what expanded access does and does not provide.
Expanded Access vs. Clinical Trial Enrollment
Expanded access and clinical trial enrollment are complementary pathways, not alternatives. A patient who cannot enroll in a trial may qualify for expanded access. A patient who does not qualify for expanded access may be eligible for a trial. The table below clarifies the structural differences between the two.
| Feature | Clinical Trial Enrollment | Expanded Access |
|---|---|---|
| Who initiates | Trial sponsor recruits patients | Treating physician submits request |
| Patient criteria | Defined by trial inclusion and exclusion criteria | Serious or life-threatening condition, no alternatives |
| FDA involvement | IND governs the trial | Individual expanded access request reviewed by FDA |
| Sponsor involvement | Sponsor runs the trial | Sponsor must agree to provide product |
| IRB oversight | Required for all trials | Required (waivable in emergencies) |
| Cost to patient | Usually none (trial-funded) | May have costs; varies by sponsor |
| Data generation | Contributes to trial dataset | Not part of primary trial data |
| Availability | Depends on open trials for the condition | Available if criteria met, sponsor agrees, FDA approves |
The FDA expanded access framework and the active adipose-derived stem cell trial pipeline together define the two pathways, and Save My Fat’s resources on what ClinicalTrials.gov phases mean and finding legitimate clinical trials help patients and physicians navigate the trial option in parallel with evaluating expanded access.
Frequently Asked Questions
How long does the expanded access application process take?
For non-emergency individual patient applications, the FDA typically completes review within thirty days of a complete submission. Emergency applications can be authorized by phone within hours when the clinical urgency warrants it. The sponsor’s agreement to provide the product is a separate step from FDA review, and the sponsor’s timeline depends on their internal process, which can take longer than the FDA review itself. Patients and physicians should expect the full end-to-end process to take weeks rather than days for non-emergency situations. The FDA’s physician-facing expanded access page covers the timeline in more detail.
Does expanded access cost anything?
The FDA does not charge for expanded access review. The sponsor may or may not charge for the investigational product, and this varies by sponsor and by product. Some sponsors provide investigational products at no cost through expanded access; others charge for manufacturing costs. The treating physician’s time and administrative burden represent a separate real cost, even when the product itself is provided without charge. The FDA patient-facing information covers the cost structure patients should expect.
Are there stem cell therapies currently available through expanded access?
Expanded access for specific investigational cell therapies requires a qualifying patient condition, a specific investigational product under active development, and sponsor agreement for the product to be provided. Whether any specific adipose-derived stem cell therapy is available through expanded access at any given time depends on what programs sponsors have established and what the FDA has authorized. The active adipose-derived stem cell trial pipeline is the starting point for identifying investigational products that might support an expanded access application.
What is Right to Try and how is it different?
Right to Try is a federal law passed in 2018 that provides an alternative pathway for terminally ill patients to access certain investigational drugs without going through the FDA’s expanded access process. The key differences are narrower scope, different oversight, and different consent and reporting requirements. Right to Try applies only to terminal patients, while expanded access applies to serious and life-threatening conditions that include but are not limited to terminal diagnoses. Right to Try does not require FDA approval, only sponsor agreement. Expanded access requires both FDA review and sponsor agreement. Right to Try provides less regulatory oversight and fewer reporting obligations; expanded access has more robust informed consent and adverse event reporting requirements. Save My Fat’s overview of Right to Try and how it differs covers the two pathways in side-by-side depth.
If I bank my cells, does that automatically qualify me for expanded access?
No. Banking preserves the autologous biological material that might be required for an autologous adipose-derived stem cell expanded access protocol. It does not create eligibility, guarantee access, or substitute for the qualifying criteria that must be met independently. The regulatory framework at 21 CFR Part 1271 governs banking as a preservation service, and expanded access is a separate framework with its own independent eligibility determination.
What should a physician do first if they think a patient might qualify for expanded access?
The Reagan-Udall Foundation Navigator is the recommended first step. It guides physicians and patients through the process, helps identify the relevant product and sponsor, and provides direct access to FDA staff for questions. Starting with the Navigator saves significant time compared to piecing the process together from the FDA website alone, and it is free to use.
Key Takeaways
Expanded access, also called compassionate use, is a formal FDA pathway for patients with serious or life-threatening conditions to access investigational therapies outside of clinical trials when no comparable alternatives exist. It is a real, documented, FDA-recognized mechanism with decades of use across a wide range of therapeutic areas, and it is not a workaround or an informal arrangement.
Three types of expanded access exist. Individual patient expanded access is the most common and is initiated by the treating physician. Intermediate-size group protocols are typically initiated by the sponsor for patient populations too large for individual requests but too small for widespread treatment programs. Widespread treatment use protocols are typically established late in the development cycle as bridges between late-phase trial completion and commercial approval.
The physician initiates and manages the request. The FDA reviews it. The sponsor must independently agree to provide the product. All three parties must align before access can proceed, and each represents an independent decision point where the request can be declined for legitimate reasons.
The Reagan-Udall Foundation’s Expanded Access Navigator is the best starting resource for patients and physicians evaluating this pathway. It is free, independent, and built specifically for this use case, and it shortens the path from question to answer for anyone new to the expanded access process.
Banking preserves the autologous cells that would be required for an autologous adipose-derived stem cell expanded access protocol. It does not create eligibility or guarantee access. The connection between banking and expanded access is that banking preserves a biological asset that some future investigational protocols may require, not that banking creates any right to access any specific therapy.
Expanded access and clinical trial enrollment are complementary pathways. Patients who qualify for an active trial are generally better served by that pathway. Patients who cannot qualify or for whom no relevant trial exists may find expanded access to be the appropriate route. Neither pathway is guaranteed, and both require the patient’s situation, the investigational product, and the regulatory landscape to align.
Resources for Patients and Physicians
Before pursuing expanded access: adipose tissue banking is a preservation service for potential future use in FDA-regulated pathways, not a treatment or a guarantee of access to any specific clinical trial, therapy, or product, including any expanded access program. Save My Fat does not provide expanded access services or administer any FDA pathway for investigational therapy access. Banking cannot be represented as a guaranteed route to any future therapy. Physicians considering partnership should independently verify applicable state licensing and informed-consent requirements, particularly in Florida, Utah, and Nevada, which have stem cell-specific statutes.
For patients, the Reagan-Udall Foundation Expanded Access Navigator is the recommended first resource, and the FDA’s own expanded access patient information covers the framework in plain language. Save My Fat’s expanded access programs overview complements both with adipose-tissue-specific context.
For physicians, the FDA’s physician-facing expanded access information is the primary reference, and the Reagan-Udall Navigator provides the most efficient working tool for evaluating and submitting individual requests. For physicians whose patients may benefit from an active trial before pursuing expanded access, the active adipose-derived stem cell trial search on ClinicalTrials.gov is the starting point.
For providers considering joining Save My Fat as a partner for adipose tissue banking specifically, the Save My Fat provider program overview and the partner sign-up page are the starting resources.
Save My Fat provides adipose tissue banking services in partnership with L2 Bio for laboratory operations. Save My Fat does not provide medical treatments, clinical trial enrollment, or Expanded Access services.
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.
