An expanded access program gives seriously ill patients access to an investigational product outside a clinical trial. The paperwork is regulated and the volume is uneven: a handful of requests, each with its own approvals, consent, supply and safety reporting. This page shows how to track all of that as structured data.
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Requests received
14
Approved
9
Pending review
3
Declined
2
What to know first
The landscape
Expanded access, sometimes called compassionate use, allows a patient to receive an investigational medical product when they cannot take part in a clinical trial and have no comparable alternative. In the United States, FDA's regulations at 21 CFR 312 Subpart I set out the general criteria, which include a serious or immediately life-threatening disease, no comparable or satisfactory alternative therapy, a judgment that the potential benefit justifies the risks, and that access will not interfere with development of the product. A manufacturer decides whether to provide its product; FDA does not compel that.
The rules are different in other regions. European countries run their own compassionate use or named-patient programmes with national procedures, and the details vary. A US federal "Right to Try" law offers a separate route that is outside the FDA expanded access process. If your programme spans several countries, build a country field and a pathway field into the record from day one, and have regulatory counsel confirm the requirements in each place.
Where does software come in? A programme usually has one controlling team, perhaps a medical affairs group or a small company, and a changing set of treating physicians at different hospitals. The sponsor needs a single view: who asked, who was approved, who has product, who is overdue with a report. That is what the clinical trial software in Capture records. For related regulatory background see 21 CFR Part 312 explained and the FDA IND application guide for academics.
Pathways
A general summary of the structure in 21 CFR Part 312, Subpart I. Check current FDA forms and guidance for details; requirements change.
| Category | Typical use | Who is the sponsor | Tracking implications |
|---|---|---|---|
| Single patient (non-emergency) | One patient, one physician, product the physician cannot get another way | The licensed physician, who acts as sponsor-investigator | One case record per patient with request, authorization, ethics approval and consent dates. |
| Emergency single patient | Patient needs treatment before the usual paperwork can be completed | The licensed physician | Record the date and form of authorization and when the written request followed. |
| Intermediate-size population | More than one patient but fewer than a broader programme, often similar patients with a shared need | A physician or a company, depending on the case | A programme record with a case record per patient, and a shared protocol version. |
| Treatment IND or protocol (widespread use) | A larger group, often when the product is close to approval | Usually the manufacturer | Programme-level protocol, many sites, aggregated safety and periodic reporting. |
Summary only. Eligibility, forms and reporting obligations under each category are set by FDA and by your regulatory advisors.
The case record
A good expanded access case record reads like a short casebook. It starts with the request: date, requesting physician and institution, condition, prior therapies, why no trial or alternative is available, and the product and dose requested. It continues with the decision: who reviewed, the date, the outcome and any conditions. Then the approvals: the regulatory authorization reference your team records, ethics committee or IRB approval date, and the informed consent date. Next the supply details: product lot or batch reference as your team assigns it, quantity sent and date received. Finally the clinical follow-up: treatment dates, adverse events, safety reports, response information as the treating physician provides it, and a closing form.
These are plain structured fields, so they translate directly to eCRFs. The visit schedule is irregular. Capture lets you define a small set of visits such as "Request", "Approval", "Treatment start", "Follow-up 1", and "Closure", with each followed by a form. Forms move from draft to approved, approved forms are locked for live use, and every change is recorded in a field-level audit trail with user, time, old value, new value and reason. That audit trail is exactly the history that helps when someone asks, months later, when an approval was entered.
Role-based access matters because the physician sees one patient, the programme manager sees all, and safety sees events. Capture's roles (researcher, site coordinator, admin) and PII segregation support that: staff can see participant names where needed, others work with coded IDs. See multi-site trial management for the site model that treating physicians fit into.
Request
Requesting physician and site
REQPHYSPathway
EAPATHCondition is serious or life-threatening
SERIOUSComparable alternative therapy available
ALTAVAILDate of request
REQDTDecision and approvals
Decision
DECISIONEthics committee approval date
IRBDTRequired before supply
Blank: needed before shipment
Informed consent date
ICDTBuild the intake form, decision log and follow-up forms in the free sandbox and test with dummy cases. Every feature, no credit card, no time limit.
Workflow
The physician's request is entered on an intake form with the fields above. Edit checks flag missing required items (for example, no consent date) as queries, so gaps are visible to the programme manager.
A reviewer records the decision, the rationale and any conditions. Keep a "pending" status explicit and put a target date on it so requests do not stall silently.
Record the regulatory authorization reference your team holds, the ethics approval date and informed consent. Consent can be collected on Capture's eConsent where it suits your process, with countersignature and an exportable PDF; see eConsent and 21 CFR Part 50 informed consent.
Record dispatch and receipt dates and the product reference. If you need full supply control, drug accountability options exist on the Full Platform plan.
Record treatment start, dose changes, adverse events and any outcome information the physician provides, on a small number of repeatable follow-up visits.
Log each safety report: event, seriousness, awareness date, due date, and the date your team sent it. Capture records and tracks; your team submits. See below.
A closure form records the last dose, reason for stopping (death, withdrawal, adverse event, supply end), and the final report date, then locks the case.
Safety reporting
Expanded access under an IND is covered by the IND safety reporting rules, including the sponsor's duties for serious and unexpected suspected adverse reactions in 21 CFR 312.32, and an annual report under 312.33 in many cases. The detail depends on the category and on who holds the IND. Do not assume a physician-sponsor and a manufacturer carry the same duties; get the allocation written into the agreement.
Capture does not submit to FDA or any regulator. It gives your team a reliable place to record and track. For each adverse event: the description, onset date, seriousness criteria, severity, relatedness, outcome, the date your team became aware, the due date calculated from your rule, and the date the report was sent. A dashboard shows reports that are due or overdue, so the deadline, not memory, drives action. The SAE reporting deadline calculator, the SAE reporting timelines guide and the SUSAR reporting software page cover the mechanics, and the SAE report form template is a base for the form itself.
Keep the line clear in your procedures: Capture is the record; the submission channel and the content of the submission are your regulatory team's responsibility. Add a procedure that says who confirms the submission was sent and where the acknowledgement is stored.
Quality
Make the ethics approval date a required field before the supply form can be approved, using an edit check.
Add a rule that compares consent date with treatment start and raises a query when consent is later.
Store awareness date, due date and sent date on every serious event and filter by overdue.
Schedule a follow-up form and a closure form; empty ones show as missing in the casebook.
Record the protocol or letter of authorization version per case.
Add pathway and country fields and filter exports by site.
Using the data
Expanded access data are not designed to prove efficacy, but they have uses: a summary for regulators and ethics committees, safety information that adds to the development programme, and an internal record of who received what. Capture exports CSV and Excel with a data dictionary, can filter by site and date range, and produces SDTM datasets as SAS XPT with Define-XML, so a programmer can fold expanded access data into safety summaries if your regulatory strategy calls for it. Export a final casebook for each closed case, with audit trail, and store it in your trial master file; see how to set up a trial master file.
For rare disease programmes, where expanded access is often tied to a small community, EDC for rare disease clinical trials and the page for rare disease foundations describe the wider context. If the programme will later turn into a trial, the structure you create here carries into EDC for investigator-initiated trials.
It is a way for patients with serious or life-threatening conditions to receive an investigational product outside a clinical trial when no comparable alternative is available. In the US it is governed by 21 CFR Part 312, Subpart I.
No. Capture tracks and stores the data; your team makes any regulatory submission through the required channels. Capture can record the submission date and reference.
The terms are used loosely and often mean the same thing. Compassionate use is the common term in Europe; programmes there follow national procedures, which differ from the US process.
Record the event, seriousness, awareness date, due date and date sent on every event, then filter by overdue. The deadline rules come from your regulatory team and the applicable regulations.
Treating physicians or their staff can be given a site role to complete forms for their own patient, while the programme manager sees all cases. Test the role set up in the free sandbox.
Yes. A single-patient case is one record; an intermediate-size or treatment programme is a set of records under one protocol version. The visit design is the same.
The data structure works anywhere; the regulatory content does not carry over. Add country and pathway fields and have local regulatory advisors confirm requirements. Capture hosts data in the EU (Frankfurt) and the US (N. Virginia).
It can be. Capture eConsent supports on-screen signing with email one-time-password verification and investigator countersignature, and exports signed consent as PDF. Confirm your ethics committee accepts electronic consent for the programme.
Keep exploring
21 CFR Part 312 explained
The IND regulations in plain language.
FDA IND application guide for academics
Sponsor-investigator IND basics.
SUSAR reporting software
Tracking serious unexpected reactions.
SAE reporting timelines guide
Deadlines for serious events.
EDC for rare disease trials
Small populations and special pathways.
21 CFR Part 50 informed consent
Consent requirements in the US.
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