Prepare an expanded-access request to charge for an investigational drug

How should expanded-access charging be justified?

Identify the expanded-access category, IND sponsor, supplier, patient scope and proposed period. Provide reasonable assurance that charging will not interfere with development for marketing approval, then justify the amount under the applicable cost rules. Distinguish calculated costs from an unchanged third-party invoice, and obtain written charging authorization separately from permission for treatment access.

Before you begin

Expanded access under IND regulations; individual, intermediate-size and treatment-use programs have consequential differences.

What you will prepare: A category-specific charging package with a justified amount and tracked authorization period.

Start with the actual expanded-access use

Record the IND/protocol, sponsor, patient category, proposed number of patients, supplier and charging period. Explain why charging will not interfere with development for marketing approval. A treatment IND/protocol under 312.320 has additional specified support concerning trial enrollment, development progress and the coming year's milestones.

Do not reuse the clinical-trial extraordinary-cost argument as the sole expanded-access justification. The charging and expanded-access permissions are distinct decisions; permission to treat a patient does not automatically authorize charging for the drug.

Apply the cost rules to the category and supply arrangement

Prepare a transparent direct-cost record. Intermediate-size and treatment IND/protocol settings may also include the specified monitoring, reporting and directly associated administrative costs under 312.8(d)(2); do not automatically apply those additional categories to single-patient use.

Where the sponsor calculates recoverable costs, obtain supporting documentation and the applicable independent CPA review/approval statement. The February 2024 guidance Q21 distinguishes an expanded-access sponsor simply passing through the amount paid to a third-party supplier: with no sponsor calculation to review, FDA describes using the supplier receipt or invoice to justify that amount. An added fee or amortization calculation changes that fact pattern and needs its own analysis.

If costs are allocated across years, preserve the assumptions, patient numbers and accounting basis. An amortization model does not create a multiyear FDA authorization.

Match the request to the allowed period and patients

Describe the requested amount, patient scope and duration; provide the evidence of noninterference and the cost attachments. Obtain written authorization before charging. Under the rule, expanded-access authorization generally lasts one year unless FDA specifies a shorter period, and reauthorization can be requested. It is limited to the authorized patient number when a limit exists.

Fictional editorial exercise: a physician sponsor passes through an unchanged supplier invoice for a single patient. The draft adds the manufacturer's clinical-development overhead because a template used for a treatment program included administrative costs. Remove unsupported additions and reassess the category. If the invoice is missing, the amount remains unsubstantiated; do not substitute a website price.

Match each cost and authorization condition to the actual use

Start with the treatment category before copying a cost model. The regulation and February 2024 guidance distinguish these situations:

Supply exercise: a physician IND sponsor receives a supplier invoice and proposes to pass through precisely that amount. Q21 describes documenting the amount with the supplier receipt or invoice rather than a CPA statement for a nonexistent sponsor calculation. If the draft adds a program fee or spreads startup costs across future patients, those new facts require a different cost and category assessment. The invoice example cannot justify every added charge.

Keep a line-by-line record of the cost, source, reason it is recoverable, allocation method, period and reviewer. Distinguish the sponsor’s own margin from fees paid to a third party to administer a qualifying intermediate-size or treatment program. Q18 addresses recovery of such third-party fees, including profit within the third party’s fee; it is not general permission for the sponsor to add a profit margin to its recoverable drug costs.

The guidance also distinguishes drug cost recovery from costs at a clinical site, such as administration, nursing and study procedures, that fall outside §312.8. Do not claim FDA charging authorization decides all treatment bills or insurance coverage. Separate those matters in the financial explanation given to patients and the request package.

Maintain an authorization register with actual FDA date, approved scope, patient limit if any, shorter conditions if specified, expiry and reauthorization status. The ordinary expanded-access term is one year from authorization, unless FDA specifies a shorter period. A cost model spanning several years does not extend that permission. Reconcile an updated model with the actual population and term before seeking further authorization.

Use the clinical-trial charging guide if the intended use is an investigation for trial evaluation rather than expanded access. The route determines the evidentiary test; the same drug and sponsor do not make the two requests interchangeable.

Frequently asked questions

Sources and revisions

Requirements, source recommendations and editorial preparation advice have different roles. Review the scope and revision of the source you use.

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