Prepare a request to charge for an investigational drug in a clinical trial

What must a clinical-trial charging request demonstrate?

For the sponsor’s investigational drug, support potential significant clinical advantage, the trial data’s importance to initial approval or a significant labeling change, and extraordinary drug cost that prevents conducting the trial without charging. Document recoverable direct costs and applicable independent CPA review, then obtain FDA’s prior written authorization. First establish the supply relationship because approved-drug exceptions have different boundaries.

Before you begin

Charging for a sponsor’s investigational drug under 21 CFR 312.8(b); not expanded-access charging.

What you will prepare: A criterion-by-criterion charging request and documented cost calculation for FDA review.

Establish the trial-specific basis

Obtain the IND/protocol identity, drug ownership and supply arrangement, clinical rationale, development plan and finance records. For the sponsor's drug, the rule requires evidence of potential significant clinical advantage, that the trial data are essential to initial approval or a significant labeling change, and that extraordinary drug cost prevents conducting the trial without charging. Address each condition separately.

Do not equate financial pressure with the regulatory test. Explain the actual manufacturing, scarcity, quantity or other relevant circumstances relative to the sponsor's resources. The regulation separately treats an approved drug obtained from an unaffiliated entity for trial evaluation; establish that fact before applying the sponsor-owned drug analysis.

Build the recoverable-cost calculation

Ask finance to separate costs specifically attributable to supplying the investigational drug from broader development or commercial costs. Show units, production or acquisition inputs, shipping/handling assumptions, the proposed amount per unit and the population/time covered. Explain exclusions, including costs that would exist independently of the authorized investigational use.

Provide the supporting cost documentation and the applicable independent CPA review/approval statement. A pricing model, target margin or invoice to the patient is not the required evidentiary calculation. Reconcile the costing quantity with protocol dose, duration and enrollment; account for assumptions transparently rather than hiding them in a single total.

Write the request and preserve the authorization boundary

Use a request outline of: IND and protocol; proposed charging scope and duration; clinical criteria; extraordinary-cost rationale; cost documentation; independent review; requested FDA action. Obtain prior written FDA authorization before charging. The default duration in the rule is the length of the trial unless FDA specifies a shorter period; record the actual authorization conditions.

Fictional editorial exercise: a sponsor includes future commercial-facility depreciation and a profit margin in the proposed amount. Return the model to finance to isolate permitted direct costs and document exclusions. If the sponsor's relationship to the supplying entity is unknown, resolve that input before choosing the relevant charging provisions.

Separate eligibility from the proposed amount

Use two reviews: one for why charging is justified and one for what amount is recoverable. Passing the arithmetic review does not establish the clinical-trial criteria.

Illustrative calculation: suppose a fictional model attributes $48,000 to manufacturing 120 units for the proposed investigational use and $6,000 to their direct shipping and storage. That produces $450 per unit before checking the underlying eligibility and allocation assumptions. A separate $18,000 estimate for general corporate activity cannot be included merely by dividing it by the same 120 units. Identify what each cost represents and whether it is specifically attributable to the authorized use. This example is arithmetic for review, not a proposed charge or agency-approved accounting method.

Reconcile units to dose, treatment duration, enrollment and the actual supply plan. Ask the finance and clinical owners to explain any difference between manufactured units and the population over which costs are allocated. Unsupported assumptions should remain visible for review, not disappear inside a per-patient price.

The February 2024 guidance defines an independent CPA as qualified to make the charging determinations and not an employee of the company or institution seeking to charge. It also distinguishes a sponsor’s own approved drug used investigationally from an approved drug obtained from an unaffiliated entity for trial evaluation. “Approved” alone does not resolve the charging question. Separately, the guidance addresses approved concomitant therapy that is not part of the investigational evaluation.

Record the actual authorization conditions. The regulation does not establish a fixed response deadline; the guidance says FDA intends to respond within 30 days when possible. Silence is not authorization. Where the proposal is treatment access rather than a trial, use the expanded-access charging guide and reassess the criteria.

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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