08/31/2022
The US Food and Drug Administration allows charging for investigational drugs that are under an investigational new drug application (IND) in certain circumstances. The “charging regulation” (21 CFR 312.8) which came into effect in October 2009, describes what costs are recoverable and circumstances where charging is permitted. Expanded access for treatment use, also known as compassionate use, is a treatment option for patients who have an “immediately life threatening condition or serious disease” when there is no viable treatment option. The investigational product is provided outside the clinical trial as treatment although it has not yet been found to be safe or efficacious. If the sponsor charges for expanded access, it needs to provide assurance to the Agency that this will not interfere with ongoing clinical trials. Also, certain clinical trials may meet the charging regulation requirements, for example, if there is evidence that the investigational drug has the potential to provide a significant benefit and it is so costly that a clinical trial could not be run without charging. The sponsor provides justification for the amount to be charged when applying for the charging authorization. The charging authorization is valid 1) for one year for expanded access or 2) for the duration of the clinical trial.
Since 2009, the FDA received questions about when the charging regulation is applicable and how to determine what costs can be charged to patients or study participants. A final guidance “Charging for Investigational Drugs Under IND — Questions and Answers” from June 2016 helped to clarify. Since the 2016 final guidance, the Agency received additional questions for clarification. The revised draft guidance “Charging for Investigational Drugs Under an IND: Questions and Answers” further answers these questions. When finalized, the draft guidance will replace the June 2016 guidance.
Three additional questions are included in the draft:
Q. 21: “Is a sponsor of an expanded access IND who seeks to recover the cost incurred from obtaining an investigational drug from another source required to include in the charging request submitted to FDA a statement that an independent certified public accountant has reviewed and approved the calculation?”
Answer summary: No. If there is no calculation of cost involved by the sponsor, a copy of the receipt or invoice from the third party vendor who provides the investigational drug to the sponsor is adequate. Also refer to the response to Q. 13 in the guidance.
Q. 22: “Can a sponsor of an intermediate or treatment IND or protocol seeking to charge for the investigational drug distribute the costs associated with monitoring the program for the intermediate or treatment IND or protocol and other administrative ‘startup’ costs over the expected duration of the IND or protocol, rather than in the first year of the treatment?”
Answer summary: Yes. It is expected that startup costs would be higher in the first year and costs would decrease afterward. A cost distribution plan that distributes the costs over the years rather than charging more to patients who receive it in the first year and less to future patients may be authorized. Calculations must be approved by an independent accountant.
Q. 23: “Can a sponsor of an expanded access IND or protocol seeking to charge for the investigational drug distribute the higher manufacturing costs associated with manufacturing the drug in the first year compared to subsequent years, over the duration of the IND or protocol, rather than in the first year of the treatment?”
Answer summary: Yes. It is expected that manufacturing costs would be higher in the first year and costs would decrease afterward. A cost distribution plan may be authorized, and calculations must be approved by an independent accountant.
Comment now through October 24, 2022 HERE.
- The Clinical Pathways Team
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