For some sick patients, receiving medication through an Expanded Access Program (EAP), also known as managed access, compassionate use, named patient, etc., is the only viable treatment option left. These programs allow the patients to gain access to investigational new drugs which are still in the clinical trial phase and are not, yet, licensed for use.
However, sometimes patients can find that they are unable to apply for such a program because the trial sponsor wishes to charge patients for the drug. To help patients better understand the charging process, this article will explain when, and how much, a sponsor can charge for expanded access use of a drug.
There are two requirements that a sponsor must satisfy in order to charge for its drug. Firstly, the sponsor must be able to assure the FDA that charging patients will not interfere with the development of the drug. Under this requirement, the sponsor must show that there is sufficient enrollment in the clinical trial, and, if not, efforts are being made to ensure that there will be. The sponsor must also provide evidence to show that the there is adequate progress in the development of the investigational drug for market approval. The sponsor should also be able to provide specific drug development milestones that it plans to meet within the next year. This is all to ensure that the trial will be completed successfully and as planned, so that the drug will be made available to patients on the market as quickly as possible.
Secondly, the sponsor must show that its calculation of the amount it will charge is consistent with the requirements in clause § 312.8(d). Clause § 312.8(d) was added to the FDA regulations in 2009 and explains the criteria for determining what costs a sponsor can recover when charging for an investigational drug. Under this clause, when charging for individual patient access to a drug, a sponsor can only recover the direct costs associated with making the drug available to the patient. This includes manufacturing or acquisition costs as well as shipping and handling costs. Indirect costs, including administrative and regulatory reporting costs, cannot be recovered.
For an intermediate-size patient population IND or protocol, the sponsor can recover the direct costs, as well as the costs associated with the monitoring of the IND or protocol, and the costs associated with meeting IND reporting requirements. The sponsor can also recover the cost of any fees paid to a third party for setting up and administering the Expanded Access Program on behalf of the sponsor.
If these two requirements are met, the FDA will provide written authorization, after which a sponsor can begin charging. A sponsor can then charge for the drug for up to one year, unless the FDA has specified a shorter period of time. After one year, the FDA must reassess the charging request to determine whether charging for the drug is interfering in any way with the development of the drug for market use. At the one-year period, if the sponsor wishes to continue charging, a new request must be sent to the FDA. The FDA recommends that this request be sent at least 60 days before the expiration date of the existing authorization.