Expanded Access Policy

Rigel Public Expanded Access Policy

The following electronic application is to request Expanded Access (Compassionate Use) to fostamatinib for your patient with immune thrombocytopenic purpura (ITP). Note that this program is currently available only in the United States. The deadline for new requests is April 27, 2018.

Rigel Pharmaceuticals, Inc. is a biotechnology company focused on the development of novel small-molecule therapies for immunological and hematological disorders, cancer, and rare diseases. A crucial step in the development of Rigel’s investigational new drugs is to conduct clinical trials, which assess the safety and efficacy of the products. Rigel uses the data it collects from clinical trials to support marketing applications to FDA and other regulatory bodies so that Rigel can ultimately gain approval to provide access to its products on a widespread basis.

What is expanded access?
Through expanded access, sometimes also referred to as compassionate use, patients may be able to access investigational new drugs outside of the clinical trial context. FDA will typically facilitate expanded access to investigational products for patients with serious or life-threatening diseases or conditions when there are no comparable or satisfactory therapeutic alternatives. Unlike the use of an investigational new drug in a clinical trial setting, the primary purpose of expanded access is to use the investigational drug for patient treatment purposes, rather than to gather data on safety and effectiveness.

What is Rigel’s basis for granting expanded access?
Rigel believes the best way to access our investigational products is through participation in one of our ongoing clinical trials. Rigel may provide expanded access to an investigational product to patients where sufficient evidence for the safety, tolerability and effectiveness of the investigational product are available. However, pre-approval access outside of a controlled clinical trial may interfere with the conduct of our ongoing trials and may also disrupt the progress of our development programs, which would, in turn, delay access to many other patients in need. Therefore, expanded access will only be considered:

1) when the grant of the request would be consistent with regulatory requirements for such a grant including that –
  • the patient is suffering from a serious or life-threatening disease or condition,
  • there is no comparable or satisfactory alternative therapy available for the patient,
  • the potential benefit to the patient justifies the potential risk of using the treatment, and any such risk is not unreasonable under the circumstances,
  • if the investigational product is in a controlled clinical trial for the disease or condition for which the patient seeks treatment, granting the expanded access would not interfere with enrollment of such trial, and
2) when the request is made by a patient’s treating physician, who has independently determined that –
  • the patient is suffering from a serious or life-threatening disease or condition,
  • the patient is not eligible for participation in one of Rigel’s ongoing clinical trials, and
  • no acceptable alternative treatment exists; and
3) Rigel believes that –
  • there is sufficient evidence of the investigational product’s safety, tolerability and effectiveness,
  • there is no opportunity for the patient to enter an appropriate, currently-enrolling clinical trial, and
  • there is sufficient supply of the investigational product so that granting the expanded access request would not jeopardize the supply needed for ongoing clinical trials, research, or regulatory submissions.
Even if all entry criteria for the program are met, Rigel cannot guarantee that expanded access will be granted.

This policy is subject to change at the discretion of Rigel, and this webpage will be updated should there be a change.

How can a request for expanded access be made?
Treating physicians can make a request via completion of an application at http://eap.rigel.com. Requesting physicians must be licensed in the state where the patient is located.

How long will it take for a decision on expanded access to be made?
Each request must be considered individually, taking into account all relevant circumstances, the above criteria and, when appropriate, information obtained from discussions between the treating physician and Rigel’s clinical team. Therefore, while Rigel anticipates acknowledging requests for expanded access within 10 business days, a definitive time for a response cannot be estimated. Whenever possible, patients will be referred to a clinical trial site as a means of accessing an investigational product.

How can I get more information about Rigel’s clinical trials?
For more information on Rigel’s clinical trials, including expanded access trials, see clinicaltrials.gov. Search “Rigel” to find Rigel clinical trials that may currently be recruiting. If you are a healthcare professional and would like more information on Rigel’s investigational products, please send an inquiry to medinfo@rigel.com. An acknowledgement of receipt can be expected within 3 business days.