Policy for Compassionate Use of Investigational Medicines

Patients are at the center of Blueprint Medicines’ mission of discovering, developing and delivering innovative medicines that address significant medical needs. Our goal at Blueprint Medicines is to determine whether our investigational medicines are safe and effective and, if so, to advance them through the clinical trial and regulatory process as quickly and responsibly as possible. It is through clinical trials and the successful registration of our medicines with global health authorities that we will be able to make our potential medicines available to the greatest number of patients who might benefit.

We appreciate the urgency felt by patients and their families facing a serious or immediately life-threatening disease when there are no standard treatment options available or all treatment options have been exhausted. We are committed to providing compassionate use of our investigational medicines for these patients most in need, while maintaining our primary focus on moving our investigational medicines through clinical trials and ultimately toward regulatory approvals.

We will consider individual compassionate use requests for our investigational medicines that meet the criteria outlined below. For our investigational medicines that are in later stages of development, we may also consider opening broader expanded access clinical programs. Our ability to provide compassionate use or expanded access, the type of program, and the timeframe for processing requests and providing investigational medicines varies from country to country for many reasons, including national and local laws as well as health authority requirements. This may be the case even for the same investigational medicine. However, all requests for compassionate use of our investigational medicines outside of clinical trials must meet the following criteria in order to be considered.


  • Requests for compassionate use of our investigational medicines must be made by a qualified physician who agrees to directly supervise treatment of the patient. The physician making the request must have made a determination that the probable risk to the patient from the investigational medicine is not greater than the probable risk from the disease. We will work with the patient’s physician to provide him or her with available information about the investigational medicine, at the time of the request, to enable this assessment;
  • The patient must have a serious or immediately life-threatening disease for which there is no comparable or satisfactory alternative therapy for the disease or condition as determined by the requesting physician;
  • The patient must not be eligible for ongoing clinical trials of the investigational medicine;
  • The investigational medicine must be in active clinical development in the disease with sufficient clinical data available to suggest that the drug may provide therapeutic benefit in that disease, and a recommended dose and dosing schedule has been determined for further studies; and
  • There must be a sufficient amount of the investigational medicine available for the requested use after ensuring adequate supply for ongoing and planned clinical trials and other supporting work required for regulatory submissions.
  • While we cannot guarantee compassionate use or expanded access to our investigational medicines, Blueprint Medicines will continuously evaluate all of our clinical programs in relation to these criteria and will evaluate all requests individually. Appropriate health authorities, such as the Food and Drug Administration (FDA) in the United States, and the Institutional Review Board (IRB) or Ethics Committee (EC) at the patient’s treating hospital or clinic must also review and approve the proposed use of the investigational medicine by the patient before Blueprint Medicines can provide it. In addition, the submitting physician must agree to comply with regulatory obligations, including obtaining patient informed consent, patient monitoring and safety reporting.

Process for Requesting Compassionate Use

In January 2018, Blueprint Medicines initiated a compassionate use program for our investigational medicine avapritinib (formerly known as BLU-285). In accordance with our criteria for providing compassionate use noted above, we have established eligibility requirements for the avapritinib program. In order for patients to be eligible, they must have advanced gastrointestinal stromal tumors (GIST) or advanced or smoldering systemic mastocytosis (SM), not be eligible for avapritinib clinical trials, and have no comparable or satisfactory alternative therapy.

Currently, we are not able to evaluate and fulfill requests for compassionate use of our other investigational medicines, BLU-554, BLU-667 and BLU-782, as these programs do not currently meet the criteria noted above.

Only a qualified physician who agrees to directly supervise treatment of the patient may submit a compassionate use request. Patients interested in seeking compassionate use of avapritinib should talk to their physician about their treatment options. Physician requests can be submitted to medinfo@blueprintmedicines.com.

Once we receive a request, we will provide the physician with detailed treatment guidelines including eligibility criteria and other documentation to enable review of the compassionate use request by Blueprint Medicines, the physician’s local institutional review board and relevant regulatory authorities. We typically respond to initial inquiries within five business days or less.

Acknowledgement of a request does not guarantee that access to the investigational drug will be granted. Medical professionals at Blueprint Medicines will individually review each request, consistent with our policy on compassionate use and the program eligibility requirements.

Additional Information

You can learn more about our ongoing clinical trials on our website at www.blueprintmedicines.com/patients/our-clinical-trials or by searching for “Blueprint Medicines” or the name of the investigational medicine on www.clinicaltrials.gov.

In addition, patients and physicians with questions about our clinical trials or compassionate use policy can contact us at medinfo@blueprintmedicines.com.