We at Blueprint Medicines Corporation are committed to providing access to our investigational medicines for patients facing a serious or immediately life-threatening disease. Our ongoing development programs in gastrointestinal stromal tumors (GIST), systemic mastocytosis (SM), hepatocellular carcinoma (HCC), and RET-altered solid tumors including non-small cell lung cancer (NSCLC) and medullary thyroid cancer (MTC) do not currently meet the criteria identified below to enable us to evaluate and fulfill requests for compassionate use at this time. If you have questions regarding the status of our development programs or regarding our criteria for evaluating requests for compassionate use, please contact us at medinfo@blueprintmedicines.com.


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 at Blueprint Medicines 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

We are not currently able to evaluate and fulfill requests for compassionate use of our investigational medicines as our programs do not currently meet the criteria above.

Patients interested in seeking compassionate use of a Blueprint Medicines investigational medicine should talk to their physician.  A treating physician may request information about Blueprint Medicines’ compassionate use policies by contacting us at medinfo@blueprintmedicines.com.  We typically will respond to inquiries within five business days.

You can also learn more about our ongoing clinical trials by searching for “Blueprint Medicines” or the name of the investigational medicine on http://www.clinicaltrials.gov.