Pre-approval access policy

Pre-Approval Access Policy for 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 pre-approval access to 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 pre-approval access 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 pre-approval access programs. Our ability to provide pre-approval 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 pre-approval access to our investigational medicines outside of clinical trials must meet the following criteria in order to be considered.


Requests for pre-approval access to 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 determined 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.

Patients must have a serious or immediately life-threatening disease; received available standard therapies for their disease, or are not an appropriate candidate for one or more of the available standard therapies as determined by the requesting physician; and must not be eligible for ongoing clinical trials of the investigational medicine;

There must be sufficient evidence to suggest that the investigational medicine could potentially provide therapeutic benefit; 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 pre-approval 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 Pre-Approval Access

For avapritinib:

In 2018, Blueprint Medicines initiated a pre-approval access program for avapritinib (formerly known as BLU-285). Avapritinib is commercially available in the United States for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations. In accordance with our criteria for providing pre-approval access noted above, we have established eligibility requirements for the avapritinib program. Eligible patients must:

  • Have advanced GIST (for patients where avapritinib is not commercially available), advanced or smoldering systemic mastocytosis (SM), or another tumor type with a mutation in exon 17 of the KIT gene or in exon 18 of the PDGFRA gene;
  • Not be eligible for avapritinib clinical trials; and
  • Have no comparable or satisfactory alternative therapy.

For pralsetinib:

In 2019, Blueprint Medicines initiated a pre-approval access program for our investigational medicine pralsetinib (formerly known as BLU-667). In accordance with our criteria for providing pre-approval access noted above, we have established eligibility requirements for the pralsetinib program. In order for patients to be eligible, they must:

  • Have advanced non-small cell lung cancer (NSCLC) or medullary thyroid cancer (MTC);
  • Have a verified mutation or fusion in the RET gene (NSCLC only);
  • Not be eligible for pralsetinib clinical trials; and
  • Have no comparable or satisfactory alternative therapy.

Currently, we are not able to evaluate and fulfill requests for pre-approval access to our other investigational medicine, fisogatinib (formerly known as BLU-554), as this program does not currently meet the criteria noted above.

Only a qualified physician who agrees to directly supervise treatment of the patient may submit a pre-approval access request. Patients interested in seeking pre-approval use of avapritinib or pralsetinib should talk to their physician about their treatment options. Physician requests can be submitted to

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 pre-approval access 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 pre-approval access and the program eligibility requirements.

Additional Information

You can learn more about our ongoing clinical trials on our website at or by searching for “Blueprint Medicines” or the name of the investigational medicine on

In addition, patients and physicians with questions about our clinical trials or pre-approval access policy can contact us at

Last updated on January 16, 2020.