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.


All requests for pre-approval access to our investigational medicines outside of clinical trials must meet the following criteria to be considered:

1) Requests 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.

2) 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 (or soon opening) clinical trials;

3) There must be sufficient evidence to suggest that potential benefit of treatment with the investigational medicine outweighs the potential risk in the context of the disease or condition to be treated; and

4) 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.

Investigational Medicines Available for Pre-Approval Access


Avapritinib is commercially available in the United States for the treatment of adults with advanced systemic mastocytosis, including aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia, and 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 addition, avapritinib is approved by the European Commission for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA D842V mutation.

In accordance with our criteria for providing pre-approval access noted above, we have established eligibility requirements for the avapritinib program. Eligible patients must:

  • For patients in countries where avapritinib is not commercially available, have advanced SM
  • Have non-advanced SM;
  • For patients in countries where avapritinib is not commercially available, have advanced PDGFRA exon 18 mutant GIST;
  • Have another non-GIST tumor type with a mutation in exon 17 of the KIT gene or in exon 18 of the PDGFRA gene;
  • Not be eligible for ongoing (or soon opening) clinical trials; and
  • Have no comparable or satisfactory alternative therapy.

An expanded access program (NCT04714086) is also open in the United States for patients with advanced SM or smoldering SM. Additional information can be found here:


For questions about the pralsetinib pre-approval access program, please contact our partner Roche using the Roche Medical Information site. A Roche Position Paper provides further information on pre-approval access to investigational medicines.

Other agents in development:

Currently, we are not able to evaluate and fulfill requests for pre-approval access to our other investigational medicines as these programs do not currently meet the criteria noted above.

Process for Requesting Pre-Approval Access

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 three 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 June 16, 2021.