This site is intended for US Healthcare Professionals only.
To report an adverse reaction or product complaint, call 1-888-BLUPRNT, option 2, or visit or contact the FDA at 1-800-FDA-1088 or

Early 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 early access programs for 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.

Our ability to provide early access, the type of program, and the timeframe for processing requests and providing investigational medicines varies depending on the country and/or indication for many reasons, including national and local laws as well as health authority requirements. We will consider individual early 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 early access programs.


Early access programs provide a pathway for physicians to request treatments that are not otherwise available for patients in their country. All requests for early access to our investigational medicines outside of clinical trials must be unsolicited and 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;
  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. 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; and
  4. There must be a sufficient amount of Blueprint Medicines’ resources available to fulfill the request after ensuring adequate supply and resources for ongoing and planned clinical trials, as well as other supporting work required for regulatory submissions.

Meeting these criteria does not guarantee early access to our medicines; Blueprint Medicines will evaluate each request to assess whether important internal and external requirements can be met.

Requesting early access

Only a qualified physician who agrees to directly supervise treatment of the patient may submit an early access request. Patients interested in seeking early use of avapritinib or pralsetinib should talk to their physician about their treatment options. Physician requests and inquiries regarding early access can be submitted to We typically respond to initial inquiries within three business days or less.

Acknowledgement of a request does not guarantee that access to the investigational medicine will be provided. Medical professionals at Blueprint Medicines will review each request in accordance with our policy 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 early access policy can contact us at

Last updated on August 17, 2023.

Contact Blueprint Medicines

Blueprint Medicines is a pharmaceutical company focusing on the research, development, and commercialization of products in oncology, hematology, and rare diseases. This website is for US healthcare professionals for educational purposes only. Scientific information and data may include investigational use(s) of compounds for which efficacy and safety have not been established. Blueprint Medicines does not promote or endorse the use of its products in a manner not consistent with the approved label.