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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.

Criteria

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.

Investigational medicines available for early access

Avapritinib:

For information on avapritinib clinical trials, go to clinicaltrials.gov.

In the United States, avapritinib is commercially available for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and for the treatment of adult patients with advanced systemic mastocytosis (Advanced SM), including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL).

Early access programs are closed in geographies where avapritinib is commercially available and/or approved for use. We recommend physicians contact medinfo@blueprintmedicines.com prior to submitting a request, so we may provide information on national and local laws, health authority requirements, and additional considerations including whether we have sufficient resources available to fulfill requests, after ensuring adequate supply for clinical trials and other supporting work required for regulatory processes.

Pralsetinib:

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

Other agents in development:

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

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 medinfo@blueprintmedicines.com. 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

Physicians with questions about our clinical trials or early access policy can contact us at medinfo@blueprintmedicines.com.

Last updated on May 31, 2022.

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Contact our medical information teammedinfo@blueprintmedicines.com or 1-888-258-7768 (Option 2)

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.