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:
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.
Avapritinib:
For information on avapritinib clinical trials, go to clinicaltrials.gov.
In certain geographies, we support a limited number of early access programs for avapritinib. We recommend physicians contact medinfo@blueprintmedicines.com prior to submitting a request so that we may review it along with 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:
Our partner Roche supports existing patients who are receiving early access to pralsetinib. Questions regarding this program should be directed to Roche using the Roche Medical Information site.
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.
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.
Physicians with questions about our clinical trials or early access policy can contact us at medinfo@blueprintmedicines.com.
Last updated on November 3, 2022.
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.
Early access policy for investigational medicines