Investigator sponsored research

Investigator-Sponsored Research Support Program

Blueprint Medicines is committed to supporting investigator-sponsored research (ISR) that contributes to the scientific and medical understanding of our products and relevant disease areas, and which enhances the practice of medicine, ultimately improving patient care.

ISR includes both clinical and non-clinical studies designed and managed by an independent investigator outside of Blueprint Medicines. The Investigator and their organization assume responsibility for the management and conduct of the study through the duration of their research. We welcome well-written, scientifically sound research proposals that align with our areas of medical and scientific interest (see below) and meet a multitude of goals, including addressing unmet medical needs, addressing gaps in existing scientific understanding, improving patient care or sparking new ideas for disease-related research.

Support for an approved ISR may come in various forms including funding, product and/or material, or in some cases, both funding and product in response to unsolicited ISR requests, as allowed under local laws and regulations. All requests will be critically reviewed, and a decision will be made based on scientific merit, feasibility, and our current research objectives and available resources. Proposals that directly compete with our current and/or future studies will not be considered.

Areas of Research Interest

Blueprint Medicines will consider IST proposals in the following areas of interest:


  • Systemic Mastocytosis (SM)
    • Advanced systemic mastocytosis: Use in advanced systemic mastocytosis and other hematologic malignancies
      • Combination/sequencing studies in SM with an associated hematologic neoplasm (AHN)/mast cell leukemia (MCL), including preclinical models; real-world experiences; and pharmacokinetic studies evaluating combinations
      • Use of avapritinib as bridge to bone marrow transplantation (BMT) and/or maintenance therapy post-BMT
      • Rational treatment sequencing/combinations in acute myeloid leukemia (AML) or other hematologic malignancies
      • Real-world experience in mitigating anaphylactic episodes, polypharmacy, health care resource utilization (HCRU) or disease burden
      • Studies to optimize dosing and adverse event management strategies, including for patients with severe thrombocytopenia
      • Immunological, histopathological or genetic evaluations of disease or response to therapeutics
    • Non-advanced systemic mastocytosis: Use in non-advanced systemic mastocytosis, including mast cell disorders
      • Proof-of-concept preclinical studies to evaluate disease biology; impact of mast cell mediator release on symptom pathology
      • New methods for evaluation of response in non-advanced SM
      • Utilization of the Indolent SM Symptom Assessment Form (ISM-SAF)
      • Impact of avapritinib on anaphylaxis in patients with non-advanced SM
    • Retrospective data analysis to characterize patient population/diagnostic journey in SM
      • Real-world data to define the diagnostic journey using institutional databases
      • Best practices to highlight multidisciplinary care, leveraging real-world evidence
  • Gastrointestinal Stromal Tumor (GIST)
    • Studies to optimize dosing strategies, including combination studies
    • Neoadjuvant/adjuvant studies for PDGFRa GIST patients
    • Novel proof-of-concept studies in KIT- or PDGFRa-driven solid tumors

We recommend that Investigator Sponsored Research support requests be submitted at least 9 months prior to planned research initiation. Please review the following information carefully to ensure all requirements are met, as this will facilitate a timely review.




How to submit an ISR request

Brief requests for ISR support (ISR Concept) must be submitted via our web-based portal. The ISR Concept will be reviewed by Blueprint Medicines’ Scientific Review Committee (SRC). If the SRC approves the ISR Concept according to the criteria described above, the investigator will be invited to submit a more detailed proposal containing the following, at a minimum:

  • A comprehensive description of the proposed ISR study, including the nature and scope of the support requested;
  • A curriculum vitae (CV) or resume that documents previous clinical research experience of the proposed investigator(s) and other key research staff;
  • Evidence of good medical standing, including no restrictions by a regulatory authority (debarment) to undertake clinical research, if applicable;
  • Detailed project budget, including itemization of costs and services;
  • Proposed research timelines and milestones, including a description of anticipated publications and/or presentations; and
  • Any additional relevant supporting documentation required by Blueprint Medicines’ Scientific Review Committee.

Last updated on March 15, 2023.