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 ISR proposals in the following areas of interest:


  • Evaluation of the natural history of disease in SM, including disease worsening or progression, utilizing biomarker and clinical features to characterize risk​.
  • Better understanding of mild, moderate, and severe disease in ISM including the correlation of underlying biology with clinical signs and specific organ/system symptoms​
  • Defining the etiology, pathophysiology, and mechanism of neuro-cognitive effects (including depression) in mastocytosis​
  • Real world data to define the diagnostic journey using institutional databases​.
  • Screening studies in high-risk populations (e.g., POTS, IBS, CM)​
  • Evaluation of novel treatment algorithms in ISM based on objective measures​.
  • New methods for evaluation of response​
  • Evaluation of the impact of avapritinib on mitigating anaphylactic episodes, polypharmacy, healthcare resource utilization, and/or burden of disease​
  • Studies to optimize dosing and adverse event management strategies, including patients with severe thrombocytopenia​.
  • Rational treatment sequencing/combination trials in SM with an associated hematologic neoplasm (AHN)/mast cell leukemia (MCL) or acute myeloid leukemia (AML), including preclinical models and pharmacokinetic studies evaluating combinations​.
  • Immunological, histopathological, or genetic evaluations of disease or response to therapeutics​

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 April 9, 2024.