Externally Sponsored Research

We support Externally Sponsored Research (ESR) in two forms.

  • Investigator-Sponsored Research (ISR), which is unsolicited independent research from an external sponsor-investigator who takes full responsibility for study design and execution.
  • Externally Sponsored Collaborations (ESC), which may either be solicited by Alexion or proposed by an external sponsor-investigator, and which is conducted in collaboration with joint decision-making authority.


The global ESR program is open to all investigators who are interested in conducting sponsored research and assuming the legal and regulatory responsibilities of the study. Alexion supports only clinical (interventional or observational) ESR studies, research utilizing patient samples, patient records, or patient chart review. Support for non-clinical studies can be requested through the separate Discovery Partnerships program. 

Alexion receives many requests for ESR support and carefully evaluates each ESR submission. Review of a submitted ESR proposal does not imply or guarantee approval.

Research Areas of Interest

Alexion is interested in research that explores the following topics:

  • Hypophosphatasia (HPP)
    • Diagnostic pathways for HPP in children and adults
    • Burden and course of disease in HPP patients across all age groups
    • Real world data on the effectiveness and safety of alkaline phosphatase enzyme replacement therapy 
  • Lysosomal acid lipase deficiency (LAL-D)
    • Natural history and complications of LAL-D
    • Diagnosis and differential diagnosis of LAL-D, including FH, fatty liver disease, HLH, failure to thrive
    • Treatment options, including supportive treatment concepts and dietary care, employed for the therapy of signs and symptoms of LAL-D and treatment results
    • Burden of disease of LAL-D or patients, care givers/families and society
    • Fatty liver disease in conjunction with very low LAL activity
    • Co-morbidities hypothesized to be linked to/caused by low levels of LAL activity
  • Neurofibromatosis type 1 (NF1)
    • Exploration of potential benefits of earlier treatment of pediatric NF1 patients prior to emergence of severe symptoms
    • Exploration of potential benefits of re-treatment with mitogen-activated protein kinase kinase 1/2 (MEK1/2) inhibitor to patients who had previously discontinued therapy for reasons other than toxicity or progression
    • Real world data on efficacy and safety of MEK1/2 inhibitor therapy in NF1 populations
  • Generalized myasthenia gravis (gMG)
    • Effectiveness and safety of early treatment with C5 complement inhibition therapy (C5IT) in gMG and patient subgroups
    • Effectiveness and safety of C5IT as monotherapy in gMG
    • Effectiveness and safety of switching to C5IT in gMG
    • Biomarkers to identify patients who may benefit the most from C5IT
    • Acute and long-term anatomical consequences of complement-mediated destruction and inflammation at the neuromuscular junction
    • Assessing disease status, with novel clinical or patient-centric outcomes
  • Neuromyelitis Optica Spectrum Disorder NMOSD
    • Use of imaging, such as MRI and OCT, to enable early and accurate NMOSD diagnosis and effective monitoring
    • Non-imaging biomarkers for diagnosis, prognosis (including relapse prediction), response to treatment for NMOSD, and patient monitoring between relapses (including relapse-independent disease activity)
    • Co-morbidities, mortality, disease characteristics of NMOSD patients
    • Characterization of full clinical impact of disease on NMOSD patients and/or caregivers
    • Real-world effectiveness and safety of C5IT across NMOSD treatment algorithm
    • Mitigation and management of meningococcal infection in patients treated with C5IT and prior to treatment

How Our Submission Process Works

Submit Proposal

Investigators interested in submitting an ESR to Alexion for consideration will first have to register on the web-based system with their profile. Once you have registered in the system, you can submit a proposal electronically. Email submissions are not accepted. To complete the submission of your concept proposal, please specify the type of support you are requesting (funding, drug product or product), and include the following information:

  • Study Title
  • Contact Information
  • Therapeutic Area
  • Indication
  • Study Duration
  • Rationale
  • Objectives
  • Endpoints
  • Curriculum Vitae and Conflict of Interest form

Proposal Review

Alexion will review each ESR proposal to confirm patient safety, scientific merit, operational feasibility, strategic alignment, portfolio, and budget fit. Decisions are typically communicated within 45 days of receipt of a complete proposal submission.

Study Start-Up

Following the approval of the proposal, you will be asked to submit a full protocol, final study budget and final clinical drug supply request. Once this information has been reviewed and approved, you will receive a study start up package, including Third Party Due Diligence financial forms, contract, and other required start-up documents.

Study Conduct

Once a study is activated, any changes or updates to a study must be submitted via the web-based ESR system. Alexion requires quarterly updates for studies. If an Alexion medication is part of the study, all adverse drug events must be reported promptly to Alexion in accordance with the reporting guidance provided by Alexion.

Study Closeout

After study completion, Alexion must receive a final study report and reconciliation of all clinical drug supplies and funding provided.


An expected outcome of the ESR study is a Final Study Report. In addition, the sponsor-investigator may choose to submit an abstract or manuscript to a conference or journal. Prior to any such submission, Alexion requires that a draft of the publication be sent to Alexion for a courtesy review. Alexion funding must be disclosed in any publication.

ESR submissions are managed by Alexion directly in some markets, and by our parent company AstraZeneca in others. Please submit at the link associated with your country below.

Australia, Austria, Belgium, Brazil, Canada, Colombia, Finland, France, Ireland, Italy, Japan, Netherlands, New Zealand, Norway, Spain, Switzerland, Turkey, United Kingdom, United States

Submit your ISR proposal

Submit your ESC proposal

Argentina, Bahrain, Bosnia-Hercegovina, Bulgaria, Chile, China, Croatia, Cyprus, Czech Republic, Ecuador, Estonia, Greece, Hong Kong, Hungary, Kuwait, Latvia, Lithuania, Macedonia, Mexico, Montenegro, Oman, Peru, Poland, Qatar, Romania, Saudi Arabia, Serbia, Slovakia, Slovenia, South Korea, Taiwan, United Arab Emirates

Submit your ESR Proposal (ISR & ESC)