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Shire Global

Investigator Initiated Research (IIR)

Areas of Interest

Shire provides IIR grants with the intention of improving medical and scientific knowledge through the generation of new or confirmatory scientific and/or clinical information. 

Although Shire may provide IIR grants in a variety of different therapeutic areas and disease states, Shire is more likely to support grants that focus on current “Areas of Interest (AOI)”.

Please find below Shire’s current areas of interest by therapeutic area questions of interest. AOIs are evaluated as needed to address shifts in scientific knowledge and the company strategy.

Areas of Interest

Click on each of the items below for more information

Neuro- science

Attention Deficit Hyperactivity Disorder

  1. C&A and Adult ADHD disease management – recognition, & treatment
  2. Functional outcomes research related to optimal and suboptimal treatment
    of ADHD symptoms
  3. Lisdexamfetamine dimesylate in C&A and Adult ADHD and other comorbid
  4. Effectiveness of Lisdexamfetamine dimesylate RWE
  5. Guanfacine use in complex ADHD patients
    • Guanfacine use in combination with stimulants RWE
    • Guanfacine effect on symptoms and functioning in ADHD patients
      across ages RWE
  6. Adherence & persistence to treatment options in ADHD
  7. Role of adjunctive psychotherapy

Binge Eating Disorder

  1. Observational Studies on patient characteristics, comorbidities, treatment
    patterns, outcomes
  2. Role of adjunctive psychotherapy

GI/Internal Medicine

Adrenal Insufficiency

  • 2018 Areas of Interest are Currently Under Development

Eosinophilic Esophagitis (EoE)

  1. Pathogenesis
  2. Biomarkers
  3. Epidemiology, diagnosis, clinical response criteria, and qualitative/quantitative management of symptoms
  4. Clinical, behavioral, burden of illness and comorbid conditions


  • 2018 Areas of Interest are Currently Under Development

Short Bowel Syndrome

  1. Real world use of teduglutide in adult and pediatric SBS-IF
  2. Long Term Efficacy Studies in adult and pediatric SBS-IF patients who have reached enteral autonomy under teduglutide
  3. Real world use of teduglutide evaluating intestinal adaptation in treated vs. untreated pediatric SBS- IF patients
  4. Prevention/avoidance of Intestinal Transplantation in stable SBS-IF chronic PN dependent adult or pediatric patients
  5. Comparison of maintenance treatment regimen in adult or pediatric patients with SBS-IF having reached enteral autonomy
  6. Predictors of intestinal adaptation in SBS adult or pediatric patients
  7. Real world QoL in adult or pediatric SBS-IF treated vs. untreated patients
  8. Real world QoL in adult SBS- IF treated vs. untreated patients by étiology and bowel anatomy types
  9. Borderline/Fragile non-Parenteral Support (PS) Dependent Short Bowel Syndrome (SBS)
  10. Reduction of Output in High Output conditions (stoma or intact Gastro-intestinal Tract)
  11. Enterocutaneous Fistula Healing
  12. Potential Anti-Inflammatory mechanisms of Teduglutide

Inflammatory Bowel Disease

  1. Further understand of the role of MAdCAM-1 and T-cell trafficking in IBD
    • Role of different T-cell types (regulatory T-cells versus activated T-cells)
  2. Confirmation of location of MAdCAM-1 expression in human tissue
  3. Factors affecting expression of MAdCAM-1 in human tissue
    • Difference between expression in different stages of disease and in different patient types
    • Impact of prior anti-TNF therapy on MAdCAM-1 expression
  4. Identification of biomarkers that predict and monitor response to therapy

Genetic Diseases

Hunter Syndrome

  1. Analyses of natural history of MPSII
  2. Studies involved in the development or evaluation of biomarkers or diagnostic capabilities in MPS II
  3. Studies examining:
    • Impact of early diagnosis, including screening, and treatment start
    • Long term treatment effectiveness of Idursulfase (also retrospective)
    • Early diagnosis of cognitive impairment
    • Neurodevelopmental assessment
    • Standardization of cognitive testing
    • Neuronopathic MPS II Disease course

Fabry Disease

  1. Screening and early diagnosis strategies in high risks populations
  2. Research projects in:
    • Monitoring disease progression
    • Impact of early treatment and outcomes
    • Agalsidase alfa effectiveness, including long-term effectiveness
      studies (also retrospective)
  3. Studies fostering understanding of inflammation and immunogenicity in
    Fabry disease

Gaucher Disease

  1. Screening for patients with Gaucher disease. Methodology aligned with evidence based global guidance, such as Delphi Consensus on earlier diagnosis, as well as innovative screening projects of high risk populations
  2. Generation of data on patient reported outcomes (PRO)
  3. Generation of data on the role of Lyso-Gb1 as a novel biomarker
  4. Studies fostering understanding of inflammation and immunogenicity in Gaucher disease
  5. ERT infusion optimization (e.g. rate of administration, location (hospital/home), infusion pumps, etc.) that could have potential positive impact on convenience and patient QoL

Ophthal- mology

Dry Eye Disease

  1. Studies to improve the understanding of Dry Eye Disease including diagnosis, risk factors, inflammation/immunopathogenesis
  2. Studies assessing the clinical benefit of lifitegrast in certain subpopulations and/or ocular conditions such as contact lens wear and cataract and refractive surgery
  3. Studies assessing the clinical benefit of lifitegrast on QoL measures, visual function, and long-term outcomes
  4. Studies that advance the understanding of lifitegrast MOA and its effect on (including, but not limited to), biomarkers, mediators, and ocular surface structures
  • Any studies involving the use of lifitegrast in humans can only be considered after registration in country where study would be conducted


Liquid Tumors

  1. Acute Lymphoblastic Leukemia (ALL)
    • Studies examining toxicity handling, combination therapies, ASP activity testing or MRD measurements in adults, adolescents and young adults with Acute Lymphoblastic Leukemia (ALL) treated with pegaspargase
    • Studies examining toxicity handling, combination therapies, ASP activity testing or MRD measurements in children with ALL treated with pegaspargase
    • Studies examining Calaspargase Pegol in adolescents and young adults with ALL (US only)

Solid Tumors

  1. Based on strong pre-clinical/clinical rationale
  2. Pancreatic cancer
    • Health economic/access oriented studies
    • Studies involving Quality of Life
    • Special populations post-gemcitabine
    • Novel therapy combinations post-gemcitabine
    • Earlier line of treatment (except first line in combination with oxaliplatin)
  3. Disease states with known irinotecan/topoisomerase-I activity
    • Safety and efficacy outside of pancreatic cancer
    • Particularly interest in gastrointestinal cancers (apart from mCRC), but not limited to
  4. Novel therapy combinations
    • Novel agents in late-stage development / active therapies (approved in other tumor types or in P3 trials)
  5. Translational research, including validation of MoD/MoA
  6. Personalized treatment strategies


Hemophilia A

  1. Studies examining the cost efficiency of recombinant Antihemophilic Factor (ADVATE) with or without myPKFiT
  2. Studies examining the cost efficiency of PEGylated –recombinant Antihemophilic Factor (ADYNOVATE/ADYNOVI) with myPKFiT
  3. Studies examining the relationship between time of bleeding and Factor VIII levels in patients using the myPKFiT mobile app
  4. Studies to investigate changes in adherence and quality of life (QoL) in patients using recombinant Antihemophilic Factor (ADVATE) myPKFiT and the myPKFiT patient app
  5. Real world utilization (dosing, frequency, etc.) of recombinant Antihemophilic Factor (ADVATE),a standard half-life rFVIII (SHL rFVIII)
  6. Real World Evidence on use of PEGylated -recombinant Antihemophilic Factor (ADYNOVATE/ADYNOVI), an extended half-life rFVIII (EHL rFVIII), in clinical practice with or without myPKFiT (including safety, efficacy, utilization, QoL, adherence, patient Satisfaction etc.)
  7. Studies on other non-coagulation effects of Factor VIII
  8. Studies looking at the GOAL-HEM (Goal Attainment Scaling for Life – Hemophilia) as a patient-centered reported outcome measure to monitor clinical progress
  9. Non-clinical studies on Polyethylenglycol (PEG) safety
  10. Role of PEGylated -recombinant Antihemophilic Factor (ADYNOVATE/ADYNOVI) for tolerization or in previously tolerized/partially tolerized patients

Hemophilia A and B Inhibitor prevention and management

  1. Pre-clinical and clinical studies examining the care and treatment of persons with inhibitors and the use of Anti-Inhibitor Coagulant Complex to control and prevent bleeding episodes and perioperative management in adults and children
  2. Studies exploring the potential immunotolerance effect of Anti-Inhibitor Coagulant Complex prophylaxis during Immuno Tolerance Induction(ITI) in hemophilia patients with inhibitors
  3. Studies examining clinical effectiveness and pharmaco-economic aspects of using Anti-Inhibitor Coagulant Complex in prophylaxis as well as in ITI
  4. Studies examining potential correlations between thrombin generation assay (TGA) parameters and clinical outcomes
  5. Investigations of differences in patient profiles for hemophilia patients with and without inhibitors, and possible correlations with prediction, prevention, eradication and prophylaxis of inhibitors
  6. Explore whether knowledge from other immunological disease states can be used to develop better management of inhibitor patients
  7. Investigate safety profile of Anti-Inhibitor Coagulant Complex to identify possible predictors of outcomes
  8. Investigate the use and cost effectiveness of low dose FVIII ITI with Anti-Inhibitor Coagulant Complex prophylaxis as compared to high dose FVIII ITI alone
  9. Evaluation of Thrombin Generation with non-factor therapies and the impact of Anti-Inhibitor Coagulant Complex co-administration

Acquired Hemophilia (AHA)

  1. Explore efficacy and safety of recombinant-porcine Antihemophilic Factor in patient subpopulations “(i.e. post-partum or patients with specific comorbidities) with AHA
  2. Relationship between treatment effectiveness, FVIII level and anti-pFVIII inhibitor titer in
    subjects with AHA receiving recombinant-porcine Antihemophilic factor
  3. Explore the potential use of recombinant-porcine Antihemophilic Factor for breakthrough bleeds in patients treated with non-factors
  4. Studies intended to develop flexible and tailored dosing regimens for recombinant porcine Antihemophilic Factor
  5. Investigate effectiveness, safety and treatment outcomes of the continuous infusion of recombinant-porcine Antihemophilic Factor
  6. Collect long term data on treatment for patients with AHA

Thrombotic Thrombocytopenic Purpura (TTP)

  1. Long-term outcomes and disease progression in TTP (congenital and or/acquired TTP) with current standard of care
  2. Studies of the epidemiology, disease burden, and/or health care utilization of TTP patients
  3. Genotype/phenotype correlations among TTP patients
  4. Correlation of ADAMTS13 levels with outcomes
  5. Investigate effects and adverse events associated with chronic plasma use
  6. Mechanistic investigations into rADAMTS13 and TTP
  7. Interactions of ADAMTS13 with other proteins that might affect clinical outcomes

Hemophilia Gene Therapy

  1. Basic science of adeno-associated virus (AAV) vectors
  2. Measures to evaluate outcomes with hemophilia gene therapy (clinical, humanistic, quality of life, economic, biomarkers, etc.)
  3. Immune response and immunosuppression with gene therapy
  4. Strategies to examining dosing in the presence of AAV antibodies

von Willebrand Disease (VWD)

  1. Studies that examine the key differentiating features of recombinant von Willebrand factor (rVWF):
    • Ultra-large Multimers (ULM)
      • Role of VWF ULM in hemostasis in vitro, in vivo and in clinical scenarios
      • Studies that examine the role of ULM in FVIII binding and half-life extension
      • Role of ULM in the mechanism and therapeutic impact of angiodysplasia related bleeding
      • Research in animal models examining role of ULMs and ULM size in angiogenesis
    • FVIII Independence
      • Investigation of safety profiles in VWD patients and clinical scenarios where risk of thrombosis is high
      • Guidance on which clinical scenarios require co-administration of rVWF with and without FVIII
  2. Studies investigating rVWF in prophylaxis, situations of heavy menstrual bleeding (HMB), post-partum hemorrhage (PPH), gastrointestinal (GI) bleeding, severe epistaxis or major surgeries.
  3. Personalization of VWD therapy using genetics, bleed prediction, and bleeding assessment tools
  4. Impact of VWD on QoL and the burden of the disease
  5. Impact of prophylaxis on long-term health outcomes
  6. Further understand the mechanism and impact of dysfunctional angiogenesis leading to vascular malformation (angiodysplasia) in VWD related bleeds.
  7. Studies to examine pharmacokinetics and pharmacodynamics of rVWF
  8. Optimizing diagnosis of VWD - screening tools and diagnostic assays
  9. Studies investigating VWF Inhibitor formation in patients having prolonged rVWF exposure


Hereditary Angioedema

  • 2018 Areas of Interest are Currently Under Development

Primary immunodeficiency


  1. We support research in primary immunodeficiency, secondary immunodeficiency and neuroimmunology including peripheral neuropathies.

    Areas of research interest include diagnosis, treatment particularly with immunoglobulin, and outcomes.


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