Investigator-Initiated Trials / Studies

• Studies with sac/val in Chronic Heart Failure with reduced ejection fraction (EF):
  • Safety and tolerability in populations not well represented in PARADIGM-HF, PIONEER-HF
    • de-novo heart failure
    • ACEi/ARB naive
• Mechanistic studies looking at:
  • Remodeling, fibrosis, inflammation
  • Cardiac function (including diastolic function)
  • Cardiac biomarkers
• Population with specific, less well studied / documented HF etiologies
• Out of scope :
  • Studies in HFmREF or HFpEF or post-MI patients
  • Comparative effectivenes studies vs other MoA, e.g. SGLT-2i
  • Studies in non-cardiovascular disease
  • Studies in patients with valvular disorders not related to HF
  • Studies focused on hypertension including resistant hypertension
  • Studies in patients with severe renal impairment (eGFR < 30 ml/min/1.73 m2)
  • Studies in children (<18 years)

• Registries with data on Familial Mediterranean Fever (FMF):
  • Real-life data on colchicine resistance, lack of tolerance and suboptimal response to colchicine (retrospective)
  • Impact on quality of life (QoL) of suboptimal response to colchicine; impact seen by patients vs that by HCPs (prospective)
• Clinical studies looking at the window of opportunity in Still’s disease:
  • Early use of canakinumab in Still’s disease
• Studies in rare systemic autoinflammatory diseases (SAID):
  • Schnitzler Syndrome
  • Periodic Fever, Aphthous stomatitis, Pharyngitis and cervical Adenitis (PFAPA)
  • Pyogenic sterile Arthritis Pyoderma gangrenosum and Acne (PAPA)
  • Chronic Recurrent Multifocal Osteomyelitis (CRMO)
  • Kawasaki
  • Behçet disease
  • Yao syndrome
  • Post-hoc analyses of existing trials
• Biomarkers in Still’s disease & IL-1ß signature
• Out of scope
  • Osteoarthritis & other high prevalence indications
  • Pre-clinical IITs

• Vaccination studies 
• Implementation studies 
• Telemedicine approaches in MS patient care
• Patient-reported/relevant outcomes and development of novel and holistic patient improvement measures
• Clinical impact in cognition, fatigue, ambulation and other functional changes
• Biomarkers in MS disease
• Neuroimaging (microglia, brain atrophy, SC, cortical, etc.)
• Progression and Predictors of response/conversion: Digital tools, clinical, biomarkers
• Differentiation of S1P Modulators
• Differentiation of ofatumumab from other RMS DMTs
• MS pathophysiology studies, including mechanism of CD20+ immune cells in disease
• Use of ofatumumab in subpopulations not specifically studied in pivotal trial programs 
• Potential role of ofatumumab in neuroscience diseases/disorders in which dysregulation of B cells may occur
• Confirmatory studies that high efficacy MS treatment early leads to better patient outcomes
• Out of scope:
  • Head to head studies
  • Studies in patients with secondary progressive MS (for ofatumumab)
  • Studies in oncological indications

• Studies with alpelisib in HR+/HER2- studies in breast cancer patients exploring:
  • Side effect management trials (e.g., hyperglycemia and rash)
  • Post progression on CDK4/6 inhibitors + endocrine therapy in adjuvant setting
  • Recycling of fulvestrant after use of CDK4/6i plus fulvestrant
  • Rash management optimization trials
• Studies with alpelisib in other tumor types with PIK3CA mutations

• Melanoma:
  • dabrafenib/trametinib treatment optimization including sequencing and/or biomarkers driven strategies 
  • Melanoma early stages (i.e., neoadjuvant, adjuvant treatment after local/regional relapse) 
  • Acral/mucosal melanoma
  • Real world evidence (i.e., patient characterization, treatment patterns, outcome predictors)
• Out of scope:
  • NRAS mutated melanoma
  • Pediatric gliomas

• Biomarker studies (e.g., TIM-3, LSC, etc.)
• Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML): Combinations, novel –novel, optimal sequence
• Burden of illness and quality of life studies in MDS and AML 
• Pattern of treatment, novel agents treatment in real world (RW) and RW data in MDS and AML
• Out of scope:
  • Solid tumors and other none-hematology indications
  • Studies in children
  • Clinical studies in COVID-19

Indication: neovascular Age-related Macular Degeneration (nAMD)

• Studies investigating innovative assessment methods:
  • functional efficacy end-points, e.g. reading speed, contrast sensitivity and anatomic end-points
  • new imaging tools e.g. angio-OCT and Ultrawide Field imaging
• Studies aimed to evaluate effectiveness with real-world usage of brolucizumab, including treatment patterns, anatomical outcomes, treatment burden and adherence/compliance, impact on QoL
• Studies investigating brolucizumab in sub-populations, e.g. CNV sub-types or PED and other VEGF-driven retinal diseases, e.g.Mac-Tel; Central Serous Chorioretinopathy and similar
• Studies investigating biomarkers or genetic markers which could better predict outcomes to improve patient care
• Studies utilizing novel PRO endpoints and evaluations
• Studies involving use of digital technology for improved patient compliance (e.g. home monitoring devices)
• Out of scope:
  • Mechanisitc/Pre-clinical studies aimed to evaluate and understand PK/PD or systemic VEGF levels and similar
  • H2H studies with insufficient statistical power (i.e. small sample size) evaluating BCVA as primary efficacy objective
  • Studies (single arm or H2H) targeting safety as primary or key secondary end-point
  • H2H studies versus unlicensed bevacizumab or licensed biosimilars of any anti-VEGF agents

• Effectiveness of IND/GLY/MF (LABA/LAMA/ICS), incl. benefits on symptom relief
• Benefits of the Breezhaler (Concept 1) and/or digital companion (sensor & app), incl. characterization of the patient population who benefits most from digital tools
• Anti-inflammatory action of GLY (LAMA)