Investigator Initiated Trials / Studies (clinical) and Investigator Initiated Research (non-clinical)

Studies within the label population​ 

• Long-term safety and tolerability​
• Health-related quality of life (HrQoL)​
• Implementation science and/or quality system improvement programs (ex. clinical care pathways)​
• Early post-event implementation
• Stroke
• PAD
• LDL-C lowering in under-represented population
• Adherence vs. other LLTs

Mechanistic Studies in secondary prevention​ 

• Remodeling, fibrosis, inflammation ​
• Plaque burden regression/modification​
• CABG graft remodeling 

Mechanistic Studies in primary prevention and/or patients with statin-intolerance

• Remodeling, fibrosis, inflammation ​
• Plaque burden regression/modification
• Assessment techniques (IVUS, echo, CCTA, OTC, MRI) must be guidelines validated (pending vascular bed assessment)

Out of scope:

• Studies in off-label populations (with respect to geographies)​
• Efficacy, safety and tolerability studies with inclisiran in pediatric population (<18 y)​
• Studies in adults with HoFH and/or different populations than ASCVD and ASCVD equivalent
• CVOT trials​ 
• Head-to-head efficacy/safety studies with other lipid lowering therapies  
• Pre-Clinical Proposals (separate process)​

Studies involving drug for any indication(s) currently in clinical development and not yet approved

With Drug

1. Mechanistic studies in IgAN, C3G, aHUS
2. Subgroups of patients that are included in the overall study population in indications pursued with iptacopan (IgAN, C3G, aHUS)

Without Drug

1. Role of complement system in complement-mediated kidney diseases
2. Additional ways to foster diagnosis of glomerulopathies beyond biopsy
3. Studies which attempt to clarify the histopathologic complexity/equipoise of C3G
4. Identification of approaches that lead to better characterization, management or correlation with outcomes in IgAN, C3G, aHUS, MN, LN – e.g. identification of biomarkers, genetic analysis or biopsy-based studies
5. Burden of disease (clinical, economic, and/or humanistic burden) - IgAN, C3G, aHUS, MN, LN
6. Epidemiology studies (incl. registries) - IgAN, C3G, aHUS, MN, LN

Out of scope:

• Pediatric studies (with drug)
• Studies exploring different dosing regimens as currently investigated
• Any study, which combines iptacopan with immunosuppressant
• Head-to-head comparisons
• Studies including patients with CKD stages 4 and 5

* Strategic areas of interest for iptacopan (PNH), please also refer to the Oncology section

Without drug 

• Epidemiology associated with elevated Lp(a)
  • Patient characterization, identification, and genetic risk across sub-groups
  • Plaque characteristics and differences across patient sub-groups
  • Association & impact on different types of CVD (ischemic stroke, CAVS, PAD), various vascular beds, and other diseases (e.g., AF, kidney disease, diabetes)
• Distinct and unique pathophysiology of Lp(a) 
  • Insights on the pro-inflammatory or pro-thrombotic mechanisms impacted by Lp(a)
  • Unique features  of Lp(a)
• Quantification of Lp(a) role in CV risk assessment tools
  • Quantification of Lp(a) contribution to global CV risk and in light of other CV risk factors 
  • Risk score calculators incorporating Lp(a) 
  • Patient perception on contribution of Lp(a) to CVD and CV risk
• Lp(a) testing and global CV risk management
  • Implementation of Lp(a) testing in CVD management pathways
  • Clinical and economic value of Lp(a) testing
  • Guidance on management of currently modifiable risk factors in the setting of elevated Lp(a)

Out of scope:

• Comparison / association with LDL-C​ 
• Studies involving drug for any indication(s) currently in clinical development and not yet approved

Heart Failure

• RWE or Implementation Science studies on improvements of HF care through increase in GDMT
• RWE studies with sac/val in Chronic Heart Failure with reduced EF
• RWE studies with sac/val in Chronic Heart Failure with mildly-reduced or preserved EF - in geographies where it is in-label
• RWE studies with sac/val in HTN - in geographies where it is in-label

Out of scope:

• Comparative effectiveness studies vs other MoA, e.g. SGLT2i, MRA, BB
• Studies in non-cardiovascular disease
• Studies in patients with valvular disorders not related to HF
• Studies in children (<18 years)
• Mechanistic Studies in HF including but not limited to those looking at:
  • Remodeling, fibrosis, inflammation
  • Cardiac function (including diastolic function)
  • Cardiac biomarkers
• Studies in populations with specific, less well studied / documented HF etiologies, e.g. chemotherapy /toxicity induced HF

Indication: Spinal Muscular Atrophy (SMA)

• Safety-related IITs demonstrating or validating care needs for SMA populations post Zolgensma treatment
• Expansion of treatment with Zolgensma for patient populations not included in clinical trials (e.g. older/heavier patients, four SMN2 copies, switch therapy, ambulatory, etc.…)
• Cost of care, quality of life and caregiver burden, cost effectiveness
• Methods/processes to assess the efficacy and durability of Zolgensma (e.g. bulbar function)
• Biomarkers for efficacy

Out of scope

• Clinical trials involving Zolgensma:
  • Re-dosing
  • Study of alternative doses/maximum dose
  • Head-to-head comparison with other therapies and combination with other disease-modifying treatments (DMTs)
• Basic science research that request use of Zolgensma

• Studies with crizanlizumab in sickle cell disease and related complications
  • e.g- renal, leg ulcer, stroke, AVN, adolescents with SCD, priapism, splenic sequestration, VOCs
• Mechanistic studies with crizanlizumab
• Predictors of response to crizanlizumab
• SCD biomarkers

Out of scope:

• IIT requests from countries outside of US, SSA, Brazil
• IIT requests in non-SCD indications

• Studies with HU in sickle cell disease and organ protection
  • e.g., spleen, lungs, kidneys
• Societal and economic impact of HU/HU-FCT on LMIC
• Studies with HU-FCT looking at treatment/stroke prevention in LMIC 
• HU-FCT preference by caregivers

Out of scope

• IIT requests from countries outside of SSA, Brazil and India
• IIT requests in non-SCD indications

Indications: axSpA (axial spondyloarthritis), incl. r-axSpA (radiographic) and nr-axSpA (non-radiographic)

Clinical data, outcomes & RWE:

• Long term RWE studies on clinical efficacy, structural progression & safety of secukinumab
• Clinical outcomes with Secukinumab across different manifestations of axSpA , by gender and race

Implementation Science/HCS research.

• Identification of Predictors of structural progression and treatment algorithm related to structural progression
• Impact of early intervention and treat-to-target on patient outcomes
• impact of Secukinumab on prevention or reduction of Comorbidities
• Research Use of Novel imaging modalities for early diagnosis, pathogenesis of disease and monitoring of Secukinumab response
• Evaluate the impact of Secukinumab and treatment strategy to reduce Fatigue and pain

Exploratory/ mechanistic studies:

• New classification criteria of AxSpA and differences in pathogenesis of axSpA vs. axial PsA.
• Role of IL-17A in the pathogenesis of axial, peripheral manifestations and comorbidities of AxSpA
• Role of IL-17A across the spectrum of spondyloarthritides (SpA)

Out of scope

• Studies on safety topics e.g. infections (tuberculosis, HIV, viral hepatitis), high-risk patients
• Studies with combination biologics
• Clinical comparative studies with other treatments

Indications: Psoriatic arthritis (PsA)

Clinical data, outcomes and RWE:

• Long term RWE studies on clinical efficacy, inhibition of structural progression & safety of secukinumab
• Long term RWE studies on efficacy, safety and treatment strategy in juvenile PsA (JPsA) and enthesitis-related arthritis (ERA
• Clinical outcomes with Secukinumab in key manifestations of PsA, by gender, race, ethnic minorities and access to health care systems
• Clinical outcomes with Secukinumab in specific phenotypes (Axial PsA, skin predominant , nail/dactylitis, Oligoarticular predominant)

Implementation Science/HCS research: cost-effectiveness, resource utilization and guideline implementation

• Impact of early treatment and treat-to-target on patient outcomes and resource utilization
• Impact of Secukinumab on prevention or reduction of Comorbidities (e.g.CV, metabolic)
• Research studies on Novel imaging for early diagnosis and monitoring of Secukinumab response
• Evaluate the impact of Secukinumab to reduce Fatigue and pain

Exploratory/ mechanistic studies:

• Role of IL-17A in the pathogenesis of Axial PsA and differences with pathogenesis of. axial PsA vs axSpA
• Roles of different cytokine pathways in the key manifestations of PsA notably axial disease, enthesitis, nail-dactylitis

Out of scope

• Studies on safety topics e.g. infections (tuberculosis, HIV, viral hepatitis), high-risk patients
• Studies with combination of other biologics
• Comparative studies with other treatments

Indications: Psoriasis (PsO)

Clinical data, outcomes and RWE:

• Long term RWE studies on clinical efficacy, & safety of secukinumab, risk factors and prevention of the transition period of PsO to PsA
• Clinical outcomes with Secukinumab by gender, race, skin of colors, ethnic minorities and access to health care systems
• Long term RWE studies on efficacy, safety and treatment strategy in pediatric PsO

Implementation Science/HCS research:

• Impact of early intervention strategy on disease modification in PSO and resource utilization
• Research program designed for early diagnosis and characterization of PSO patients at risk of PsA : disease burden, risk factors, screening tools/app, novel imaging

Exploratory/ mechanistic studies:

• role of IL-17A in the pathogenesis of the transition period PsO to PsA
• Mechanistic study of Secukinumab in Early PsO

Out of scope

• Studies on safety topics e.g. infections (tuberculosis, HIV, viral hepatitis), high-risk patients
• Studies with combination other biologics
• Comparative studies with other treatments

Indications: Hidradenitis Suppurativa

Clinical data, outcomes and RWE:

• Disease progression and impact of early intervention (including imaging techniques, such as ultrasound)
• Clinical outcomes in subpopulations (e.g. disease phenotypes, comorbidities, Black / African American, super-responders,..)
• Safety of integration of surgical interventions during secukinumab treatment
• Safety and efficacy / effectiveness of combination therapy with secukinumab
• Effects of lifestyle intervention on HS treatment with secukinumab

Implementation Science / HCS research:

• Quality of care, cost-effectiveness, resource utilization, and guidelines implementation
• Artificial Intelligence/Machine Learning algorithms and big data approach to improve diagnosis and treatment of HS
• Development and validation of scoring tools / PROs

Exploratory/ mechanistic studies:

• Translational research on pathophysiology - role of IL-17A and other pathways in HS over the course of the disease
• Biomarkers to predict disease and treatment outcomes

Out of scope

• Comparative studies with other treatments
• Combination studies for secukinumab with other biologic agents
• IV dosing for HS

• Focus on prognosis and diligent monitoring of patients with MS (including data and digital):
  • Markers for disease prognosis, disease monitoring, and/or risk mitigation
  • New or improved quantitative outcome measures in MS, including next-generation technology and patient assessment technologies
  • Integration of markers/outcome measures to establish disease stability or disease control, disease progression
• Mechanistic studies looking at differentiating Novartis compounds from other DMTs

BREAST CANCER

• HR+ / HER2 - studies in breast cancer patients exploring:
  • Post adjuvant CDK4/6 inhibitors + endocrine therapy
  • Patient care, safety and tolerability related studies (e.g., AEs with special interest, Quality of Life, Digital health solution)
• Studies in advanced breast cancer an ovarian indications:
  • Combination of Alpelisib with other compounds in view of the evolving landscape
  • Differentiation from established/future compounds, clinically relevant patients groups, etc

RARE

• Alpelisib in non-cancer pathologies with evidence of a PIK3CA mutation and/or an activation of the PI3K/AKT/mTOR pathway

Out of scope:

• BREAST CANCER - Any area outside of advanced breast and ovarian cancer
• RARE - Alpelisib in PROS

Studies in the label population (adult patients with Ph+ CML-CP, previously treated with two or more tyrosine kinase inhibitors):

• Clinical efficacy and safety in real-world setting
• Long-term safety and tolerability
• Treatment optimization in 3rd line

Studies exploring additional patient populations in CML:

• Use of asciminib in earlier treatment lines, such as 2^nd line and newly diagnosed CML
• Treatment Free Remission
• High need populations such as Ph+ALL, CML-AP/BC 
• Use of combinations

Studies providing insight into mechanistical action of asciminib, potential on- and off target effects and its use against additional mutations in patients with CML.

Out of scope:

• Use of asciminib in ABL-independent diseases

With Drug

1. Mechanistic studies in Paroxysmal Nocturnal Hemoglobinuria (PNH);
2. Studies evaluating factors associated with or predictive of treatment outcome in PNH;
3. Studies exploring preferences in oral treatment administration approaches in PNH

Without Drug

1. Role of complement system in complement-mediated PNH, Immune Thrombocytopenia Purpura (ITP) and Cold Agglutinin Disease (CAD);
2. Approaches to facilitating and expediting diagnosis of PNH and CAD;
3. Identification of biomarkers that leads to better characterization, management or correlation with outcomes in PNH, ITP and CAD;
4. Burden of disease (clinical, economic, and/or humanistic burden) – PNH and CAD;
5. Epidemiology studies (incl. registries) – PNH and CAD

Out of scope:

• Pediatric studies
• Studies exploring different dosing regimens as currently investigated
• Any study, which combines iptacopan with immunosuppressant and anti-C5 treatments
• Head-to-head comparisons
• Studies in other hematology diseases

** Strategic areas of interest for iptacopan (IgAN, C3G, aHUS, MN, LN), please also refer to the Cardiovascular, Renal & Metabolism section

• Translational research on determinants and mechanisms of response and/or resistance to JDQ443
• Combinations of JDQ443:
  • 1L or 2L combinations
  • 2L combination with chemotherapy
• JDQ443 in early-stage resectable NSCLC (neoadjuvant and/or adjuvant)
• Efficacy of 2L JDQ443 monotherapy vs. other 2L line combinations (e.g. chemo plus VEGFi)
• JDQ443 combinations in KRAS^G12C inhibitor-pretreated, refractory patients
• JDQ443 in other KRAS G12C mutated tumours (e.g. GI or GYN)

Out of scope:

• H2H interventional studies versus other KRAS G12C inhibitors
• Any study in overlap with ongoing Novartis-sponsored/supported studies

• Studies (other than prospective design) describing optimal timing and sequence of treatment in advanced or metastatic GEP-NET patients
• Studies of Lutathera in advanced or metastatic NET patients in combination with other anti-cancer treatments, including chemotherapy (also bolus 1L), immuno-oncology therapies, tyrosine kinase inhibitors (TKIs), PARP-inhibitors, CDK4/6 inhibitors, or other upcoming treatments (if supported by MoA rationale)  
• Retrospective studies describing long-term safety or health economic aspects 
• Studies on biomarkers to predict and prognosticate treatment in GEP-NET

• Studies on tumor types beyond prostate cancer
  • Solid tumors expressing PSMA and/or are PSMA PET avid
• Studies on 1/2L mCRPC and nm (localized, loco-regional) and mHSPC (De Novo and Relapse)
  • Chemo-unfit /frail patients population
  • Head to head vs chemotherapy in 1L mHSPC
  • Combination and sequencing strategies
    • with PSMA enhancers / sensitizers in mHSPC
    • to reduce and control AE management
    • with PARP inhibitor or Immunotherapeutic in mHSPC
  • Pattern of treatment sequencing to understand potential cross-resistance
  • Re-treatment strategies from localized, loco-regional or mHSPC to mCRPC
  • Long-term safety studies and/or post-progression evaluations
  • Burden of illness and quality of life studies
• Biomarker / translational studies
  • Predictive to efficacy and safety, as well as disease aggressiveness
  • Informative (PSMA-PET)
  • Resistance
  • Regulation of PSMA expression in different disease stages

Out of scope:

• Clinical studies in COVID-19

• HR+/HER2- studies in breast cancer patients: Exploring strategies to inform treatment sequencing across early to advanced breast cancer
• HR+/HER2- studies in breast cancer patients: Utilizing real world data and/or digital health technologies
• HR+/HER2- studies in breast cancer patients: Utilizing patient reported outcomes (PRO)
• Mechanistic studies looking at CDK4 and CDK6 function in cellular senescence, immunomodulation, and endocrine resistance

Out of scope:

• Any topic outside HR+/HER2- breast cancer

• Biomarker studies (e.g. TIM-3, Leukemic Stem Cell, etc.) 
• Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML): novel-novel combinations, optimal sequence
• Burden of Illness and Quality of Life studies in MDS and AML
• Pattern of treatment (incl. transplant), Real world (RW) agents treatment in MDS and AML

Out of scope:

• Solid tumors and other non-hematology indications 
• Pediatric studies
• Clinical studies in COVID-19