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

Areas of interest by product

• Demonstrating or validating care needs for SMA populations post OAV101 Treatment-safety related items
• Expansion of treatment with OAV101 to patient populations not included in clinical trials (e.g. older/heavier, 4 copies, switch therapy, ambulatory)
• Value of OAV101: Cost of care, Quality of life, and Caregiver Burden-Cost effectiveness
• Methods/Processes to assess the efficacy and durability of OAV101 (e.g. bulbar function)
• Biomarkers for efficacy

Out of scope

• Clinical Trials involving OAV 101 re-dosing
• Study of OAV101 alternative doses/maximum dose
• Head-to-head comparison with other therapies and combination with other MDT
• Basic Science research that request use of OAV101

Areas of interest by product 

• Interventional Studies of OAV IT in patients not included in clinical trials (e.g. ambulant SMA patients, severe scoliosis)
• Non-interventional Studies of OAV IT assessing sleep, bulbar function, scoliosis and respiratory function, head steadiness and independence.
• Studies on biomarkers assessing clinical response to OAV IT

Out of scope

• Clinical Trials involving OAV 101 re-dosing
• Study of OAV101 alternative doses/maximum dose
• Head-to-head comparison with other therapies and combination with other MDT
• Basic Science research that request use of OAV101

• 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:

• Early intervention with Sec and impact on disease progression (including imaging techniques, such as ultrasound)
• Clinical outcomes in subpopulations (e.g. disease phenotypes, Black / African American, super-responders,..)
• Integrating surgical procedures with the administration of Secukinumab for the treatment of HS. (effectiveness and Safety)
• HS comorbidities (Mental Health, Obesity, CV)
• Effects of lifestyle intervention on HS treatment with Secukinumab

Implementation Science / HCS research:

• Quality of care, cost-effectiveness, resource utilization, and guidelines implementation
• AI/ML 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 , and in specific aspects of the disease e.g., Fistula/tunnel development"
• 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

Indications: GCA and PMR

Pathophysiology and biomarkers

• Biomarkers to monitor subclinical disease activity, predict prognosis and treatment outcomes
• Effects of a mechanism-based approach to therapy
• Pathways involved in refractory/flaring GCA
• Biomarkers to predict drug/GC toxicity

Diagnosis and classification

• Standardization of clinical trial endpoints
• Validation of the definition of remission, response, relapse and disease subtypes of importance
• Use of the different imaging techniques for vascular activity, damage assessment and follow-up

Treatment and treatment outcomes

• Implementation Science/HCS research: quality of care, cost-effectiveness, resource utilization, and guidelines implementation
• Disease characteristics and clinical outcomes in patients with skin of color, ethnic minorities, or populations so far underrepresented in clinical trials. Gender related differences
• Assessment of prognosis by demographic, clinical and histological data
• Predictors of response, remission or relapse
• Validation of patient-reported outcomes
• Predictive models for IL-17A inhibition responses across disease phenotypes
• Effect of secukinumab on the development of future vascular complications

Out of scope

• Effect of secukinumab on the development of future vascular complications
• Combination studies of secukinumab with other biologic agents
• The role of ultrasound for guiding temporal artery biopsy, specific treatment of organ complications

• 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

Disease: Relapsing Multiple Sclerosis

Product: Remibrutinib

Areas of interest by product

Follow the science 

• Impact on the immune system in and outside the CNS – clinical and preclinical studies
• Direct CNS effects (i.e. microglial activity, synaptogenesis, neuronal function) and correlation with clinical outcomes beyond relapses (e.g. PIRA, disability improvement) and with some patient outcomes (e.g. cognition, fatigue) 
• Impact on chronic inflammation and correlation with linked clinical and paraclinical outcomes (disability measures, such as EDSS/MSFC, imaging measures, fluid biomarkers)

Safety related:

• BTKi – vaccine response
• Pregnancy registries (in line with initiatives already in place) 

Identify unmet need under current DMTs:

• Patient preference
• Tolerability and safety concerns (what, when, to whom – patient profile-)
• Effectiveness gaps under HET (what, when, to whom – patient profile -)

Out of scope

Progressive phenotypes of MS (naSPMS or PPMS); hepatotoxicity

Disease: Chronic Urticaria – CSU (chronic spontaeous urticaria) and CindU (chronic inducible urticaria)

Product: Remibrutinib

Areas of interest by product

Disease Related Research:

• Population-based epidemiology studies; studies investigating the impact of urticaria on patients; comorbidities; studies of real-world treatment patterns (incl. e.g. overuse of corticosteroids, impact on sleep)
• Studies investigating biomarkers in urticaria aiming to identify predictors of chronic urticaria in patients with acute episodes, biomarkers predictive of treatment response, biomarkers correlating with the time-course of CSU, biomarkers of permanent remission or relapse, including with remibrutinib
• Studies employing digital technology e.g. in silico models, machine learning techniques, telemedicine etc. to predict disease trajectories, treatment response, disease modification, etc. 
• Studies investigating innovative tools to support urticaria management, e.g. digital applications, sleep related
• Long-term CSU observational registries and secondary use of data
• Studies investigating patient preference

Clinical Studies:

• Studies with remibrutinib in CindU
• Studies with remibrutinib focusing on angioedema only

Mechanistic Studies

• Mechanistic studies assessing the effects of remibrutinib on mast cells, basophils, B-cells in vitro or ex vivo
• Studies investigating the disease modifying potential of remibrutinib, including pre-clinical in vitro or ex vivo research

Out of scope

• Head-to-head comparisons of remibrutinib with other active treatments
• Studies investigating remibrutinib in combination with biologics
• Alternative dosing regimens to 25 mg b.i.d developed in the CSU phase 3 clinical program

Studies in adult patients Chronic Myeloid Leukemia in Chronic Phase (CML-CP) in early treatment lines, investigating:

• Long-term safety and tolerability
• Clinical efficacy and safety in real-world setting
• Treatment sequencing
• Patient-reported outcomes (PROs) and Quality of Life (QoL)
• Patients with CML-CP and additional T315I mutation
• Response to asciminib in patients with pre-existing mutations other than T315I or treatment approaches in patients with emerging mutations under asciminib, including compound mutations

Studies exploring additional high-need patient populations other than CML-CP:

• Patients with Philadelphia-positive acute lymphoblastic leukemia (Ph+ALL)
• Exploratory high risk CML populations such as patients with additional genomic alterations
• CML-AP/BC

Studies in Treatment Free Remission (TFR)

• Studies aiming to improve deep molecular responses, increase the eligibility for TFR attempts or reduce the risk of relapse after treatment discontinuation
• Combination approaches of asciminib with non-ATP-TKI compounds

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

• 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

Indication: Prostate Cancer

• Sequential use of different radioligand therapies (alpha- or beta-emitter)
• Efficacy and safety of ¹⁷⁷Lu-PSMA-617 combinations to overcome resistance and to improve efficacy outcomes / Efficacy and safety of ²²⁵Ac-PSMA-617/R2 combinations (any disease stage)
• Efficacy and safety of ¹⁷⁷Lu-PSMA-617 in low volume disease (mHSPC, OMPC)
• Adaptive and alternative treatment regimens with ¹⁷⁷Lu-PSMA-617 monotherapy or in combinations (mHSPC, mCRPRC)
• Efficacy and safety of radioligand therapy (alpha- or beta-emitter) in neoadjuvant setting (HRLPC)
• Impact of ¹⁷⁷Lu-PSMA-617 efficacy and safety in patient populations with sub-optimal outcomes, including patients distinct mutations (e.g., PTEN-loss, AKT, DDR), patients CNS mets, liver mets etc. (mHSPC, mCRPRC)
• Retrospective analysis to predict long-term safety events (mCRPC)
• Treatment effect on disease biology (HRLPC, mHSPC, 1L-2L mCRPC)
• Understanding PSMA expression in different stages of prostate cancer (HRLPC, mHSPC, 1L-2L mCRPC)

Beyond GU

• Brain Metastasis (secondary malignancies)
• Ovarian Ca
• NSCLC
• GBM: microenvironment and translational research (MoA deeper understanding)
• GBM: Other mode of administration in GBM (not IV)
• Hepatocellular Carcinoma
• High grade gliomas
• Others PSMA-expressing/PET-avid tumors
• Imaging studies
• Pediatric indications

• HR+/HER2- studies in breast cancer
  • Exploring data on CDK4/6 inhibitor rechallenge
  • Exploring ribociclib with novel/emergent compounds
  • Utilizing real world data and/or digital health technologies
  • Utilizing patient reported outcomes (PRO)

Out of scope:

• Any area outside HR+/HER2- breast cancer
• Any study in overlap with ongoing Novartis-sponsored/supported studies

Disease: Prostrate Cancer

Product: RAG) ²²⁵Ac-PSMA-617 

Areas of interest by product:

• Combinations (taxane based chemotherapy, PARPi with or without ARPI, Combination in DDR+ vs DDRwt patients, others)
• Retreatment or rechallenge or extension of treatment
• Safety: protecting salivary gland or preventing xerostomia.
• Tandem dosing schedule between ¹¹⁷Lu-PSMA-617 and ²²⁵Ac-PSMA-617
• Head-to-Head ²²⁵Ac-PSMA-617 vs PARPi +/- ARPI in BRCA2mut vs ITT
• Head-to-Head ²²⁵Ac-PSMA-617 vs ¹¹⁷Lu-PSMA-617
• Impact of prior ARPI exposure on ²²⁵Ac-PSMA-617 combination with ARPI (ARPI resistant vs sensitive)
• Impact of prior treatment on Dosimetry and Adaptive dosing

For consideration:

• Concordance rate of PSMA positivity/negativity between different PSMA PET imaging agents
• Define new categories of volume of disease/burden of disease in mHSPC based on PSMA – PET based imaging
• Metachronous Oligometastatic CRPC detected by PSMA-PET but being non-metastatic by conventional imaging
• Prospective study to correlate PET parameters with treatment outcomes.
• Biomarker correlation of acquired resistance and response to ²²⁵Ac-PSMA-617
• Evaluation Method of Intra-tumoral Heterogeneity by PET Imaging and Molecular expression analysis

Out of scope:

• Outside prostrate cancer.