## [IIT Global Areas of Interest](/node/662876 "IIT Global Areas of Interest") Cardiovascular, Renal & Metabolism Atrasentan (Vanrafia) Without drug (IgAN, FSGS) - Diagnosis and classification - Additional ways to foster (earlier) diagnosis of IgAN and FSGS beyond biopsy - Validate outcome measures (endpoint validation, validation of definitions) including patient-related outcomes, treatment targets, partial remission / remission and relapse criteria in IgAN - Pathophysiology and biomarkers - Studies evaluating the mechanism of anemia with ERAs - Role of the endothelin system in IgAN and FSGS - Identification of approaches that lead to better characterization, management and/or correlation with outcomes in IgAN and FSGS (eg identification of biomarkers, genetic analysis, novel imaging technologies or biopsy-based studies) - Disease burden - Burden of disease (clinical, economic and/or humanistic burden) in IgAN and FSGS - Epidemiological studies - Prevalence, treatment patterns and RWE, sex/geography differences (incl. registries) in IgAN and FSGS - Disease characteristics and clinical outcomes in patients with skin of color, ethnic minorities, or populations so far underrepresented in clinical trials. Gender related differences. - Gaps in the optimal care and management of IgAN and FSGS patients - Impact of patient education programs - Patient perspective on disease, treatment options and QoL With Drug (IgAN, FSGS) - Mode of action - Mechanistic studies using atrasentan in IgAN and FSGS, including biomarkers, biopsy and novel imaging techniques (eg MRI) - Studies evaluating non-hemodynamic effects of atrasentan in IgAN and FSGS - Studies evaluating the effects of atrasentan on pain in IgAN and FSGS - Treatment optimization - Studies evaluating novel implementation protocols in IgAN - Evaluation/biomarker analysis of subgroups of patients included in the overall study populations in the atrasentan CDP - Studies in IgAN utilising SGLT2i and/or other novel treatments in combination with atrasentan - Studies evaluating the early use of atrasentan initiated alongside RASi +/- SGLT2i, or atrasentan alone in IgAN - Expanded populations in IgAN - Studies of atrasentan in patients with IgAN with less severe disease (eg. lower proteinuria >0.5g/d) - Studies of atrasentan in patients with IgAN in more severe disease (eg. CKD stage 4, eGFR 15-29 ml/min/1.73m2) - Studies evaluating atrasentan in transplant patients with recurrent IgAN Out of scope (IgAN, FSGS) - Pediatric studies in IgAN and FSGS - Studies exploring different dosing regimens to those currently being evaluated in current clinical development programs for IgAN and FSGS - Head-to-head comparisons with other treatments for IgAN and FSGS - Studies in IgAN including patients with eGFR <15 ml/min/1.73m2 (CKD stage 5) Inclisiran (Leqvio®) 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 Iptacopan (Fabhalta) **Without drug** (IgAN, C3G, IC-MPGN, aHUS, LN, AAV, FSGS) - Additional ways to foster (earlier) diagnosis of glomerulopathies, beyond biopsy including diagnostic research leveraging use of artificial intelligence in IgAN, C3G, IC-MPGN, aHUS, LN, AAV, FSGS - Identification of approaches that lead to better characterization, management and/or correlation with outcomes in IgAN,C3G, IC-MPGN, aHUS, LN, AAV, FSGS eg identification of biomarkers, genetic analysis or biopsy-based studies - Burden of disease (clinical, economic and/or humanistic burden in IgAN, C3G, IC-MPGN, aHUS, LN, AAV - Epidemiological studies - Prevalence, treatment patterns and RWE, sex/geography differences (incl. registries) in IgAN, C3G, IC-MPGN, aHUS, LN, AAV - Role of the complement system in complement mediated kidney diseases **With drug** (IgAN, C3G, IC-MPGN) - Mechanistic studies using iptacopan including biopsy in IgAN C3G, IC-MPGN - Subgroups of patients that are included in the overall study population in indications in the iptacopan development program in IgAN, C3G, IC-MPGN - Studies evaluating iptacopan in transplant patients with recurrent IgAN or recurrent C3G - Evaluation/biomarker analysis for subgroups of patients included in the overall study population in IgAN, C3G and IC-MPGN - Preclinical research studies in IgAN, C3G and IC-MPGN - Studies investigating combination, sequencing of targeted treatments in IgAN **Out of scope** (with drug) - Pediatric studies - Studies exploring different dosing regimens to those currently being evaluated in current clinical development program - Head-to-head comparisons - Studies including patients with GFR <20 ml/min/1.73m2 Pelacarsen 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 Sacubitril/Valsartan (Entresto) **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 Zigakibart Without drug - Diagnosis and classification - Additional ways to foster (earlier) diagnosis of glomerulopathies (IgAN), beyond biopsy and its impact on outcomes - Validate outcome measures (endpoint validation, validation of definitions) including patient-related outcomes, treatment targets, partial remission / remission and relapse criteria in IgAN - Pathophysiology and biomarkers - Role of APRIL system in IgAN, Gd-IgA1 and autoantibodies in IgAN Including the subtype characterisation - Identification of approaches that lead to better characterization, management and/or correlation with outcomes in IgAN eg identification of biomarkers, genetic analysis or biopsy-based studies - Predictive models for APRIL inhibition responses across disease phenotypes/genotypes - Disease burden - Burden of disease (clinical, economic and/or humanistic burden) in IgAN - Epidemiological studies - Prevalence, treatment patterns and RWE, sex/geography differences (incl. registries) in IgAN Disease characteristics and clinical outcomes in patients with different racial and ethnic backgrounds, or populations so far underrepresented in clinical trials. Gender related differences. - Gaps in the optimal care and management of IgAN patients - Impact of patient education programs - Patient perspective on disease, treatment options and QoL With drug - Pre-clinical - Mechanistic studies in IgAN - Preclinical evaluations aimed at demonstrating MoA-pathway activity and/or differentiation - Clinical - Evaluation of zigakibart in expanded IgAN patient populations: Transplant patients with recurrent IgAN, IgA vasculitis with nephritis patients, Low proteinuria (0.5 – 1 g/d) Out of scope - Pediatric studies - Studies exploring different dosing regimens to those currently being evaluated in current clinical development program - Head-to-head comparisons - The value of tight control (treat to target) versus conventional management strategies in IgAN - Risk prediction models for which patients may develop hypogammaglobulinemia - Preclinical (comparison) studies (vs APRIL/BAFF or APRIL/BLyS) and other IgAN treatments - Duration and withdrawal of therapy / impact of treatment breaks - Combination evidence - Studies including patients with GFR <20 ml/min/1.73m2 - Vaccine titer studies or b-cell subset characterization Gene Therapies Zolgensma IV **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 OAV101 IT **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 Global Health Adakveo, Ryverna (Crizanlizumab, SEG101) - 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 HU, HU-FCT (Hydroxyurea) - 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 Ganaplacide/Lumefantrine - Efficacy against non-falciparum malaria (blood stage) - Efficacy when used in combination with tafenoquine - Special patient groups- >65 years, >70kg - Population impact of use in low transmission settings **Out of scope** - Use in traveler prophylaxis Special populations - Treatment of malaria in pregnancy - Treatment Studies in malnourished individuals Immunology Ianalumab **Clinical data, outcomes & RWE (with drug):** - RWE studies on clinical effectiveness and safety of Ianalumab in Sjogren’s disease - Clinical outcomes with Ianalumab across different manifestations and domains of Sjogren’s disease - Clinical outcomes with Ianalumab by gender, race, skin of colors, ethnic minorities and access to health care systems in Sjogren’s disease - Research investigating clinical impact of Ianalumab on Sjogren’s disease systemic and organ complications such as neuropathies, interstitial lung disease, renal disease, vasculitis, etc **Implementation Science/HCS research.** - Population-based epidemiology studies investigating the impact of autoimmune diseases on patients; comorbidities; studies of real-world treatment patterns (incl. e.g. overuse of corticosteroids, delays in systemic therapies initiation or Treat-to-Target approaches, etc) - Identification of predictors and patient characteristics associated with rapid progression or poor outcomes in autoimmune diseases - Impact of early intervention and treat-to-target on patient outcomes - Impact of systemic treatments on prevention or reduction of long-term comorbidities and complications in autoimmune diseases - Research utilizing novel imaging modalities for early diagnosis and monitoring of treatment responses - Research evaluating treatment impact on patients' symptoms, such as fatigue, pain, mental and sexual well-being, - Research evaluating patients perspectives in terms of unmet needs, current treatment satisfaction, disease management, treatment adherence, PROs, and shared decision-making across autoimmune diseases **Exploratory/ mechanistic studies:** - Role of B-cells in the pathogenesis of Sjogren’s disease, Systemic Lupus Erythematosus, Systemic Sclerosis, and other autoimmune conditions - Impact of BAFF blockade on Sjogren’s disease, Systemic Lupus Erythematosus, Systemic Sclerosis, and other autoimmune conditions - Identification and validation of new clinical outcome measures or PROs for clinical trials or real-world practice use across autoimmune diseases - Studies employing digital or AI based technologies e.g. in silico models, machine learning techniques, telemedicine etc. to support clinical diagnosis, clinical management, prediction of disease trajectories, prediction of treatment responses **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 - Pediatric studies - Studies exploring different dosing regimens as currently investigated Secukinumab (Cosentyx) ### 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 Remibrutinib in Chronic Urticaria **Disease:** Chronic Urticaria – CSU (chronic spontaneous 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 understanding acute -> chronic urticaria transition, biomarkers, identify patients at risk 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, AI / 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 - Remi in acute or early chronic urticaria / prevention of chronic urticaria - Remi in other potentially BTK mediated conditions like allergic conditions, urticaria vasculitis etc. Mechanistic Studies: - Clinical Mechanistic studies assessing the effects of remibrutinib on mast cells, basophils, B-cells - Clinical mechanistic studies investigating the disease modifying potential of remibrutinib - Clinical mechanistic studies investigating the impact of remibrutinib on peripheral neuroinflammation **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 bid developed in the CSU phase 3 clinical program Neuroscience Ofatumumab (Kesimpta) and siponimod (Mayzent) - Multiple sclerosis (MS) - 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 Remibrutinib in RMS **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* Oncology Asciminib (Scemblix) 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 Iptacopan (LNP023) **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 \[177Lu\] Lu-DOTA-TATE (Lutathera®) - 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 \[177Lu\] Lu-PSMA-617 (Pluvicto®) **Indication:** Prostate Cancer - mHSPC, mCRPC - **Sequencing RWE** - Sequential use of different radioligand therapies (alpha- or beta-emitter); Treatment optimization - HRLPC, BCR - **Combinations** - Efficacy and safety of 177Lu-PSMA-617 combinations to overcome resistance and to improve efficacy outcomes - mHSPC (OMPC) - **Low volume disease** - Efficacy and safety of 177Lu-PSMA-617 in low volume disease - mHSPC - **Alternative dosing** - Adaptive and alternative treatment regimens with 177Lu-PSMA-617 monotherapy or in combinations - HRLPC, BCR - **Treatment effect in earlier stages** - Efficacy and safety of radioligand therapy (alpha- or beta-emitter) - mHSPC, mCRPC - **Subpopulations** - 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. - All disease stages - **Long-term safety** - Retrospective analysis to predict long-term safety events - HRLPC, mHSPC, 1L-2L mCRPC - **Translational Research** - Treatment effect on disease biology - HRLPC, mHSPC, 1L-2L mCRPC - **Imaging** - Understanding PSMA expression in different stages of prostate cancer **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 Ribociclib (Kisqali) - 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 - [Print](/node/662876/printable/print) - [PDF](/node/662876/printable/pdf)