Lymphoid
  • Essential factors for selecting patients for CAR-T therapy to improve safety and/or response
  • Essential factors for sequencing CAR-T therapy with other therapies and determining outcomes
  • Novel combinations of therapies with CAR-T to improve response
  • Study outcomes of CAR-T administered at various sites (e.g. in-patient, out-patient, community hospital, community practice)
Myeloid/Malignant Hematology

CML in Earlier Lines (1L & 2L)

  • Sequencing of TKIs, clinical efficacy, and safety in real-world setting
  • Patient – reported outcomes (PROs) and Quality of life issues with current CML therapies
  • TFR and safety biomarkers
  • Long-term safety and tolerability
  • 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
  • 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

CML- BC and Ph+ ALL

  • Efficacy and safety of Asciminib in selected ALL settings (PH+, Ph-Like)
  • Exploratory high risk CML populations such as patients with additional genomic alterations
  • TKI- based combinations addressing high unmet need populations (CML-AP/BC)
Non-Malignant Hematology

With Drug:

  • Mechanistic studies in PNH
  • Studies evaluating complement Factors associated with or predictive of treatment outcome in PNH
  • Studies exploring Factor B inhibition in other disease states where complement plays a role

Without drug

  • Role of complement system in complement-mediated PNH
  • Approaches to facilitating and expediting diagnosis of PNH;
  • Identification of biomarkers that leads to better characterization, management or correlation with outcomes in PNH
  • Epidemiology studies (incl. registries) – PNH
Breast and Gynecological Cancers

Breast Cancer

  • Ribociclib in early breast cancer and metastatic breast cancer (limited scope)

Out of Scope

  • All other studies and tumor types
RLT
  • Investigating alternative dosing regimens (cycles, frequency) with 177Lu-PSMA-617 in mCRPC
  • Radioligand therapy in neoadjuvant setting for localized prostate cancer
  • Use of 177Lu-PSMA-617 in adjuvant setting in combination with EBRT + ADT +/- abiraterone in patients with localized prostate cancer post prostatectomy with N1M0 on PSMA PET
  • Use of 177Lu-PSMA-617 post definitive therapy for localized prostate cancer with biochemical recurrence and PSMA-PET M0 disease
  • Use of PSMA-targeted PET imaging agents in prostate cancer (e.g., patient selection, treatment assessment)
  • Use of 177Lu-PSMA-617 in combination with other agents in mHSPC or mCRPC
  • Treatments up-regulating PSMA expression in prostate cancer 
  • Use of >6 cycles of 177Lu-PSMA-617 in patients with mHSPC or mCRPC
  • Use of 177Lu-PSMA-617 in prostate cancer patients with distinct mutations (e.g., PTEN-loss, AKT, DDR)
  • Use of 177Lu-PSMA-617 in patients with low or no PSMA expression in mCRPC
  • Safety and efficacy of 177Lu-PSMA-617 treatment in solid tumors other than prostate cancer
  • Real-world evidence in prostate cancer for 177 Lu-PSMA-617
  • Health disparities in advanced prostate cancer

GEP & Bronchopulmonary NET

  • Re-treatment/Re-challenge with Lutathera (after initial 4 cycles) (HEOR)
  • Combinations with other agents with proven efficacy in GEP-NETs
  • Sequencing studies (HEOR)
  • Long-term safety (HEOR)
  • Bronchopulmonary NET
  • Efficacy/Safety of Lutathera in specific patient subgroups

Other SSTR+ Tumors

  • Role of Lutathera in the management of patients with other SSTR-positive tumors

NETSPOT for Imaging

  • Role of Netspot in other non-GEP NET SSTR2+ tumors
CVM

Non-drug IITs

Epidemiology associated with elevated Lp(a)

  • Patient characterization, identification, and genetic risk across sub-groups
  • Association & impact on different types of CVD (ischemic stroke, PAD), polyvascular disease, and other CV-related diseases (e.g. kidney disease, diabetes)

Distinct and unique pathophysiology of Lp(a) related to CVD

  • 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
  • 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 risk evaluation
  • 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
  • Non-cardiovascular related diseases
  • Mechanistic studies in post ACS/ symptomatic PAD
  • Real world utilization & implementation of inclisiran post ACS/symptomatic PAD
  • Inclisiran in under-represented patient population (eg, women, pts with treatment disparities in LDL-C lowering, patient types who tend to have worst outcomes)
  • Characterization of the effect of inclisiran on lipoprotein metabolism: particle synthesis/secretion, including Lp(a)
  • Population modeling of diverse populations in various health care settings (including HCRU,...)
  • Effects of inclisiran in high-risk patient population (e.g., diabetes,...)
  • Differentiating attributes of inclisiran versus other LLTs (e.g., safety, drug interaction, adherence,..)
  • Preclinical studies evaluating non-LDL-C-lowering effects
Neuroscience

Multiple Sclerosis

  • Novel short-term efficacy outcomes and long-term efficacy outcomes in treatment naive RMS population
  • Studies collecting data on patients’ treatment experience and preference
  • Studies looking at differentiating ofatumumab from other DMTs
  • RWE (prospective, retrospective, primary, secondary data collection, registry) studies on MS patients’ / subgroup of patients/ special populations and/ or outcomes
  • Focus on prognosis and diligent monitoring of disease activity in RMS patients (incl. data and digital):
    • Markers for disease prognosis, disease activity or disease monitoring
    • New or improved quantitative outcome measures in MS, incl. next-generation technology and patient assessment technologies
    • Integration of markers/outcome measures to establish disease stability or disease control, disease progression

Multiple Sclerosis

  • The impact on CNS – BBB transmigration, microglial impact (activation)
  • The impact on biology of progression – PET imaging impact, cognition, fatigue, depression outcomes
  • The impact on imaging – SELs, PRLs, cortical lesions impact
  • The role for remibrutinib in sequencing of treatments
  • The proteome profiling effects of remibrutinib

Myasthenia Gravis:

  • Impact of remibrutinib on gMG
Gene Therapy

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