Investigator-Initiated Studies

2025 IIS Strategic Priorities will be posted soon.

DURYSTA® (bimatoprost intracameral implant); OZURDEX® (dexamethasone intravitreal implant); REFRESH® Portfolio of Artificial Tears; XEN® (gel stent)

Durysta Priorities 

• Real World Evidence (RWE) on effectiveness and safety of Durysta
• Risk factors leading to corneal adverse and methods to decrease such events with Durysta (e.g., anatomic profile, placement, etc.)
• Identification of predictors of long-term Intraocular Pressure (IOP) lowering effect with Durysta
• Impact on topical AEs profile (including Ocular Surface Disease (OSD)) of switch from drops to Durysta
• Impact of Durysta on adjunctive medication use beyond PGA drops
• Identification of predictors of response to Durysta
• Understand how to best transition number and use of glaucoma drops to Durysta
• Effectiveness and transition to subsequent treatments after Durysta
• Effectiveness of Durysta after Selective Laser Trabeculoplasty (SLT), including non-responders to SLT
• Effectiveness of Durysta before and/or after prior interventions (e.g., Glaucoma Surgeries)
• QoL, patient and ECP satisfaction surveys
• Patient Selection and Clinical Outcomes including diverse patient population groups

Refresh Artificial Tear Portfolio

• Quantification of the duration of relief of symptoms with Refresh artificial tears use
• Effectiveness of combination of Refresh products
• Real world outcomes in aqueous deficient, mixed, and evaporative dry eye disease (i.e. meibomian gland dysfunction) patient subtypes with the use of Refresh artificial tears
• Real world outcomes of patients with dry eye symptoms due to digital device use with lipid-containing Refresh artificial tears
• Real world outcomes related to preservative-free Refresh artificial tears

XEN 45/63

• Long term and improvement of patient outcomes via novel surgical techniques 
• Data after failure of other MIGS procedures 
• Data comparing with other bleb forming procedures (eg Trabeculectomy, Preserflo) or with Tube shunts 
• Trabeculectomy or repeat XEN after previously failed XEN surgery. 
• Rate of Progression in Glaucoma and Visual Field forecasting algorithm 
• Study when implanted earlier in treatment paradigm (e.g. after 2 drops) 
• Impact of COVID 19 on Glaucoma progression and IOP control 
• Data in Angle closure glaucoma 

Ozurdex

• Biomarkers in DME and RVO: Identifying and validating predictors of inflammation or Ozurdex therapeutic benefit (with or without AI). 
• Use of Ozurdex in phakic patients with DME 
  • First and second line use of Ozurdex 
• Sequential use of Ozurdex and Anti-Vascular Endothelial Growth Factor (a-VEGF) (e.g. Ozurdex as 1^st line). 
• Safety profile of Ozurdex in relationship to shorter or extended treatment intervals"  
• Efficacy and safety of Ozurdex across indications (e.g. DME, RVO, Uveitis) in 1^st line. 
• AI’s role in diagnosing, classifying, monitoring, and/or treating DME, RVO, and/or Uveitis. 
• Limitations of standards of care in DME  
  • e.g. focusing on functional and anatomical outcomes switching between different a-VEGF agents 
  • e.g. identifying patient sub-groups/clinical characteristics where limitations in aVEGF standard of care are more pronounced. 
• RWE in patients with suboptimal response to anti-VEGF therapy in DME/RVO. 
• Combination use of Ozurdex and anti VEGF. 

IMMUNOLOGY – GENERAL:
Adalimumab, Risankizumab & Upadacitinib

Pre-clinical and clinical proposals for diseases with high unmet need/burden, few to no suitable approved therapeutic options and strong rationale for target engagement in causality of disease may be submitted for consideration.

Clinical interventional studies will only be considered for compounds that have regulatory approval for use in at least one indication in that country.

For preclinical studies utilizing upadacitinib and risankizumab outside of approved indications only ex-vivo, in-vitro or in-silico studies will be considered.

Studies documenting treatment patterns and outcomes in under-represented populations.

Studies utilizing adalimumab will be considered only for investigating conditions without suitable alternative treatment options, where sufficient evidence exists to support a hypothesis for life-altering outcomes.

IMMUNOLOGY – Gastroenterology
Risankizumab & Upadacitinib

1. Prevention and treatment of different subsets of IBD, including but not limited to Acute Severe Ulcerative Colitis, pouchitis, ileal disease, fistulizing and stricturing disease, and post-operative recurrence in Crohn’s disease
2. Further the understanding of mechanisms of diseases in IBD, predictive and prognostic factors of disease severity and progression, response to therapy
3. Optimal monitoring of disease, including symptoms and inflammation in IBD, non-invasive mucosal healing monitoring tools (e.g., ultrasound, MRI) and proposals of validation of monitoring tools (e.g., AI of endoscopic scoring)
4. Broadening and exploration of drug mechanisms, including JAK and IL-23, evaluation of biomarkers geared towards identifying different drivers of response, targeted therapy approaches and developing and testing patient stratification hypotheses.
5. Broadening the knowledge on early and sustained control of inflammation in IBD to prevent disease worsening, adverse disease outcomes or optimize long term outcomes
6. Optimizing strategies to mitigate the holistic burden of IBD (total cost of illness, quality of life, co-morbidities, unmet needs) with special focus on underserved populations
7. Understanding of dosing and sequence strategies to optimize disease control

IMMUNOLOGY – RHEUMATOLOGY:
Rinvoq (Upadacitinib-UPA) and Skyrizi (Risankizumab-RISA)
  

 Rheumatology / Overarching 

1. Use of JAKi and/or IL-23i in inflammatory disease related to but not limited to adherence, persistence, treatment patterns/strategies and comparative effectiveness (observational or interventional)
2. Biomarkers predictive of response to treatment and/or prognostic of disease progression
3. Outcomes associated with patients achieving and/or maintaining stringent disease control 
4. Addressing elements of standard of care, specifically, early and appropriate diagnosis, treatment targets, healthcare disparities, workplace shortages and understanding the barriers to implementing goal directed therapy
5. Effectiveness of switching to JAKi or IL-23i, versus cycling through the same previous MOA (e.g., in patients with lack of response/intolerance to TNFi switching to a JAKi vs another TNFi)
   

Compound: Rinvoq (Upadacitinib-UPA)

Indication: UPA in GCA/TAK

1. Understanding the burden of GC treatment and characterize the unmet need in GCA/TAK Role of the JAK/STAT pathway in the pathogenesis of large vessel vasculitis
2. Characterizing patients not responding to current therapies (e.g., IL-6)
3. Impact of therapies in vascular tissue damage (imaging / biopsy studies)

Indication: UPA in SLE

1. Understanding pathogenic mechanism involving JAK/STAT pathway in Cutaneous lupus Erythematosus (CLE), Lupus Nephritis (LN) and Neuropsychiatric SLE (NPSLE)
2. Understanding what Lupus Arthritis is (e.g., MRI and/or ultrasound imaging findings) and the role of JAK/STAT pathway. Characterization of joint involvement in SLE, including the role of JAK/STAT, disease progression and disease monitoring (MRI/Ultrasound etc.)
3. Understanding patient-reported neuropsychiatric symptoms (e.g., brain fog, cognitive dysfunction, fatigue, etc.) in SLE and the role of JAK/STAT pathway
4. Identification of SLE endotypes, patients susceptible to flare, and gene signatures, soluble factors, or other biomarkers that can predict early response to treatment and/or characterize patients who will not respond to current treatment modalities

Indication: UPA in axSpA

1. Real world impact of upadacitinib on core axSpA domains with a focus on stringent disease activity, pain, functioning, and structural damage
2. Impact of upadacitinib on extra-musculoskeletal manifestations across the axSpA spectrum
3. Use of imaging modalities to assess the effectiveness of upadacitinib across the axSpA spectrum

Indication: UPA in PsA

1. Real world impact of upadacitinib treatment on PsA domains (effectiveness including patient reported outcomes, safety and tolerability)
2. Impact of upadacitinib treatment on imaging confirmed axial PsA
3. Impact of upadacitinib on extra-musculoskeletal manifestations in PsA

Compound: Skyrizi (Risankizumab-RISA)

Indication: Risa in PsA

1. Impact of risankizumab treatment on PsA domains and related conditions with a focus on musculoskeletal symptoms, axial manifestations, and structural joint damage
2. Adherence and persistence with risankizumab in PsA
3. Understanding novel treatment strategies involving risankizumab

IMMUNOLOGY – DERMATOLOGY:
Risankizumab (SKYRIZI), Upadacitinib (RINVOQ)

Overarching
Indications: PsO, PsA, AD, HS, Vitiligo, AA

Priority consideration will be given to applications proposing to investigate the following areas of interest:

1. Impact of treat-to-target strategies, treatment goals and initiatives to advance quality of care in managing disease
2. Understanding treatment patterns, including dosing, sequencing, and compliance, including persistence, adherence, adverse events, and outcomes in diverse populations including patients with skin of color
3. Burden of disease, including co-morbidities, psychosocial impact (stigma, mental health), access to care, and health care resource utilization (HCRU), as well as the cumulative long-term impact of disease across varying patient types (age, extent of disease, skin phototype)
4. Disease prevalence and natural course of disease, including progression
5. Disease pathogenesis
   
   
   Indication: Psoriatic Disease (PsO, PsA)
   Compound: Risankizumab (Skyrizi)
   
   
6. Evaluation of the impact of Psoriatic Disease on the Cumulative Life Course Impairment (CLCI)
7. Identification of predictors of early and durable response with Risankizumab
8. Evaluate the impact of Risankizumab in in specific understudied sub-populations and on Psoriatic-associated comorbidities (i.e., PsA, metabolic syndrome)
9. Real-world effectiveness and treatment patterns of Risankizumab in Psoriatic Arthritis
   
   
   Indication: Atopic Dermatitis (AD)
   Compound: Upadacitinib (Rinvoq)
   
   
10. Effectiveness and safety of Upadacitinib in AD, in specific understudied sub-populations
11. Understanding biomarkers of systemic inflammatory response in Atopic Dermatitis and how these may impact therapeutic response.
12. Understanding the importance, implementation and/or validation of optimal disease control (e.g., minimal disease activity, EASI 90, NRS 0/1) and its impact on patients
13. Impact of Upadacitinib on AD co-morbidities
    
    
    Indication: Hidradenitis Suppurativa (HS)
    
    
14. Practical diagnostic, assessment, monitoring and imaging tools that aid in disease classification, assessment of disease severity, activity progression, flares, response to treatment (including higher level efficacy)
15. Patient types/segments, including the role of specific cytokine pathways (IL-1, JAK), as well as their needs with respect to disease management
16. Impact of early referral, diagnosis and treatment in HS including impact on progression, and the consequences of underdiagnosed / undertreated disease
    
    
    Indication: Vitiligo
    
    
17. Disease scoring and assessment measures, including QoL measures and characterization of disease severity beyond the extent of lesions (e.g., patient impact)
    
    
    Indication: Alopecia Areata (AA)
    
    
18. Disease scoring and assessment measures, including QoL measures, and quality of hair growth
    
    
    Other Inflammatory Skin Diseases
    
    
19. Research to understand the pathogenesis, disease course, burden (including co-morbidities, CLCI), disease assessments and, treatment goals and patterns of other inflammatory skin diseases
20. Efficacy/effectiveness and safety of approved AbbVie assets in other inflammatory skin diseases

NEUROSCIENCE – MIGRAINE: 
OnabotulinumtoxinA/BoNT/A (BOTOX®), ubrogepant (Ubrelvy), atogepant (Qulipta/Aquipta)  

Things to consider:

• Studies will only be considered for compounds that have received regulatory approval for at least one indication in the country of interest.

Proposals for the topics listed below will NOT be considered:

• OnabotulinumtoxinA vs placebo, vs other toxins, vs mAbs (monoclonal antibodies)
• Ubrogepant vs placebo, vs other acute treatments for migraine
• Atogepant vs placebo, vs other preventive treatments for migraine
• Double-blind, placebo controlled pediatric studies
• Placebo controlled studies for treatment during premonitory/prodrome phase

Priority will be given to proposals investigating the following areas:

Migraine Disease State

• Characterize and describe disease state across migraine
• Impact of disease management on disease state including acute and preventive treatment patterns
• Data describing the burden of menstrual migraine (defined as Pure Menstrual Migraine AND Menstrually-Related Migraine) including how patients treat or prevent these attacks
• Impact of early intervention (e.g. first line of preventive treatment or early post-migraine onset) with migraine-specific treatment on migraine disease progression
• Impact of triptan unsuitability on clinical and economic outcomes

OnabotulinumtoxinA/BoNT/A (BOTOX®) - Chronic Migraine

• OnabotA MOA in CM, including preclinical research to understand potential synergistic effect of onabotA used in combination with CGRP-targeted treatments (e.g., atogepant)
• Real-world effectiveness and safety of the combination treatment of onabotA and atogepant in CM patients
• Evidence on how onabotA treatment could improve patient outcomes for those CM patients who are not achieving optimal outcomes on CGRP mAbs
• Safety and efficacy of BOTOX use in patients with CM having concomitant conditions treated with additional injection paradigm (bruxism, CD, allodynia, peri-orbital pain)
• Impact of onabotA on migraine associated co-morbidities, including the prevention of associated comorbidities
• Impact of onabotA on functional outcomes, quality of life, health resource utilization and work productivity/return to daily activities
• Impact of onabotA on non-headache symptoms (e.g., cognition, brain fog, fatigue, etc)
• Long-term (>2 yr) data to demonstrate sustained onabotA efficacy and safety
• Real-world evidence demonstrating medico-economic impact of delaying treatment of patients with chronic migraine, including due to access limitations.
• Patient experience and satisfaction with onabotA tx, including the benefits of regular HCP touchpoints
• Data demonstrating patient’s benefit to expand injection procedure to non-neurologist i.e., pain specialists, nurses, clinical specialists (efficacy/safety/persistence/satisfaction)

Ubrogepant (Ubrelvy) - Acute Treatment of Migraine

• Evaluation of effectiveness of ubrogepant in combination with other migraine treatments
• Data demonstrating the effectiveness of ubrogepant across all phases of migraine
• Impact of ubrogepant use and Medication Overuse Headache (MOH)
• Ubrogepant efficacy and safety in new/different indications

Atogepant (Qulipta®/Aquipta®) – Preventive Treatment of Migraine

• Evaluation of effectiveness of atogepant and other migraine treatments in people with EM or CM
• Further characterize atogepant MOA with pre-clinical and clinical evidence
• Evidence for how patient outcomes can improve after early intervention ((e.g. first line of preventive treatment or early post-migraine onset) with atogepant in their treatment journey
• Impact on ictal/interictal QoL and non-HA symptoms (including function, daily activities, etc.), and work productivity and/or cognition
• Impact on co-morbidities associated with migraine, e.g. obesity, depression, anxiety, etc.
• Evidence that atogepant impacts disease progression in people with EM or CM
   

NEUROSCIENCE – NEUROTOXIN THERAPEUTICS: Botox (OnabotulinumtoxinA/BoNTA)

For the purposes of this document the IIS Strategic Priorities will be described for these indications:

• Toxin Science and New Indications
• Spasticity and Movement Disorders
• Adult Spasticity and Cervical dystonia
• Urology (focus on OAB and NDO)

For Migraine and for Botox Cosmetics indications, please refer to the applicable sections

Things to consider:

• Limited funds may be available to support proposals

Proposals for the topics listed below will NOT be considered:

• Studies conflicting with current research and development programs and strategies
• For Spasticity – Assessment of anticoagulants pre/post injection procedure
• For Urology – Evaluation of Botox for Premature Ejaculation (PE) or Benign Prostatic Hypertrophy (BPH)

Priority will be given to proposals investigating the following areas:

Toxin Science and Novel Indications

• Real world evidence of clinical and health economic outcomes of Botox utilization versus other toxins, including the consequences of toxin switching
• Botox utilization in multi-indication patients
• Novel indications, applications and treatment paradigms in alignment with areas of company focus.
• Pain, sensory, anti-inflammatory mechanisms of action of Botox
• Non-classical mechanism of action of Botox
• Preclinical studies assessing Botox versus other toxins

Spasticity

• Studies assessing the applicability of tools and biomarkers as a measure of efficacy and/or predictors for early spasticity diagnosis and treatment
• Impact of early diagnosis and intervention on overall outcomes when treated with Botox
• Impact of treatment adherence/discontinuation
• Real world safety, efficacy and utilization of Botox (assessing muscles, doses and retreatment)
• Impact of Botox treatment on comorbidities (e.g., depression/anxiety)
• Impact of Botox on patient relevant outcomes (including but not limited to patient satisfaction, functional impairment, pain relief, quality of life etc.)
• Assessment of improvement of function and/or specific functional goal attainment after treatment with Botox in spasticity due to any etiology (beyond post stroke etiology)
• Optimization of Botox in combination with other therapies
• Optimization of Botox therapy with utilization of new technologies and training tools
• Studies that assess Health Equity and Social Determinants of Health in Spasticity Management
• Studies looking at the use ultrasound to improve clinical outcomes of Botox

Movement Disorders

• Novel indications, applications and treatment paradigms in alignment with areas of company focus.
• Studies that assess Health Equity and Social Determinants of Health in Movement Disorders

Cervical Dystonia

• Botox for treatment of pain associated with cervical dystonia
• Clinical and/or Pharmacoeconomic impact of early diagnosis and intervention 
• Studies assessing approaches/predictors for prompt diagnosis and early intervention with Botox for Cervical Dystonia to obtain optimized patient outcomes 
• Real world observations describing impact of Botox on comorbidities (e.g., depression/anxiety, migraine) and/or patient reported outcomes (including but not limited to patient/physician satisfaction, functional impairment, pain relief, quality of life etc.)
• Optimization of Botox therapy with utilization of new technologies and training tools
• Studies that assess pharmacodynamics and/or clinically meaningful duration of Botox effects
• Impact of treatment adherence/discontinuation on pharmacoeconomic and/or patient outcomes

Essential Tremor

• Studies that assess diagnosis and disease classification in essential tremor
• Studies focusing on the treatment goals and patient outcomes associated with essential tremor

Urology Indications

• Studies to assess long-term real-world economics, safety and/or effectiveness of Botox in OAB and/or NDO
• Mechanism of action of Botox in the bladder for overactive bladder conditions or painful pelvic floor conditions
• Assessment of procedure setting or utilization of comfort techniques during the procedure and treatment adherence and/or patient satisfaction
• Studies to assess variables that impact time between diagnosis and advancement to minimally invasive therapies (including prolonged cycling on oral medications)
• Impact of Botox treatment adherence/discontinuation on pharmacoeconomic and/or patient outcomes
• Studies showing benefit of Botox specifically in men with OAB/NDO
• Studies that assess Health Equity and Social Determinants of Health in Urology
  
  

NEUROSCIENCE – PARKINSON’S DISEASE:
Foslevodopa/Foscarbidopa (LDp/CDp), Levodopa Carbidopa Intestinal Gel/Carbidopa Levodopa Enteral Suspension (LCIG/CLES)

Things to consider:

• Studies will only be considered for compounds that have received regulatory approval for at least one indication in the country of interest.

Proposals for the topics listed below will NOT be considered:

• Head-to-head proposals between device aided therapies

Priority will be given to proposals investigating the following areas:

Parkinson's Disease

• Disease burden/Progression:
  • Further understand the characteristics and/or burden in different stages of advancing Parkinson’s disease (PD) inadequately controlled by optimized oral therapy including but not limited to the impact on: nighttime sleep disturbances (e.g., nighttime mobility/OFF symptoms, Further understand the characteristics and/or burden in different stages of advancing Parkinson’s disease (PD) inadequately controlled by optimized oral therapy including but not limited to the impact on: nighttime sleep disturbances, nighttime mobility/OFF symptoms/, /morning akinesia), 
  • patients, caregivers, and healthcare resource utilization
• Disease Management:
  • Clinical utility of diagnostics or technology to identify signs and symptoms of Advancing Parkinson’s Disease and resultant changes in care.
  • Understand the right time for intervention (and provide referral, if needed) using different tools (i.e., Tools/Criteria such as “5-2-1”, My PD-Care, and MANAGE-PD; biomarkers, technology based objective measures (TOM) for use by HCP, patient/caregiver, and patient foundations/associations.)
  • Challenges with current treatment options
• Patient Perspective:
  • Evaluate patient barriers to refusal or initiation of invasive treatment (with a focus on subcutaneous infusion options) despite insufficient symptom control

Continuous dopaminergic stimulation (CDS) with or without Foslevodopa/Foscarbidopa (LDp/CDp)

• Short and long-term assessment (including but not limited to laboratory, clinical, imaging) of CDS vs pulsatile stimulation, including but not limited to use in earlier stages of PD to assess the ability of continuous therapies for disease modification and prevention/delay of motor complications
• Assessment of CDS therapies to partially or completely reverse synaptic plasticity / widen the therapeutic window (including but not limited to laboratory, clinical, imaging)
• Pharmacological effects of CDS over 24 hrs.
• Investigate indicators/markers (including but not limited to laboratory, clinical, imaging) to provide evidence to support whether continuous drug delivery translates to continuous dopaminergic stimulation in patients.
• Evaluate preclinical impact of CDS on neuroinflammation

Foslevodopa/Foscarbidopa (LDp/CDp)

Patient Selection or Characteristics

• Evaluate LDp/CDp efficacy and safety in subpopulations of advanced PD (APD) patients uncontrolled on orals including, but not limited to: age <65 years, Body-Mass-Index subgroups (e.g. BMI <18.5, 18.5-25, >25), cognitively impaired, patients with impulse control disorders (ICDs), and underserved populations such as diverse ethnicities/race/gender
• Evaluate device-aided therapy sequencing patterns (including but not limited to LDp/CDp to LCIG or vice versa, or LDp/CDp to other device-aided therapies and vice versa)
• Evaluate efficacy and safety in PD patients earlier within “advanced” PD (e.g., time to motor fluctuations < 3 years, less daily motor complication burden, shorter disease duration), “active” patients (e.g., with regards to exercise levels and work ability)

Efficacy and Safety Measures

• Further understand the efficacy and safety (including responder analyses/characteristics), including but not limited to:
  • Motor efficacy including but not limited to dyskinesia, freezing of gait, and axial symptoms.
  • Patient and caregiver-related outcomes, preferences, and satisfaction (patient/caregiver experience), activities of daily living (ADL), quality of life (QoL), perceived independence/self-efficacy.
  • General or specific Non-Motor Symptoms (NMS) including but not limited to sleep, apathy, fatigue, pain, gastrointestinal dysfunction, sexual dysfunction, nocturia, orthostatic hypotension
  • Morning akinesia/early-morning OFF or early-morning dystonia
  • Using Technology based Objective Measurements (e.g., wearable devices, biosensors, and others) as surrogates of efficacy and safety of LDp/CDp in APD patients, including but not limited to (nighttime) mobility, sleep.
  • Health care resource utilization, caregiver burden, work productivity, cost-effectiveness, societal impact (e.g., return to activity, outreach/life space, social participation, lifestyle), stigma, delay in nursing home admission
• Long-term data on effectiveness and safety, including infusion site events root cause evaluation (i.e., inflammatory process), factors leading to discontinuations and interventions to improve adherence / development and evaluation of protocols for the structured routine support of patients on treatment.

Initiation/Titration/Practical Outcomes

• Characterize APD comedication management to achieve monotherapy with LDp/CDp, including pill burden reduction
• Understand titration and the impact of different settings for the treatment initiation with LDp/CDp, including but not limited to evaluation by patient/caregiver-related outcomes (like satisfaction, self-efficacy, QoL, ADL) in APD, under the following circumstances
  • Outpatient versus inpatient titration setting
  • General Neurologists (GN)-initiated versus Movement Disorder Specialist (MDS)- initiated titration
  • Different nurse care models
  • Use of telemedicine (including but not limited to partial or full remote)
• Understand practical aspects of long-term therapy, including evaluating handling of the infusion device (e.g., infusion site rotation, alternative infusion sites, frequency of needle placement, use of different infusion sets, cannula lengths, vial consumption), regimens or algorithms for the prevention or treatment of infusion site events
• Use of and outcomes with alternative flow rates (e.g., high rate for periods of increased activity and low rates for nighttime)

Other Indications

• Evaluate LDp/CDp in other Parkinsonian Syndromes.

Levodopa Carbidopa Intestinal Gel/Carbidopa Levodopa Enteral Suspension (LCIG/CLES) – applicable to countries who do not have LDp/CDp approved/available

• Evaluate LCIG/CLES efficacy and safety in subpopulations of advanced PD (APD) patients uncontrolled on orals where currently limited data exists
• Understanding the use of LCIG/CLES in combination with or after failure with other device aided therapies (DATs)
• Further understand the characteristics of responders to LCIG/CLES therapy
• Evaluate effects of 24-hour use of LCIG, especially on nighttime sleep quality, nighttime mobility / Off symptoms, freezing of gait and quality of life
• Potential use of LCIG after LDp/CDp from the clinical perspective, infusion duration (24h==>16h), transition process, patient, and caregiver adoption
• LCIG use in later stages of the APD (after LCIG), older patients, more cognitive impairment, mobility limitations
• Additional areas:
  • Evaluate LCIG/CLES in other Parkinsonism disorders
     

NEUROSCIENCE PSYCHIATRY – VRAYLAR (CARIPRAZINE):
Bipolar Disorder I (BP-I) depressive, manic, and mixed episodes; Schizophrenia; and US only: Adjunctive treatment of MDD (aMDD) :

VRAYLAR (cariprazine) is an orally active atypical antipsychotic. It is a partial agonist at central dopamine D3/D2 and serotonin 5-HT1A receptors and has antagonist activity at serotonin 5-HT2A receptors.  

We welcome proposals from Investigators from under-represented groups.   

Vraylar is approved for adults with:  

• Bipolar Disorder I (BP-I) - depressive, manic, and mixed episodes   
• Schizophrenia  
• US only: Adjunctive treatment of MDD (aMDD)  

Studies will only be considered for compounds that have received regulatory approval for at least one indication in the country of interest.    

Priority will be given to proposals to investigate the following areas:  

• Investigation of disease states where Vraylar’s pharmacology matches with the hypothesized underlying biology of psychiatric illnesses
• Real world experience with and implementation of Rapid Mood Screener  
• Understanding Vraylar:
  • Impact on functioning and quality of life
  • Potential to improve cognitive deficits in patients   
  • Efficacy on anhedonia
  • Efficacy on anxiety
  • Long-term efficacy data
  • Efficacy in MDD and BP Depression with Mixed Features
  • Treatment in first episode psychosis  
  • Combination treatment in schizophrenia and bipolar-I disorder
  • Treatment in schizophrenia prodrome
  • Use in patients with schizophrenia, bipolar disorder, and/or MDD early in the disease course
• Alternate dosing and titration strategies in approved indications
• Outcomes related to switching to/from cariprazine
• Impact on novel outcomes related to treatment
• Efficacy and safety in patients with substance use disorders  

Proposals in the following areas will NOT be considered:  

• Indications for which Cariprazine (VRAYLAR) has been or is being evaluated:   
  • Pediatric adjunctive Major Depressive Disorder (aMDD)  
  • Pediatric BP-I disorders  
  • Pediatric schizophrenia  
  • Maintenance treatment of BP-I disorder in adults  
  • Irritability associated with autism spectrum disorder (ASD)  
  • Negative symptoms in Schizophrenia  
  • Populations at high risk for impulse control dysfunction/compulsive behavior   
• Patients at high risk for seizures  
• Populations and/or indications under consideration by AbbVie   

Epcoritamab, Venetoclax (ABT-199), Etentamig (ABBV-383), Telisotuzumab vedotin (Teliso-V, ABBV-399), Elahere, ABBV-400, Livmoniplimab (ABBV 151)

AbbVie Oncology compounds with areas of interest for investigator-initiated studies are listed below.  AbbVie is accepting preclinical and clinical applications in 2025.

Things to consider for the investigator:

• Priority will be given to drug only applications for the below Oncology compounds.
• A clinical interventional proposal may be considered only after the safety profile and dose finding has been established for an Abbvie compound or non-AbbVie compound for research in combination therapies. Dosage and dosing interval optimization studies may be considered.
• AbbVie’s medical science liaisons are available to provide guidance throughout this process.
• The funding of research must not exceed local fair market value, nor be used for expenses not associated with the conduct of the research.
•  Submissions that duplicate or overlap with Abbvie clinical development or other external research are not a priority.

In addition to our areas of interest for oncology IIS, we have a diverse clinical trial program. Details of our AbbVie oncology pipeline can be found at this link.

Epcoritamab

Epcoritamab, a subcutaneous CD3xCD20 bispecific antibody, is approved in some regions as monotherapy with treatment until disease progression for 3L+ R/R DLBCL FL. 

Epcoritamab is being investigated in a number of hematological indications including diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL), and chronic lymphocytic leukemia (CLL).

Priority will be given to Epcoritamab applications proposing to investigate the following areas:

• Evaluation of Epcoritamab management in various patient care settings
• Evaluation of real-world data including but not limited to treatment patterns and patient-reported outcomes
• Evaluation of novel and additional clinical endpoints and/or exploratory predictive models or biomarkers

Guidance for Pre-clinical proposals:

Pre-clinical and translational applications will be reviewed to prioritize proposals aligned to the following goals:

• Investigate rationale for combinations that improve efficacy, safety and synergize with Epcoritamab’s mechanism of action, using relevant models
• Evaluate relationship between Epcoritamab and the tumor immune micro-environment
• Evaluate Epcoritamab activity and biomarkers of response/resistance in patient-derived samples/tumor tissues

Venetoclax (ABT-199)

Venetoclax is a BCL2 inhibitor with scientific rationale for evaluation across a broad variety of hematologic malignancies/disorders including, but not limited to chronic lymphocytic leukemia (CLL) and acute myeloid leukemia (AML).

Priority will be given to applications proposing to investigate the following areas:

• Novel therapeutic combinations
• Evaluation of mechanisms to overcome resistance
• Evaluation of novel and additional clinical endpoints and/or exploratory predictive models or biomarkers

Notable considerations for the investigator:

• Adult solid tumor proposals will not be accepted.

Etentamig (ABBV-383) 

Etentamig (ABBV-383) is a BCMAxCD3 bispecific T-Cell Engager with scientific rationale for evaluation in Multiple Myeloma. 

Priority will be given to Etentamig (ABBV-383) applications proposing to investigate the following areas:

• Novel therapeutic combinations in Multiple Myeloma
• Evaluation of novel and additional clinical endpoints and/or exploratory predictive models or biomarkers
• Exploration in special patient populations
• “Response adaptive and MRD guided approaches” OR “Patient-centric trial designs”

Notable considerations for the investigator: 

• Solid tumor proposals will not be accepted
• Low risk / Intermediate Risk SMM; MGUS proposals will not be accepted
• PK studies will not be accepted

Guidance for Pre-clinical proposals: 

Pre-clinical and translational applications will be reviewed to prioritize proposals aligned to the following goals:

• Provide rationale for combinations that improve efficacy, safety and synergize with Etentamig (ABBV-383) MOA
• Evaluate relationship between Etentamig (ABBV-383) and the tumor immune microenvironment   
• Evaluate novel combinations and mechanistic studies in relevant models 
• Evaluate novel combinations and mechanistic studies in relevant models to better differentiate 383 and other T-Cell engagers

Telisotuzumab vedotin (Teliso-V, ABBV-399)

Teliso-V is an antibody drug conjugate (ADC) targeting c-Met protein overexpression with scientific rationale for evaluation in non-small cell lung cancer and other solid tumor malignancies.

Priority will be given to Teliso-V applications proposing to investigate the following areas:

• Clinical proposals evaluating Teliso-V based novel combinations (e.g; IO, TKI, RT or others) for EGFR wt Non-Squamous Non-Small Cell Lung Carcinoma, based on robust preclinical evidence demonstrating combinatorial efficacy. 
• Evaluate potential Teliso-V based combinations (IO and others) with the rationale for extrapolating treatment strategies to NSCLC patients with unmet needs (e.g., brain metastases).

For non-clinical IIS:

• Proposals that evaluate overlap between c-Met and other genetic aberrations, as well as alternate c-Met testing modalities, will be prioritized. 

Notable considerations for the investigator:

• Pediatric proposals will not be accepted. 
• Proposals with squamous NSCLC histology will not be accepted.
• Monotherapy proposals in NSCLC patients with EGFR actionable mutations will not be accepted.
• Proposals with translational/biomarker components will be assessed on a case-by-case basis based on unmet scientific questions.

Elahere

Mirvetuximab Soravtansine is an antibody drug conjugate (ADC) targeting Folate Receptor α found overexpressed on certain tumour cells with scientific rationale for evaluation in ovarian cancer and other solid tumour malignancies.

Priority will be given to Elahere applications proposing to investigate the following areas:

High:​

• Combination therapies to overcome PARPi resistance​
• Combinations with novel agents​
• MIRV retreatment​
• Concordance/Quality of FRα testing​
• Consistency/stability of FRα expression

Medium:​

• Platinum Refractory​
• Overlap of ADC target biomarkers​
• Therapies post MIRV​
• Strategies to manage compliance and tolerability​
• Heterogeneity of FRα expression​
• Alternate methodologies and sample types (FRα)​

ABBV-400

ABBV-400 is an antibody drug conjugate (ADC) targeting c-Met that is being investigated to treat colorectal cancer, gastroesophageal adenocarcinoma, non-small cell lung cancer and other malignancies.

Priority will be given only to applications proposing to investigate ABBV-400 in metastatic colorectal cancer with a focus on the following areas:

• Clinical proposals evaluating ABBV-400 based combinations with SOC therapy or with novel agents (e.g; IO, TKI, RT or others) in colorectal cancer based on robust preclinical and clinical evidence demonstrating efficacy and safety. 

• Clinical concepts with ABBV-400 monotherapy or in combinations (IO and others) with the rationale for extrapolating treatment strategies to CRC patients with unmet needs (e.g., chemo ineligible, frail, brain metastases).

• Proposals assessing activity of ABBV-400 (either as monotherapy or in combination) in earlier lines of therapy for CRC patients.

• Proposals in combinations with localized therapies for treatment of liver mets (e.g. radio/chemo embolization)

For non-clinical IIS:

• Overlap between c-Met protein expression status and oncogenic and non-oncogenic CRC biomarkers/signatures

• Evolution and stability of c-Met protein expression from diagnosis to later lines of therapy, across different sample types (archived versus post-progression/recent sample; cytologic versus tissue biopsy)

• Concordance between c-Met protein expression and c-Met amplification status, and c-Met protein expression between primary and metastatic site across lines of therapy

Notable considerations for the investigator:

• Pediatric proposals will not be accepted. 

• Monotherapy or combination therapy proposals in 3L+ mCRC setting will be evaluated on a case-by-case basis based on the final optimized dose.

• Proposals in Neoadjuvant or adjuvant setting will not be accepted.

• Proposals with combination therapy with a risk of unacceptable toxicity will not be accepted.

• Proposals with translational/biomarker components will be assessed on a case-by-case basis based on unmet scientific questions.

• Proposals comparing ABBV-400 with other SOC therapies in head-to-head trials will not be accepted.

Livmoniplimab (ABBV 151)

Livmoniplimab (ABBV-151) is an antibody that targets the GARP-TGF-β1 complex and is being investigated for the treatment of solid tumors, including hepatocellular carcinoma, non-small cell lung cancer, urothelial cancer, ovarian granulosa tumors, and colorectal cancer. 

Priority will be given to drug only applications proposing to investigate livmoniplimab in the following areas:   

• Novel therapeutic combinations  
• Other disease states not referenced in the description above  
• Earlier Stage disease  
• Exploration of multiple areas of interest in a proposal are welcome 

Guidance for non-clinical proposals: 

• Spatial localization of GARP-expressing cells in tumor microenvironment   
• Evolution of GARP-expression and localization from diagnosis to later lines of therapy  
• Ex vivo human tumor models or humanized animal models for monotherapy or combination treatment  
• Mechanisms of resistance and novel biomarkers   

SPECIALTY – Hepatology (HCV) – Maviret/Mavyret:
Hepatitis C Virus

AbbVie is committed to support global efforts to meet WHO target of HCV elimination as a major public health threat by 2030.  

In this context, AbbVie is interested in scientific study proposals addressing any of the following priority areas: 

1. Scalable and sustainable models of care that: A) successfully incorporate non-liver/non-ID specialists (e.g., addiction specialists, psychiatrists, pharmacists, primary care and NP/PAs) into HCV care, and/or B) enhance linkage to care in incarcerated populations. 
2. Clinical and economic outcomes of 8-week treatment in patients with HCV to enable further simplification of HCV care across disease stages.
    

SPECIALTY – EPIC– INFeD: 
Iron Deficiency

AbbVie is interested in scientific study proposals requesting product only addressing the following priority area for INFeD:

Iron Deficiency/Anemia

• Evidence characterizing the outcomes and of patients treated with INFeD administered by total dose infusion for iron deficiency who have an intolerance to oral iron.
   

Endo-Metabolic: CREON

Abbvie is interested in scientific study proposals that would address the following priority clinical areas: 

1. Burden of exocrine pancreatic insufficiency (EPI) disease and/or impact of pancreatic enzyme replacement therapy (PERT) treatment in patients with EPI and:
   
   a.  Diabetes mellitus type 2 in the absence of other pancreatic disease (e.g., chronic or acute pancreatitis, cystic fibrosis, pancreatic cancer)
   
   b.  Fatty pancreas
   
   c.  GI surgeries that could result in EPI
   
   d.  Celiac Disease 
   
   
2. Outcomes in EPI patients treated with CREON® (pancrelipase) with underlying conditions of EPI, excluding chronic pancreatitis in adults 
   
   
3. Novel approaches (e.g., clinical tools, biomarkers, devices) that:
   
   a.  Accelerate/ease the diagnosis of EPI 
   
   b.  Improve adequate PERT dosing
   
   c.  Improve PERT adherence
   
   d.  Assess outcome of PERT treatment beyond stool fat measurements 
    

ANTI-INFECTIVES:
Avycaz (ceftazidime/avibactam) 

Key Focus Areas: Avycaz (ceftazidime/avibactam) 

• Studies (pre-clinical and clinical) evaluating emergence of resistance of Gram-negative bacteria, including prevalence of resistance, mechanisms of resistance, and strategies for prevention 
• Clinical outcomes in special populations treated with CAZ-AVI (e.g., immune-compromised, transplant, continual renal replacement therapy) 
   

GI CARE: 
Linaclotide

AbbVie is interested in scientific study proposals that would address the following priority areas: 

1. Real world effectiveness and safety of linaclotide in patients with constipation, including alternative doses, special populations (including but not limited to patients with developmental delays, autism, anxiety, depression).  
2. Real-world effectiveness and safety of linaclotide in specific patient populations (e.g. GI hypomotility disorders, comorbid disorders of the gut-brain interaction, Cystic Fibrosis, IBS-C/CIC and concomitant use of GLP-1s) suffering from constipation with/or without abdominal symptoms.  
3. Novel devices, clinical tools and/or biomarkers that: 
   a.  Increase the ease of confident diagnosis of IBS-C and CIC
   
   b.   Improve patient clinical experience, outcomes and/or adherence to linaclotide treatment
   
   c.  Assess novel outcomes of IBS-C and CIC treatment