Investigator-Initiated Studies

IISs are clinical studies and non-clinical studies which have been solely conceived, initiated and conducted by a nonpharmaceutical institution (ie, academic), associations (medical professionals or patients) or investigator/physician/academic.

The aim of an IIS is to generate key data relevant to a specific therapeutic area or a drug – this will help address certain areas such as supporting clinicians in their day-to-day practice. These studies also tend to capture unanswered questions or areas that haven’t been studied during Phase I-III of drug development.

Because IISs are studies that generate data in the real world, setting up these studies could potentially assist in:

• The development of hospital or country policies as well as support the training of Healthcare Personnel
• The generation of more safety data as well as data that can support benefit-risk assessments
• Providing real-world data in patient populations outside the controlled populations of clinical trial studies
• Answering daily practice research questions with less commercial conflicts of interest. 

• Ensure the overall study has scientific merit, that the research protocol and methodology is robust
• Ensure study is able to meet necessary ethical and regulatory requirements
• Ensure the investigator leading the study has the necessary qualifications as well as the necessary motivation and commitment to drive the study to its completion.

• Interventional (clinical) studies (which may include the use of approved and investigational UCB products)
• Non-interventional studies such as clinical observational studies or RWE studies (which may include the use of approved and investigational UCB products)
• Non/Pre-clinical studies irrespective of marketing license authorisation status
• Population health research, Health Economics and Outcomes Research (HEOR) studies
• Non-product related (where a drug intervention is not a direct objective of the study)
• Patient Registry-based studies where data are used to conduct the study and answer research hypothesis.

Study

• Has scientific merit and it aligns with UCB’s areas of interest (listed below)
• Is well designed with a robust study proposal that can meet all the ethical and regulatory requirements
• Has realistic timelines
• Has a clear budget and publication plan
• Is not duplicative of existing research in the public domain

Investigator

• Has the required qualifications and experience to conduct and manage the study being proposed
• Is motivated and committed to ensure the study meets its aim and is completed according to agreed timelines
• Is being assisted by a team that can support all stages of the study
• Is responsible for preparing a study progress updated final report and publication(s)
• Is responsible for reporting any safety events to UCB (if applicable).

Myasthenia Gravis (MG)

• Biomarker research related to gMG
• In vitro studies investigating neuromuscular junction
• On-label population effectiveness studies

Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD)

• Earlier diagnosis and diagnostic criteria validation
• Disease course and relapse prediction
• Outcome measures
• Biomarker identification
• Burden of disease studies

Thymidine Kinase 2 deficiency (TK2d)

• Earlier diagnosis of patients with TK2d (including screening initiatives)
• TK2d patient-relevant outcome measures of disease progression (including biomarker-related studies)
• Robust epidemiology prevalence data

MG and MOGAD: Submissions are welcomed throughout the year however there will be strict review cycles (see below). If your study proposal is submitted during the one of three yearly review periods it will have to wait until the next review cycle to be evaluated. 

TK2d: Submissions are welcomed throughout the year and they will be reviewed within one month after submission of the proposal. You can expect the outcome within 3 weeks after the review meeting.
 

Developmental Epileptic Encephalopathies and Neurodevelopmental disorders

• Perceptions, outcomes, and healthcare resource utilization for pediatric and adult patients and their caregivers in Dravet and Lennox-Gastaut syndromes, CDKL5 deficiency disorder and Rett syndrome
• Real-world evidence to support diagnosis and natural history of Developmental Epileptic Encephalopathies (DEEs) and neurodevelopmental disorders, including non-seizure outcomes
   

Acute Seizure Management

• Perceptions, outcomes, and healthcare resource utilization with emergency medications for patients with epilepsy
• Real-world interventions to support identification and diagnosis of seizure clusters, prolonged seizures and/or the use of seizure action plans among patients and caregivers
• Impacts of undertreatment in people with epilepsy with seizure clusters and prolonged seizures, including the impact of seizure duration and progression on quality of life and health care utilization
• Perception and data on the relationship between prolonged seizures, seizure clusters and status epilepticus, including the reduction of inter-seizure intervals
• Piloting the use of seizure detection devices or other early ictal or pro-ictal activities to reduce the seizure burden and their impact on people with epilepsy
   

Focal Epileptic Seizures

• Real-world evidence on outcomes with chronic treatments in vulnerable patient populations (e.g., elderly, pediatrics, patients with comorbidities or requiring hospitalization)
• Perceptions, outcomes, and insights specific to women of childbearing age with epilepsy
   

Submissions are welcomed throughout the year and they will be reviewed within one month after submission of the proposal. You can expect the outcome within 3 weeks after the review meeting. 

Dermatology: Psoriasis (PsO), Hidradenitis Suppurativa (HS)

• Impact of early intervention with Bimekizumab on disease progression, clinical and socioeconomic outcomes
• Impact on understudied sub-populations and comorbidities 
• Demonstrating the role of earlier inflammation resolution in enhancing clinical outcomes and patient well-being
• Identification of predictors of disease progression and how these can influence the impact of earlier treatment and clinical outcome
• Evaluation of the impact of disease on cumulative life course impairment (CLCI)

Axial spondyloarthritis (axSpA)  & Psoriatic Arthritis (PsA)

• Real-world evidence data on the use of BKZ relevant to clinicians and patients
• Innovative techniques to evaluate the impact of BKZ in axSpA & PsA
• IL-17A and IL-17F role in axSpA and PsA pathobiology 
• Identification of predictors of disease progression and how these can influence earlier treatment and clinical outcome
• Impact of early treatment on clinical outcomes and patient well-being
• Data on use of BKZ in axSpA subpopulations
• Generating persistence data with BKZ in PsA
   

Submissions are welcomed throughout the year however there will be strict review cycles (see below). If your study proposal is submitted during the one of four yearly review periods it will have to wait until the next review cycle to be evaluated. 

• Addressing the critical gaps in fracture care and enhancing post-fracture coordination
• Optimizing Bone Health in osteoporotic patients undergoing spinal fusion
• Generating real-world evidence on the effectiveness of romosozumab within its approved indication using non-interventional designs where clinical data are limited. Key focus areas include gathering patient experience data, such as patient preferences and reported outcomes, to inform clinical decision-making and enhance patient-centered care
   

Submissions are welcomed throughout the year; however there will be strict review cycles (see below). If your study proposal is submitted during the one of four yearly review periods it will have to wait until the next review cycle to be evaluated.