Investigator-Initiated Studies

What is an IIS?

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.
 

What does an IIS aim to achieve?

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.
 

What are the benefits of an IIS?

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
   

What shall I consider when submitting an IIS proposal?

• 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.
   

Types of studies that are in scope when applying for an IIS with UCB

• 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.

When submitting an IIS request with UCB, please ensure that the

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).

UCB’s IIS submission and review process
 

Rare Diseases
Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD), Autoimmune encephalitis (AIE), Thymidine kinase 2 deficiency (TK2d)

• Early diagnosis, genetic testing, long-term effectiveness, safety, outcomes and healthcare resource utilization in patients with TK2D
   

Myasthenia Gravis (MG)

• gMG biomarkers research
• In vitro studies investigating Neuromuscular Junction
• gMG studies in adult population

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. 

Epilepsy

Developmental Epileptic Encephalopathies

• Perceptions, outcomes, and healthcare resource utilization for patients and their caregivers in Dravet and Lennox-Gastaut syndromes
• Real-world evidence to support diagnosis and natural history of Developmental Epileptic Encephalopathies (DEEs), 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. 

Chronic Treatment and Overarching Themes

• 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 however there will be strict review cycles (see below). If your submission is submitted during the one of three yearly review periods it will have to wait until the next review cycle. 
 

Immunology

Psoriatic disease (PsA) 

Studies that focus on:

• The underlying mechanisms and causes of psoriatic arthritis
• Markers for early detection, disease progression, and response to treatment
• Factors that influence the progression of psoriatic arthritis
• Imaging advancements in PsA
• The link between psoriatic arthritis and other inflammatory conditions, such as cardiovascular disease

Moderate to severe Psoriasis (PsO)

Studies that focus on:

• Evaluating the benefits of skin clearance
• Understanding the value of speed of onset and the benefit of early treatment
• Evaluating the effect in high-impact areas (e.g., nail, genital and PP)
• Understanding early signs of PsA in PsO patients and impact on disease progression
• The Impact of relevant treatments on comorbidities of psoriatic disease 
• Treatment efficacy in patients with inadequate response to current therapies

Axial spondyloarthritis (axSpA)
Studies that focus on:

• The pathobiology and mechanisms of disease in axSpA
• Treatment strategies in axSpA
• Understanding axSpA subpopulations
• Imaging advancements in axSpA
• Exploring axSpA comorbidities

Moderate to severe Hidradenitis Suppurativa (HS)
Studies that focus on:

• Understanding the additional benefit of IL-17F and its inhibition in the pathobiology over the course of the disease and how this is translated into clinical outcomes
• Evaluating the effectiveness and tolerability in subpopulations (out-of-scope paediatric, adolescent, out-of-label population)
• Understanding disease epidemiology, burden of disease, cost of care and comorbidities
• Developing and validating scoring tools/PROs
• Using machine learning and big data approach to improve diagnosis and treatment in HS
• Evaluating the impact of treatment on surgical interventions, on lesions in different anatomical areas and on health care resource utilisation.

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. 

Bone

• Advancing the understanding of sclerostin modulation in the bone
• Implementation of Science Projects for post-fracture care
• Use and impact of romosozumab in the clinical setting (within approved indication).

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.