Decision Tree for Selecting the Right Research …...Introduction This Learner Guide is a resource to supplement the eLearning module: Decision Tree for Selecting the Right Research

Decision Tree for Selecting the Right Research ApproachLearner Guide

Learning Resources: Supplementary Materials

Introduction

This Learner Guide is a resource to supplement the eLearning module: Decision Tree for Selecting the Right Research Approach

Confidential. For Internal Training Use Only. Do Not Distribute.

1- Course Overview & Learning Objectives

2- Key Concepts

3- Definitions

Learner Guide Content

Course Link

This course can be found on Talent Share.

4- Learning-to-Action & Coaching Tips

Overview & Learning Objectives


Learning Objectives

Describe the UCB process for determining the approach to answering a business question

Define Non-interventional Studies (NIS), Market Research Programs (MRP) and Patient Support Programs (PSP)

Differentiate the implementation path for NIS, MRP, PSP

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4 Explain the implications/consequences of not adhering to the UCB Decision Tree

This module, Decision Tree for Selecting the Right Research Approach is part of the Real-World Evidence learning series.

The UCB Decision Tree is designed to facilitate the selection of the right research/study or program approach. The Decision Tree elements are first described/defined and then explored through a series of 6 case examples.

4Confidential. For Internal Training Use Only. Do Not Distribute.

Key Concepts

Choosing the right approach to answer a business question is not only essential to ensure the approach effectively addresses the business question, but it also has important other implications.

Choosing the Right Approach

The consequences of not selecting the right approach include:• The business question may not be effectively addressed• Loss of investment & other resources• Compliance concerns resulting in fines/ damage to our company

reputation• Ethical concerns – e.g. using the wrong approach to answer a

research question to avoid stringent reporting obligations

Considerations

Here are some considerations that will help when selecting an approach that will expedite the review and implementation processes downstream:• What is the business question?• What is the intended purpose of the research/study or program?• What is the potential impact of the evidence generated?• Is there a link to company strategy?• Who is the target audience?

Key ConceptsDecision Tree


The Decision Tree focuses on the purpose of the research/study or program:

To assess benefit/risk of a UCB product and/or to produce evidence for scientific purposes.

To gather data to gain an insight or support decision making for the business, that is, to inform brand strategy.

To provide a service, solution or initiative that is externally oriented and targeted towards UCB patients and caregivers.

NIS MRP PSP

RWE (NIS) vs MRP vs PSP


Real World Evidence (RWE) Study Non-Interventional Study (NIS) Market Research Program Patient Support Program

Data source

RWE studies utilize “data collected under real life practice conditions”. Such data may already exist (e.g. registries, EHR]EMR administrative (claims) data) and can be analyzed secondarily or they can be collected prospectively. Other terms used for RWE studies are observational studies, epidemiological studies and/or NIS as by design, no influence on the course of action regarding patients’ treatment is taken.

Qualitative and quantitative data generated prospectively through interaction with patients or other healthcare decision makers where a personal perspective is reported and therefore is subjective in nature by default.

PSPs are structured where interactions or contact between the patient, caregiver and /or healthcare professional are foreseen in the program, whereby UCB or the vendor acting on behalf of UCB may collect information in relation to UCB medicinal product or therapeutic area/disease.

Research focus

Main focus includes understanding the natural course of disease, real life treatment patterns & patient outcomes (safety & effectiveness) in the wider ecosystem of care.

Main focus is determine broad patterns amongst collective interviewees (patients and/or other stakeholders) experiences

To assist patients in managing their disease, medication or behaviors through either gaining access to medication, administration of medication, monitoring or following up on treatment or providing information related to the medication or disease.

Business question

Designed to address internal and external business questions. The studies designed for external business questions are usually publishable in peer reviewed journals and therefore rely on recognized and accepted methods capable of standing up to evidence-based critical appraisal.

Designed to address internal business questions.

Methods

Various methodological approaches applied to structured data underpinned by epidemiological sciences such as cox regression / proportional hazard modelling, with a mix of agile and flexible analysis methods for internal business questions and high scientific rigor (e.g. GxP related) in approaches to respond to external business questions.

Various methodologies used such as conjoint analysis, factor analysis and cluster analysis.

Various methodologies are applies to analyze data to gain insight, though main purpose is the service to the patients.


Definitions - StudiesApproaches In Scope for the Decision Tree

Non-interventional Study (NIS) is a study where the medicinal product is prescribed in the usual manner in accordance with the terms of marketing authorization and the assignment of the patient to a particular therapeutic strategy falls within current practice. An NIS is also referred to as a Real-World Evidence or RWE Study.

Interventional Study/Clinical Trial is any research study that prospectively assigns people to one or more health-related interventions (e.g., preventive care, drugs, surgical procedures, behavioral treatments, etc.) to evaluate their effects on health-related outcomes

For more information relating to RWE (non-interventional) Studies vs Clinical Trials (interventional studies), refer to the RWE eLearningHow do Clinical Trials and Real-World Evidence (RWE) Studies Differ?(TalentShare course id: 31281)


Individual Patient Engagement refers to engaging directly with patients in order to ensure a lively and connected ecosystem where the patient voice is broadly heard and taken into account. Patient Engagement involves engaging directly with patient communities, individual patients, patient advocates and patient groups.

Social Listening is the process of monitoring and tracking social media channels for conversations and mentions of your brand, competitors, product, and any other ideas or themes that are relevant to your business and leveraging your insights to discover opportunities or create content for those audiences.

Definitions - MRPsApproaches In Scope for the Decision Tree

Market Research Program (MRP) is the systematic and objective identification, collection, analysis, and dissemination of information for the purposes of assisting management in decision making related to the identification and solution of problems and opportunities. It is not a commercial communication or selling or promotional opportunity.


Patient Support Program (PSP) is a structured service, solution or initiative for patients and/or caregivers to assist patients in managing their disease, medication or behaviors through either gaining access to medication, administration of medication, monitoring or following up on treatment, or providing information related to the medication or disease. The intent of a PSP is to provide a service, solution or initiative and not research. Therefore, unlike NIS and MRPs, PSPs are not specifically referred to as a research approach.

Patient Value Initiatives (PVI) are disease, product, therapeutic area related activities which are externally orientated and targeted towards our patients, and/or caregivers to ensure they receive maximum benefit during the course of their journey and within their ecosystem of care. In this module, Patient Value Initiatives and Patient Support Programs are both referred to as Patient Support Programs.

Definitions – PSPsApproaches In Scope for the Decision Tree


Investigator Initiated Studies (IIS) are research-sponsored, conducted and managed by parties other than UCB but for which UCB provides financial and/or product support. They explore key medical and scientific hypotheses related to UCB’s products and related therapeutic areas. They must be unsolicited on the part of UCB.

Collaborative Agreement is a collaboration that occurs between UCB and other parties to combine skills, experiences and/or resources to work jointly, during a certain term on a project whilst the independence of each party is maintained, each party complying with all applicable laws, rules and regulations.

Grants are a type of financial support from UCB to an Institution for the purpose of furthering medical and scientific knowledge and for the benefit of the patient. They aim to foster science and medical practice and are to be unsolicited and without tangible benefit for UCB. They are made directly to an Institution and not to an individual, are transparent (i.e. declared), and are not linked to promotional activity.

DefinitionsApproaches Out of Scope for the Decision Tree


Definitions - General

Adverse event is any undesirable experience associated with the use of a medical product in a patient

Business questions are research questions that emerge during the drug development process due to gaps in knowledge or missing information that is needed to support business units to develop effective and safe medicines for patients. Refer to the Business Questions eLearning module for more information

Claims databases or administrative databases are electronic records of transactions for reimbursement for services provided during a health encounter. They include information entered on bills (claims) submitted by hospitals, clinics, nursing homes, pharmacies, individual providers, and other medical professionals to public and private insurance entities

Compliance is adhering to laws, regulations and company policies that apply to one’s area of responsibility

Homogenous management guidelines: Management guidelines are systematically developed statements to assist practitioner and patient decisions about appropriate health care for specific clinical circumstances. Homogenous management guidelines provide general guidelines without differentiating needs for specific patient subpopulations

Individual Case Safety Report Quality Forum (IQF) is a forum where multidisciplinary team reviews, assesses, and resolves issues that have an impact on the quality and reporting of individual case safety reports to drive the improvement of quality and compliance with external regulations and collaborate on the UCB standard for patient safety processes that is agreed upon by relevant stakeholders and communicated throughout the company

Patient journey is a series of events that a patient follows when navigating through the healthcare system from time of diagnosis through to a health outcome

Patient segments are distinct groups of patients with different characteristics, for which intervention programs can be designed

Definitions – General (2)


Phenotype is a measurable biological (physiological, biochemical, and anatomical features), behavioral (psychometric pattern), or cognitive marker that is found more often in individuals with a disease or condition than in the general population

Prospective is a study design where the investigators conceive and design the study, recruit subjects, and collect baseline exposure data on all subjects, before any of the subjects have developed any of the outcomes of interest. The subjects are then followed into the future to record the development of any of the outcomes of interest

Real World Dosing refers to the dosage of medication prescribed to patients in clinical practice

Registry is an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves one or more predetermined scientific, clinical, or policy purposes

Retrospective is study design that uses existing data. The outcome of interest has already occurred at the time the study is initiated

Standard operating procedure (SOP) is a set of written instructions that document a routine or repetitive activity followed by an organization

Safety reporting obligations are the requirements for sponsors investigating a drug to notify regulatory authorities and all relevant stakeholders of any adverse experience associated with the use of the drug and any finding from tests in laboratory animals that suggested a significant risk for human subjects


Additional Resources

o Standard Operating Procedures (SOPs)o SOPs related to this course include:

o Link to SOPs in this course and all other UCB SOPs – Source CDo https://ucb-source-cd.veevavault.com/

Both of these situations require escalation.

Reach out to:[email protected]: NIS/MRP/PSP Assessment

In the email, include the following:• Business question / intent of the research• Key stakeholders • Target audience

A response will be provided to you within 5 business days of receipt.

Can’t classify the approach or formulate the business question?

SOP-016162 Market Research Program Initiation and Adverse Event and Product Complaint Reporting

SOP-af-011237 Drug Safety Individual Case Report (ICSR) and Product Quality Compliant (PQC) Reporting Template – Market Research

SOP-015682 Development and Management of UCB Patient Value Solutions

SOP-015032 Study Management – Interventional and Prospective Non-Interventional Studies

SOP-014704 Retrospective Studies

SOP-016275 Digital Initiatives

SOP-015032 Study Management – Interventional and Prospective Non-Interventional Studies

SOP-009229 Company-Sponsored Post Authorization Safety Studies

Learning to ActionLearner Reflection and Application


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What did I learn? List 3 key takeaways:

How can I apply what I have learned?

If I applied these learnings, what benefits could I achieve?

Follow-up items:

Coaching Tips


1. Set the Stage for the Transfer Discussion

Goal: To discuss learner key takeaways from the RWE e-learning and to explore how these learnings might be best applied.

o How will you apply the learnings from this module? Were there new learnings for you? What were they?

o Do you have any questions about any of the content that you would like me to address with you?

o One of the key take-homes from the e-module is that it is important to systematically assess the business question and purpose or intent of the research, in order to identify the appropriate research approach. Do you have any questions about the Decision Tree and how to use it?

o Are there other resources or tools that you, or your team, might need to better understand the approaches and how to select the right approach?

2. Questions to guide the discussion:

Learning transfer is the process of putting learning to work in a way that improves performance.

Learning Activity

Application of Learning

(on-the-job)

Behavior Change

Any questions, comments or suggestions?

Please don’t hesitate to reach out to us via e-mail @ [email protected]

Documents

Decision Tree for Selecting the Right Research …...Introduction This Learner Guide is a resource to supplement the eLearning module: Decision Tree for Selecting the Right Research