Project Structure

The overall project consists of eight Work Packages. The project management and scientific coordination (WP1), four core applications (WP2-5) and three cross-cutting themes (WP6-8).

WP1
Project Management and Coordination
WP2
Outcome Definition
WP3
Identification, mapping and integration of RWE
WP4
Disease Modelling and Simulation
WP5
Health Economics
WP6
Regulatory and HTA Engagement
WP7
Communication and Patient/Healthcare Provider Engagement
WP8
Ethical, Legal and Social Implication (ELSI)
WP1: Project Management and Coordination
Carlos Diaz - Synapse Research Management Partners S.L (SYNAPSE)
Frédéric De Reydet De Vullpilliers - Novartis Pharma AG (Novartis)
WP1 will provide scientific coordination and project management to ensure progress and successful completion of the Project.
WP1 will provide scientific coordination and project management to ensure progress and successful completion of the Project.
Overall objective: Develop a full plan for phase 2 of the ROADMAP initiative that addresses identified gaps and pitfalls, and exploits promising solutions to their full potential for development of a European RWE platform in AD (WP1)

Objectives

  • Establish a project management and governance structure enabling efficient execution of the Project, including decision making, conflict resolution, reporting and accountability
  • Manage resources, procedures and tools to deliver the Project to plan, within time and budget, including risk management and quality control procedures on deliverables
  • Ensure effective communication and work dynamics between consortium members to help drive the whole Consortium as a team towards successful completion of the Project, with special attention to interactions between WPs
  • Ensure appropriate administration, financial allocation/reporting, legal management and logistics in support of the project
  • Develop the necessary mechanisms to ensure coordination with the Big Data for Better Outcomes programme, and collaboration with other relevant projects and initiatives
  • Integrate results from all other WPs into a credible roadmap for phase 2 of ROADS
WP2: Outcome Definition
Catherine Sudlow - University of Edinburgh (UEDIN)
Christin Bexelius - F. Hoffmann‐La Roche Ltd (ROCHE)
WP2 aims to obtain consensus on a set of real-world AD focused outcomes across the spectrum of the patient journey from pre‐clinical to severe stages of the disease.
WP2 aims to obtain consensus on a set of real-world AD focused outcomes across the spectrum of the patient journey from pre‐clinical to severe stages of the disease. The set of outcomes generated will be those of relevance to patients, carers, scientists, clinicians, regulators, HTA bodies, payers and policy makers.
Overall objective: Define and catalogue scales and consensus‐based methodologies for identifying AD outcomes from routinely collected data (WP2)

Objectives

  • Identify the ‘universe’ of outcomes relevant to the analysis (including cognition, behaviour, mental health, quality of life, service delivery, cost, social acceptability, etc.). Outcomes will be classified according to stakeholder relevance (patient, carer, clinician, scientist, policy maker) and prioritised according to scientific, service delivery, and economic impacts
  • Evaluate and determine what constitutes a meaningful delay in disease progression from clinical and economic perspectives and how this can be expressed using real-world Data, including the translation from clinical trial data to the real-world setting
  • For markers of priority outcomes and delayed progression, evaluate what is known of the validity and reliability of existing measures, and identify gaps between data requirements and currently available data
WP3: Identification, mapping and integration of RWE
Pieter-Jelle Visser - Universiteit Maastricht (UM)
Antje Hottgenroth - Eli Lilly and Company Ltd (Eli Lilly)
WP3 will address the challenges related to finding, extracting, harmonising, integrating and analysing data from RWE sources relevant to AD.
WP3 will address the challenges related to finding, extracting, harmonising, integrating and analysing data from RWE sources relevant to AD.
Overall objective: Identify and pool AD‐related RWE data and establish solution options for how to combine different RWE sources with RCT data supporting pharmacoeconomic evaluation (WP3)

Objectives

  • Identify and assess data sources across the disease stages of AD that include clinical/health outcomes and economic outcomes (e.g., costs, utilities, QOL) of relevance to stakeholders
  • Develop pilot exercises of data extraction, harmonisation, integration and analysis supporting the project Use Cases, evaluating different methodologies and tools and helping assess data availability and suitability, by leveraging existing platforms, technologies and data management methodologies, which are in alignment with the Provisional Recommendation on Data Management Technology for projects in the Big Data for Better Outcomes Program (BD4BO)
  • Establish guidelines on how to best combine data from different RWE sources
  • Establish guidelines on how to best combine RCT data with pragmatic and EHR‐based research into a holistic data package for key decision‐makers
  • Develop  data  solution  options  applicable  to  different  real‐world  outcome  measures  supporting pharmacoeconomic evaluation
  • Explore opportunities of mobile health solutions and perform a pilot project in specified geographies
WP4: Disease Modelling and Simulation
Billy Amzal - Novartis Pharma AG (Novartis)
Johan van der Lei - Erasmus Universitair Medisch Centrum Rotterdam (EMC)
WP4 will identify AD disease models and test them using outcomes and datasets identified through WP2 and WP3.
WP4 will identify AD disease models and test them using outcomes and datasets identified through WP2 and WP3.
Overall objective: Develop and validate a core disease progression model combining diverse datasets to facilitate analysis of disease trajectories and effect of interventions on disease trajectories (WP4)

Objectives

  • Identify existing AD disease models and risk factors from the literature focusing on understanding and prediction of disease incidence and prediction of disease incidence and progression
  • Establish a sequence of disease stages as a reference, and support Use Case 1 via a heat map ‘data cube’ of outcomes/variables, data sources and disease stage definitions, to be populated in collaboration with WP2 (outcome definitions) and WP3 (available data), in order to assess availability (cohorts, RWE, and possibly also trial data) and suitability of data for disease modelling in a way that is acceptable to stakeholders
  • Select disease models for validation based on availability of data
  • Validate selected disease models through confirmatory analyses (ideally in diverse settings) using independent datasets provided by ROADMAP partners
  • Support Use Case 2 with ‘hypothesis‐free’ analysis of disease trajectories
  • Support Use Case 3 by analysing pharmacological interventions from the modelling perspective
  • Use results in the three Use Cases to progressively enrich complex disease progression models in dialogue with other WPs
  • Establish guidelines for best practice on combining datasets for modelling and simulation in the domain of AD
  • In collaboration with WP2 and 3, document capabilities and actual use of available disease coding systems (e.g. ICD‐10, ICPC, DSM) to capture AD throughout the entire disease spectrum and, if needed, propose changes or additions
WP5: Health Economics
Alastair Gray - The Chancellor, Masters and Scholars of the University of Oxford (UOXF)
Antje Tockhorn - Eli Lilly and Company Ltd (Eli Lilly)
The focus of WP5 is the development of a proof‐of‐concept core health economic cost‐effectiveness model for the asymptomatic and early symptomatic AD stages (MCI due to AD, mild and moderate AD).
The focus of WP5 is the development of a proof‐of‐concept core health economic cost‐effectiveness model for the asymptomatic and early symptomatic AD stages (MCI due to AD, mild and moderate AD).
Develop a proof of concept AD cost‐effectiveness and budget impact model for HTA agencies, regulators, service providers, industry, payers and carers (WP5)

Objectives

  • Identify the specific data and analytical requirements of robust state‐of‐the‐art cost‐effectiveness (C‐E) analysis for Alzheimer Disease (AD) interventions (both symptomatic and disease‐modifying agents) initiated in the asymptomatic and early symptomatic AD stages (Mild cognitive impairment (MCI) due to AD, mild AD and moderate AD). Determine availability and suitability of specifically health economic data (e.g. resource use, unit costs, quality of life, treatment pathways and event probabilities) contributing to Use Case 1
  • Use relevant RWE datasets to improve the evidence base of long‐term AD C‐E Identify and review existing C‐E models for symptomatic and disease modifying interventions in the asymptomatic and early symptomatic AD stages (MCI due to AD, mild AD and moderate AD) and evaluate against manufacturer, academic and reimbursement requirements and current modelling standards. Evaluate emerging disease trajectory hypotheses across the entire AD spectrum within Use Case 2, in relation to quality of life, treatment costs, resource utilization and HTA modelling requirements in different European jurisdictions
  • Develop an integrated proof‐of‐concept core health economic model of healthcare use/costs and health outcomes, contributing to Use Case Use model as platform to evaluate medium/long‐term data requirements, requirements for HE models in the asymptomatic and early symptomatic AD stages (MCI due to AD, mild AD and moderate AD), ability to capture heterogeneity across patients and health care systems. The core health economic model shall be adaptable to the needs of various European jurisdictions (e.g. payer, societal perspective) and manufacturer requirements (e.g. modelling pre‐clinical and other stages of Alzheimer Disease)
WP6: Regulatory and HTA Engagement
Jacoline Bouvy - National Insititute for Health and Care Excellence (NICE)
Robin Thompson - Biogen (BIOGEN)
WP6 will provide the mechanism and platform for engagement of ROADMAP’s different work packages with experts from the regulatory, HTA and payer environment through the Expert Advisory Group (EXAG).
WP6 will provide the mechanism and platform for engagement of ROADMAP’s different work packages with experts from the regulatory, HTA and payer environment through the Expert Advisory Group (EXAG). The EXAG will enable ROADMAP to develop outputs that are not merely of high scientific quality but might have meaningful applicability in the regulatory, HTA, and payer contexts.
Overall objective: Develop guiding principles and recommendations for the development and incorporation of RWE into clinical and market access development plans for AD (WP6)

Objectives

  • Provide the mechanism for providing technical and process recommendations on the potential use of RWE for AD from the regulatory, HTA, and payer perspectives.
  • Provide a platform for liaising with regulatory, HTA, and payer experts on the type and quality of RWE that might be appropriate in the context of new interventions for AD.
  • Identify a possible RWE-specific regulatory/HTA pathway for AD with input from all healthcare system stakeholders.
  • Identify current challenges in marketing authorisation, pricing and reimbursement of interventions in AD and identify how ROADMAP activities might address these.
WP7: Communication and Patient/Healthcare Provider Engagement
Jean Georges - Alzheimer Europe (AE)
Laura Campo - Eli Lilly and Company Ltd (Eli Lilly)
WP7 will lead and coordinate external communication and dissemination activities, as well as involvement with and outreach to specific external stakeholders in particular the broader dementia community of patients, carers and healthcare professionals.
WP7 will lead and coordinate external communication and dissemination activities, as well as involvement with and outreach to specific external stakeholders in particular the broader dementia community of patients, carers and healthcare professionals.
Overall objective: Develop and implement a communication strategy focusing on the needs of patients and professionals (WP7)

Objectives

  • To design a plan that optimises communication within the project Consortium and the dissemination of information and knowledge generated by the project to the scientific community, other relevant stakeholders and society at large.
  • To collaboratively design and produce the communication framework including targeted channels and tools, that will be needed to implement the communication plan.
  • To effectively communicate project aims, progress and results to external stakeholders including patients and their caregivers, healthcare practitioners, regulators, payers, policy makers and the wider dementia community.
WP8: Ethical, Legal and Social Implication (ELSI)
Andrew Turner - The Chancellor, Masters and Scholars of the University of Oxford (UOXF)
Zuzanna Angehrn - Novartis Pharma AG (Novartis)
WP8 will map critical ethical, legal and social issues which arise from the core ROADMAP activity – creating a real-world Evidence platform to enable access and secondary uses of health data.
WP8 will map critical ethical, legal and social issues which arise from the core ROADMAP activity – creating a real-world Evidence platform to enable access and secondary uses of health data. WP8 also addresses ethical concerns and topics arising during the course of the project.
Overall objective: Develop an ELSI framework with extensive patient involvement for the development and application of RWE in AD (WP8)

Objectives

  • Map the requirements and critical issues for an ELSI framework for an RWE platform for AD.
  • Establish and manage an Ethics Advisory Board (EAB) to identify ethical and legal issues embedded in data integration tools developed by ROADMAP, and the recommendations for an RWE platform for phase 2 of the ROADS call.
  • Review of ethical issues in sensitive medical data re‐purposing and pooling for UC1, UC2 and UC3, including assessment and integration of prior work on the ethical and legal governance of biobanks and other biomedical Big Data platforms, as well as empirical studies of AD patient and carer attitudes towards an RWE approach and ethics and privacy challenges for an RWE approach in AD introduced by the EU General Data Protection Regulation.
  • Conduct stakeholder focus groups on ELSI issues arising in a RWE approach to inform UC1, UC2 and UC3.
  • Provide input on WP2, WP5 and WP7 stakeholder engagement activities concerning ethical dimensions of social outcomes (e.g. quality of life measurements) arising in developing the preference matrix.