Six work packages have been defined to achieve the objectives of NEED. WP1 will be dedicated to the project coordination, project management and reporting. WP2 will develop the scientific methods and procedures for a solid research infrastructure. WP3 will select topics for the proof-of-concept of the NEED approach. WP4 will perform case studies according to the methodology described in WP2 on the topics selected in WP3. WP5 will address the data management aspects of NEED, and WP6 will develop and execute a dissemination and exploitation plan for the NEED database. WP1, WP5 and WP6 are transversal packages.
WP1. Coordination, project management and reporting
NEED is coordinated by KCE (Irina Cleemput) and Sciensano (Robby De Pauw). Principal investigator is Charline Maertens de Noordhout and project manager is Nathalie Swartenbroekx. The coordination team meets weekly to discuss the progress and plan next steps.
WP1 comprises 2 tasks:
- General project management: To steer and monitor the progress of the different work packages, all consortium partners form a steering committee that meets at least twice a year.
- Reporting: Reporting is done as per BELSPO requirements, including the elaboration of annual progress reports.
WP2. Development of the scientific bases for a solid research infrastructure using transparent and scientifically sound methods and procedures
This WP provides the conceptual basis of the NEEDs infrastructure (i.e. the NEED framework) and creates process notes and methodological guidance for the different steps in the selection of needs research topics and in the actual assessment of patient and societal needs.
WP2 comprises 4 tasks:
- NEED framework development: An “unmet needs framework” will be developed, building on existing or ongoing scientific work in the area of unmet needs. This framework will include criteria to define individual patient and societal needs (e.g. severity of the disease, prevalence of the disease), types of evidence for each of the criteria (e.g. quantitative or qualitative data), and methods to generate (new) scientific evidence to assess these criteria (e.g. literature review or the analysis of primary or routine data).
- Definition of prioritisation criteria and development of processes for the identification and prioritisation of health conditions for unmet needs research: Because not all health conditions can be examined at the same time, a procedure for assessing and prioritising health conditions, based on specific prioritisation criteria will be developed.
- Assessment processes: Methodological approaches for the data analysis will be adapted according to the selected criteria. In general, the data analysis will include a step-wise approach including (1) a data quality check, (2) the identification of subpopulations (e.g., specific age groups or sexes), and (3) final calculations. For data collection through the survey and interviews and subsequent analysis of these data, the methodology as proposed in KCE report 348 will be followed. This encompasses (1) a literature review on the health condition, its symptoms, its treatment(s) and the effectiveness of this (these) treatment(s), (2) the use of a generic questionnaire (to be adapted to the condition under consideration, based on knowledge about treatments and symptoms from the available literature) to survey patients with that condition (or their representatives if they are unable to participate themselves), and (3) qualitative research for an in-depth examination of the results of the survey.
- Guidance for updating the evidence in the NEED database: The regular updating of the database is necessary, as new health-related interventions will be developed over time and certain needs included in the database might no longer be relevant/applicable. NEED will develop guidance on how to update the information in the database.
WP3. Selection of research topics for the proof-of-concept of the NEED approach
Based on the data related to the prioritisation criteria for the research topics defined in WP2, health conditions will be selected for the case studies that will serve as a proof-of-concept.
WP3 comprises 3 tasks:
- Analysis and improvement of databases containing (an) indicator(s) for patient and societal unmet needs, as a tool for the prioritisation of research topics: The prioritisation of research topics will be based on the minimum set of criteria as defined under WP2 (task 2.2). Within the Belgian Burden of Disease (BeBOD) framework, criteria related to disease burden and costs are calculated for a limited set of health conditions with yearly updates. The current list of health conditions will be updated based on the results of the stakeholders meeting; whereby suggested health conditions will be added to the BeBOD framework. The addition of health conditions will be based on the methods already developed and published by Sciensano.
- Call for topics (patients, patient organisations, healthcare providers, general public): A call for proposals will be launched at regular time intervials via the NEED website. The call will invite patients, patient organisations, healthcare professionals and the general public to highlight health conditions for which there are high patient and/or societal unmet needs.
- Prioritisation of topics/diseases/populations: submitted topics will be mapped with the results of the analysis of databases (see above) if possible. Prioritisation of topics to be tackled will happen via a standardised procedure as developed in WP2, involving the scoring of topics according to predefined criteria. A separate ranking will be made for patient needs and societal needs.
WP4. Case studies
Case study topics will be selected from the short list generated by WP3. They will be performed according to the procedures and using the methodology described by WP2.
WP4 comprises 5 tasks:
- Analysis of databases: An in-depth analysis of the selected health conditions will be carried out to estimate the unmet needs for each health condition. This process will be based on the criteria that were defined and developed in WP2 (Task 2.1). This task includes four steps: (i) Information Security Council (ISC) and Ethical Committee (EC) approval, (ii) data source identification, (iii) data linkage and (iv) data analysis.
- Literature review: For the selected topics, standard approaches to systematic literature review as used by KCE will be applied (KCE process book for literature reviews). The objectives of the literature review are: (1) to identify existing evidence on patient and societal needs and the burden of the condition, (2) to collect information on the available treatments, their effectiveness, the inconvenience of current treatment and possibly other disease- or treatment related aspects as defined by WP2; and (3) collect input for the required modifications to the generic patients' unmet needs questionnaire.
- Survey to explore patient needs and analysis of responses: The generic patient needs survey developed by KCE will be adapted to the health condition under consideration, based on the results of the literature review. If the population size allows it, pre-testing of the survey with a limited number of patients will be performed. KCE procedures for patient involvement will be applied for the recruitment of patients to take part in the survey (KCE report 340). Everything will happen in accordance with the General Data Protection Regulation (GDPR) and principles of ethical conduct of research. The data resulting from the survey will mainly be analysed quantitatively.
- Qualitative data collection and analysis for an in-depth investigation of the meaning of the quantitative results from the survey and databases (for patient needs only): Needs identified through the survey will be further explored via qualitative research techniques, such as individual interviews, focus groups, an online forum, etc. It might also be worthwhile also to include healthcare providers and caregivers in this step as key informants, especially if they experience large needs linked to the condition of the patients or if the patients have limited ability to express themselves. The same principles for patient involvement as specified for the survey will be applied for the qualitative data collection.
- Input in the NEED database: The data obtained from the database analysis, the literature review, survey and qualitative research will be introduced and stored in the NEED database.
WP5. Data management
The NEED project will use a variety of data sources. Ethical and legal clearances will be submitted to the concerned parties. The main task in WP5 is to follow the required steps as described in the Data Management Plan. Soma data will be required at patient level, other data will be aggregated.
Figure: Example of information flow between different data holders
WP6. Dissemination and exploitation
Dissemination and awareness raising activities regarding the NEED approach will be organised and active presentation to the networks of all consortium partners will be pursued along the duration of the project.
WP6 comprises 3 tasks:
- Organisation of a high-level conference with ministerial debate and annual meetings: In May 2024 a high-level conference with ministerial debate will be organised by the Belgian Presidency of the European Council. The conference will be organised by the working group on unmet medical needs, which is led by FAMHP, NIHDI and KCE. Sciensano is involved as member of the working group. The objectives and achievements of NEED will be presented. Three annual meetings open to all stakeholders and the advisory committees will be organised to present the progress and intermediary results of the project. At the end of the project, a final meeting will be organised for all stakeholders to present and disseminate the research infrastructure and its sustainability plan.
- Creation of a website
- Presentations at (inter)national scientific conferences and publications in peer-reviewed journals
