Stakeholder Actions to Generate Better Real-World Evidence for HTA/Payers
The RWE4Decisions Learning Network has developed a new set of Stakeholder Actions to support stakeholders across the ecosystem in better RWE generation and use that respond to HTA/Payer needs.
We want to hear from you!
New Stakeholder Actions
Building on the success of the 2020 Stakeholder Actions, the RWE4Decisions Learning Network collaborated throughout 2024 to co-create updated recommendations for seven key stakeholder groups: national HTA/Payers, HTA/Payer collaborations, the pharmaceutical industry, clinical teams, patient groups, disease registry holders, and RWD analytics experts.
These revised Actions reflect changes in the decision-making environment, progress made, and remaining challenges to RWE adoption. They emphasize the need for cross-sector collaboration and partnerships to generate robust, value-based RWE.
The updated Stakeholder Actions were launched at the 2024 RWE4Decisions Symposium in Brussels and presented at the ISPOR EU RWE Summit in Barcelona.
Four pillars for better RWE in HTA/Payer decision-making
Four pillars were identified in a previous paper as key to shaping the generation of robust RWE for HTA and Payers.
With more opportunities for secondary use of RWD, as well as methodological advancements in building/reporting RWE, stakeholders across the health ecosystem have worked together to improve RWD quality and accessibility, guidance for researchers generating RWE, data analytics through digital transformation. Nonetheless, the lack of harmonisation among regulators, HTA bodies and Payers calls for a joint vision of RWE – what can be produced, by whom and for what purposes.
The new Stakeholder Actions represent the RWE4Decisions Learning Network’s vision for closer collaboration – the actions are highly intertwined and are designed to meet the specific challenges of each stakeholder group from an ecosystem approach.
National Payers/HTA Bodies
POLICY and PARTNERSHIPS
1.1 Shared vision on use of RWD
Create a shared vision that conceptualizes a future state for the use of real-world data (RWD) in health technology assessment (HTA)/Payer processes. Ensure that clear roles and responsibilities are designated for oversight and achievement of the shared vision.
1.2 Overcome fragmentation and lack of collaboration
Overcome fragmentation and lack of collaboration between HTA bodies and payers by implementing the necessary infrastructures, aligning processes, and upskilling competencies for effectively requesting, producing, and utilizing real-world evidence.
DATA AVAILABILITY and GOVERNANCE
1.3 Collaborate via HTA/Payer networks to align PLEG requirements
Collaborate with other HTA bodies and Payers (e.g. via networks such as the National Competent Authorities for Pricing and Reimbursement (NCAPR) or the Medicine Evaluation Committee (MEDEV)) and regulatory authorities to align post-launch evidence generation (PLEG) requirements needing national data collection. Focus on critical endpoints and requirements for data quality.
1.4 Engage with companies on RWE generation plans
Initiate and engage in multistakeholder discussions with companies about RWE generation plans. Determine which data are transferable and identify additional local RWD that may be needed to inform decision-making, aligning with other jurisdictions when possible.
1.5 Influence the development of national secondary use governance frameworks
Influence national developments on the secondary use of health data. Communicate HTA/Payer needs regarding, for example, types of data, data linkage and data quality, ensuring these needs are considered and integrated into national governance frameworks.
1.6 Require feasibility assessments, study protocols, etc. to be made public
Require that feasibility assessments, study protocols, details of data extractions and study reports are made publicly available.
1.7 Share case studies on RWE influence over P&R and reassessments
Publish examples where RWE has influenced pricing and reimbursement decisions or reassessments. Also share case studies that identify methodological areas requiring development.
Payer/HTA Collaborations
POLICY and PARTNERSHIPS
2.1 Leverage regional collaborations for coordinated use of RWD in decision-making
Use HTA/Payer collaboratives (such as Joint Nordic HTA-Bodies, the Beneluxa initiatve etc) to encourage and enhance joint work on use of RWD in initial access decisions, managed entry agreements and re-assessments.
2.2 Develop joint voice on RWD issues to feed into EU-level policy issues
Develop a joint voice on RWD issues to feed into policy and system developments such as the European Health Data Space (EHDS) and the Data Analysis and Real-World Interrogation Network (DARWIN-EU) and the implementation of the HTA Regulation, with clear accountability for this task potentially assigned to an entity such as NCAPR.
2.3 Co-create RWD/E learning platforms with other stakeholders
Co-create joint learning platforms with other decision-makers and stakeholders in the RWD/E community.
2.4 Work with regulators to align on RWD/E methods and guidance
Work with Regulators to understand and influence their international activities to develop harmonized methods and guidance for RWD collection. In Europe, incorporate RWD/E needs and guidance in the implementation of the HTA Regulation through Joint Scientific Consultations and Joint Clinical Assessments.
2.5 Provide early scientific advice on RWE generation over the product lifecycle
Collaborate to expand Early Dialogue/scientific advice/Joint Scientific Consultation to cover RWE generation throughout the product life cycle to address uncertainties in clinical and cost effectiveness. Recognise the need for agile processes to adapt to evolving knowledge, treatment options and RWD and RWE requirements.
2.6 Support industry in expanding post-authorisation studies to also address HTA/Payer needs
Encourage and support industry to consider possibilities to expand post-authorisation studies and data collection plans intended to support regulatory decision making to also address HTA/Payer evidence needs.
2.7 Promote open access to findings to design a shared understanding of HTA requirements with other stakeholders
Collaborate with companies, clinical teams, academia and other stakeholders on study protocols, study governance, analyses, and reporting to encourage a common understanding of HTA requirements and to promote open access to documents and findings.
DATA AVAILABILITY and GOVERNANCE
2.8 Co-design and streamline shared frameworks for data quality assessment and standardisation with regulators
Collaborate with regulators on common frameworks for data quality assessment, data standardisation efforts and methodologies for feasibility assessment. Advise health data holders of the common requirements so that they can develop their datasets accordingly.
METHODOLOGY
2.9 Pilot the use of RWD/E methods guidance in assessment and share learnings
Pilot the use of existing RWD/E methods guidance and tools in assessments. Share feedback with guidance authors and HTA/Payer community.
2.10 Co-develop harmonised RWE guidance with academia and industry to support HTA Regulation implementation
After piloting existing guidance, develop harmonized RWE guidance jointly with academia and industry, particularly to support implementation of the HTA Regulation. Review the guidance regularly to take account of new methodological developments and experience of use (living guidance).
TRUST and TRANSPARENCY
2.11 Build a portal of publicly available examples of post-launch RWD collection and RWE generation requirements
Compile available documents (protocols and reports) describing examples of post launch RWD collection and RWE generation required by HTA/payers, utilizing an existing portal if available.
TRUST AND TRANSPARENCY
3.1 Engage with HTA/Payers throughout the product lifecycle on integrating RWE into evidence generation plans
Discuss plans for RWE generation as part of industry (integrated) evidence plans at scientific advice meetings. Do this at several points during the lifecycle of the medicine, to develop understanding of what RWE may be valuable to HTA/Payers as clinical evidence and knowledge about the technology/disease evolves.
3.2 Ensure transparency in pivotal RWE study design, execution and analysis
Ensure transparency around the design, conduct, and analysis of RWE studies that are agreed to be pivotal to health technology assessment (HTA)/Payer decision making, e.g. using published tools to document data capture, management and analysis, following RWE guidance/frameworks.
POLICY and PARTNERSHIPS
3.3 Vertically and horizontally integrate in-house knowledge to map national/regional HTA & Payer RWE needs
Work together across company functions and national affiliates involved in RWE generation and analysis to develop a common understanding of national/regional HTA/Payers needs for RWE.
3.4 Cooperate on RWD transportability alignment across borders
Lead discussions about transportability of real-world data (RWD) across borders and support efforts to align data collection requirements across jurisdictions.
3.5 Drive multi-stakeholder discussions about OBMEA and PLEG alignment
Continue to drive discussions about use of, and alignment of, Outcomes-Based Managed Entry Agreements (OBMEA)/Post-Launch Evidence Generation (PLEG).
DATA AVAILABILITY and GOVERNANCE
3.6 Digitise the collection of patient-relevant outcomes
Explore use and analysis of digital apps to capture patient-relevant outcomes, particularly to inform OBMEA.
3.7 Collaboratively explore with other developers synergies in RWD collection and analysis
Collaborate across companies (e.g. in a specific disease area or for a class of medicines) to agree on synergies in methods and processes for RWE in that specific case (e.g. core datasets, analytical approaches, etc).
3.8 Ensure data quality and interoperability through early engagement with clinical networks and registries
At an early stage of product development, engage with clinical networks to create or further develop registries and databases following unified data standards, to collect high quality and interoperable data.
METHODOLOGY
3.9 Support the implementation of the EU HTA Regulation by advocating for clear guidance on RWE in JCA dossiers
Engage and support operationalisation of the HTA Regulation to highlight need for RWE in the first two tranches of JCAs and encourage development of clear guidance about assessment of RWE in the EU HTA context.
3.10 Enable exchanges of best practices and learnings from submissions of RWE to HTA or post-launch evidence requirements
Share, publish and enable discussion of case studies to show how RWE has been assessed and used in HTA/Payer decision-making (in assessment and post-launch), focussing on specific issues (e.g. external control arms, transportability), and challenging cases scenarios (e.g. rare disease) etc.
POLICY and PARTNERSHIPS
4.1 Support the European Health Data Space to raise awareness about the value of secondary use of health data
EU clinical networks should engage in developments related to the European Health Data Space to support public and political awareness of the value of secondary use of health data.
4.2 Involve patients and collaborate with other clinical networks, regulators and HTA/Payer bodies in the development of fit-for-purpose data collection systems for patient-relevant outcomes
EU clinical networks and clinical trials collaborative groups (such as European Reference Networks, the EU cancer mission networks, the European Organisation for Research and Treatment of Cancer and other disease specific study groups) should systematically involve patients to collect patient relevant outcomes including nutritional status and co-morbidities, and collaborate with regulators and Payer/health technology assessment (HTA) bodies to ensure data collection systems are fit for all purposes, including HTA requirements pre and post launch.
4.3 Support educating medical practitioners and hospital staff on the importance of health data collection and use
Medical faculties should include educational programmes on the value of health system data to improve delivery of care, patient outcomes and inform health system decision making, such as HTA evaluations of new healthcare interventions.
DATA AVAILABILITY and GOVERNANCE
4.4 Advise health systems on the most efficient and high-quality collection of RWD by clinical teams
Clinical networks and study groups should advise on the most suitable and efficient way for health systems to collect real-world data (RWD) to avoid multiplicity of data entry and clarify the support clinical teams require to collect good quality RWD.
4.5 Involve clinical teams and patients in the design of data collection systems and governance structures
Clinical networks and clinical trials collaborative groups should encourage health systems to involve clinical teams and patients in the design of data collection systems and associated governance structures to ensure processes are efficient and clarify the support clinical teams require to collect good quality real world data.
4.6 Ensure patient-centricity in local information governance systems for informed consent-based processes
Clinicians should seek to ensure that local information governance systems are designed to deliver optimal care for patients (across providers and departments), and to encourage informed consent processes based on unified ethical principles that are intelligible to patients, in all required languages.
POLICY and PARTNERSHIPS
5.1 Empower patients’ role in RWE generation for informed HTA and policy decision-making
Ensure that opportunities and resources to develop patient expertise in the field of RWD are clearly communicated to the patient community to develop skills that support multi-stakeholder and patient-centred generation of real-world evidence (RWE) to inform health technology assessment (HTA) and to engage in policy and system developments relating to use of health data.
5.2 Engage in international consortia, collaborations and policies relevant to the RWD space
International patient groups should continue to engage in real-world data (RWD) initiatives, such as Innovative Health Initiatve (IHI) projects, regulatory and HTA led activities, and policy developments such as the European Health Data Space (EHDS).
5.3 Actively engage in the roll-out of the EHDS to shape data collection infrastructures for the benefit of patients’ access to their data
Seek to influence the implementation of the EHDS and understand its implications for national data collection systems, in particular to ensure that patients have access to their own data.
5.4 Encourage alignment on RWD identification, collection and evaluation for HTA/Payer decision-making
Support development of a process for iterative multi-stakeholder dialogues throughout the lifecycle of a medicine to encourage alignment of views on identification, collection, analysis and evaluation of RWD for decision-making.
DATA AVAILABILITY and GOVERNANCE
5.5 Enhance patient participation in secondary health data uses by ensuring efficient consent-based processes
Support the development of efficient informed consent processes for secondary use of health data. This will necessitate a rethinking of the type of consent model in use from ‘broad to ‘dynamic’ to encourage accelerated and increased patient participation.
5.6 Educate patient communities on the importance of RWE/RWD for secondary uses
Disseminate clear, unbiased, patient-relevant information about RWD and RWE to patient communities, including the value of secondary use of data and information to support Post-Launch Evidence Generation.
METHODOLOGY
5.7 Engage in multistakeholder discussions on novel collection methods for patient-relevant data
Engage with clinicians, academics and decision-makers to discuss how patient-relevant data from novel collection methods (such as wearables, apps etc) can be used in decision-making, to help build a predictable pathway for use of these novel RWD collection approaches.
5.8 Explore synergies between patient group-owned disease registries and other data repositories for highly innovative medicines
Patient Groups who might also be disease registry holders or have developed disease specific data collection approaches, should agree with stakeholders how to integrate such evidence with other data for highly innovative medicines.
DATA AVAILABILITY and GOVERNANCE
6.1 Explore the potential of disease registries to support regulatory and HTA/Payer decision-making
Participate in multi-stakeholder dialogues about real-world evidence (RWE) generation for a specific medicine, or group of medicines, to discuss the potential for a disease registry to be used for regulatory and health technology assessment (HTA)/Payer purposes and ensure realism about data quality (availability for main outcomes and trade-offs for different data capture algorithms).
6.2 Form multistakeholder partnerships to support use of data for HTA/Payer purposes
Form multi-stakeholder partnerships to support use of data for HTA/payer purposes, including public/private partnerships.
TRUST and TRANSPARENCY
6.3 Support multistakeholder alignment on the role of disease registries and RWE studies in HTA in light of existing tools
Engage with HTA/Payers and industry to explain the construct and purposes of disease registries, discuss their potential and limitations, and agree with HTA/Payers how disease registries and RWE studies will be assessed, building on existing tools (e.g. tools to report registry quality overall, and fitness-for-purpose evaluations for individual RWE studies).
6.4 Profile examples of disease registries RWD supporting HTA/Payer decision-making
Share and publish case studies of where real-world data (RWD) from disease registries has been used in to support HTA/Payer decision making.
DATA AVAILABILITY and GOVERNANCE
6.5 Ensure appropriate data governance and interoperability of registry-based health data for HTA/Payer purposes
Develop governance processes that enable sharing of disease registry data and linkage to other data sources (e.g. administrative data) for HTA/Payer purposes (including patient consent mechanisms, protocols, data management etc).
6.6 Align RWD standards to ensure data meets HTA needs for quality standards
Align to RWD standards to ensure data meets quality standards required by HTA bodies.
METHODOLOGY
7.1 Contribute to exchanges of knowledge and best practices on fit for HTA purposes RWE
Support development of a repository of empirical evidence/case law about what real-world evidence (RWE) was fit for purpose in health technology assessment (HTA) and what was not, as well as RWE case studies that highlight data quality and methodology examples.
7.2 Promote and disseminate methodologies fit for HTA/Payer concerns in RWE uptake
Continue to operationalise and share/publish methodologies that address known HTA/Payers’ concerns with use of RWE through HTA-friendly tools, demonstration projects, case studies etc.
7.3 Build RWE analytics knowledge base and support RWE assessment under EU HTA
Build RWE analytics knowledge base and support RWE assessment and generation within HTA bodies linking to new policy initiatives such as the HTA Regulation.
7.4 Collaborate with HTA bodies on demonstration projects to help build a mutual understanding and trust in RWE
Work with HTA bodies to share and operationalize the implications from available demonstration projects (e.g., emulation studies, newly developed methods, tools, repositories etc) to build mutual understanding and trust in RWE; explore which are most helpful or need adaptation and support collaboration to create a harmonized RWE toolkit.
7.5 Contribute to a mapping of HTA/Payer needs across jurisdictions and promote the harmonisation of fit-for-purpose methods
Raise awareness of differences between HTA body RWE frameworks and how these differences relate to differences in HTA body remits and RWE needs. Where appropriate, engage in dialogue with HTA bodies to encourage harmonization of fit-for-purpose methods.
7.6 Support HTA bodies in developing clear guidance articulating broad multistakeholder agreement
Where there is substantial evidence and agreement across stakeholders on use cases, standards, and analytical methods and where articulating guidance would benefit researchers, support HTA bodies in developing detailed published guidance.
7.7 Consolidate validated definitions of key aspects covering diagnoses, outcomes, covariates into a world-wide library
Create a standard world-wide library of definitions for key aspects covering diagnoses, outcomes, covariates etc and algorithms that have been validated.
TRUST and TRANSPARENCY
7.8 Support industry in following published standards for RWE generation
Work with industry to ensure they are following published standards/best practices for RWE generation (e.g., transparency, data quality, etc).
7.9 Support data custodians in standardising documentation for RWE studies
Work with data custodians to explain the requirements of decision-makers to clearly demonstrate data quality and encourage standardised documentation they can use for all RWE studies.
Stakeholder Implementation Stories
RWE4Decisions will continue to monitor the fitness of the Stakeholder Actions, feeding information from their real-world application into multistakeholder discussions and subsequent updates.
We are eager to learn from you about your journey implementing the Stakeholder Actions – share your story to help us ensure better RWE generation and use in the future.
Your Implementation Stories to be shared here soon!
HTA bodies and Payers
Pharmaceutical Industry
Clinical Teams
Patient Groups
Disease Registry Holders
RWD Analytics Groups
Glossary of terms
Beneluxa
Collaboration to ensure patients’ access to expensive medicines through horizon scanning, information sharing, HTA, and pricing and reimbursement involving Belgium, Netherlands, Luxembourg, Austria and Ireland https://beneluxa.org/collaboration.
DARWIN EU
Data Analysis and Real World Interrogation Network https://darwin-eu.org/
EHDS
European Health Data Space https://health.ec.europa.eu/ehealth-digital-health-and-care/european-health-data-space_en
EU
European Union
EUnetHTA
European Network for HTA
HTA
Health Technology Assessment
HTAR
EU Regulation on Health Technology Assessment https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX:32021R2282
IHI
Innovative Health Initiative https://www.ihi.europa.eu/
JCA
Joint Clinical Assessment (under the HTAR)
NCAPR
National Competent Authorities on Pricing and Reimbursement
OBMEA
Outcome-Based Managed Entry Agreement
P&R
Pricing and Reimbursement
PLEG
Post-Launch Evidence Generation
RWD
Real-World Data
RWE
Real-World Evidence