Payers and HTA Bodies
National Payers / HTA Bodies
Vision: The HTA/Payer Community needs to work together to inform the health system of the specific RWD needs essential for their decision making and procurement processs.
Payers and HTA bodies need to work together to:
POLICY and PARTNERSHIPS
1.1. Create a shared vision that conceptualizes a future state for the use of RWD in HTA/Payer processes, ensuring a specific body is designated as accountable for overseeing and achieving this vision.
1.2. Overcome the lack of collaboration and fragmentation between HTA bodies and payers by developing joint processes and upskilling competencies for requesting, producing, and utilizing RWE effectively. RWD collection for individual assessments should be aligned in terms of critical endpoints and data requirements for value assessment, decision-making, managed entry agreements and regulatory needs.
1.3. Collaborate across jurisdictions to align PLEG requirements (e.g. via NCAPR/MEDEV) and develop a shared interpretation of the HTA Regulation to understand how to collaborate to support PLEG.
TRUST and TRANSPARENCY
1.4. Communicate details about how RWD/RWE has been assessed, gather case studies of what RWE has been deemed fit for purpose and clarify what methodological areas need development for HTA purposes.
1.5. Enhance transparency of PLEG through sharing of feasibility assessments, data collection protocols, and case-studies showing how RWE has influenced decisions (in assessment/P&R deliberation and reassessment/revised P&R).
1.6. Build trust by encouraging all stakeholders involved in HTA to engage in design, management and reporting of additional RWD collection.
DATA
1.7. Influence national developments on secondary use of health data so that the needs of managed entry are considered and integrated into national governance frameworks.
1.8. Discuss global evidence generation plans with HTD, to determine which data are transferable and what additional local RWD (that could be aligned with other jurisdictions) may be needed.
Payer/HTA Collaborations
POLICY and PARTNERSHIPS
2.1. HTA/Payers need to develop a joint voice on issues to feed into policy and system developments such as EHDS, DARWIN EU, with clear accountability for this task potentially assigned to an entity such as NCAPR.
2.2. Use HTA/Payer collaboratives (such as FINOSE, BENELUXA etc) to encourage and enhance joint work on use of RWD.
2.3. Improve relationship with Regulators to co-design RWD collection to suit the needs of regulators and Payers.
2.4. Encourage MAHs to discuss PASS/PAES plans with HTA/Payers to consider if the studies can be expanded to include HTA/Payer needs.
2.5. Collaborate with academia and clinicians on study protocols, study governance, analyses, and reporting to encourage common understanding of HTA requirements
2.6. 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, recognising the need for agile processes to adapt to evolving knowledge, treatment options and data and evidence requirements.
METHODOLGY
2.7. Collaborate with regulators on frameworks for data quality and methodologies for feasibility assessment so that registry holders can develop their datasets to meet common requirements.
2.8. National HTA bodies or Collaboratives should pilot use of trusted RWE methods guides and tools in their assessments, and share feedback with guidance authors and HTA/Payer community.
2.9. After piloting existing guidance, harmonized RWE guidance should be developed jointly with academia, particularly to support implementation of the HTAR. This should be reviewed regularly to take account of new methodological developments and experience of use (living guidance).
TRUST and TRANSPARENCY
2.10. Create a public portal for publicly available documents relating to PLEG (protocols and reports).
Pharmaceutical Industry
3.1. 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 around the design, conduct and analysis of RWE studies that are agreed to be pivotal to HTA/Payer decision making, e.g. using published tools to document data capture, management and analysis, following RWE guidance/ frameworks.
3.3. Work together across company functions involved in RWE generation and analysis to develop a common understanding of national/regional HTA/Payers needs for RWE.
3.4. Lead discussions about transportability of RWD across borders and support efforts to align data collection requirements across jurisdictions.
3.5. Explore use and analysis of digital apps to capture patient-relevant outcomes, particularly to inform innovative pricing agreements.
3.6. Continue to drive discussions about use of, and alignment of, OBMEA/PLEG.
3.7. 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.
3.8. 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).
3.9. Engage and support operationalisation of the HTAR 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. At an early stage of product development, engage with clinical networks to create or further develop registries and databases that collect high quality and interoperable data.
Clinical Teams
4.1. 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. EU clinical networks (such as European Reference Networks and the EU cancer mission networks) should systematically involve patients to collect patient relevant outcomes and collaborate with regulators and Payer/HTAs to ensure data collection systems are fit for all purposes, including HTA requirements pre and post launch.
4.3. Clinical networks should advise on the most suitable and efficient way for health systems to collect RWD (to avoid multiplicity of data entry) and clarify the support clinical teams require to collect good quality real world data.
4.4. Clinical networks should encourage health systems to involve clinical teams 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.
4.5. 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 that are intelligible to patients.
4.6. 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.
Patient Groups
5.1. 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 RWE to inform HTA and to engage in policy and system developments relating to use of health data.
5.2. International patient groups should continue to engage in RWD initiatives, such as IHI projects, regulatory and HTA led activities, and policy developments such as the European Health Data Space.
5.3. 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.
5.4. 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 Licensing Evidence Generation.
5.5. 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.
5.6. 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.
5.7. 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.
5.8. Patient Groups who might also be Disease Registry Holders or have undertaken QoL surveys, or PRO data collection, or be holders of patient-relevant data from novel collection methods, should agree with stakeholders how to integrate such evidence with other data for highly innovative medicines.
Disease Registry Holders
6.1. Engage with HTA/Payers 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 (develop REQuEST v2.0?), and fitness-for-purpose evaluations for individual RWE studies (e.g. use SPIFD)).
6.2. Participate in multi-stakeholder dialogues about RWE generation for a specific medicine, or group of medicines, to discuss the potential for a disease registry to be used for regulatory and HTA/Payer purposes and ensure realism about data quality (availability for main outcomes and trade-offs for different data capture algorithms).
6.3. 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).
6.4. Share and publish case studies of where RWD from disease registries has been used in to support HTA/Payer decision making.
RWD / Analytics Groups
7.1. Support development of a repository of empirical evidence/case law about what RWE was fit for purpose in HTA and what was not, as well as RWE case studies that highlight data quality and methodology examples (e.g. building on EURECCA).
7.2. Operationalise and share/publish methodologies that address 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 and generation within HTA bodies linking to new policy initiatives such as the HTAR.
7.4. Work with HTAbs 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. 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. 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. Work with HTD to ensure they are following published standards/best practices for RWE generation (e.g., transparency, data quality, etc).
7.8. 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.
7.9. Create a standard world-wide library of definitions for key aspects covering diagnoses, outcomes, covariates etc and algorithms that have been validated.
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 |
| EURECCA | European Initiative for New Reimbursement and Access Approaches https://www.valueinhealthjournal.com/article/S1098-3015(23)00012-8/pdf |
| FINOSE | Nordic collaboration to provide efficient and transparent evaluations of medicinal products with a view to reimbursement or procurement in the four countries Denmark, Finland, Norway and Sweden https://www.dmp.no/en/public-funding-and-pricing-of-medicines/single-technology-assessments/finose |
| HTA | Health Technology Assessment |
| HTAR | EU Regulation on Health Technology Assessment https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX:32021R2282 |
| HTD | Health Technology Developers |
| JCA | Joint Clinical Assessment (under the HTAR) |
| JSC | Joint Scientific Assessment (under the HTAR) |
| MAH | Marketing Authorisation Holder |
| NCAPR | National Competent Authorities on Pricing and Reimbursement |
| OBMEA | Outcome-Based Managed Entry Agreement |
| PAES | Post-Authorisation Efficacy Study |
| PASS | Post-Authorisation Safety Study |
| P&R | Pricing and Reimbursement |
| PLEG | Post-Launch Evidence Generation |
| PRO | Patient-Reported Outcome |
| QoL | Quality of Life |
| REQuEST | Registry Evaluation and Quality Standards Tool (from EUnetHTA) https://www.eunethta.eu/request-tool-and-its-vision-paper/ |
| RWD | Real-World Data |
| RWE | Real-World Evidence |
| SPIFD | Structured Process to Identify Fit-for-Purpose Data https://www.ncbi.nlm.nih.gov/pmc/articles/pmid/34716990/ |