D2.6 - First report on stakeholder engagement focused on patient-centered and patient-informed ethical guidance

STRONG-AYA – No. 101057482 – D2.6 A new, interdisciplinary, multistakeholder European network to improve healthcare services, research and outcomes for Adolescents and Young Adults with cancer. STRONG-AYA – No. 101057482 – D2.6 2 Deliverable Report WP2 – Governance, data security and ethics Deliverable D2.6 First report on stakeholder engagement focused on patient-centered and patient-informed ethical guidance Due date of deliverable: 30/09/2024 Actual submission date: 24/10/2024 Project: STRONG-AYA Lead Contributor Flora Lysen (UNIMAAS) Email [email protected] Other Contributors Darian Meacham (UNIMAAS); Urska Kosir (YCE); Norbert Couespel (E.C.O.); Anna Kiss (E.C.O.); Dan Stark (UOL) Emails [email protected]; urska.kosir2[email protected]om; [email protected]; anna.kis[email protected]; [email protected] Due date 30 09 2024 Delivery date 24 10 2024 Deliverable type R Dissemination level PU Description of Work Version Date STRONG-AYA – No. 101057482 – D2.6 3 V1.0 30 09 2024 Description: First report on stakeholder engagement focused on patient-centered and patientinformed ethical guidance Publishable summary (max ½ page) This report provides a targeted literature review of patient involvement practices and patient involvement evaluation practices focused on the field of AYA cancer research. In recent years, efforts to involve patients in AYA research have been introduced within the AYA population, though the number of initiatives is still relatively small. Despite the widespread calls for involvement practices, implementation, monitoring and evaluation practices for AYA participation are lacking in many European countries. There are currently no European standards for involvement practices with AYAs with lived experience of cancer: context-dependent policies and best practices should be developed. This report offers a preliminary mapping of the type of patient involvement practices that are part of the STRONG AYA project, showing examples of ‘consent’, ‘consultation’ and ‘cooperation’ practices. The report also discusses the topic of evaluation frameworks for patient involvement in relation to STRONG AYA’s involvement efforts. A wide variety of frameworks and guidelines exist in academic and professional literature, with differing approaches and focus. Recently, two guidelines specifically centered on improving patient involvement in AYA research have been published. The report ends with recommendations for improving evaluation frameworks for patient involvement in the context of research projects such as STRONG AYA. STRONG-AYA – No. 101057482 – D2.6 4 1 Table of Contents PUBLISHABLE SUMMARY (MAX ½ PAGE) .................................................................................................................... 3 1 TABLE OF CONTENTS .......................................................................................................................................... 4 2 DEFINITIONS....................................................................................................................................................... 5 3 ABBREVIATIONS ................................................................................................................................................. 6 4 INTRODUCTION .................................................................................................................................................. 7 PROJECT BACKGROUND ........................................................................................................................................... 7 DELIVERABLE INTRODUCTION .................................................................................................................................... 8 5 MAPPING PATIENT INVOLVEMENT PRACTICES IN STRONG AYA ......................................................................... 8 BACKGROUND TO THIS REPORT ................................................................................................................................. 8 5.1.1 STRONG AYA’s aims for patient involvement as part of Horizon Europe ...................................................... 9 5.1.2 STRONG AYA and European ethical principles for digital health ................................................................ 10 5.1.3 Scope of this report ..................................................................................................................................... 11 6 TARGETED LITERATURE REVIEW: PRACTICING AND EVALUATING PATIENT INVOLVEMENT IN AYA CANCER RESEARCH ..................................................................................................................................................................11 PATIENT INVOLVEMENT IN AYA CANCER RESEARCH ..................................................................................................... 12 EXAMPLE OF INVOLVEMENT PRACTICES IN STRONG AYA ............................................................................................ 14 FRAMEWORKS FOR IMPROVING AND EVALUATING PATIENT INVOLVEMENT IN (AYA CANCER) RESEARCH ................................ 18 6.3.1 Expanding current frameworks for evaluating patient involvement to fit with STRONG AYA .................... 20 7 CONCLUSION .....................................................................................................................................................23 8 BIBLIOGRAPHY ..................................................................................................................................................23 STRONG-AYA – No. 101057482 – D2.6 5 2 Definitions STRONG AYA consortium members are referred to as following within this text: 1. NKI-AVL – Stichting het Nederlands Kanker Instituut – Antoni van Leeuwenhoek Ziekenhuis (NL) 2. YCE – Youth Cancer Europe (RO) 3. INT – Fondazione IRCCS Instituto Nazionale dei Tumori (IT) 4. FFUND – FFUND BV (NL) 5. CLB – Centre de Lutte Contre le Cancer Leon Berard (FR) 6. ECO – European Cancer Organisation (BE) 7. UNIMAAS – Universiteit Maastricht (NL) 8. IKNL – Stichting Integraal Kankercentrum Nederland (NL) 9. EORTC – European Organisation for Research and Treatment of Cancer AISBL (BE) 10. IGR – Institut Gustave Roussy (FR) 11. MSCNRIO – Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie – Panstwowy Instytut Badawczy (Marie Sklodowska-Curie National Research Institute of Oncology) (PL) 12. UOM – University of Manchester (UK) 13. UOL – University of Leeds (UK) 14. LTHT – Leeds Teaching Hospitals National Health Service Trust (UL) 15. SOUTHAMPTON – University of Southampton (UK) • Grant Agreement (including its annexes and amendments): the agreement signed between the beneficiaries of the HORIZON Research and Innovations Actions (hereafter referred to as Horizon) and the European Health and Digital Executive Agency (hereafter referred to as HADEA) for the undertaking of the STRONG AYA project (Grant Agreement no. 101057482). • Beneficiary: Signatories of the Grant Agreement • Associated Partner: Entities which participate in the action but without the right to charge costs or claim contributions. • Project: the sum of all activities carried out in the framework of the Grant Agreement. • Consortium: the STRONG AYA consortium, including all the aforementioned partners. • Consortium Agreement: The agreement made between STRONG AYA members for the implementation and execution of the action outlined in the Grant Agreement. The agreement shall not affect the parties’ obligations to HADEA on behalf of the European Union, and/or to one another arising from the Grant Agreement. STRONG-AYA – No. 101057482 – D2.6 6 3 Abbreviations Acronym/Abbreviation Meaning HCP Health Care Provider PRO Patient Reported Outcome PROM Patient Reported Outcome Measure COS Core Outcome Set WP Work Package WPL Work Package Lead(s) WP1 Work Package 1 (Development Core Outcome Set AYA with cancer & data collection) WP2 Work Package 2 (Governance, Data Security and Ethics) WP3 Work Package 3 (Infrastructure and Interoperability) WP4 Work Package 4 (Operation of STRONG AYA ecosystems, stakeholder and patient involvement, dissemination, exploitation, communication) WP5 Work Package 5 (Scientific coordination and project managemen t) KPI Key Performance Indicator OA Open Access PAB Patient Advisory Board EC European Commission HADEA European Health and Digital Executive Agency SC Steering Committee MT Management Team STRONG-AYA – No. 101057482 – D2.6 7 4 Introduction Project background Cancer at adolescent and young adult (AYA) age (15-39 at primary diagnosis) is rare. However, this rarity does not reflect the significant personal and societal costs of cancer in this population, as reflected in the potential years of life lost or saved, the decreased productivity and quality-of-life due to the impact of the disease during formative years, and the long-term complications or disabilities1. AYAs with cancer form a unique group; they face age-specific issues (e.g. Infertility, unemployment, financial problems) and decreased quality of life due to cancer and its treatment. Unlike dedicated healthcare and trials for paediatric cancer patients, AYA-specific healthcare services are scarce and vary across Europe. AYAs who are at the core of society and the economy need access to age-appropriate and high-quality healthcare. The STRONG-AYA project aims to tackle the underrepresentation of AYA’s experiences and outcomes when navigating the healthcare system and in clinical care by developing national infrastructures for outcome data management and clinical decision-making within a pan-European ecosystem and establishing communication feedback for AYAs with cancer and the healthcare systems. This will be key to improving healthcare services, research, outcomes and policies for AYAs and to ultimately better cancer care for this patient group. The project brings together an international multi-disciplinary consortium (academic/research, clinical, stakeholder and patient organisations) across seven European countries. Building on previous initiatives, a STRONG-AYA data ecosystem will be set up for value-based care, research and policy for AYA with cancer by: 1. Developing a Core Outcome Set (COS) specifically for AYAs with cancer, via a participative consensus process defining most important aspects for those directly affected by AYA cancer, including patients and healthcare professionals. 2. Implementing the COS across several national European healthcare systems. Data will be collected at local level and will then be included in a data integration platform. An overall ecosystem framework for data analytics and output will be created supporting federated analyses and the creation of reports across clinical and patient-reported data and making national repositories of (patient-reported) health data, available to individual patients, patient organisations, regulatory authorities, as well as the patients’ health care providers to inform clinical decision-making. The five resulting national ecosystems will be connected to each other into the pan-European ecosystem using a federated approach also utilizing the overall ecosystem framework. 3. Disseminating the COS to a wide range of local as well as pan-European stakeholders, in particular by developing analytical tools to process and present patient outcome data and establish feedback loops that inform patients and clinicians. Implementing these project objectives are five work packages within the STRONG-AYA project: • WP1: Development Core Outcome Set AYA with cancer and Data Collection (Lead: SOUTHAMPTON) • WP2: Governance, Data Security and Ethics (Lead: EORTC) • WP3: Infrastructure and Interoperability (Lead: UNIMAAS) • WP4: Operation of STRONG-AYA ecosystems, Stakeholder and Patient involvement, Dissemination, Exploitation, Communication (Lead: E.C.O.) 1 Stoneham SJ. AYA survivorship: The next challenge. Cancer 2020; 126: 2116-2119. STRONG-AYA – No. 101057482 – D2.6 8 • WP5: Scientific Coordination and Project Management (Lead: NKI) STRONG-AYA will enable AYA care and research to benefit from collection and pooling of patient-centered data and collaboration among all stakeholders: patients, healthcare professionals, scientists, and policymakers. More widely, the project will leverage the network of interested organisations and networks established under STRONG-AYA for long-term strengthened promotion of the necessary implementation of specialist AYA cancer services across Europe. This will ultimately bring novel insights into AYA cancer care, research and policy, contributing to the long-term improvement of outcomes for people with AYA cancer. Deliverable introduction The report focuses on reviewing patient involvement and evaluation practices specifically within AYA (Adolescent and Young Adult) cancer research. The report provides a targeted literature review on patient involvement and involvement evaluation practices with special attention to AYA (Adolescent and Young Adult) cancer research. Key take away points: • Although efforts to involve patients in AYA research have begun, the number of such initiatives remains relatively small. • No standardized practices exist in Europe for involving AYAs with lived experience of cancer, highlighting the need for context-specific policies and best practices. • A wide range of evaluation frameworks and guidelines for patient involvement are available in the literature, each with different approaches and focuses. • Two guidelines aimed at improving patient involvement specifically in AYA research have been recently published. Preliminary Mapping of Involvement Practices: The report maps out the types of patient involvement practices within the STRONG AYA project, categorizing them into 'consent,' 'consultation,' and 'cooperation' practices. Recommendations for Evaluation Improvement: The report concludes with suggestions for enhancing evaluation frameworks for patient involvement in research projects like STRONG AYA. 5 Mapping patient involvement practices in STRONG AYA Background to this report This report builds on the first STRONG AYA Stakeholder Forum Meeting on 27 September 2023: ‘Caught in the Middle: Identifying Gaps for Adolescents and Young Adults with Cancer. This Stakeholder Forum meeting brought together 70 participants (including healthcare professionals, policy makers and patient representatives) to provide feedback on the consortium’s ongoing activities and to bring AYA Community’s attention to the project. In engaging with stakeholders during this event, an additional aim was to signal emerging issues that need further elucidation and examination during the development of STRONG AYA. During the event, the issue of patient involvement surfaced as a returning topic of conversation. In tandem, in 2023, the STRONG AYA Ethics and Regulatory advisory board (ERAB), also asked to clarify STRONG AYA’s approach to patient involvement. The current report jointly responds to these demands for clarification and provides a mapping of ongoing efforts within STRONG AYA to realise different forms of patient involvement. Another aim is to summarise relevant literature that can help to suitably evaluate AYA patient involvement practices. As the STRONG AYA consortium is currently developing, this report will not comprehensively STRONG-AYA – No. 101057482 – D2.6 9 evaluate patient involvement but will conclude with pointers for strengthening the evaluation of patient involvement during the further course of the project as well as future research development. 5.1.1 STRONG AYA’s aims for patient involvement as part of Horizon Europe From its inception, STRONG AYA underwrites the importance of patient involvement in (data-intensive) health research. STRONG AYA’s attention to involvement builds on decades of emerging patient involvement and patient activism in Europe, which have led to the institutionalization and professionalization of patient involvement practices in European research projects, including in HORIZON EUROPE.2 Patient involvement has been suggested as a way to improve the effectiveness, sustainability, and quality of medical innovations, to expand patient choice, democratize healthcare decision-making, emancipate patients, and promote fairness and justice in medicine.3 Definitions of patient involvement, engagement or participation vary widely. 4 A basic definition is “an activity that is done ‘with’ or ‘by’ patients or members of the public rather than ‘to’, ‘about’ or ‘for’ them.”5 Many different kinds of involvement practices proliferate: from informing patients about research and consulting with patients by means of questionnaires, to co-designing research design and tools and actively seeking representation by patients on governance bodies.6 This variation in types of involvement is also expressed by an expanding involvement vocabulary, such as ‘patient & public involvement’ (PPI), ‘public involvement and engagement’ (PI&E) ‘public engagement’ (PE), ‘patient engagement in research’ (PER), ‘patient partners in research’ (PPR), ‘patient engagement in research’ (PEIR), ‘patient research partner engagement’ (PRPE) and participatory design (PD). STRONG AYA draws on extensive experience and expertise by our consortium members in the domain of patient involvement, spearheaded by the core expertise of The European Cancer Organization (ECO) and Youth Cancer Europe (YCE). Within the STRONG AYA consortium, ECO takes the lead on stakeholder involvement activities. ECO also has experience working on patient involvement activities, including through its Patient Advisory Committee and role in other EU-funded projects - in that regard, ECO contributed to a very recent handbook on the topic. YCE is leading a consortium-specific STRONG-AYA Patient Advisory Board (PAB), which is responsible for co-creating platforms for patient engagement and co2 The HORIZON EUROPE funding scheme has underlined the importance of civil society participation in “co-designing and co-creating responsible research and innovation agendas and contents, promoting science education, making scientific knowledge publicly accessible, and facilitating participation by citizens and civil society organisations in its activities.” Regulations. Establishing Horizon Europe – the Framework Programme for Research and Innovation, laying down its rules for participation and dissemination, and repealing Regulations (EU) No 1290/2013 and (EU) No 1291/2013. (2021). Official Journal of the European Union, 170, 51. 3 For an overview of envisioned effects of patient involvement, see for example Prainsack, B. (2017). Personalized Medicine: Empowered Patients in the 21st Century? NYU Press. Madden, M., & Speed, E. (2017). Beware Zombies and Unicorns: Toward Critical Patient and Public Involvement in Health Research in a Neoliberal Context. Frontiers in Sociology, 2. 4 Baines et al. (2022) found 133 terms to describe forms of involvement, with ‘user-centred design’, ‘participatory design’ and ‘codesign’ most commonly used. Baines, R., Bradwell, H., Edwards, K., Stevens, S., Prime, S., Tredinnick-Rowe, J., Sibley, M., & Chatterjee, A. (2022). Meaningful patient and public involvement in digital health innovation, implementation and evaluation: A systematic review. Health Expectations, 1–14. https://doi.org/10.1111/hex.13506. Castro, E. M., Van Regenmortel, T., Vanhaecht, K., Sermeus, W., & Van Hecke, A. (2016). Patient empowerment, patient participation and patient-centeredness in hospital care: A concept analysis based on a literature review. Patient Education and Counseling, 99(12), 1923–1939. https://doi.org/10.1016/j.pec.2016.07.026 5 Ocloo, J., & Matthews, R. (2016). From tokenism to empowerment: Progressing patient and public involvement in healthcare improvement. BMJ Quality & Safety, 25(8), 626–632. https://doi.org/10.1136/bmjqs-2015-004839. Additionally, a useful distinction has been made between individual and collective patient participation: “Individual patient participation revolves around a patients’ rights and opportunities to influence and engage in decision making about their care through a dialogue attuned to a patient’s preferences, potential and a combination of his experiential and the professional’s expert knowledge. Collective patient participation is the contribution of patients or their representing organizations in shaping health and social care services by means of active involvement in a range of activities at the individual, organizational and policy level that combine experiential and professional knowledge.” Castro, E. M., Van Regenmortel, T., Vanhaecht, K., Sermeus, W., & Van Hecke, A. (2016). Patient empowerment, patient participation and patient-centeredness in hospital care: A concept analysis based on a literature review. Patient Education and Counseling, 99(12), 1923–1939. https://doi.org/10.1016/j.pec.2016.07.026. 6 Ocloo, J., & Matthews, R. (2016). From tokenism to empowerment: Progressing patient and public involvement in healthcare improvement. BMJ Quality & Safety, 25(8), 626–632. https://doi.org/10.1136/bmjqs-2015-004839. STRONG-AYA – No. 101057482 – D2.6 16 data will be exchanged between institutions, no data transfer agreements or additional patient consent will be needed.” “If or when PRO data beyond the outcomes collected as part of clinical care and previous studies are desired for STRONG AYA in accordance with our finalized COS, a new protocol or amendment (in accordance to IRB guidelines) will be developed.” STRONG-AYA – No. 101057482 – D2.6 17 323334 Figure 2: types of patient involvement in STRONG AYA, continued STRONG-AYA – No. 101057482 – D2.6 18 Frameworks for improving and evaluating patient involvement in (AYA cancer) research In the previous section we have listed examples of patient involvement practices currently part of the STRONG AYA project. These practices abide by general guidelines set in the STRONG-AYA information governance and ethics framework.35 In past decades, patient organizations have rallied for formulating and monitoring specific criteria for the evaluation of patient involvement practices. The European Patient Forum, for example, reflecting on HORIZON EUROPE, has called for “clear criteria for meaningful patient involvement in calls to be developed together with patient organisations, to avoid researchers treating ‘patient involvement’ as a tick-box exercise as they still too often do, and to reward those projects that practice genuine co-creation and co-production.”36 The evaluation of involvement practices is a complex issue: a wide variety of frameworks and guidelines exist in academic and professional literature, with differing approaches and focus.37 A 2019 systematic review (Greenhalgh et al. 2019) of tools, frameworks, benchmarks, guidelines and checklists for patient involvement in research found 56 relevant frameworks. Analysing these frameworks for involvement practices, Greenhalgh et al. distinguished five main types: power-focused frameworks, priority-setting frameworks, study-focused frameworks, report-focused frameworks and partnership-focused frameworks. Recently, two guidelines specifically centred on improving patient involvement in AYA research have been published.38 Burgers et al. (2022) developed a list of tips for the involvement of patients as research partners in the context of a research that conducted qualitative interviews with AYA patients with uncertain and/or poor cancer prognosis (UPCP) to better understand their daily life challenges, coping, and healthcare needs.39 32 Re: patient involvement in PROM design, see D1.1. Digital technology requirements, Report including specification for digital technology selection, adaptation and/or development. 33 Re: patient involvement in overall consortium work, see D 4.1 Stakeholder involvement and engagement plan. 34 Re: patient involvement in overall governance, see D4.2 Bylaws of the Patient Advisory Board (PAB). at 25EU/hour but are free to opt out from any compensation. Individual contracts will be drafted in agreement with YCE, the lead beneficiary who will remunerate PAB members’ time (up to 1,500EU per member for the duration of the project). In case members of the PAB will be asked to participate in any in-person activities or General Assembly, YCE may compensate up to 8 hours per day of attendance. See also Monge-Montero, C., O’Callaghan, S., Rizvi, K., Kosir, U., & Girbu, V. (2023). Recommendations for Equitable, Diverse, and Inclusive Cancer Care in Europe. Youth Cancer Europe. European Commission co-funded project: EU-CAYAS-NET EU4H-2021-PJ-04101056918. https://www.youthcancereurope.org/the-european-network-of-youth-cancer-survivors-launches-its-recommendations-forequitable-diverse-and-inclusive-cancer-care-in-europe. 35 D2.2. STRONG-AYA information governance and ethics framework. 36 Horizon Europe must invest more in health to support high-quality, accessible and equitable health systems. (2018). European Patients Forum. https://www.eu-patient.eu/news/latest-epf-news/2018/horizon-europe-must-invest-more-in-health-to-supporthigh-quality-accessible-and-equitable-health-systems/. 37 Greenhalgh, T., Hinton, L., Finlay, T., Macfarlane, A., Fahy, N., Clyde, B., & Chant, A. (2019). Frameworks for supporting patient and public involvement in research: Systematic review and co-design pilot. Health Expectations, 22(4), 785–801. https://doi.org/10.1111/hex.12888. 38 van Ham, C. R., Burgers, V. W. G., Sleeman, S. H. E., Dickhout, A., Harthoorn, N. C. G. L., Manten-Horst, E., van Eenbergen, M. C., & Husson, O. (2022). A qualitative study on the involvement of adolescents and young adults (AYAs) with cancer during multiple research phases: “Plan, structure, and discuss.” Research Involvement and Engagement, 8(1), 30. https://doi.org/10.1186/s40900022-00362-w. Oveisi, N., Cheng, V., Taylor, D., Bechthold, H., Barnes, M., Jansen, N., McTaggart-Cowan, H., Brotto, L. A., Peacock, S., Hanley, G. E., Gill, S., Rayar, M., Srikanthan, A., & De Vera, M. A. (2024). Meaningful Patient Engagement in Adolescent and Young Adult (AYA) Cancer Research: A Framework for Qualitative Studies. Current Oncology, 31(4), Article 4. https://doi.org/10.3390/curroncol31040128. 39 Burgers, V. W. G., Dickhout, A., Harthoorn, N. C. G. L., Frissen, S. A. M. M., Noordhoek, M. J., Franssen, S. A., Reuvers, M. J. P., van der Graaf, W. T. A., & Husson, O. (2023). Involving adolescents and young adults (AYA) with an uncertain or poor cancer prognosis as research partners. Acta Oncologica, 62(8), 961–968. https://doi.org/10.1080/0284186X.2023.2238554. STRONG-AYA – No. 101057482 – D2.6 19 Oveisi et al. (2024) published a framework for patient research partners (PRPs) in the context of a research that employs qualitative methods (focus groups) to explore the impacts of cancer diagnosis and treatment on the sexual and reproductive health of AYA cancer patients [figure 3].40 Figure 3: Collaborative and iterative framework for meaningful patient engagement in adolescent and young adult (AYA) cancer qualitative research. Developed by and reproduced from Oveisi et al. 202441 The studies of Burgers et al. 2022 and Oveisi et al. 2024 are close to what Greenhalgh et al. have typified as “study-focused” frameworks for evaluating involvement practices, whereby researchers distinguish different phases in a research project linked to various potential involvement challenges and issues. Both guidelines focus on patients as research partners - i.e. on consultation and cooperation by means 40 Oveisi, N., Cheng, V., Taylor, D., Bechthold, H., Barnes, M., Jansen, N., McTaggart-Cowan, H., Brotto, L. A., Peacock, S., Hanley, G. E., Gill, S., Rayar, M., Srikanthan, A., & De Vera, M. A. (2024). Meaningful Patient Engagement in Adolescent and Young Adult (AYA) Cancer Research: A Framework for Qualitative Studies. Current Oncology, 31(4), Article 4. https://doi.org/10.3390/curroncol31040128. 41 Oveisi, N., Cheng, V., Taylor, D., Bechthold, H., Barnes, M., Jansen, N., McTaggart-Cowan, H., Brotto, L. A., Peacock, S., Hanley, G. E., Gill, S., Rayar, M., Srikanthan, A., & De Vera, M. A. (2024). Meaningful Patient Engagement in Adolescent and Young Adult (AYA) Cancer Research: A Framework for Qualitative Studies. Current Oncology, 31(4), Article 4. https://doi.org/10.3390/curroncol31040128. STRONG-AYA – No. 101057482 – D2.6 20 of direct involvement of individual patients. Not part of the focus of these AYA guidelines are involvement through patient representative organizations, the role of patients in governance of research consortia, as well as procedures of patient consent. As Greenhalgh et al. 2019 have emphasized, frameworks for evaluating patient involvement developed in the context of one research may not be (entirely) fitting for other contexts. Research projects may need to develop a tailor-made evaluation framework based, for example, on a participatory workshop in which patients are involved in designing the evaluation criteria and framework for patient involvement practices.42 In the next section, we list issues related to patient involvement and evaluation of involvement that deserve special attention in the context of the STRONG AYA project. 7 Expanding current frameworks for evaluating patient involvement to fit with STRONG AYA As outlined above, frameworks for evaluating patient involvement in the domain of AYA care and research are still rare (our research turned up two guidelines, Burgers et al. 2022 & Oveisi et al. 2024) and need to be adapted to the variety of involvement practices that are part of the STRONG AYA consortium. Based on analysis of AYA research literature, as well as experience by consortium members, particularly within the European Cancer Organisation and Youth Cancer Europe, below is a non-exhaustive list of aspects that deserve further attention in relation to improving and monitoring the quality of involvement practices in AYA work. Adequate structures for financially compensating AYA involvement work Within STRONG AYA, Youth Cancer Europe has set up platforms for patient involvement, including a Patient Advisory Board. In these various tasks, financial compensation for work by patient advocates is outlined, i.e. the process is made transparent and sets expectations of how much time they can devote.43 However, even when financial compensation is secured for patient involvement work, current structures and mechanisms for compensation might not fit the financial circumstances in which AYA patients find themselves. For example, adolescents and young adults in treatment or off treatment may be receiving disability pay, which can conflict with the financial payments they receive because of involvement work. AYAs will need access to experts with knowledge of these financial regulations in their home country. At present, EU research projects are often not equipped with adequate consultation on this issue. 42 ”Appendix S2: Facilitator notes for PPI build-your-own-framework workshop.” in Greenhalgh, Trisha, Lisa Hinton, Teresa Finlay, Alastair Macfarlane, Nick Fahy, Ben Clyde, and Alan Chant. 2019. “Frameworks for Supporting Patient and Public Involvement in Research: Systematic Review and Co-Design Pilot.” Health Expectations 22 (4): 785–801. https://doi.org/10.1111/hex.12888. 43 D4.2. Bylaws of the Patient Advisory Board (PAB) (M6): Terms of reference and principles for the selection of participants, operation and governance of the PAB. “All members of the PAB will be offered monetary compensation for their time and work at 25EU/hour but are free to opt out from any compensation. Individual contracts will be drafted in agreement with YCE, the lead beneficiary who will remunerate PAB members’ time (up to 1,500EU per member for the duration of the project). In case members of the PAB will be asked to participate in any in-person activities or General Assembly, YCE may compensate up to 8 hours per day of attendance. Any travel arrangements will need to be agreed upon with the entire Consortium. In order to provide adequate justification and eligibility of any corresponding costs, PAB members will have the responsibility of keeping their own record of time spent on the project (with guidance from the PAB secretariat). The PAB secretariat will take responsibility to check all correspondence between the time declared by the PAB members and their actual participation in the project.” STRONG-AYA – No. 101057482 – D2.6 21 Equity, Diversity and Inclusion (EDI) in AYA involvement work Researchers have pointed to the barriers and burdens faced by marginalised AYA patients in participating in patient representation and involvement practices.44 To realise the existing recommendations and expertise with EDI in AYA care, adequate compensation is vital, but not enough. Improving the funding of participation of professionals with EDI expertise is necessary, as well as supporting the participation of marginalised groups within which we might be able to identify AYAs with lived experience that can enhance inclusivity in practice, particularly to promote EDI for racial and ethnic minority AYAs.45 EDI criteria are already part of STRONG AYA’s involvement practices, for example in the recruitment process for the Patient Advisory Board.46 Going forward, it is important to employ similar EDI criteria and standards for AYA cancer research teams as well. By ensuring diversity across all facets of the research process, EDI in patient involvement can be more readily and satisfactorily accomplished. Moreover, recent research into the attitudes toward EDI in the AYA population has showed that diversity work should include categories beyond gender, race and ethnicity, such e.g. disability and neurodiversity.47 Adequate patient representation As mentioned during the 2023 STRONG AYA stakeholder forum, “adequate patient representation” is important, as patient involvement in larger research consortiums often takes the shape of consultation through patient representatives as well as professionals working at patient advocacy organizations.48 The evaluation of patient representation practices is a complex and contested issue: who is regarded as the proper representative at specific involvement moments in health research is context-dependent and contingent on shifting values and perspectives about the aims of representation work.49 To be able to do representation work, for example as a person with lived experience of AYA, requires substantial levels of knowledge, adaptive skills (to adjust to variable demands by a diverse consortium), free time, financial 44 Cheung, C. K., Tucker-Seeley, R., Davies, S., Gilman, M., Miller, K. A., Lopes, G., Betz, G. D., Katerere-Virima, T., Helbling, L. E., Thomas, B. N., & Lewis, M. A. (2021). A call to action: Antiracist patient engagement in adolescent and young adult oncology research and advocacy. Future Oncology, 17(28), 3743–3756. https://doi.org/10.2217/fon-2020-1213. Cheung, C. K., Miller, K. A., Goings, T. C., Thomas, B. N., Lee, H., Brandon, R. E., Katerere-Virima, T., Helbling, L. E., Causadias, J. M., Roth, M. E., Berthaud, F. M., Jones, L. P., Ross, V. A., Betz, G. D., Simmons, C. D., Carter, J., Davies, S. J., Gilman, M. L., Lewis, M. A., … Tucker-Seeley, R. D. (2024). BIPOC Experiences of (anti-)Racist Patient Engagement in Adolescent and Young Adult Oncology Research: An Electronic Delphi Study. Future Oncology, 20(9), 547–561. https://doi.org/10.2217/fon-2023-0771. 45 Cheung, C. K., Tucker-Seeley, R., Davies, S., Gilman, M., Miller, K. A., Lopes, G., Betz, G. D., Katerere-Virima, T., Helbling, L. E., Thomas, B. N., & Lewis, M. A. (2021). A call to action: Antiracist patient engagement in adolescent and young adult oncology research and advocacy. Future Oncology, 17(28), 3743–3756. https://doi.org/10.2217/fon-2020-1213. 46 D. 4.2 Bylaws of the Patient Advisory Board (PAB). Mentions the selection criteria for PAB members, with the aim of maximizing diverse knowledge, experience and expertise while employing principles of Equity, Diversity and Inclusion (EDI). ”The EDI principles consider diversity of thought, experience, expertise, geographical representation, gender identity, educational status, health status and disability as essential aspects of delivering work fit-for-purpose of this multinational project.“ 47 Monge-Montero, Carmen, Stewart O’Callaghan, Katie Rizvi, Urska Kosir, and Victor Girbu. 2023. “Recommendations for Equitable, Diverse, and Inclusive Cancer Care in Europe.” Yout Cancer Europe. European Commission co-funded project: EU-CAYAS-NET EU4H-2021PJ-04101056918. https://www.youthcancereurope.org/the-european-network-of-youth-cancer-survivors-launches-its-recommendationsfor-equitable-diverse-and-inclusive-cancer-care-in-europe/. 48 L’Hôte, M., Lorenzo i Sunyer, N., & Couespel, N. (2024). Caught in the Middle: Identifying Gaps for Adolescents and Young Adults (AYAs) with Cancer. European Cancer Organisation. https://www.europeancancer.org/resources/publications/reports/identifying-gapsadolescents-young-adults-ayas-cancer.html 49 Halloy, Arnaud, Emmanuelle Simon, and Fabienne Hejoaka. 2023. “Defining Patient’s Experiential Knowledge: Who, What and How Patients Know. A Narrative Critical Review.” Sociology of Health & Illness 45 (2): 405–22. https://doi.org/10.1111/1467-9566.13588. Mankell, Anna, and Mio Fredriksson. 2021. “Federative Patient Organizations in a Decentralized Health-Care System: A Challenge for Representation?” Health 25 (6): 722–38. https://doi.org/10.1177/1363459320912807. STRONG-AYA – No. 101057482 – D2.6 22 resources and willpower.50 This set of requirements often means that a small group of available, trained and experienced patients becomes frequently approached by stakeholders and turn into recurrent representatives or ’expert patients’.51 In past years, with increasing demands for patient representation practices, patient organisations have become important intermediaries for representation.52 They have become endowed with increasing responsibilities in recruiting fitting representatives, ensuring diversity through recruiting a broad group of representatives, facilitating training of representatives and preventing forms of fatigue and bias within the pool of representatives. To play this intermediary role, patient organisations should be facilitated and financially supported, for example by providing structural funding (beyond project-based funding) to build up a continuous training program for AYA cancer patient representatives. Patient involvement with secondary use of data in data-intensive health research Research on AYA-cancer issues in STRONG AYA depends on the ability to pool personal health data from different countries to perform retrospective studies, i.e. the ‘secondary use’ of personal health data for research. At present, ethical use of secondary data in STRONG AYA is ensured through existing national legislations regarding patient consent and GDPR. At the same time, a recent EU-proposal for the European Health Data space proposes new legislation that would increase secondary data use to facilitate research.53 Within this emerging legislation, the role of patient involvement is left undefined. While patient control is explicitly mentioned for primary use of healthcare data, the EHDS is not specific about how and what type of information about secondary use of data should be provided to patients, nor how patients can partake in governance or development practices.54 The STRONG AYA project has developed efforts to inquire into AYA patients’ attitudes towards data protection (see section on consultation). Moreover, the consortium can draw on recent studies about health data sharing attitudes towards secondary use of data, which show that patients’ approval of secondary data use is dependent on context and purpose of use and type of user.55 50 Egher, Claudia, and Olga Zvonareva. 2024. “Knowledge-Based Representation: Patient Engagement in Drug Development.” Health Expectations 27 (1): e13912. https://doi.org/10.1111/hex.13912. 51 Pols, Jeannette. 2014. “Knowing Patients: Turning Patient Knowledge into Science.” Science, Technology, & Human Values 39 (1): 73–97. https://doi.org/10.1177/0162243913504306. 52 Moreira, Tiago. 2015. “Understanding the Role of Patient Organizations in Health Technology Assessment.” Health Expectations 18 (6): 3349–57. https://doi.org/10.1111/hex.12325. 53 European Commission. Proposal for a regulation of the European Parliament and of the Council on the European Health Data Space. 3 May 2022. https:// eur-lex.europa.eu/resource.html?uri=cellar:dbfd8974-cb79-11ec-b6f401aa75ed71a1.000102/DOC_1&format=PDF. 54 Member states are requested to develop health data access bodies (HDABs, a public sector body) to create a governance structure that facilitates secondary use. Saelaert, Marlies, Louise Mathieu, Wannes Van Hoof, and Brecht Devleesschauwer. 2023. “Expanding Citizen Engagement in the Secondary Use of Health Data: An Opportunity for National Health Data Access Bodies to Realise the Intentions of the European Health Data Space.” Archives of Public Health 81 (1): 168. https://doi.org/10.1186/s13690-023-01182-4. 55 A 2024 systematic review of health data sharing attitudes found more than a dozen studies examining attitudes towards sharing personal health data and information.Studies found a higher intention to share data for secondary use correlated with variables, younger age, female gender, higher education level, health care occupation, being white and region of residence (Europe and Middle East were more willing than North Americans and Asians). Other studies found that younger patients were more uncomfortable than older patients to share data. Generally, for many patients, sharing secondary data was conditional upon specific use purposes and specific users, being informed, having a choice, perceived use, trust in data protection, use by a trustworthy actor, such as a government agency or public institution. Cascini, Fidelia, Ana Pantovic, Yazan A. Al-Ajlouni, Valeria Puleo, Lucia De Maio, and Walter Ricciardi. 2024. “Health Data Sharing Attitudes towards Primary and Secondary Use of Data: A Systematic Review.” eClinicalMedicine 71 (May). https://doi.org/10.1016/j.eclinm.2024.102551. STRONG-AYA – No. 101057482 – D2.6 23 However, as researchers have emphasized, experiments with new ways of involving citizens and patients with the issue of secondary health data are necessary.56 Understanding patient attitudes towards patient involvement work in the AYA cancer field Research on (AYA cancer) patient attitudes towards involvement in research practices has shown that attitudes vary according to different age groups. Adolescents are found to want to take a less proactive and involved role compared to older (20+) patients.57 Other variables may also be important in attitudes towards patient involvement, such as gender, income, education level, having a family. While there is research into participation attitudes of patients as research subjects, including reasons of response or non-response by AYA patients to invitations to enrol in PRO-studies, this research does not address the topic of patient involvement in research.58 More research is necessary to understand motivations for – and attitudes towards – patient involvement on the part of patients. Future research should also include a study into the way patients are informed about the value of patient involvement (before participating) as well as methods of reflection and evaluation with patients after involvement practices. 8 Conclusion This report has provided a targeted literature review of patient involvement practices and patient involvement evaluation practices focused on the field of AYA cancer research. In recent years, efforts to involve patients in AYA research have been introduced within the AYA population, though the number of initiatives is still relatively small. Despite the widespread calls for involvement practices, implementation, monitoring and evaluation practices for AYA participation are lacking in many European countries. There are currently no European standards for involvement practices with AYAs with lived experience and contextdependent policies and best practices should be developed. This report has offered a preliminary mapping of the type of patient involvement practices that are part of the STRONG AYA project, showing examples of ‘consent’, ‘consultation’ and ‘cooperation’ practices. The report has also discussed the topic of evaluation frameworks for patient involvement in relation to STRONG AYA’s involvement efforts. A wide variety of frameworks and guidelines exist in academic and professional literature, with differing approaches and focus. Recently, two guidelines specifically centered on improving patient involvement in AYA research have been published. The report ends with recommendations for improving evaluation frameworks for patient involvement in the context of research projects such as STRONG AYA. 9 Bibliography • “Peer Visit as Research Method in EU-CAYAS-NET. Description of Methodology –Final draft Developed under Task 4.2 in Work Package 4: AYA Care.” 2024. 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