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Background and Aims: To sustainably address challenges in implementing precision medicine (PM), coordinated efforts of different stakeholders are required. Understanding their expectations represents a first key step toward aligning on future actions and strategies. Here, we aimed to explore the expectations of different stakeholders from themselves and each other regarding PM., Methods: This collaborative qualitative study was initiated by the global multistakeholder consortium From Testing to Targeted Treatments (FT3). Structured interviews were conducted with participants from five stakeholder groups: patients/patient advocates, healthcare providers (HCPs), researchers, policymakers/regulators/payers and industry representatives. A broad reach across geography, roles, experiences, and disease areas was sought. Results were analyzed by grounded theory methodology., Results: All stakeholders stated that optimal implementation of PM can only be achieved through collaboration; industry representatives were the biggest promoters of collaboration. Stakeholders agreed that PM should be implemented focusing on the patient's best interest; HCPs were seen as important gatekeepers for PM by interacting directly with patients, and policymakers/payers were perceived as the most important drivers of access to PM. Areas of misalignment included the role of industry in clinical trial design and in access to PM (perceived as important by patients, HCPs and policymakers but not by industry representatives), and the stakeholders responsible for elaborating guidelines on PM use (patients indicated policymakers, while researchers indicated themselves). Priorities for optimal PM implementation and suggested actions included the need for enhancing high-level policy focus, improving genomic literacy, optimizing the health technology assessment for PM, advocating for equitable access, promoting collaboration between industry and other stakeholder groups and development of reliable research standards., Conclusion: Stakeholder expectations revealed in this study suggested that no stakeholder group can drive change on its own; a global, multistakeholder collaborative approach that brings together current programs and best practices to support universal access to PM is needed., Competing Interests: Tanya Knott reports support for meeting attendance/speaker from Roche Products Ireland. Roche & Illumina grants for World CUP Awareness Week, and a leadership or fiduciary role in the FT3 board. Benjamin Horbach is an employee of and holds stock options in F. Hoffmann‐La Roche Ltd. James Creeden is an unpaid independent advisor to the FT3 board and owns Roche stock. Maximiliane Rauch‐Zumbrägel, Lidewij Vat, and Helena Harnik are collaborating with The Synergyst, a non‐profit organization, of which programs, including the FT3 program, are sponsored by the industry (list available at https://www.fromtestingtotargetedtreatments.org/). Zorana Maravic's organization received grants from BMS, Astellas, Daiichi‐Sankyo, Bayer, BI, Pierre Fabre and Seagen., (© 2023 The Authors. Health Science Reports published by Wiley Periodicals LLC.)

Real-world evidence (RWE) research helps determine whether outcomes observed in clinical trials happen in real-life clinical practice. RWE research may help patients receive more appropriate treatment, closer to their needs and wishes. RWE for metastatic colorectal cancer is currently limited. The PROMETCO RWE study is an important example of an ongoing initiative that focuses on patient-reported outcomes in metastatic colorectal cancer. Patients play an active role throughout the RWE research process, including study design, participation and results dissemination. This involvement can encourage greater patient empowerment through active engagement, potentially resulting in various benefits that can lead to improved clinical outcomes. Greater patient engagement can increase involvement in RWE, helping more patients to access the benefits of RWE research. Clinical Trial Registration: NCT03935763 (ClinicalTrials.gov).

Introduction: Meaningful patient involvement in health technology assessment (HTA) is essential in ensuring that the interests of the affected patient population, their families, and the general public are accurately reflected in coverage and reimbursement decisions. Central and Eastern European (CEE) countries are generally at less advanced stages of implementing HTA, which is particularly true for patient involvement activities. As part of the Horizon2020 HTx project, this research aimed to form recommendations for critical barriers to patient involvement in HTA in CEE countries., Methods: Built on previous research findings on potential barriers, a prioritisation survey was conducted online with CEE stakeholders. Recommendations for prioritised barriers were formed through a face-to-face workshop by CEE stakeholders and HTx experts., Results: A total of 105 stakeholders from 13 CEE countries completed the prioritisation survey and identified 12 of the 22 potential barriers as highly important. The workshop had 36 participants representing 9 CEE countries, and 5 Western European countries coming together to discuss solutions in order to form recommendations based on best practices, real-life experience, and transferability aspects. Stakeholder groups involved in both phases included HTA organisation representatives, payers, patients, caregivers, patient organisation representatives, patient experts, health care providers, academic and non-academic researchers, health care consultants and health technology manufacturers/providers. As a result, 12 recommendations were formed specified to the CEE region's context, but potentially useful for a broader geographic audience., Conclusion: In this paper, we present 12 recommendations for meaningful, systematic, and sustainable patient involvement in HTA in CEE countries. Our hope is that engaging more than a hundred CEE stakeholders in the study helped to spread awareness of the importance and potential of patient involvement and that the resulting recommendations provide tangible steps for the way forward. Future studies shall focus on country-specific case studies of the implemented recommendations., Competing Interests: Patient involvement should be open to all and non-discriminative on the grounds of previous experience and presumed support time needed. There are multiple initiatives locally and internationally aiming to ease patient recruitment with education, coordination and/or databases. We recommend organisations leading these initiatives to come together and join forces on the base of commonly agreed principles. We also recommend local HTA/payer organisations to set up their open call for local patients, carers, patient advocates, patient experts and patient organisation representatives to be able to express interest. We suggest to actively promote this opportunity to harder to reach patient communities and a periodical revision of the registry. Regarding ethical and compliance issues, a clear policy on financial and other conflicts of interests (how interests are declared, assessed and addressed) should be in place. Those registered should complete a declaration of interest form both on personal and organisational level conflict of interests and update it periodically. It should be clarified what kind of involvement with industry (e.g., attending a single advisory board meeting with a company versus only in case of direct conflict of interest) would impose restrictions on how a person can be involved in the HTA and decision-making process. We argue that in situations where patient experts and/or patient organisation representatives are difficult to identify, a softer approach should be taken and special measures could be proposed, equivalent to the “expert witness” status at the European Medicines Agency (EMA). Expert witnesses can be heard or participate in the deliberations but are not allowed to take part in the vote. However, the consequences of not being transparent with potential conflict of interests should be serious and communicated clearly from the beginning.KrT, RH, ZP, BN, and ZK were employed by Syreon Research Institute. At the time of the study IJ was the President of the European Patients’ Forum Youth Group, a Board of Trustees member at the EUPATI Foundation and employed by Syreon Research Institute. MN is the founder and co-owner of HealthQuest, a health technology assessment and market access consulting company. DaD is a Trustee of Thrombosis UK. TB was employed by Patvocates GmbH. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2023 Jakab, Dimitrova, Houÿez, Bereczky, Fövényes, Maravic, Belina, Andriciuc, Tóth, Piniazhko, Hren, Gutierrez-Ibarluzea, Czech, Tesar, Niewada, Lorenzovici, Kamusheva, Manova, Savova, Mitkova, Tachkov, Németh, Petykó, Dawoud, Delnoij, Knies, Goettsch and Kaló.)

Background: Artificial intelligence (AI) has attracted much attention because of its enormous potential in healthcare, but uptake has been slow. There are substantial barriers that challenge health technology assessment (HTA) professionals to use AI-generated evidence for decision-making from large real-world databases (e.g., based on claims data). As part of the European Commission-funded HTx H2020 (Next Generation Health Technology Assessment) project, we aimed to put forward recommendations to support healthcare decision-makers in integrating AI into the HTA processes. The barriers, addressed by the paper, are particularly focusing on Central and Eastern European (CEE) countries, where the implementation of HTA and access to health databases lag behind Western European countries., Methods: We constructed a survey to rank the barriers to using AI for HTA purposes, completed by respondents from CEE jurisdictions with expertise in HTA. Using the results, two members of the HTx consortium from CEE developed recommendations on the most critical barriers. Then these recommendations were discussed in a workshop by a wider group of experts, including HTA and reimbursement decision-makers from both CEE countries and Western European countries, and summarized in a consensus report., Results: Recommendations have been developed to address the top 15 barriers in areas of (1) human factor-related barriers, focusing on educating HTA doers and users, establishing collaborations and best practice sharing; (2) regulatory and policy-related barriers, proposing increasing awareness and political commitment and improving the management of sensitive information for AI use; (3) data-related barriers, suggesting enhancing standardization and collaboration with data networks, managing missing and unstructured data, using analytical and statistical approaches to address bias, using quality assessment tools and quality standards, improving reporting, and developing better conditions for the use of data; and (4) technological barriers, suggesting sustainable development of AI infrastructure., Conclusion: In the field of HTA, the great potential of AI to support evidence generation and evaluation has not yet been sufficiently explored and realized. Raising awareness of the intended and unintended consequences of AI-based methods and encouraging political commitment from policymakers is necessary to upgrade the regulatory and infrastructural environment and knowledge base required to integrate AI into HTA-based decision-making processes better., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2023 Zemplényi, Tachkov, Balkanyi, Németh, Petykó, Petrova, Czech, Dawoud, Goettsch, Gutierrez Ibarluzea, Hren, Knies, Lorenzovici, Maravic, Piniazhko, Savova, Manova, Tesar, Zerovnik and Kaló.)

Aim: Real-world data and real-world evidence (RWE) are becoming more important for healthcare decision making and health technology assessment. We aimed to propose solutions to overcome barriers preventing Central and Eastern European (CEE) countries from using RWE generated in Western Europe. Materials & methods: To achieve this, following a scoping review and a webinar, the most important barriers were selected through a survey. A workshop was held with CEE experts to discuss proposed solutions. Results: Based on survey results, we selected the nine most important barriers. Multiple solutions were proposed, for example, the need for a European consensus, and building trust in using RWE. Conclusion: Through collaboration with regional stakeholders, we proposed a list of solutions to overcome barriers on transferring RWE from Western Europe to CEE countries.

Background: Health inequities lead to low rates of cancer screening in certain populations, such as low-income and ethnic minority groups. Different interventions to address this have been developed with mixed results. However, interventions are not always developed in collaboration with the people they target. The aim of our article is to present the viewpoint of patients, survivors, advocates, and lay persons on interventions to increase cancer screening from a health inequity perspective., Methods: We prepared talking points to guide discussions between coauthors, who included representatives from nine patient and survivor advocacy groups, organizations working for citizen/patient empowerment, and health equity experts. Perspectives and opinions were first collected through video conferencing meetings and a first draft of the paper was prepared. All authors, read through, revised, and discussed the contents to reach an agreement on the final perspectives to be presented., Results: Several themes were identified: it is important to not view screening as a discrete event; barriers underlying an individual's access and willingness to undergo screening span across a continuum; individually tailored interventions are likely to be more effective than a one-size fits-all approach because they may better accommodate the person's personal beliefs, knowledge, behaviors, and preferences; targeting people who are unknown to medical services and largely unreachable is a major challenge; including professional patient advocacy groups and relevant lay persons in the cocreation of interventions at all stages of design, implementation, and evaluation is essential along with relevant stakeholders (healthcare professionals, researchers, local government and community organizations etc)., Conclusions: Interventions to address cancer screening inequity currently do not adequately solve the issue, especially from the viewpoint of patients, survivors, and lay persons. Several core pathways should be focused on when designing and implementing interventions: advancing individually tailored interventions; digital tools and social media; peer-based approaches; empowerment; addressing policy and system barriers; better design of interventions; and collaboration, including the involvement of patients and patient advocacy organizations., (© 2023. The Author(s).)

Nutritional intervention is an essential part of cancer treatments. Research and clinical evidence in cancer have shown that nutritional support can reduce length of hospitalisation, diminish treatment-related toxicity, and improve nutrient intake, quality of life, and physical function. Nutritional intervention can improve outcomes and help patients in the successful completion of oncological treatments by preventing malnutrition. Malnutrition is a very common hallmark in patients with cancers. Almost one-fourth of cancer patients are at risk of dying because of the consequences of malnutrition, rather than cancer itself. Patients with digestive cancers are at higher risk of suffering malnutrition due to the gastrointestinal impairment caused by their disease. They are at high nutritional risk by definition, yet the majority of them have insufficient or null access to nutritional intervention.Inadequate resources are dedicated to implementing nutritional services in Europe. Universal access to nutritional support for digestive cancer patients is not a reality in many European countries. To change this situation, health systems should invest in qualified staff to reinforce or create nutritional teams' experts in digestive cancer treatments. We aim to share the patient community's perspective on the status and the importance of nutritional intervention. This is an advocacy manuscript presenting data on the topic and analysing the current situations and the challenges for nutrition in digestive cancers. It highlights the importance of integrative nutrition in the treatment of digestive cancers and advocates for equitable and universal access to nutritional intervention for all patients., (© 2022. The Author(s).)

Hepatocellular carcinoma (HCC) is a major public health problem worldwide for which the incidence and mortality are similar, pointing to the lack of effective treatment options. Knowing the different issues involved in the management of HCC, from risk factors to screening and management, is essential to improve the prognosis and quality of life of affected individuals. This document summarises the current state of knowledge and the unmet needs for all the different stakeholders in the care of liver cancer, meaning patients, relatives, physicians, regulatory agencies and health authorities so that optimal care can be delivered to patients. The document was commissioned by the International Liver Cancer Association and was reviewed by senior members, including two ex-presidents of the Association. This document lays out the recommended approaches to the societal management of HCC based on the economic status of a given region., Competing Interests: Please refer to the accompanying ICMJE disclosure forms for further details., (© 2022 The Authors.)

Pancreatic cancer is a disease requiring urgent attention from governments and policymakers. Recently, a state of emergency has been declared for this cancer-being the fourth most common cause of cancer deaths in the European Union, it has the lowest survival rate of all common cancers. One of the major reasons pancreatic cancer is associated with such poor outcomes is because it is usually diagnosed at a late stage. Also, investment in research for effective targeted therapies is lacking. This is the perspective of a white paper developed by Digestive Cancers Europe, an umbrella organisation representing European patient organisations. It has been developed after consultation with pancreatic cancer patients, representatives of cancer patient organisations and leading pancreatic cancer healthcare professionals. The purpose of the paper is to highlight the key urgent unmet needs in pancreatic cancer from the patient perspective, ultimately with a view to improve patient care and outcomes in this very challenging disease., Competing Interests: Declaration of Conflicting Interests: The authors declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article., (© The Author(s) 2022.)

Rapid and continuing advances in biomarker testing are not being matched by take-up in health systems, and this is hampering both patient care and innovation. It also risks costing health systems the opportunity to make their services more efficient and, over time, more economical. This paper sets out the potential of biomarker testing, the unfolding precision and range of possible diagnosis and prediction, and the many obstacles to adoption. It offers case studies of biomarker testing in breast, ovarian, prostate, lung, thyroid and colon cancers, and derives specific lessons as to the potential and actual use of each of them. It also draws lessons about how to improve access and alignment, and to remedy the data deficiencies that impede development. And it suggests solutions to outstanding issues - notably including funding and the tangled web of obtaining reimbursement or equivalent coverage that Europe's fragmented health system implies. It urges a European evolution towards an initial minimum testing scenario, which would guarantee universal access to a suite of biomarker tests for the currently most common conditions, and, further into the future, to an optimum testing scenario in which a much wider range of biomarker tests would be introduced and become part of a more sophisticated health system articulated around personalised medicine. For exploiting genomics to the full, it argues the need for a new policy framework for Europe. Biomarker testing is not an issue that can be treated in isolation, since the purpose of testing is to improve health. Its use is therefore always closely linked to specific health challenges and needs to be viewed in the broader policy context in the EU and more widely. The paper is the result of extensive engagement with experts and decision makers to develop the framework, and consequently represents a wide consensus of views on how healthcare systems should respond from push and pull factors at local, national and cross-border and EU level. It contains strong views and clear recommendations springing from the convictions of patients, clinicians, academics, medicines authorities, HTA bodies, payers, the diagnostic, pharmaceutical and ICT industries, and national policy makers., Competing Interests: Benjamin Horbach is a Roche employee and has stock options in Roche. Stephen Hall is a Novartis employee and has stock options in Novartis. Benjamin Gannon is a Myriad Genetics employee and has stock options in Myriad. Rocio Varea Menendezis Eli Lilly employee and has stock options in Eli Lilly. Sarah Mee is an AstraZeneca employee and has stock options in AstraZeneca. Susana Banerjee receives grants from Astrazeneca, GSK & Honoraria − Astrazeneca/MSD, Amgen, Clovis, GSK, Mersana, Seattle Genetics, Merck Sereno, Genmab, Roche, Immunogen., (Copyright © 2020 by S. Karger AG, Basel.)

Background: Despite being highly preventable and treatable if diagnosed early, colorectal cancer (CRC) remains the second leading cause of cancer-related death in Europe. Limited information is available from the patient perspective on the persisting unmet needs of the journey of the patient with CRC., Objective: To capture European metastatic CRC (mCRC) patients' insights during the patient journey (prediagnosis; diagnosis; postdiagnosis) through a patient survey., Methods: In total, 883 patients from 15 European countries participated. Participants were divided into four groups from Hungary, Poland, Serbia and 'other European countries' (n=103, 163, 170 and 447 patients, respectively)., Results: General awareness of CRC and its symptoms prediagnosis varied among groups, with patients from Poland recording the lowest levels. Screening practices and attitudes also varied; while more patients from Serbia had been invited to CRC screening (~15%) compared with the other groups, the ones not invited claimed mostly (~20%) that would not have attended if they had been invited. Whereas most patients were diagnosed within a month after the first consultation/positive screening, the percentages varied substantially being lowest among patients in Poland (~30%) and Serbia (~25%). Although CRC-related information provision varied, with most informed patients from Hungary (~90%) and least from Serbia (~50%), all groups requested an easier-to-understand language by the healthcare team. Approximately 50% of patients from Eastern Europe had to wait longer than a month to receive treatment, in contrast to ~30% from other European countries. All groups emphasised the unmet need for support from psychologists and other patients., Conclusions: Our survey reveals the key aspects of the journey of the patient with mCRC and highlights the areas of similarities and differences between patients with mCRC from Eastern Europe versus those from other European countries as well as among patients from different Eastern European countries, calling for improvement particularly around awareness, screening, treatment availability, communication and support networks., Competing Interests: Competing interests: None declared., (© Author (s) (or their employer(s)) 2020. Re-use permitted under CC BY-NC. No commercial re-use. Published by BMJ on behalf of the European Society for Medical Oncology.)