Bengt Jönsson

The cancer problem is expanding, particularly in low- and middle-income countries (LMICs). Preventive measures can reduce the incidence by 40-50%, and cure rates have increased during the past decades in a number of cancers. However, optimizing prevention programmes and increasing cure rates of cancer remain significant research challenges. The main focus of the conference was on P4 Cancer Medicine (Predictive, Preventive, Personalized and Participatory), a comprehensive strategy encompassing Health-Related Quality of Life (HRQoL) research, aiming to enhance the well-being of patients and individuals at risk. Addressing the cancer problem requires two key elements: translational cancer research and the development of relevant infrastructures. A Comprehensive Cancer Centre (CCC) acts as an innovation hub by integrating high-quality, multidisciplinary therapy and care, with healthcare-dependent prevention, research, and education. The United States has been at the forefront, providing quality-assured CCCs and the Cancer Moonshot for strategic cancer research. The EU has followed with the European Research Council for basic research, the European Innovation Council to boost disruptive innovation, and two EU initiatives on cancer, Europe's Beating Cancer Plan (EBCP) and the Mission on Cancer. The increasing complexity of cancer biology and technologies presents both a research challenge and a healthcare demand. For most patients, a CCC is not available. A critical discussion focused on quality assurance of healthcare outside the catchment area of a CCC and involving patients in clinical research. The strategic deployment of resources to support collective healthcare efforts and research aimed at reducing the cancer problem was discussed with representatives from the United States, EU, Africa, China, India and Taiwan. Analyses of translational cancer research have revealed important gaps in implementing innovations, assessment of clinical effectiveness, HRQoL, outcome and health economics research. The increased release of new anticancer agents over the last 25 years, accompanied by insufficient information on clinical benefits, presents both an economic and ethical problem. Direct healthcare costs have increased due to expenses for anticancer agents for the treatment of patients with incurable diseases. Evidence-based treatment based on HRQoL research is an unmet need. Basic/preclinical research aimed at increasing the cure rate should identify new, broader targets for therapy and develop extended diagnostic technologies for stratifying patients, to inform innovative clinical trials. Present research strategies convert cancer to a chronic disease, a growing burden for the healthcare systems. The increasing complexity of cancer biology and technology, the growing need for translational cancer research, and the demand for supporting infrastructures underscore the importance of international collaborations between CCCs. However, funding for cancer research is not currently aligned to reduce the cancer problem. While public funding for cancer research doubled between 2005 and 2024, the pharmaceutical industry's spending on cancer research increased tenfold. Increasing funding by public and non-profit funding organizations is mandatory. Education is another significant need, but it is currently fragmented and underfunded. The last session of the conference summarized the strategies in a Statement with a strong emphasis on global collaboration addressing the growing cancer burden and pronounced inequalities.
Expanding partnerships and fostering innovative, multidisciplinary approaches to cancer prevention, therapeutics/care, as well as research, are not just urgent but essential steps towards reducing incidence, increasing cure rates and enhancing the well-being of cancer patients. Data-driven cancer medicine is currently under development, and modern communication technologies for diagnostics may facilitate interactions across geographical distances. A global cancer research agenda can become a model of solidarity, sustainability, and ethical responsibility.

The development of new anticancer treatments, their clinical evaluation and introduction into the healthcare system need improvement. New drugs and cell therapies often come with significant costs for society while only marginally improving patients' survival and health-related quality-of-life. Therefore, bold, innovative clinical trials with critical assessment of the efficacy and cost-effectiveness of new preventive measures and medical treatments are needed to ensure that patients and society benefit. Drug development programmes controlled by pharma should be complemented with initiatives such as stop studies, dose reduction, combination and repurposing trials. These should be validated in academia-initiated trials supported by societal funds. Special attention should be devoted to paediatric and rare adult cancers. Comprehensive Cancer Centres (CCCs) covering the entire cancer research continuum, present throughout the EU, are critical for this. More of such centres must be established concomitantly with a robust accreditation methodology to ensure that they meet appropriate quality standards. It is crucial that funding for these initiatives, now temporarily and partially provided by the EU Cancer Mission and Europe's Beating Cancer Plan, is secured for a much longer period.

Analyses of inequalities related to prevention and cancer therapeutics/care show disparities between countries with different economic standing, and within countries with high Gross Domestic Product. The development of basic, technological and biological research provides clinical and prevention opportunities that make their implementation into healthcare systems more complex, mainly due to the growth of Personalized/Precision Cancer Medicine (PCM). Initiatives like the US‐Cancer Moonshot and the EU‐Mission on Cancer and Europe´s Beating Cancer Plan are initiated to boost cancer prevention and therapeutics/care innovation and to mitigate present inequalities. The conference organised by the Pontifical Academy of Sciences in collaboration with the European Academy of Cancer Sciences discussed the inequality problem, dependent on the economic status of a country, the increasing demands for infrastructure supportive of innovative research and its implementation in healthcare and prevention programs. Establishing translational research and a coherent cancer research continuum is still a challenge. Research has to cover the entire continuum from basic to outcomes research for clinical and prevention components. Comprehensive Cancer Centres (CCCs) are of critical importance for integrating research innovations to preclinical and early clinical research, as for ensuring state‐of‐the‐art patient care within healthcare systems. International collaborative networks between CCCs are necessary to reach the critical mass of infrastructures and patients for PCM research, and for introducing prevention modalities and new treatments effectively. Outcomes and health economics research are required to assess the cost‐effectiveness of new interventions, currently a missing element in the research portfolio. Data sharing and critical mass are essential for innovative research to develop PCM. Despite advances in cancer research, cancer incidence and prevalence is growing. Making cancer research infrastructures accessible for all patients, considering the increasing inequalities, requires science policy actions incentivising research aimed at prevention and cancer therapeutics/care with an increased focus on patients´ needs and cost‐effective healthcare.

Introduction EQ-5D-3L preference-based value sets are predominately based on hypothetical health states and derived in cross-sectional settings. Therefore, we derived an experience-based value set from a prospective observational study. Methods The International Costs and Utilities Related to Osteoporotic fractures Study (ICUROS) was a multinational study on fragility fractures, prospectively collecting EQ-5D-3L and Time trade-off (TTO) within two weeks after fracture (including pre-fracture recall), and at 4, 12, and 18 months thereafter. We derived an EQ-5D-3L value set by regressing the TTO values on the ten impairment levels in the EQ-5D-3L. We explored the potential for response shift and whether preferences for domains vary systematically with prior impairment in that domain. Finally, we compared the value set to 25 other EQ-5D-3L preference-based value sets. Results TTO data were available for 12,954 EQ-5D-3L health states in 4683 patients. All coefficients in the value set had the expected sign, were statistically significant, and increased monotonically with severity of impairment. We found evidence for response shift in mobility, self-care, and usual activities. The value set had good agreement with the only other experience- and preference-based value set, but poor agreement with all hypothetical value sets. Conclusions We present an experience- and preference-based value set with high face validity. The study indicates that response shift may be important to account for when deriving value sets. Furthermore, the study suggests that perspective (experienced versus hypothetical) is more important than country setting or demographics for valuation of EQ-5D-3L health states.

Cancer research is a crucial pillar for countries to deliver more affordable, higher quality, and more equitable cancer care. Patients treated in research-active hospitals have better outcomes than patients who are not treated in these settings. However, cancer in Europe is at a crossroads. Cancer was already a leading cause of premature death before the COVID-19 pandemic, and the disastrous effects of the pandemic on early diagnosis and treatment will probably set back cancer outcomes in Europe by almost a decade. Recognising the pivotal importance of research not just to mitigate the pandemic today, but to build better European cancer services and systems for patients tomorrow, the Lancet Oncology European Groundshot Commission on cancer research brings together a wide range of experts, together with detailed new data on cancer research activity across Europe during the past 12 years. We have deployed this knowledge to help inform Europe's Beating Cancer Plan and the EU Cancer Mission, and to set out an evidence-driven, patient-centred cancer research roadmap for Europe. The high-resolution cancer research data we have generated show current activities, captured through different metrics, including by region, disease burden, research domain, and effect on outcomes. We have also included granular data on research collaboration, gender of researchers, and research funding. The inclusion of granular data has facilitated the identification of areas that are perhaps overemphasised in current cancer research in Europe, while also highlighting domains that are underserved. Our detailed data emphasise the need for more information-driven and data-driven cancer research strategies and planning going forward. A particular focus must be on central and eastern Europe, because our findings emphasise the widening gap in cancer research activity, and capacity and outcomes, compared with the rest of Europe. Citizens and patients, no matter where they are, must benefit from advances in cancer research. This Commission also highlights that the narrow focus on discovery science and biopharmaceutical research in Europe needs to be widened to include such areas as prevention and early diagnosis; treatment modalities such as radiotherapy and surgery; and a larger concentration on developing a research and innovation strategy for the 20 million Europeans living beyond a cancer diagnosis. Our data highlight the important role of comprehensive cancer centres in driving the European cancer research agenda. Crucial to a functioning cancer research strategy and its translation into patient benefit is the need for a greater emphasis on health policy and systems research, including implementation science, so that the innovative technological outputs from cancer research have a clear pathway to delivery. This European cancer research Commission has identified 12 key recommendations within a call to action to reimagine cancer research and its implementation in Europe. We hope this call to action will help to achieve our ambitious 70:35 target: 70% average 10-year survival for all European cancer patients by 2035.

Efficiency in healthcare is crucial since available resources are scarce, and the cost of inefficient allocation is measured in prior outcomes. This is particularly relevant for cancer. The aim of this paper is to gain a comprehensive overview of the areas and dimensions to improve efficiency, and establish the indicators, different methods, perspectives, and areas of evaluation, to provide recommendations for how to improve efficiency and measure gains in cancer care. Methods: We conducted a two-phase design. First, a comprehensive scoping literature review was conducted, searching four databases. Studies published between 2000 and 2021 were included if they described experiences and cases of efficiency in cancer care or methods to evaluate efficiency. The results of the literature review were then discussed during two rounds of online consultation with a panel of 15 external experts invited to provide insight and comments to deliberate policy recommendations. Results: 46 papers met the inclusion criteria. Based on the papers retrieved we identified six areas for achieving efficiency gains throughout the entire care pathway and, for each area of efficiency, we categorized the methods and outcomes used to measure efficiency gain. Conclusion: This is the first attempt to systemize a scattered body of literature on how to improve efficiency in cancer care and identify key areas of improvement. Policy summary: There are many opportunities to improve efficiency in cancer care. We defined seven policy recommendations on how to improve efficiency in cancer care throughout the care pathway and how to improve the measurement of efficiency gains. © 2022

Significant progress has been achieved in human health in the European Union in recent years. New medicines, vaccines, and treatments have been developed to tackle some of the leading causes of disease and life-threatening illnesses. It is clear that investment in research and development (R&D) for innovative medicines and treatments is essential for making progress in preventing and treating diseases. Ahead of the legislative process, which should begin by the end of 2022, discussions focus on how Europe can best promote the huge potential benefits of new science and technology within the regulatory framework. The challenges in European healthcare were spelled out by the panellists at the roundtable organised by European Alliance for Personalised Medicine (EAPM). Outcomes from panellists’ discussions have been summarized and re-arranged in this paper under five headings: innovation, unmet medical need, access, security of supply, adapting to progress, and efficiency. Some of the conclusions that emerged from the panel are a call for a better overall holistic vision of the future of pharmaceuticals and health in Europe and a collaborative effort among all stakeholders, seeing the delivery of medicines as part of a broader picture of healthcare. © 2022 by the authors.

Background In 2015, the Swedish government in an unprecedented move decided to allocate 150 million euro to provide funding for new drugs for hepatitis C. This was triggered by the introduction of the first second generation of direct-acting antivirals (DAAs) promising higher cure rates and reduced side effects. The drugs were cost-effective but had a prohibitive budget impact. Subsequently, additional products have entered the market leading to reduction in prices and expansions of the eligible patient base. Methods We estimated the social surplus generated by the new DAAs in Stockholm, Sweden, for the years 2014-2019. The actual use and cost of the drugs was based on registry data. Effects on future health care costs, indirect costs and QALY gains were estimated using a Markov model based primarily on Swedish data and using previous generations of interferon-based therapies as the counterfactual. Results A considerable social surplus was generated, 15% of which was appropriated by the producers whose share fell rapidly over time as prices fell. Most of the consumer surplus was generated by QALY gains, although 10% was from reduced indirect costs. QALY gains increased less rapidly than the number of treated patients as the eligibility criteria was loosened. Conclusions The transfer of funds from the government to the regions helped generate substantial surplus for both consumers and producers with indirect costs playing an important role. The funding model may serve as a model for the financing of innovative treatments in the future.

Key stakeholders from the cancer research continuum met in May 2021 at the European Cancer Research Summit in Porto to discuss priorities and specific action points required for the successful implementation of the European Cancer Mission and Europe's Beating Cancer Plan (EBCP). Speakers presented a unified view about the need to establish high-quality, networked infrastructures to decrease cancer incidence, increase the cure rate, improve patient's survival and quality of life, and deal with research and care inequalities across the European Union (EU). These infrastructures, featuring Comprehensive Cancer Centres (CCCs) as key components, will integrate care, prevention and research across the entire cancer continuum to support the development of personalized/precision cancer medicine in Europe. The three pillars of the recommended European infrastructures – namely translational research, clinical/prevention trials and outcomes research – were pondered at length. Speakers addressing the future needs of translational research focused on the prospects of multiomics assisted preclinical research, progress in Molecular and Digital Pathology, immunotherapy, liquid biopsy and science data. The clinical/prevention trial session presented the requirements for next-generation, multicentric trials entailing unified strategies for patient stratification, imaging, and biospecimen acquisition and storage. The third session highlighted the need for establishing outcomes research infrastructures to cover primary prevention, early detection, clinical effectiveness of innovations, health-related quality-of-life assessment, survivorship research and health economics. An important outcome of the Summit was the presentation of the Porto Declaration, which called for a collective and committed action throughout Europe to develop the cancer research infrastructures indispensable for fostering innovation and decreasing inequalities within and between member states. Moreover, the Summit guidelines will assist decision making in the context of a unique EU-wide cancer initiative that, if expertly implemented, will decrease the cancer death toll and improve the quality of life of those confronted with cancer, and this is carried out at an affordable cost. © 2021 The Authors. Molecular Oncology published by John WileyXX1Sons Ltd on behalf of Federation of European Biochemical Societies

Background Aims: Non-alcoholic steatohepatitis (NASH) leads to cirrhosis and is associated with a substantial socioeconomic burden, which, coupled with rising prevalence, is a growing public health challenge. However, there are few real-world data available describing the impact of NASH. Methods: The Global Assessment of the Impact of NASH (GAIN) study is a prevalence-based burden of illness study across Europe (France, Germany, Italy, Spain, and the UK) and the USA. Physicians provided demographic, clinical, and economic patient information via an online survey. In total, 3,754 patients found to have NASH on liver biopsy were stratified by fibrosis score and by biomarkers as either early or advanced fibrosis. Per-patient costs were estimated using national unit price data and extrapolated to the population level to calculate the economic burden. Of the patients, 767 (20%) provided information on indirect costs and health-related quality of life using the EuroQOL 5-D (EQ-5D; n = 749) and Chronic Liver Disease Questionnaire - Non-Alcoholic Fatty Liver Disease (CLDQ-NAFLD) (n = 723).Results: Mean EQ-5D and CLDQ-NAFLD index scores were 0.75 and 4.9, respectively. For 2018, the mean total annual per patient cost of NASH was (sic)2,763, (sic)4,917, and (sic)5,509 for direct medical, direct non-medical, and indirect costs, respectively. National per-patient cost was highest in the USA and lowest in France. Costs increased with fibrosis and decompensation, driven by hospitalisation and comorbidities. Indirect costs were driven by work loss. Conclusions: The GAIN study provides real-world data on the direct medical, direct non-medical, and indirect costs associated with NASH, including patient-reported outcomes in Europe and the USA, showing a substantial burden on health services and individuals.