Working to improve the management of sarcoma patients across Europe: a policy checklist.

BACKGROUND: The Sarcoma Policy Checklist was created by a multidisciplinary expert group to provide policymakers with priority areas to improve care for sarcoma patients. MAIN BODY: This paper draws on this research, by looking more closely at how France, Germany, Italy, Spain, Sweden and the United Kingdom are addressing each of these priority areas. It aims to highlight key gaps in research, policy and practice, as well as ongoing initiatives that may impact the future care of sarcoma patients in different European countries. A pragmatic review of the published and web-based literature was undertaken. Telephone interviews were conducted in each country with clinical and patient experts to substantiate findings. Research findings were discussed within the expert group and developed into five core policy recommendations. The five identified priority areas were: the development of designated and accredited centres of reference; more professional training; multidisciplinary care; greater incentives for research and innovation; and more rapid access to effective treatments. Most of the countries studied have ongoing initiatives addressing many of these priorities; however, many are in early stages of development, or require additional funding and resources. CONCLUSION: Gaps in access to quality care are particularly concerning in many of Europe's lower-resourced countries. Equitable access to information, clinical trials, innovative treatments and quality specialist care should be available to all sarcoma patients. Achieving this across Europe will require close collaboration between all stakeholders at both the national and European level.

Burden of Illness in Hereditary Angioedema: A Conceptual Model.

The objective of the Hereditary Angioedema Burden of Illness Study in Europe was to assess the real-world experience of hereditary angioedema (HAE) from the patient perspective. Based on open-ended qualitative interviews with 30 patients from Spain, Germany and Denmark, 5 key themes emerged characterizing the impact of HAE on health-related quality of life (HRQoL): (i) unnecessary treatments and procedures, (ii) symptom triggers, (iii) attack impacts, (iv) caregiver impacts, and (v) long-term impacts. Patients for example experience unnecessary medical procedures due to diagnostic delays; anxiety and fear about attacks, and passing HAE to children; reduced work/school productivity; and limited career/educational achievement. Patient caregivers also experience worry and work/activity interruption during the attacks. In conclusion, a conceptual model was developed illustrating the hypothesized relationships among the wide-ranging short- and long-term HRQoL impacts of HAE. These findings can be used to highlight important issues in clinical management, raise awareness of the patients' experience among policymakers and help guide measurement of HRQoL outcomes in future studies in HAE.

The administration of rescue medication to children with prolonged acute convulsive seizures in a non-hospital setting: an exploratory survey of healthcare professionals' perspectives.

UNLABELLED: Timely administration of rescue medication for prolonged convulsive seizures lasting more than 5 min is necessary to prevent progression to status epilepticus in children. The Practices in Emergency and Rescue Medication for Epilepsy Managed with Community Administered Therapy (PERFECT™) initiative was set up to gain a better understanding of how prolonged convulsive seizures in children are managed when they occur outside of the hospital. We present the findings from an exploratory telephone survey of 128 healthcare professionals (HCPs) (85 pediatric neurologists and neurologists, 28 community pediatricians, and 15 epilepsy nurses) from six EU countries, conducted as part of the PERFECT™ initiative. Among HCPs, there was greater awareness of local protocols and lesser awareness of national or international guidelines. HCPs were not very aware of how prolonged convulsive seizures were managed outside of the hospital and had few professional links to schools or other settings where these seizures were most likely to occur. Approximately one third believed that lack of confidence and fear of liability were barriers to caregivers administering treatment in schools, as was insufficient training of caregivers in the wider community on the management of prolonged convulsive seizures. CONCLUSION: Results of this HCP survey have identified several clear gaps that need to be addressed: clearer guidance that spans all settings of care, greater dissemination of such guidelines across the chain of care, more open communication and better links between HCPs and schools, and systematic training of all relevant caregivers on the appropriate management of prolonged convulsive seizures.

The humanistic burden of hereditary angioedema: results from the Burden of Illness Study in Europe.

Hereditary angioedema (HAE) is a rare but potentially life-threatening disease marked by spontaneous, recurrent attacks of swelling. The broad range of consequences of HAE on patients' lives is not well understood. The study objective was to comprehensively characterize the burden of illness and impact of HAE types I and II from the patient perspective. The HAE Burden of Illness Study in Europe was conducted in Spain, Germany, and Denmark to assess the real-world experience of HAE from the patient perspective via a one-time survey, which included items on clinical characteristics and physical and emotional impacts. One hundred eighty-six patients participated; 59% reported having an attack at least once a month, 67% reported moderate-to-severe pain during their last attack, and 74% reported moderate-to-severe swelling. The most common sites of the last attack were the abdomen and extremities; 24% experienced an attack in more than one site. The impact of HAE on daily activities was high during attacks and did not vary significantly by body site affected; patients also reported that HAE impacted their daily activities between attacks. Patients reported substantial anxiety about future attacks, traveling, and passing HAE to their children. Based on Hospital Anxiety and Depression Scale scores, 38 and 14% had clinically meaningful anxiety and depression, respectively. Despite standard of care, HAE patients still have frequent and painful attacks. Patients experience substantial impairment physically and emotionally both during and between attacks. A better understanding of these effects may help in the clinical management of HAE patients.

Report from a Viral Hepatitis Policy Forum on implementing the WHO Framework for Global Action on viral hepatitis in North Asia.

BACKGROUND & AIMS: The World Health Organisation (WHO) Prevention & Control of Viral Hepatitis Infection: Framework for Global Action offers a global vision for the prevention and control of viral hepatitis. In October 2012, the Coalition to Eradicate Viral Hepatitis in Asia Pacific (CEVHAP) organised the North Asia Workshop on Viral Hepatitis in Taipei to discuss how to implement the WHO Framework in the North Asia region. This paper presents outcomes from this workshop. METHODS: Twenty-eight representatives from local liver associations, patient organisations, and centres of excellence in Hong Kong, Japan, Korea, and Taiwan participated in the workshop. FINDINGS: Priority areas for action were described along the four axes of the WHO Framework: (1) awareness, advocacy and resources; (2) evidence and data; (3) prevention of transmission; and (4) screening and treatment. Priorities included: axis 1: greater public and professional awareness, particularly among primary care physicians and local advocacy networks. Axis 2: better economic data and identifying barriers to screening and treatment uptake. Axis 3: monitoring of vaccination outcomes and targeted harm reduction strategies. Axis 4: strengthening links between hospitals and primary care providers, and secure funding of screening and treatment, including for hepatocellular carcinoma. CONCLUSIONS: The WHO Framework provides an opportunity to develop comprehensive and cohesive policies in North Asia and the broader region. A partnership between clinical specialists, primary care physicians, policy makers, and people with or at risk of viral hepatitis is essential in shaping future policies.

The hereditary angioedema burden of illness study in Europe (HAE-BOIS-Europe): background and methodology.

BACKGROUND: Hereditary angioedema (HAE) is a rare but serious disease marked by swelling attacks in the extremities, face, trunk, airway, or abdominal areas that can be spontaneous or the result of trauma and other triggers. It can be life-threatening due to the risk of asphyxiation. While there have been major advancements in our understanding of the immunogenetics of HAE, there are significant gaps in the literature regarding understanding of the humanistic and economic impact of the disease, particularly in Europe. The purpose of the HAE Burden of Illness Study-Europe (HAE-BOIS-Europe), the development and methodology of which is described here, is to better understand the management and impact of HAE from the patient perspective in Europe. METHODS/DESIGN: This is a cross-sectional study in which retrospective data were also collected being conducted in Denmark, Germany and Spain. The study is open to patients ages 12 and older with a diagnosis of HAE-I or HAE-II. Data collection includes: (i) a survey on individuals' health care resource use, direct and indirect medical costs, impact on work and school, treatment satisfaction, and emotional functioning (via the Hospital Anxiety and Depression Scale); and (ii) one-on-one interviews to collect detailed descriptive data and patient testimonials on the impact of HAE on patients' health-related quality of life. DISCUSSION: The present manuscript describes the development and plans for implementing a multi-country European study with the aim of characterizing the humanistic and economic burden of HAE from the patient perspective. This study will help raise awareness of HAE as a rare but debilitating condition with wide-ranging impacts.

The need for a comprehensive and integrated approach to lung cancer policy in Europe.

Lung cancer is the leading cause of cancer-related deaths in Europe. Europe's Beating Cancer Plan calls for a comprehensive approach to the disease in general but not specifically to lung cancer. Such a comprehensive approach, integrating efforts to strengthen anti-tobacco policies, early detection and underlying models of care, is sorely needed for lung cancer - particularly considering disruptions to care during the COVID-19 pandemic. In a recently published think piece, a multidisciplinary group of experts proposed four key policy priority areas. First, to reduce stigma and improve awareness of potential symptoms, there is a need to foster a better understanding of lung cancer - among the public and healthcare professionals. Second, opportunities for early detection should be enhanced, and the implementation of targeted screening through low-dose computed tomography should be encouraged as a complement to smoking cessation services. This complementarity should be recognised and built into joint policy proposals, with development and better integration of screening and smoking cessation programmes on the ground. Third, the socioeconomic inequalities underpinning disparities in outcomes in people with lung cancer must be addressed, with targeted approaches to overcome barriers to access Finally, the overall quality of lung cancer care must be improved, making multidisciplinary care available to all and ensuring survivorship is given due attention.

Implementing Lung Cancer Screening in Europe: Taking a Systems Approach.

Lung cancer is the leading cause of cancer death in Europe. Screening by means of low-dose computed tomography (LDCT) can shift detection to an earlier stage and reduce lung cancer mortality in high-risk individuals. However, to date, Poland, Croatia, Italy, and Romania are the only European countries to commit to large-scale implementation of targeted LDCT screening. Using a health systems approach, this article evaluates key factors needed to enable the successful implementation of screening programs across Europe. Recent literature on LDCT screening was reviewed for 10 countries (Belgium, Croatia, France, Germany, Italy, the Netherlands, Poland, Spain, Sweden, and United Kingdom) and complemented by 17 semistructured interviews with local experts. Research findings were mapped against a health systems framework adapted for lung cancer screening. The European policy landscape is highly variable, but potential barriers to implementation are similar across countries and consistent with those reported for other cancer screening programs. While consistent quality and safety of screening must be ensured across all screening centers, system factors are also important. These include appropriate data infrastructure, targeted recruitment methods that ensure equity in participation, sufficient capacity and workforce training, full integration of screening with multidisciplinary care pathways, and smoking cessation programs. Stigma and underlying perceptions of lung cancer as a self-inflicted condition are also important considerations. Building on decades of implementation research, governments now have a unique opportunity to establish effective, efficient, and equitable lung cancer screening programs adapted to their health systems, curbing the impact of lung cancer on their populations.

Creating a better journey of care for patients with heart valve disease.

Heart valve disease has been described as 'the next cardiac epidemic', with prevalence expected to double by 2040 and triple by 2060 due to the ageing of the population. Yet until now, it has been characterized by scarce data, limited research, and low general awareness compared with other cardiovascular diseases. Effective treatment options exist for heart valve disease, and early detection and treatment can dramatically change disease progression, improve quality of life, and reduce mortality. Unfortunately, in too many patients, heart valve disease is undetected, undiagnosed, untreated, or treated too late, leading to avoidable deaths and costs, and significant compromises to people's quality of life. These gaps in the patient pathway can be remedied through appropriate policy action, with a focus on: early detection and diagnosis; timely intervention; patient-centred follow-up care; patient engagement and empowerment; psychological support; and better data to guide practice. Ensuring all patients have access to appropriate diagnosis and care without delays is imperative as we look towards rebuilding stronger and more resilient health systems, and 'build back better' after the coronavirus disease-19 pandemic.

Assessing the economic merits of managing cytomegalovirus infection in organ and stem cell transplantation.

Two preventative approaches exist to manage cytomegalovirus (CMV), a common infection in recipients of organ and stem cell transplants: prophylaxis--the prevention of viraemia--and pre-emptive therapy--the prevention of manifestation of disease in patients who have viraemia. Economic evaluation may provide a helpful framework to inform the choice between these two approaches. However, several issues arise. Direct comparisons of prophylaxis and pre-emptive therapy are rare and there are few epidemiological data that depict the full natural history of CMV infection and disease. There is a need for large, prospective randomised trials that directly compare these two strategies and are of sufficient duration to assess their overall impact on direct and indirect effects of CMV as well as patient quality of life. These methodological issues are relevant to the economic evaluation of preventative measures in other clinical settings and highlight the need for a rigorous evaluative framework to best inform decision making about the optimal strategy for patients.

The life-course approach to vaccination: Harnessing the benefits of vaccination throughout life.

Vaccination beyond childhood brings significant benefits at the individual, community and socio-economic levels. Despite this, immunisation programmes often fail to deliver the vaccines which could protect those at risk of vaccine-preventable diseases. In this commentary, we argue that the benefits of vaccination beyond childhood must be more widely understood and furthermore, that action must be taken by policymakers, healthcare professionals and patient and civil society organisations to ensure that the benefits of vaccination are fully realised. We outline five areas where change is needed to ensure vaccination across the life-course becomes truly embedded in national immunisation programmes. This includes investing in robust data collection and analysis; ensuring coordinated, multidisciplinary leadership from the top; engaging healthcare professionals; changing public perceptions of vaccination; and integrating vaccination into schools and workplaces.

Identifying critical steps towards improved access to innovation in cancer care: a European CanCer Organisation position paper.

In recent decades cancer care has seen improvements in the speed and accuracy of diagnostic procedures; the effectiveness of surgery, radiation therapy and medical treatments; the power of information technology; and the development of multidisciplinary, specialist-led approaches to care. Such innovations are essential if we are to continue improving the lives of cancer patients across Europe despite financial pressures on our healthcare systems. Investment in innovation must be balanced with the need to ensure the sustainability of healthcare budgets, and all health professionals have a responsibility to help achieve this balance. It requires scrutiny of the way care is delivered; we must be ready to discontinue practices or interventions that are inefficient, and prioritise innovations that may deliver the best outcomes possible for patients within the limits of available resources. Decisions on innovations should take into account their long-term impact on patient outcomes and costs, not just their immediate costs. Adopting a culture of innovation requires a multidisciplinary team approach, with the patient at the centre and an integral part of the team. It must take a whole-system and whole-patient perspective on cancer care and be guided by high-quality real-world data, including outcomes relevant to the patient and actual costs of care; this accurately reflects the impact of any innovation in clinical practice. The European CanCer Organisation is committed to working with its member societies, patient organisations and the cancer community at large to find sustainable ways to identify and integrate the most meaningful innovations into all aspects of cancer care.

Hepatitis B and hepatitis C in southeast and southern Asia: challenges for governments.

In 2015, the Coalition to Eradicate Viral Hepatitis in Asia Pacific gathered leading hepatitis experts from Bangladesh, India, Indonesia, Malaysia, Pakistan, the Philippines, and Thailand to discuss common challenges to the burden posed by hepatitis B virus (HBV) and hepatitis C virus (HCV), to learn from each other's experience, and identify sustainable approaches. In this report, we summarise these discussions. Countries differ in their policy responses to HBV and HCV; however, substantial systemic, cultural, and financial barriers to achievement of elimination of these infections persist in all countries. Common challenges to elimination include limited availability of reliable epidemiological data; insufficient public awareness of risk factors and modes of transmission, leading to underdiagnosis; high rates of transmission through infected blood products, including in medical settings; limited access to care for people who inject drugs; prevailing stigma and discrimination against people infected with viral hepatitis; and financial barriers to treatment and care. Despite these challenges, promising examples of effective programmes, public-private initiatives, and other innovative approaches are evident in all countries we studied in Asia Pacific. The draft WHO Global Health Sector Strategy on Viral Hepatitis 2016-21 provides a solid framework upon which governments can build their local strategies towards viral hepatitis. However, greater recognition by national governments and the international community of the urgency to comprehensively tackle both HBV and HCV are still needed. In all countries, strategic plans and policy goals need to be translated into resources and concrete actions, with national governments at the helm, to enable a sustainable response to the rising burden of hepatitis B and C in all countries.

An international survey of patients with thalassemia major and their views about sustaining life-long desferrioxamine use.

BACKGROUND: Management of thalassemia major requires patients to have life-long access to a treatment regimen of regular blood transfusions coupled with iron chelation therapy. The objective of this study was to investigate patients' reasons for missing iron chelation therapy with desferrioxamine, and the support to sustain life-long adherence to treatment. METHODS: From October 1999 to May 2000 a survey of patients with thalassemia major was conducted in ten countries: Cyprus, Egypt, Greece, Hong Kong, India, Iran, Italy, Jordan, Taiwan, and the United States. RESULTS: 1,888 questionnaires (65%) were returned. Most patients (1,573) used desferrioxamine, and 79% administered a dose at least 4 days a week. Inaccessibility of the drug was a common reason for missing a dose in India (51%), and in Iran (25%), whereas, in any other country, it was a reason for less than 17% of patients. Overall, 58% reported reasons for missing a dose related to their beliefs or feelings about the medication, and 42% drug-related side effects. CONCLUSION: Many patients miss doses of desferrioxamine and an opportunity remains to develop interventions that provide more support to sustain use of desferrioxamine.

Socioeconomic burden of hereditary angioedema: results from the hereditary angioedema burden of illness study in Europe.

BACKGROUND: Hereditary angioedema (HAE) due to C1 inhibitor deficiency is a rare but serious and potentially life-threatening disease marked by spontaneous, recurrent attacks of swelling. The study objective was to characterize direct and indirect resource utilization associated with HAE from the patient perspective in Europe. METHODS: The study was conducted in Spain, Germany, and Denmark to assess the real-world experience of HAE via a cross-sectional survey of HAE patients, including direct and indirect resource utilization during and between attacks for patients and their caregivers over the past 6 months. A regression model examined predictors of medical resource utilization. RESULTS: Overall, 164 patients had an attack in the past 6 months and were included in the analysis. The most significant predictor of medical resource utilization was the severity of the last attack (OR 2.6; p < 0.001). Among patients who sought medical care during the last attack (23%), more than half utilized the emergency department. The last attack prevented patients from their normal activities an average of 4-12 hours. Patient and caregiver absenteeism increased with attack severity and frequency. Among patients who were working or in school (n = 120), 72 provided work/school absenteeism data, resulting in an estimated 20 days missing from work/school on average per year; 51% (n = 84) indicated that HAE has hindered their career/educational advancement. CONCLUSION: HAE poses a considerable burden on patients and their families in terms of direct medical costs and indirect costs related to lost productivity. This burden is substantial at the time of attacks and in between attacks.

The administration of rescue medication to children with prolonged acute convulsive seizures in the community: what happens in practice?

This paper presents the findings of a review of existing clinical and non-clinical guidance on the management of children with prolonged acute convulsive seizures (PCS) and the administration of rescue medication in community settings. Findings are based on desk- and web-based research in 6 countries. Published clinical guidelines are mostly limited to the hospital setting and offer few explicit recommendations for community settings. Non-clinical guidance on the management of medicines at school exists at the national or regional level in all 6 countries, however rescue epilepsy medication is often not mentioned specifically. Existing legal frameworks are vague and open to interpretation. As a result, whether a child receives rescue medication at school depends primarily on the availability of a willing teacher who accepts responsibility for administering it to that child during school hours. Comprehensive guidelines are clearly needed that provide practical guidance to ensure that children with PCS are treated as quickly as possible in all community settings. Recommendations for future action include: providing clearer information on PCS and rescue medication to parents and schools; putting in place an individual healthcare plan for every child with a history of PCS at his or her school; collecting more empirical data to gain a better understanding of the experience of children with PCS at school, their parents and teachers; and finally, implementing systematic training for all carers of children with PCS. The epilepsy specialist may play an important role in ensuring that these recommendations are put into place for their patients.

Are we failing to provide adequate rescue medication to children at risk of prolonged convulsive seizures in schools?

OBJECTIVE: This paper explores the issues that arise from the discussion of administering rescue medication to children who experience prolonged convulsive seizures in mainstream schools in the UK. SITUATION ANALYSIS: Current guidelines recommend immediate treatment of children with such seizures (defined as seizures lasting more than 5 min) to prevent progression to status epilepticus and neurological morbidity. As children are unconscious during prolonged convulsive seizures, whether or not they receive their treatment in time depends on the presence of a teacher or other member of staff trained and able to administer rescue medication. However, it is thought that the situation varies between schools and depends mainly on the goodwill and resources available locally. RECOMMENDATIONS: A more systematic response is needed to ensure that children receive rescue medication regardless of where their seizure occurs. Possible ways forward include: greater use of training resources for schools available from epilepsy voluntary sector organisations; consistent, practical information to schools; transparent guidance outlining a clear care pathway from the hospital to the school; and implementation and adherence to each child's individual healthcare plan. IMPLICATIONS: Children requiring emergency treatment for prolonged convulsive seizures during school hours test the goals of integrated, person-centred care as well as joined-up working to which the National Health Service (NHS) aspires. As changes to the NHS come into play and local services become reconfigured, every effort should be made to take account of the particular needs of this vulnerable group of children within broader efforts to improve the quality of paediatric epilepsy services overall.

Towards the eradication of hepatitis B in Taiwan.

Viral hepatitides are important public health problems in humans. The etiologic agents were not identified until 1965, when Baruch S. Blumberg found the relationship of Australia antigen to serum hepatitis. The antigen was found to be the surface antigen of the hepatitis B virus (HBV). This observation launched a new era in the diagnosis, prevention and treatment of hepatitis B. Over 15-20 years, the natural history of HBV infection was elucidated, and more importantly, an effective vaccine became available. The routes of transmission were also made clear, rendering effective interruption of the transmission possible. The vaccine together with effective interruption of the transmission contributed greatly to the control of HBV infection. However, these measures do very little for those who have already been chronically infected. Fortunately, specific therapies against chronic hepatitis B started to appear about 10-15 years before, and the treatments have improved substantially in the last few years. Although far from perfect, effective means to treat those who are chronically infected now exist. In Taiwan, acute and chronic liver diseases were rampant as early as the beginning of the last century. Studies around 1975, showed an extremely high prevalence of chronic hepatitis B infection in the general population (15-20%), and 80-90% of the chronic liver diseases and hepatocellular carcinoma were caused by chronic infection with the HBV. This important health problem caught the attention of the government in the late 1970s, and a government-sponsored control program was finalized in 1981. Accordingly, a mass vaccination program against hepatitis B, primarily aiming at immunizing newborn infants, was launched on July 1, 1984. Twenty years after implementation of the program, the hepatitis B carrier rate in children covered by the program decreased by 85%, from ∼ 15% to <1%. Most importantly, the deadly sequela of hepatocellular carcinoma in the vaccinees was also found to decrease in parallel. This is the first time that a human cancer was prevented by vaccination. Despite the success, there are still some who were born after implementation of the program but were not prevented from developing chronic hepatitis B infection and hepatocellular carcinoma. Non-compliance to the vaccination schedule, breakthrough infection and intrauterine infection are the causes of the failure. At present, we have effective measures for immunizing susceptible individuals, interrupting the routes of transmission and treating the chronically infected. The time for considering the elimination or even the eradication of HBV infection has come. This is especially true for countries where hepatitis B infection is not endemic. Nevertheless, with the admirable results achieved in the past, Taiwan should also think about elimination/eradication of hepatitis B, even though it will certainly be much more difficult than in the non-endemic countries. In exploring the possibility of eliminating/eradicating hepatitis B in Taiwan, we reviewed the epidemiology of hepatitis B, and analyzed the problems that remain to be tackled in Taiwan. We hope that Taiwan can take further steps towards the elimination or eradication of hepatitis B.