Can a Joint Assessment Provide Relevant Information for National/Local Relative Effectiveness Assessments? An In-Depth Comparison of Pazopanib Assessments

BackgroundIn many European jurisdictions, relative effectiveness assessments (REAs) of pharmaceuticals are performed during the reimbursement decision-making process. International collaboration in the production of these assessments may prevent the duplication of information in various jurisdictions. A first pilot of a joint REA (pazopanib for the treatment of renal cell carcinoma) was published in 2011.ObjectiveThe objective was to investigate how well the methods used in the joint REA match the methods used in the national/local assessments on the same topic.MethodsNational/local assessments from European jurisdictions, available in English language, were identified through a literature search and an e-mail request to health technology assessment organizations. Data were abstracted from joint and national/local assessments using a structured data abstraction form. Results were compared for differences and similarities.ResultsIn total, five national/local reports were included (Belgium, England/Wales, France, The Netherlands, and Scotland). The general methods (indication, main comparator, main end points, main trial) were similar. The details of the assessment (e.g., exact wording of indication, additional comparators, additional trials included, and method of indirect comparison), however, varied. Despite these differences, the joint REA included nearly all comparators, end points, trials, and methods of analysis that were used in national/local REA reports.ConclusionsThis study has shown overlap in the methods national/local REA bodies in Europe have chosen for a pazopanib REA for renal cell carcinoma, except for the use and methods of indirect comparisons. Although some additional comparators and outcomes differed between national/local REAs, they can be captured in a comprehensive joint REA.     

Introducing the patient's perspective in hospital health technology assessment (HTA): the views of HTA producers,hospital managers and patients

Background

The recent establishment of health technology assessment (HTA) units in University hospitals in the Province of Quebec (Canada) provides a unique opportunity to foster increased participation of patients in decisions regarding health technologies and interventions at the local level. However, little is known about factors that influence whether the patient''s perspective is taken into consideration when such decisions are made.

Objective

To explore the practices, perceptions and views of the various HTA stakeholders concerning patient involvement in HTA at the local level.

Method

Data were collected using semi‐structured interviews with 24 HTA producers and hospital managers and two focus groups with a total of 13 patient representatives.

Results

Patient representatives generally showed considerable interest in being involved in HTA. Our findings support the hypothesis that the patient perspective contributes to a more accurate and contextualized assessment of health technologies and produces HTA reports that are more useful for decision makers. They also suggest that participation throughout the assessment process could empower patients and improve their knowledge. Barriers to patient involvement in HTA at the local level are also discussed as well as potential strategies to overcome them.

Discussion and conclusion

This study contributes to knowledge that could guide interventions in favour of patient participation in HTA activities at the local level. Experimenting with different patient involvement strategies and assessing their impact is needed to provide evidence that will inform future interventions of this kind.     

Methods for Early Assessment of the Societal Value of Health Technologies: A Scoping Review and Proposal for Classification

ObjectivesEarly assessments of health technologies help to better align and integrate their development and assessment. Such assessments can take many forms and serve different purposes, hampering users in their selection of the most appropriate method for a specific goal. The aim of this scoping review was to structure the large set of methods according to their specific goal.MethodsA scoping review was conducted using PubMed and reference lists of retrieved articles, to identify review studies with a methodological focus. From the included reviews, all individual methods were listed. Based on additional literature and examples, we extracted the specific goal of each method. All goals were clustered to derive a set of subclasses and methods were grouped into these subclasses.ResultsOf the 404 screened, 5 reviews were included, and 1 was added when searching reference lists. The reviews described 56 methods, of which 43 (77%) were included and classified as methods to (1) explore the nature and magnitude of the problem, (2) estimate the nature and magnitude of the expected (societal) value, (3) identify conditions for the potential value to materialize, and (4) help develop and design the type of research that is needed.ConclusionsThe wide range of methods for exploring the societal value of health technologies at an early stage of development can be subdivided into a limited number of classes, distinguishing methods according to their specific objective. This facilitates selection of appropriate methods, depending on the specific needs and aims.     

Challenges and Solutions for the Benefit Assessment of Tumor-Agnostic Therapies in Germany

ObjectivesPrecision medicine is increasingly important in cancer treatment. Tumor-agnostic therapies are used regardless of tumor entity because they target specific biomarkers in tumors. In Germany, the benefit assessment of oncological pharmaceuticals has traditionally been entity specific. Thus, the assessment of tumor-agnostic therapies leaves stakeholders with various challenges. Our aim was to systematically identify challenges and possible solutions for the benefit assessment of therapies in tumor-agnostic indications using a 2-step sequential qualitative approach.MethodsTo identify relevant challenges, we conducted qualitative interviews with different stakeholders who were involved in previous benefit assessments of tumor-agnostic therapies in Germany. To identify possible solutions for these challenges, we systematically searched MEDLINE, Embase, and the websites of European health technology assessment bodies for relevant literature.ResultsWe identified 9 categories of challenges of which the following were deemed particularly relevant: the absence of direct comparative studies, challenges regarding the use of basket studies and indirect comparisons, challenges in determining the appropriate comparative therapy in a tumor-agnostic indication, and challenges on the system side. Seven categories of solutions were identified, including an increased use of real-world evidence, making conditional decisions in the context of systematic reassessments, splitting the field of application, and finding (new) ways to design and analyze basket studies.ConclusionA range of possible solutions, which can help to meet the identified challenges in Germany, have been found. Future research should investigate the acceptance and feasibility of these solutions.     

Policies for Use of Real-World Data in Health Technology Assessment (HTA): A Comparative Study of Six HTA Agencies

Background

Randomized controlled trials provide robust data on the efficacy of interventions rather than on effectiveness. Health technology assessment (HTA) agencies worldwide are thus exploring whether real-world data (RWD) may provide alternative sources of data on effectiveness of interventions. Presently, an overview of HTA agencies’ policies for RWD use in relative effectiveness assessments (REA) is lacking.

妇科手术患者健康教育的实施和效果评价

目的 探讨对妇科手术患者实施健康教育的效果.方法 随机选取我院2010年6月-2013年6月期间收治的妇科手术患者120例,随机平均分为实验组和对照组,对实验组患者进行健康教育,对照组不采取健康教育措施.采用调查问卷的方式对两组患者手术前后疾病相关知识的掌握情况、对护理质量的满意度、情绪稳定情况、术后体重变化情况及术后肠功能情况进行调查统计.结果 实验组患者的疾病相关知识掌握度、对护理质量的满意度及情绪稳定情况均明显优于对照组,差异具有显著性（P＜0.05）;实验组患者的肛门排气时间、肠鸣音的恢复时间以及出院时体重变化程度明显小于对照组,差异具有显著性（P＜0.05）.结论 对妇科手术患者实施健康教育,有利于改善患者的手术依从性,降低术后并发症的发生率,促进患者术后健康恢复.     

Designing and Implementing Deliberative Processes for Health Technology Assessment: A Good Practices Report of a Joint HTAi/ISPOR Task Force

ObjectivesDeliberative processes for health technology assessment (HTA) are intended to facilitate participatory decision making, using discussion and open dialogue between stakeholders. Increasing attention is being given to deliberative processes, but guidance is lacking for those who wish to design or use them. Health Technology Assessment International (HTAi) and ISPOR—The Professional Society for Health Economics and Outcomes Research initiated a joint Task Force to address this gap.MethodsThe joint Task Force consisted of 15 members with different backgrounds, perspectives, and expertise relevant to the field. It developed guidance and a checklist for deliberative processes for HTA. The guidance builds upon the few, existing initiatives in the field, as well as input from the HTA community following an established consultation plan. In addition, the guidance was subject to 2 rounds of peer review.ResultsA deliberative process for HTA consists of procedures, activities, and events that support the informed and critical examination of an issue and the weighing of arguments and evidence to guide a subsequent decision. Guidance and an accompanying checklist are provided for (i) developing the governance and structure of an HTA program and (ii) informing how the various stages of an HTA process might be managed using deliberation.ConclusionsThe guidance and the checklist contain a series of questions, grouped by 6 phases of a model deliberative process. They are offered as practical tools for those wishing to establish or improve deliberative processes for HTA that are fit for local contexts. The tools can also be used for independent scrutiny of deliberative processes.     

Reported Challenges in Health Technology Assessment of Complex Health Technologies

ObjectivesWith complex health technologies entering the market, methods for health technology assessment (HTA) may require changes. This study aimed to identify challenges in HTA of complex health technologies.MethodsA survey was sent to European HTA organizations participating in European Network for HTA (EUnetHTA). The survey contained open questions and used predefined potentially complex health technologies and 7 case studies to identify types of complex health technologies and challenges faced during HTA. The survey was validated, tested for reliability by an expert panel, and pilot tested before dissemination.ResultsA total of 22 HTA organizations completed the survey (67%). Advanced therapeutic medicinal products (ATMPs) and histology-independent therapies were considered most challenging based on the predefined complex health technologies and case studies. For the case studies, more than half of the reported challenges were “methodological,” equal in relative effectiveness assessments as in cost-effectiveness assessments. Through the open questions, we found that most of these challenges actually rooted in data unavailability. Data were reported as “absent,” “insufficient,” “immature,” or “low quality” by 18 of 20 organizations (90%), in particular data on quality of life. Policy and organizational challenges and challenges because of societal or political pressure were reported by 8 (40%) and 4 organizations (20%), respectively. Modeling issues were reported least often (n = 2, 4%).ConclusionsMost challenges in HTA of complex health technologies root in data insufficiencies rather than in the complexity of health technologies itself. As the number of complex technologies grows, the urgency for new methods and policies to guide HTA decision making increases.     

Acceptability and Perceived Benefits and Risks of Public and Patient Involvement in Health Care Policy: A Delphi Survey in Belgian Stakeholders

BackgroundIn systems with public health insurance, coverage decisions should reflect social values. Deliberation among stakeholders could achieve this goal, but rarely involves patients and citizens directly.ObjectivesThis study aimed at evaluating the acceptability, and the perceived benefits and risks, of public and patient involvement (PPI) in coverage decision making to Belgian stakeholders.MethodsA two-round Delphi survey was conducted among all stakeholder groups. The survey was constructed on the basis of interviews with 10 key stakeholders and a review of the literature on participation models. Consensus was defined as 65% or more of the respondents agreeing with a statement and less than 15% disagreeing. Eighty stakeholders participated in both rounds. They were defined as the Delphi panel.ResultsBelgian stakeholders are open toward PPI in coverage decision processes. Benefits are expected to exceed risks. The preferred model for involvement is to consult citizens or patients, within the existing decision-making structures and at specific milestones in the process. Consulting citizens and patients is a higher level of involvement than merely informing them and a lower level than letting them participate actively. Consultation involves asking nonbinding advice on (parts of) the decision problem. According to the Delphi panel, the benefits of PPI could be increasing awareness among members of the general public and patients about the challenges and costs of health care, and enriched decision processes with expertise by experience from patients. Potential risks include subjectivity, insufficient resources to participate and weigh on the process, difficulties in finding effective ways to express a collective opinion, the risk of manipulation, and lobbying or power games of other stakeholders.ConclusionsPPI in coverage decision-making processes is acceptable to Belgian stakeholders, be it in different ways for different types of decisions. Benefits are expected to outweigh risks.     

上海市彭浦社区卫生服务中心高血压监测效果及患者医疗费用研究

目的研究彭浦社区卫生服务中心高血压患者实施监测其控制效果及对患者医疗费用的影响。方法在3个级别监测管理的高血压目标对象中随机选择研究对象，以调查表形式进行入户调查，并对2000-2003年社区所有监测管理的高血压患者登记资料进行整理统计。结果一级监测管理目标人群血压控制效果达到50、30％、二级为35、52％、三级为24、8l％：一级监测管理人群年人均就诊医疗费用为2016、31元、二级为1556．18元、三级为106．64元；4年的社区高血压监测管理，估计减少患者就诊支付费用达1218206．74元。结论社区高血压监测管理对控制高血压患者病情与并发症的发生、发展起到积极作用，并且减轻患者与国家的疾病经济负担。     

我国区域卫生规划政策的实施效果评价

区域卫生规划作为我国卫生改革的重大政策,从理论研究到地区试点,再到全国的推广历经十几年,特别是在1999年以后,区域卫生规划在全国推广。但是,对于区域卫生规划的实施效果却没有系统的评价。利用卫生费用在不同卫生服务和不同层次医疗机构的流向、政府卫生经费在不同层次医疗机构的分配和大型医用设备控制政策的执行效果等指标,对区域卫生规划执行效果进行了初步评价。其结论是,区域卫生规划调整卫生资源配置和病人流向以及控制大型医用设备过快增长的目标没有实现。     

Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group

Background

Successful development of new treatments for rare diseases (RDs) and their sustainable patient access require overcoming a series of challenges related to research and health technology assessment (HTA). These impediments, which may be unique to RDs or also apply to common diseases but are particularly pertinent in RDs, are diverse and interrelated.

Exploring a collaborative approach to the involvement of patients,carers and the public in the initial education and training of healthcare professionals: A qualitative study of patient experiences

ObjectiveThis study aimed to explore patients'' experiences of their involvement in the design and delivery of interprofessional education interventions focussing on mental ill‐health for students studying in undergraduate healthcare and healthcare‐related programmes.DesignA qualitative methodology using a Grounded Theory approach was used to undertake an iterative series of focus groups with members of a university''s Patient, Carer and Public Involvement (PCPI) Group who have a history of mental ill‐health and were involved in the development and delivery of educational interventions for students on undergraduate healthcare and healthcare‐related programmes. Their experiences of being involved in teaching and learning activities, collaboration with academic staff and integration into the academic faculty were explored. Constant comparative analysis facilitated the identification and prioritisation of salient themes.ResultsFive salient inter‐related themes emerged from the data: (1) reduced stigma and normalisation of experience of illness; (2) enhanced self‐worth; (3) improved well‐being; (4) community and connection; and (5) enduring benefits.ConclusionsA supportive university community and a designated academic PCPI co‐ordinator facilitate a supportive environment for patients and carers to develop as educators, contribute to the training of future healthcare professionals and improve their own personal well‐being. Appropriately resourced and well‐supported initiatives to integrate patients, carers and the public into the functions of an academic faculty can result in tangible benefits to individuals and facilitate meaningful and enduring connections between the university and the wider community within which it is situated.Patient and Public InvolvementPatients have been involved in the design of the teaching and learning initiatives that this study was primarily focused on. Patients were given autonomy in determining how their experiences should be incorporated into teaching and learning experiences.     

中国重庆市避孕技术引入需求的战略评估

为了扩大避孕选择和改善服务质量,使避孕技术引入的决策更符合实际和具科学依据,系统地评估重庆地区育龄群众对避孕节育和生殖健康的需求、重庆市服务提供系统和避孕节育技术的现状,进而对改善计划生育/生殖健康服务质量提出建议,包括引入避孕技术过程中的服务质量。以世界卫生组织生殖健康和科学研究署的避孕技术引入的战略方案作为本次定性研究的基本研究方法,结合我国实际,2000年10～11月间,从重庆市43个区县中选取有代表性并能反映重庆地区特点的一个区和三个县,进行系统战略评估。共访问了14个乡镇,其中由评估组随机选择了7个乡镇,7个乡镇由县计生委选择。评估中访问了各级政府和行政管理人员、技术服务人员和服务对象,并在村、乡、县和市访谈了妇女主任等社区领导,妇女、男人和未婚青少年,共425人。观察了县/乡计划生育服务站、区县妇幼保健院、乡卫生院、村卫生室和零售药店,共49个服务机构。多数育龄群众愿意接受现行的‘一对夫妇生育一个孩子为好’的政策,但部分山区和以农业为主的群众仍希望有一个男孩。评估中发现年龄30岁以下、女性、居住在城市、初中以上文化或有外出务工经历的育龄群众避孕知识相对了解得多些。人们对于生殖道感染和性传播感染方面的知识均非常有限。由于宫内节育器使用?     

The Oral Health Needs of Wisconsin Farmers and the Need for Patient Activation: A Pilot Study

Objectives: This study aimed to determine the level of oral health need of Wisconsin farmers based on sociodemographic variables, perceived oral health, and actual oral health, and to evaluate the effectiveness of a dental hygiene patient activation intervention. Methods: Oral health screenings were administered by calibrated dental hygienists to 60 Wisconsin farmers attending the 2016 Wisconsin Farm Technology Days. Study participants self-administered the Oral Health Inventory Profile-14 survey and participated in an Adult Basic Screening Survey. Validity and reliability of both instruments have been established in previous studies. A follow-up phone call for Wisconsin farmers with a moderate- to high-risk oral health condition determined study participants followed the dental hygiene recommendations signified if study participants followed the dental hygiene recommendations and if patient activation had been achieved. Results: Study participants represented Wisconsin farmers (N = 60; n = 32 men, n = 28 women) with an average age range between 50 and 60 years old. The Oral Health Impact Profile-14 survey results indicated that this group of Wisconsin farmers did not perceive themselves to have an oral health problem. The Adult Basic Screening Survey results also indicated that the majority of study participants did not currently have active oral disease. There were 32% (n = 19) who qualified for follow-up phone call, with 15% (n = 3) seeking oral health care based on the dental hygiene recommendation. Significant associations between sociodemographic variables and actual oral health were not found, and the null hypotheses were not rejected. Conclusions: Findings suggest that this group of Wisconsin farmers is receiving regular oral health care and patient activation despite literature suggesting that farmers and rural individuals might face unique barriers to health and oral health care.     

Towards a stakeholders' consensus on patient payment policy: the views of health‐care consumers,providers, insurers and policy makers in six Central and Eastern European countries

Background

Although patient charges for health‐care services may contribute to a more sustainable health‐care financing, they often raise public opposition, which impedes their introduction. Thus, a consensus among the main stakeholders on the presence and role of patient charges should be worked out to assure their successful implementation.

Aim

To analyse the acceptability of formal patient charges for health‐care services in a basic package among different health‐care system stakeholders in six Central and Eastern European countries (Bulgaria, Hungary, Lithuania, Poland, Romania and Ukraine).

Methods

Qualitative data were collected in 2009 via focus group discussions and in‐depth interviews with health‐care consumers, providers, policy makers and insurers. The same participants were asked to fill in a self‐administrative questionnaire. Qualitative and quantitative data are analysed separately to outline similarities and differences in the opinions between the stakeholder groups and across countries.

Results

There is a rather weak consensus on patient charges in the countries. Health policy makers and insurers strongly advocate patient charges. Health‐care providers overall support charges but their financial profits from the system strongly affects their approval. Consumers are against paying for services, mostly due to poor quality and access to health‐care services and inability to pay.

Conclusions

To build consensus on patient charges, the payment policy should be responsive to consumers'' needs with regard to quality and equity. Transparency and accountability in the health‐care system should be improved to enhance public trust and acceptance of patient payments.