Uncertainty management in regulatory and health technology assessment decision-making on drugs: guidance of the HTAi-DIA Working Group.

OBJECTIVES: Uncertainty is a fundamental component of decision making regarding access to and pricing and reimbursement of drugs. The context-specific interpretation and mitigation of uncertainty remain major challenges for decision makers. Following the 2021 HTAi Global Policy Forum, a cross-sectoral, interdisciplinary HTAi-DIA Working Group (WG) was initiated to develop guidance to support stakeholder deliberation on the systematic identification and mitigation of uncertainties in the regulatory-HTA interface. METHODS: Six online discussions among WG members (Dec 2021-Sep 2022) who examined the output of a scoping review, two literature-based case studies and a survey; application of the initial guidance to a real-world case study; and two international conference panel discussions. RESULTS: The WG identified key concepts, clustered into twelve building blocks that were collectively perceived to define uncertainty: "unavailable," "inaccurate," "conflicting," "not understandable," "random variation," "information," "prediction," "impact," "risk," "relevance," "context," and "judgment." These were converted into a checklist to explain and define whether any issue constitutes a decision-relevant uncertainty. A taxonomy of domains in which uncertainty may exist within the regulatory-HTA interface was developed to facilitate categorization. The real-world case study was used to demonstrate how the guidance may facilitate deliberation between stakeholders and where additional guidance development may be needed. CONCLUSIONS: The systematic approach taken for the identification of uncertainties in this guidance has the potential to facilitate understanding of uncertainty and its management across different stakeholders involved in drug development and evaluation. This can improve consistency and transparency throughout decision processes. To further support uncertainty management, linkage to suitable mitigation strategies is necessary.

Patients as research partners in preference studies: learnings from IMI-PREFER.

BACKGROUND: There is growing recognition of the importance of patient and public stakeholder involvement (PPI) in patient preference research. However, limited evidence exists regarding the impact, barriers and enablers of PPI in preference studies. The Innovative Medicines Initiative (IMI)-PREFER project conducted a series of preference case studies which incorporated PPI. OBJECTIVE: To describe: (1) how PPI was operationalized in the PREFER case studies, (2) the impact of PPI, and (3) factors that served to impede and facilitate PPI. METHODS: We reviewed the PREFER final study reports to determine how patient partners were involved. We conducted a thematic framework analysis to characterize the impact of PPI and then administered a questionnaire to the PREFER study leads to identify barriers and facilitators to effective PPI. RESULTS: Eight PREFER case studies involved patients as research partners. Patient partners were involved in activities spanning all phases of the patient preference research process, including in study design, conduct and dissemination. However, the type and degree of patient partner involvement varied considerably. Positive impacts of PPI included improvements in the: (1) quality of the research and research process; (2) patient partner empowerment; (3) study transparency and dissemination of results; (4) research ethics, and (5) trust and respect between the research team and the patient community. Of the 13 barriers identified, the 3 most frequently reported were inadequate resources, insufficient time to fully involve patient partners, and uncertainty regarding how to operationalize the role of 'patient partner. Among the 12 facilitators identified, the two most frequently cited were (1) having a clearly stated purpose for involving patients as research partners; and (2) having multiple patient partners involved in the study. CONCLUSION: PPI had many positive impacts on the PREFER studies. Preference study leads with prior PPI experience reported a greater number of positive impacts than those with no such experience. In light of the numerous barriers identified, multi-faceted implementation strategies should be considered to support adoption, integration and sustainment of PPI within preference research. Additional case studies of patient partner involvement in preference research are needed as well to inform best practices in this area.

Research about patients' preferences for medicinal products and treatments is growing. Such research could be improved if patients were involved as 'research partners,' that is, as active members of the study team itself. To date, however, little is known about the actual experience of involving patients as partners in such research. This paper presents learnings from involving patients as partners in 8 case studies conducted as part of IMI-PREFER, a big, European-based project which aimed to develop recommendations about how to conduct preference research. Involving patients as partners led to improvements in the: (1) quality of the research and research process; (2) recruitment of participants; (3) content and design of patient-facing informational materials; and, (4) how and what study results were shared with patient communities. Our findings showed that it is important to plan for patient partners' involvement early on in the design of the preference study so as to ensure that they are fully integrated into the research team and their opportunity to contribute to all stages of the research is optimized. Such planning should address how patient partners will be paid, what their role responsibilities will include, how and when they will be trained and educated, and how they will be supported throughout the course of the study. Having a clearly stated purpose for involving patients as research partners, selecting patient partners who have had prior research experience and relationships with the researchers, and having multiple patient partners on the study team are all also helpful in supporting successful patient involvement. We need more people to share their experiences with involving patient partners in preference research so that we can continue to improve how this is done.

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

OBJECTIVES: Deliberative 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. METHODS: The joint Task Force consisted of fifteen 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 two rounds of peer review. RESULTS: A 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. CONCLUSIONS: The guidance and the checklist contain a series of questions, grouped by six 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.

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

OBJECTIVES: Deliberative 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. METHODS: The 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. RESULTS: A 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. CONCLUSIONS: The 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.

Improving the quality of care for people with chronic diseases: translating recommendations to practice.

INTRODUCTION: Aim of this paper is to present a guide for translating to practice an evidence-based set of Quality Criteria and Recommendations (QCR) to promote the implementation of policies and practices in the field of health promotion, disease prevention and care for people with chronic diseases. METHODS: The guide is based on real-world experiences of eight European pilot actions using QCR as a framework for practice design, development, implementation, monitoring and evaluation. All partners implemented their respective practices by following the same agreed process. RESULTS: The implementation method was summarized in seven steps where each of one outline a particular phase of the process. The guide provides a step-by-step tutorial for the implementation of QCR. CONCLUSIONS: Practical experiences from the pilot actions show the potential value of using the QCR in designing and implementing practices to improve the quality of care for people with chronic diseases.

The development of a core outcomes set for self-management interventions for patients living with obesity.

Self-management interventions (SMIs) can improve the life of patients living with obesity. However, there is variability in the outcomes used to assess the effectiveness of SMIs and these are often not relevant for patients. In the context of COMPAR-EU, our aim was to develop a core outcome set (COS) for the evaluation of SMIs for patients with obesity. We followed a four steps multimethod approach: (1) the development of the initial catalogue of outcomes; (2) a scoping review of reviews on patients' values and preferences on outcomes of self-management (SM); (3) a Delphi survey including patients and patient representatives to rate the importance of outcomes; and (4) a 2-day consensus workshop with patients, patient representatives, healthcare professionals and researchers. The initial catalogue included 82 outcomes. Ten patients and patient's representatives participated in the Delphi survey. We identified 16 themes through the thematic synthesis of the scoping review that informed 37.80% of the outcomes on initial catalogue. Five patients, five healthcare professionals, and four researchers participated in the consensus workshop. After the consensus process, 15 outcomes were selected to be part of the final COS, and five supplementary outcomes were also provided. We developed a COS for the evaluation of SMIs in obesity with a significant involvement of patients and other key stakeholders. This COS will help improving data synthesis and increasing the value of SM research data in healthcare decision making.

Self-management interventions for adults living with Chronic Obstructive Pulmonary Disease (COPD): The development of a Core Outcome Set for COMPAR-EU project.

BACKGROUND: A large body of evidence suggests that self-management interventions (SMIs) may improve outcomes in chronic obstructive pulmonary disease (COPD). However, accurate comparisons of the relative effectiveness of SMIs are challenging, partly due to heterogeneity of outcomes across trials and uncertainty about the importance of these outcomes for patients. We aimed to develop a core set of patient-relevant outcomes (COS) for SMIs trials to enhance comparability of interventions and ensure person-centred care. METHODS: We undertook an innovative approach consisting of four interlinked stages: i) Development of an initial catalogue of outcomes from previous EU-funded projects and/or published studies, ii) Scoping review of reviews on patients and caregivers' perspectives to identify outcomes of interest, iii) Two-round Delphi online survey with patients and patient representatives to rate the importance of outcomes, and iv) Face-to-face consensus workshop with patients, patient representatives, health professionals and researchers to develop the COS. RESULTS: From an initial list of 79 potential outcomes, 16 were included in the COS plus one supplementary outcome relevant to all participants. These were related to patient and caregiver knowledge/competence, self-efficacy, patient activation, self-monitoring, adherence, smoking cessation, COPD symptoms, physical activity, sleep quality, caregiver quality of life, activities of daily living, coping with the disease, participation and decision-making, emergency room visits/admissions and cost effectiveness. CONCLUSION: The development of the COPD COS for the evaluation of SMIs will increase consistency in the measurement and reporting of outcomes across trials. It will also contribute to more personalized health care and more informed health decisions in clinical practice as patients' preferences regarding COPD outcomes are more systematically included.

The fourth edition of the European Network for Health Technology Assessment Forum: highlights and outcomes.

The European Network for Health Technology Assessment (EUnetHTA) organizes an annual Forum with stakeholders to receive feedback on its activities, processes, and outputs produced. The fourth edition of the EUnetHTA Forum brought together representatives of HTA bodies, patient organizations, healthcare professionals (HCPs), academia, payers, regulators, and industry. The aim of this paper is to provide an overview of the highlights presented at the 2019 EUnetHTA Forum, reporting the main items and themes discussed in the plenary panel and breakout sessions. The leading topic was the concept of unmet medical need seen from different stakeholders' perspectives. Breakout sessions covered the joint production of assessment reports and engagement with payers, patients, and HCPs. Synergies, pragmatism, and inclusiveness across Member States and stakeholders were emphasized as leading factors to put in place a collaboration that serves the interest of patients and public health in a truly European spirit.