Projections of Public Spending on Pharmaceuticals: A Review of Methods.

Background: Forecasting future public pharmaceutical expenditure is a challenge for healthcare payers, particularly owing to the unpredictability of new market introductions and their economic impact. No best-practice forecasting methods have been established so far. The literature distinguishes between the top-down approach, based on historical trends, and the bottom-up approach, using a combination of historical and horizon scanning data.

How can patient preferences be used and communicated in the regulatory evaluation of medicinal products? Findings and recommendations from IMI PREFER and call to action.

Patients have unique insights and are (in-)directly affected by each decision taken throughout the life cycle of medicinal products. Patient preference studies (PPS) assess what matters most to patients, how much, and what trade-offs patients are willing to make. IMI PREFER was a six-year European public-private partnership under the Innovative Medicines Initiative that developed recommendations on how to assess and use PPS in medical product decision-making, including in the regulatory evaluation of medicinal products.

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.

[Difficulties encountered by long COVID patients in the Belgian health system].

Introduction: After contracting COVID-19, many people have continued to experience various symptoms for several weeks and months, even after a mild acute phase. These people with ‘long COVID’ faced difficulties when confronted with the healthcare system.

Purpose Of Research: In order to better understand their experience, we supplemented the information obtained in an online survey with a mixed qualitative approach based on 33 individual interviews and discussions with 101 participants in a forum in March 2021.

An EQ-5D-5L Value Set for Belgium.

Objective: This study aimed to establish a Belgian EQ-5D-5L value set based on the preferences of the adult Belgian general population.

Methods: The most recent EuroQol Valuation Technology (EQ-VT 2.1) protocol for EQ-5D-5L valuation studies was followed.

Belgian population norms for the EQ-5D-5L, 2018.

Purpose: Health-related quality of life outcomes are increasingly used to monitor population health and health inequalities and to assess the (cost-) effectiveness of health interventions. The EQ-5D-5L has been included in the Belgian Health Interview Survey, providing a new source of population-based self-perceived health status information. This study aims to estimate Belgian population norms for the EQ-5D-5L by sex, age, and region and to analyze its association with educational attainment.

Developing an agency's position with respect to patient involvement in health technology assessment: the importance of the organizational culture.

Objectives: The objective of this study was to map the PI culture at KCE in the context of the development of organization-wide supported position statements about PI.

Methods: A nominal group technique was used to measure the PI culture at KCE. Arguments for and against PI and conditions for PI in different phases of the HTA process were collected.

An overview of critical decision-points in the medical product lifecycle: Where to include patient preference information in the decision-making process?

Background: Patient preference (PP) information is not effectively integrated in decision-making throughout the medical product lifecycle (MPLC), despite having the potential to improve patients' healthcare options. A first step requires an understanding of existing processes and decision-points to know how to incorporate PP information in order to improve patient-centric decision-making.

Objectives: The aims were to: 1) identify the decision-making processes and decision-points throughout the MPLC for industry, regulatory authorities, and reimbursement/HTA, and 2) determine which decision-points can potentially include PP information.

Design, Conduct, and Use of Patient Preference Studies in the Medical Product Life Cycle: A Multi-Method Study.

To investigate stakeholder perspectives on how patient preference studies (PPS) should be designed and conducted to allow for inclusion of patient preferences in decision-making along the medical product life cycle (MPLC), and how patient preferences can be used in such decision-making. Two literature reviews and semi-structured interviews (n = 143) with healthcare stakeholders in Europe and the US were conducted; results of these informed the design of focus group guides. Eight focus groups were conducted with European patients, industry representatives and regulators, and with US regulators and European/Canadian health technology assessment (HTA) representatives.

Multicriteria Decision Analysis to Support Health Technology Assessment Agencies: Benefits, Limitations, and the Way Forward.

Objective: Recent years have witnessed an increased interest in the use of multicriteria decision analysis (MCDA) to support health technology assessment (HTA) agencies for setting healthcare priorities. However, its implementation to date has been criticized for being "entirely mechanistic," ignoring opportunity costs, and not following best practice guidelines. This article provides guidance on the use of MCDA in this context.

Factors and Situations Affecting the Value of Patient Preference Studies: Semi-Structured Interviews in Europe and the US.

Patient preference information (PPI) is gaining recognition among the pharmaceutical industry, regulatory authorities, and health technology assessment (HTA) bodies/payers for use in assessments and decision-making along the medical product lifecycle (MPLC). This study aimed to identify factors and situations that influence the value of patient preference studies (PPS) in decision-making along the MPLC according to different stakeholders. Semi-structured interviews (n = 143) were conducted with six different stakeholder groups (physicians, academics, industry representatives, regulators, HTA/payer representatives, and a combined group of patients, caregivers, and patient representatives) from seven European countries (the United Kingdom, Sweden, Italy, Romania, Germany, France, and the Netherlands) and the United States.

Opportunities and challenges for the inclusion of patient preferences in the medical product life cycle: a systematic review.

Background: The inclusion of patient preferences (PP) in the medical product life cycle is a topic of growing interest to stakeholders such as academics, Health Technology Assessment (HTA) bodies, reimbursement agencies, industry, patients, physicians and regulators. This review aimed to understand the potential roles, reasons for using PP and the expectations, concerns and requirements associated with PP in industry processes, regulatory benefit-risk assessment (BRA) and marketing authorization (MA), and HTA and reimbursement decision-making.

Methods: A systematic review of peer-reviewed and grey literature published between January 2011 and March 2018 was performed.

Patient Preferences in the Medical Product Life Cycle: What do Stakeholders Think? Semi-Structured Qualitative Interviews in Europe and the USA.

Background: Patient preferences (PP), which are investigated in PP studies using qualitative or quantitative methods, are a growing area of interest to the following stakeholders involved in the medical product lifecycle: academics, health technology assessment bodies, payers, industry, patients, physicians, and regulators. However, the use of PP in decisions along the medical product lifecycle remains limited. As the adoption of PP heavily relies on these stakeholders, knowledge of their perceptions of PP is critical.

Factors and situations influencing the value of patient preference studies along the medical product lifecycle: a literature review.

Industry, regulators, health technology assessment (HTA) bodies, and payers are exploring the use of patient preferences in their decision-making processes. In general, experience in conducting and assessing patient preference studies is limited. Here, we performed a systematic literature search and review to identify factors and situations influencing the value of patient preference studies, as well as applications throughout the medical product lifecyle.

What Does the Public Want? Structural Consideration of Citizen Preferences in Health Care Coverage Decisions.

Multi-criteria decision analysis can improve the legitimacy of health care reimbursement decisions by taking societal preferences into account when weighting decision criteria. This study measures the relative importance of health care coverage criteria according to the Belgian general public and policy makers. Criteria are structured into three domains: therapeutic need, societal need, and new treatments' added value.

A multi-criteria decision approach for ranking unmet needs in healthcare.

Early temporary reimbursement (ETR) schemes for new interventions targeting high unmet needs are increasingly applied in pharmaceutical policy. Crucial for these schemes is the assessment of unmet healthcare needs of patients and society. This study develops and tests a multi-criteria decision approach (MCDA) for assessing therapeutic and societal needs.

EXPLORING VALUES OF HEALTH TECHNOLOGY ASSESSMENT AGENCIES USING REFLECTIVE MULTICRITERIA AND RARE DISEASE CASE.

Objectives: Tackling ethical dilemmas faced by reimbursement decision makers requires deeper understanding of values on which health technology assessment (HTA) agencies are founded and how trade-offs are made. This was explored in this study including the case of rare disease.

Methods: Representatives from eight HTA explored values on which institutions are founded using a narrative approach and reflective multicriteria (developed from EVIDEM, criteria derived from ethical imperatives of health care).

Pharmaceutical regulation in 15 European countries review.

In the context of pharmaceutical care, policy-makers repeatedly face the challenge of balancing patient access to effective medicines with affordability and rising costs. With the aim of guiding the health policy discourse towards questions that are important to actual and potential patients, this study investigates a broad range of regulatory measures, spanning marketing authorization to generic substitution and resulting price levels in a sample of 16 European health systems (Austria, Belgium, Denmark, England, Finland, France, Germany, Greece, Ireland, Italy, the Netherlands, Poland, Portugal, Scotland, Spain and Sweden). All countries employ a mix of regulatory mechanisms to contain pharmaceutical expenditure and ensure quality and efficiency in pharmaceutical care, albeit with varying configurations and rigour.

The determinants of defensive medicine practices in Belgium.

In 2010 the Belgian government introduced a low cost administrative procedure for compensating medical injuries to overcome the major shortcomings of the existing tort system. This paper examines, for the first time, to what extent this reform had an impact on physician specialists' defensive practices and what are the relevant determinants affecting physicians' clinical decision making. Based on a survey of 508 physicians, we find evidence of a relatively modest increase in defensive practices among physicians in various specialties.

Assessing cancer patients' quality of life and supportive care needs: Translation-revalidation of the CARES in Flemish and exhaustive evaluation of concurrent validity.

Background: The prevalence of cancer increases every year, leading to a growing population of patients and survivors in need for care. To achieve good quality care, a patient-centered approach is essential. Correct and timely detection of needs throughout the different stages of the care trajectory is crucial and can be supported by the use of screening and assessment in a stepped-care approach.

Barriers and facilitators related to the implementation of surgical safety checklists: a systematic review of the qualitative evidence.

Objective: The objective of this review is to obtain a better understanding of the user-related barriers against, and facilitators for, the implementation of surgical safety checklists.

Methods: We searched MEDLINE for articles describing stakeholders' perspectives regarding, and experiences with, the implementation of surgical safety checklists. The quality of the papers was assessed by means of the Qualitative Assessment and Review Instrument.