HTA challenges for appraising rare disease interventions viewed through the lens of an institutional multidimensional value framework.

Introduction: Evaluating rare disease interventions poses challenges for HTA agencies, including uncertainties and ethical issues and tensions. INESSS has recently adopted a Statement of Principles and Ethical Foundations which proposes a multidimensional approach to value appraisal as well as five principles to frame the evaluation process.

Areas Covered: Our aim was to identify and analyze HTA challenges for appraising interventions for rare diseases, using the Statement's approach to value appraisal as an analytical framework, and outline how the Statement's principles can help address these challenges.

Can reflective multicriteria be the new paradigm for healthcare decision-making? The EVIDEM journey.

Background: Multiple technologies, procedures and programs call for fairly-based decisions for prioritization of healthcare interventions. There is a diversity of perspectives of what constitutes a legitimate decision, which depends on both the process and the reasoning applied. Current approaches focus on technical aspects while methods to support alignment of decisions with the compassionate impetus of healthcare systems is lacking.

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).

DRUG EVALUATION AND DECISION MAKING IN CATALONIA: DEVELOPMENT AND VALIDATION OF A METHODOLOGICAL FRAMEWORK BASED ON MULTI-CRITERIA DECISION ANALYSIS (MCDA) FOR ORPHAN DRUGS.

Objectives: The aim of this study was to adapt and assess the value of a Multi-Criteria Decision Analysis (MCDA) framework (EVIDEM) for the evaluation of Orphan drugs in Catalonia (Catalan Health Service).

Methods: The standard evaluation and decision-making procedures of CatSalut were compared with the EVIDEM methodology and contents. The EVIDEM framework was adapted to the Catalan context, focusing on the evaluation of Orphan drugs (PASFTAC program), during a Workshop with sixteen PASFTAC members.

Pragmatic Multicriteria Decision Analysis (MCDA) Combined With Advanced Pharmacoepidemiology for Benefit-Risk Assessments of Medicines Adapted to the Real-Life Constraints of Regulators: Development and Case Study.

Background: Multicriteria decision analysis (MCDA) represents a promising method for benefit-risk assessment. Our goal was to develop features of pragmatic MCDA (EVIDEM [Evidence and Value: Impact on DEcisionMaking]) addressing real-life regulatory decision-making needs, incorporate advanced pharmacoepidemiology, and test the resulting benefit-risk framework using a case study.

Methods: The Intervention Outcomes domain of EVIDEM was transformed into a generic benefit-risk framework including clinical efficacy, patient-reported outcomes, and adverse event (AE) criteria.

Which criteria are considered in healthcare decisions? Insights from an international survey of policy and clinical decision makers.

Objectives: The aim of this study was to gather qualitative and quantitative data on criteria considered by healthcare decision makers.

Methods: Using snowball sampling and an online questionnaire with forty-three criteria organized into ten clusters, decision makers were invited by an international task force to report which criteria they consider when making decisions on healthcare interventions in their context. Respondents reported whether each criterion is "currently considered," "should be considered," and its relative weight (scale 0-5).

From efficacy to equity: Literature review of decision criteria for resource allocation and healthcare decisionmaking.

Objectives: Resource allocation is a challenging issue faced by health policy decisionmakers requiring careful consideration of many factors. Objectives of this study were to identify decision criteria and their frequency reported in the literature on healthcare decisionmaking.

Method: An extensive literature search was performed in Medline and EMBASE to identify articles reporting healthcare decision criteria.

Burden of community-acquired and nosocomial rotavirus gastroenteritis in the pediatric population of Western Europe: a scoping review.

Background: Rotavirus affects 95% of children worldwide by age 5 years and is the leading cause of severe dehydrating diarrhea. The objective of this review was to estimate the burden of rotavirus gastroenteritis (RVGE) in the Western European pediatric population.

Methods: A comprehensive literature search (1999-2010) was conducted in PubMed and other sources (CDC; WHO, others).

Field testing of a multicriteria decision analysis (MCDA) framework for coverage of a screening test for cervical cancer in South Africa.

Background: Systematic and transparent approaches to priority setting are needed, particularly in low-resource settings, to produce decisions that are sound and acceptable to stakeholders. The EVIDEM framework brings together Health Technology Assessment (HTA) and multi-criteria decision analysis (MCDA) by proposing a comprehensive set of decision criteria together with standardized processes to support decisionmaking. The objective of the study was to field test the framework for decisionmaking on a screening test by a private health plan in South Africa.

Bridging health technology assessment (HTA) with multicriteria decision analyses (MCDA): field testing of the EVIDEM framework for coverage decisions by a public payer in Canada.

Background: Consistent healthcare decision making requires systematic consideration of decision criteria and evidence available to inform them. This can be tackled by combining multicriteria decision analysis (MCDA) and Health Technology Assessment (HTA). The objective of this study was to field-test a decision support framework (EVIDEM), explore its utility to a drug advisory committee and test its reliability over time.

Bridging health technology assessment (HTA) and efficient health care decision making with multicriteria decision analysis (MCDA): applying the EVIDEM framework to medicines appraisal.

Background: Health care decision making is complex and requires efficient and explicit processes to ensure transparency and consistency of factors considered.

Objectives: To pilot an adaptable decision-making framework incorporating multicriteria decision analysis (MCDA) in health technology assessment (HTA) with a pan-Canadian group of policy and clinical decision makers and researchers appraising 10 medicines covering 6 therapeutic areas.

Methods: An appraisal group was convened and participants were asked to express their individual perspectives, independently of the medicines, by assigning weights to each criterion of the MCDA core model: disease severity, size of population, current practice and unmet needs, intervention outcomes (efficacy, safety, patient reported), type of health benefit, economics, and quality of evidence.

Burden of rotavirus gastroenteritis in the pediatric population in Central and Eastern Europe: serotype distribution and burden of illness.

Background: Rotaviral gastroenteritis (RVGE) is the leading cause of severe diarrhea in children under five years of age worldwide. This comprehensive review aims to estimate the burden of RVGE among children in Central and Eastern Europe.

Methods: An extensive search of the biomedical literature (1999-2009) was conducted in major databases.

Burden of rotavirus gastroenteritis in the Middle Eastern and North African pediatric population.

Background: Rotavirus gastroenteritis (RVGE) is the most common cause of severe childhood diarrhea worldwide. Objectives were to estimate the burden of RVGE among children less than five years old in the Middle East (Bahrain, Iran, Iraq, Israel, Jordan, Kuwait, Oman, Qatar, Saudi Arabia, Syria, UAE, Yemen), North Africa (Algeria, Egypt, Libya, Morocco, Tunisia) and Turkey.

Methods: A comprehensive literature search was conducted in major databases on the epidemiology and burden of rotavirus among children less than five years old between 1999 and 2009.

Combining multicriteria decision analysis, ethics and health technology assessment: applying the EVIDEM decision-making framework to growth hormone for Turner syndrome patients.

Objectives: To test and further develop a healthcare policy and clinical decision support framework using growth hormone (GH) for Turner syndrome (TS) as a complex case study.

Methods: The EVIDEM framework was further developed to complement the multicriteria decision analysis (MCDA) Value Matrix, that includes 15 quantifiable components of decision clustered in four domains (quality of evidence, disease, intervention and economics), with a qualitative tool including six ethical and health system-related components of decision. An extensive review of the literature was performed to develop a health technology assessment report (HTA) tailored to each component of decision, and content was validated by experts.

Evidence and Value: Impact on DEcisionMaking--the EVIDEM framework and potential applications.

Background: Healthcare decisionmaking is a complex process relying on disparate types of evidence and value judgments. Our objectives for this study were to develop a practical framework to facilitate decisionmaking in terms of supporting the deliberative process, providing access to evidence, and enhancing the communication of decisions.

Methods: Extensive analyses of the literature and of documented decisionmaking processes around the globe were performed to explore what steps are currently used to make decisions with respect to context (from evidence generation to communication of decision) and thought process (conceptual components of decisions).

Medicare coverage and reimbursement of outpatient prescription drugs in the US: history, recent changes and outlook for the future.

US Medicare provides health insurance to 41.5 million disabled and elderly Americans. Outpatient coverage, including a limited pharmacy benefit, is provided by Medicare Part B.

The societal value of universal childhood vaccination.

Availability of new vaccines preventing infectious diseases in healthy children populations is increasing worldwide. In Canada, despite the current recommendation of the National Advisory Committee on Immunization to include recent vaccines in routine schedule, only a few provinces have incorporated some of the newer vaccines in routine vaccination programs. A review was undertaken of economic evaluations of childhood vaccination strategies performed from the societal point of view in industrialized countries, to gain perspective on their global benefits.

Understanding the underlying drivers of inpatient cost growth: a literature review.

Background: After the declining growth in inpatient hospital spending that occurred from 1994 through 1998, the recent trend in increased spending has been of concern to many. Understanding the underlying reasons for this new growth will aid decision makers in finding best means to manage inpatient costs.

Objective: To identify potential contributors to recent growth in inpatient spending.