Separate registration required.
This short course will focus on the recent ISPOR Special Task Force Report, “A Health Economics Approach to US Value Frameworks.” It will begin with an overview of recent US value assessment frameworks, with emphasis on the importance of perspective and decision context in the construction and use of value frameworks. It will then review how a health economics approach from a societal or health plan perspective leads to use of cost-effectiveness analysis (CEA) to help guide efficient resource allocation.
There will be in-depth discussion of how measuring some aspects of the value of health benefits could augment the standard cost-per-quality-adjusted-life-year metric for CEA. Elements such as value of insurance, value of “hope,” real option value, severity of illness, and several others, have the potential to better capture how patients and/or society value the benefits of some treatments; each one is based on some research findings and some case examples will be shown.
The course will then review how budget considerations, cost-effectiveness thresholds, and opportunity costs enter CEA-based decision-making. Next faculty will review broader approaches to cost-benefit aggregation and value-based decision-making, including extended CEA, augmented CEA (introduced by this Report), and multi-criteria decision analysis (MCDA), with an overview of issues and new approaches to MCDA. It then discusses the strengths and weaknesses of recent US value assessment frameworks from this health economic perspective and closes with a review of the high-level recommendations of this Special Task Force.
The forum aims to provide insight into the diverse roles patients and caregivers have and provides examples of these roles in patient-centered research. Case studies will be drawn from health preference and patient-reported outcome (PRO) research to enrich the discussion.
Siu Hing Lo will provide a broad overview of the roles patients and caregivers have as engaged research partners versus research participants in patient-centered research. She will then discuss how the form of patient/caregiver engagement could differ depending on the study objectives, existing evidence, practical considerations, and the intended use of the study findings. She will also highlight similarities and differences in patient/caregiver roles between health preference and PRO studies.
Holly Peay will discuss case studies in which patients and caregivers participated in a community engaged process for the development, interpretation, and dissemination of qualitative and quantitative HPR research. She will summarize barriers and facilitators to engagement and approaches to match engagement to stated preference research objectives.
Ryan Fischer will discuss his experience collaborating with academic and industry partners for advocacy-led patient preference and experience studies, and how advocacy organizations use the results generated from such studies in their strategic planning, advocacy, education, and research funding.
Fraser Bocell will discuss the roles of patients and the use of patient engagement in selecting, modifying, and adapting meaningful PRO instruments, from a regulator’s perspective.
Speakers will use polling questions to engage the audience in discussion.
This forum is a collaboration presented by the Health Preference Research and the Patient-Centered Special Interest Groups.
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Fraser Bocell, PhD
U.S. Food and Drug Administration, Silver Spring, MD, USA
Fraser D. Bocell, MEd, PhD is the Patient-Science Team Lead and a Psychometrician with the Division for Patient Centered Development in the Center for Devices and Radiological Health at the US FDA. At CDRH he provides expertise and training, leads Patient-Centered research, and helps develop policy on the evaluation and use of COAs in regulatory decision-making. His work helps bring patient voices to regulatory decision making.
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Ryan Fischer, BA
Foundation for Angelman Syndrome Therapeutics, Marble Falls, TX, USA
Ryan serves as Chief Operating Officer for the Foundation for Angelman Syndrome Therapeutics or FAST. Ryan works in collaboration with FAST’s President to establish, direct, and achieve the organization’s short and long-term strategic goals and objectives across all operational areas, including general administration, programs, grants, and external relations. He maintains and builds effective relationships with key stakeholders, including Congress, regulators, pharmaceutical companies, other foundation leaders, and the broader rare disease advocacy community. Prior to joining FAST, Ryan served as Chief Advocacy Officer for Parent Project Muscular Dystrophy (PPMD). Ryan held various roles within PPMD over 18 years helping to grow the organization into one of the leading rare diseases advocacy nonprofits globally. In 2012, Ryan was a member of the core team (in partnership with Johns Hopkins School of Public Health) that developed and produced a novel patient preference study on caregiver benefit-risk preferences and priorities for emerging treatments in Duchenne. Since then, Ryan has co-led several additional preference studies and advocates for advancing the science of patient input within Patient-Focused Drug Development.
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Holly Peay, PhD
RTI International, Research Triangle Park, NC, USA
Holly Peay is a Senior Research Scientist at RTI International. She conducts research on the ethical application of new technologies in research, clinical, and public health settings. Her research includes patient and stakeholder preference studies, decision support interventions, and engagement science.
OBJECTIVES: The study's main goal was to assess both the quality of life and economic outcomes associated with rare diseases within the scope of the Journey of Valued Care for Patients with Rare Diseases (JAV-RARAS) study. JAV-RARAS aims to comprehend and measure the value-based care journey of patients in Brazil by establishing disease records, defining value parameters, and evaluating the cost-effectiveness relationship to optimize resource allocation across different regions of the country.
METHODS: Data were analyzed from patients diagnosed with the following diseases: Osteogenesis Imperfecta (OI), Prader-Willi Syndrome (PWS), Acromegaly (ACRO), Hereditary Angioedema, Mucopolysaccharidosis type II (MPS2), Classical Homocystinuria (HC), Phenylketonuria (PKU), Spinal Muscular Atrophy (SMA), Cystic Fibrosis (CF), Familial Amyloidotic Polyneuropathy (FAP), Duchenne Muscular Dystrophy (DM), Gaucher Disease, and Amyotrophic Lateral Sclerosis (ALS).
Quality of life was assessed using the EQ-5D questionnaire, which evaluates health across five dimensions: mobility, personal care, daily activities, pain/discomfort, and anxiety/depression. Costs were determined using Time-Driven Activity-Based Costing (TDABC), a method that, based on Clinical Protocols and Therapeutic Guidelines (PCDT), maps care processes and actual costs.
RESULTS: Initial findings suggest that among the diseases examined, Prader-Willi Syndrome (PWS) has the highest Quality-Adjusted Life Years (QALY) score, while Familial Amyloidotic Polyneuropathy (FAP) has the highest average treatment cost based on the PCDT. When assessed through the Time-Driven Activity-Based Costing (TDABC) method, Spinal Muscular Atrophy (SMA) exhibits the highest average treatment cost. In contrast, Gaucher disease demonstrates the lowest average cost of treatment and the lowest cost per incremental Quality-Adjusted Life Year (QALY). This highlights a more efficient protocol compared to the other diseases under evaluation, positioning Gaucher disease as a reference point for cost-effectiveness.
CONCLUSIONS: The evaluation of treatment protocols conducted by JAV-RARAS allows for ranking the technical allocative efficiency of PCDTs as a policy intervention, through a cost-utility analysis based on the relationship between cost and QALY outcomes.
