Lawrenceville, NJ, USA—October 28, 2026—Value in Health, the official journal of ISPOR—The Professional Society for Health Economics and Outcomes Research, announced today the publication of a themed section examining new approaches to measuring meaningful change for patients with rare diseases.
The section includes 5 studies examining conditions including Angelman syndrome, inherited optic neuropathies, idiopathic short stature, idiopathic pulmonary fibrosis, systemic sclerosis, myositis, and nephrotic syndrome.
The articles were published in the October 2026 issue of Value in Health, which also features the editorial, “The Application of Food and Drug Administration Patient-Focused Drug Development Guidance in Rare Patient Populations: Innovation and Flexibility,” by the Guest Editor Stacie Hudgens, MA.
Patient-focused drug development is a US Food and Drug Administration priority, with agency guidance encouraging flexible approaches to developing and interpreting clinical outcome assessments for rare and orphan diseases.
“As we develop new approaches to bring the patient voice into clinical development programs, clinical practice, and real-world disease management, it is imperative that we continue to develop innovative qualitative, quantitative, and mixed methods for interpreting patient-focused outcomes,” wrote Hudgens, chief scientific and strategy officer at Clinical Outcomes Solutions in Tucson, AZ.
Interpreting and communicating meaningful change in clinical studies can be particularly challenging in rare diseases because of small patient populations, differences in symptoms and disease progression, and the need to consider perspectives from patients, caregivers, and clinicians.
The themed section includes 5 research papers exploring 3 key areas:
Topic 1: Defining Meaningful Change With Patients and Caregivers
“Caregiver Meaningful Score Differences and Meaningful Score Regions for the Observer-Reported Communication Ability Measure for Individuals With Angelman Syndrome,” by A Rader, L Lin, A Sadhwani, and colleagues. The study presents a novel method for determining whether changes in communication are meaningful to caregivers.
“Developing Meaningful Score Differences for the Bayley-4 and Vineland-3 in Angelman Syndrome Using a Delphi Panel,” by S Powers, KN Anderson, W-H Tan, and colleagues. Caregivers helped researchers identify how much change on commonly used developmental measures would make a meaningful difference in daily functioning or family quality of life, with meaningful change varying according to patients’ baseline functioning.
“How Much Change Is Meaningful in Global Impression Anchors? Evidence From 5 Rare Diseases,” by C Hurt, M Kuharic, S Hashemi, and colleagues. Through interviews with patients and caregivers, researchers examined how much change they considered meaningful.
Topic 2: Accounting for Differences in Treatment Response
“Longitudinal Modeling and Simulation of Growth Hormone Efficacy in Children With Idiopathic Short Stature,” by H Cho, H Jeon, C Kang, and colleagues. Researchers developed a model and web-based tool to predict how individual children may respond to growth hormone treatment.
Topic 3: Understanding Outcomes That Matter to Patients
“‘Adrift From the World’: Exploring the Lived Experiences of Individuals Affected by an Inherited Optic Neuropathy in the United Kingdom—A Qualitative Study,” by BS Chen, C Seikus, J Ferguson, and colleagues. Interviews with patients explored how inherited optic neuropathies affect daily life and what matters most to patients’ quality of life.
Taken together, the studies offer researchers different ways to address the challenges of measuring meaningful change in small, diverse patient populations.
“Each approach is thoughtfully developed and enhances the scientific framework for evaluating what matters most to patients with rare and orphan diseases,” Hudgens wrote.
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ABOUT ISPOR
ISPOR—The Professional Society for Health Economics and Outcomes Research (HEOR), is an international, multistakeholder, nonprofit dedicated to advancing HEOR excellence to improve decision making for health globally. The Society is the leading source for scientific conferences, peer-reviewed and MEDLINE®-indexed publications, good practices guidance, education, collaboration, and tools/resources in the field.
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ABOUT VALUE IN HEALTH
Value in Health (ISSN 1098-3015) is an international, indexed journal that publishes original research and health policy articles that advance the field of health economics and outcomes research to help healthcare leaders make evidence-based decisions. The journal’s current impact factor score is 6.0 and its 5-year impact factor score is 5.7. Value in Health is ranked 5th of 124 journals in Health Policy and Services, 12th of 185 journals in Health Care Sciences & Services, and 37th of 617 journals in Economics. Value in Health is a monthly publication that circulates to more than 55,000 readers around the world.
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