Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies
Bibliographic record
Abstract
Ryan Fischer,1 Pat Furlong,1 Annie Kennedy,2 Kelly Maynard,3,4 Marissa Penrod,4,5 Debra Miller,6 Chamindra G Laverty,7 Linda P Lowes,8,9 Nancy L Kuntz,10,11 Perry B Shieh,12 Jane Kondejewski,13 Peter J Neumann,14 Jason Shafrin,15 Richard J Willke16 1Parent Project Muscular Dystrophy, Washington, DC, USA; 2EveryLife Foundation for Rare Diseases, Washington, DC, USA; 3Little Hercules Foundation, Dublin, OH, USA; 4Duchenne Family Assistance Program, Detroit, MI, USA; 5Team Joseph, West Bloomfield, MI, USA; 6Cureduchenne, Newport Beach, CA, USA; 7Department of Neuroscience, University of California San Diego, San Diego, CA, USA; 8Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children’s Hospital, Columbus, OH, USA; 9Department of Pediatrics, the Ohio State University, Columbus, OH, USA; 10Division of Neurology, Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL, USA; 11Division of Neurology, Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, USA; 12Department of Neurology and Pediatrics, University of California Los Angeles, Los Angeles, CA, USA; 13SNELL Medical Communication, Inc., Montreal, QC, Canada; 14Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA; 15FTI Consulting, Los Angeles, CA, USA; 16Scintegral Health Economics, Chattanooga, TN, USACorrespondence: Richard J Willke, Scintegral Health Economics, Chattanooga, TN, USA, Tel +1-908-672-4156, Email willkerj@aol.comPurpose: Traditional value assessment frameworks are challenged in comprehensively assessing the societal value new therapies bring to individuals with rare, progressive, genetic, fatal, neuromuscular diseases such as Duchenne muscular dystrophy (DMD). The objective of this study was to identify how value assessment frameworks may need to be adapted to measure the value to society of DMD therapies.Patients and Methods: Three stakeholder groups (6 patient advocates, 4 clinicians, 3 health economists; N = 13) participated in semi-structured interviews around the International Society for Pharmacoeconomics and Outcomes Research’s Value Flower, which includes elements to consider within value assessments of healthcare technologies.Results: All stakeholders agreed that traditional value assessment frameworks based on the quality-adjusted life year (QALY) are narrow and will undervalue new DMD therapies. All stakeholders expressed some level of concern that using the QALY as a key metric of value discriminates against patients with severe progressive diseases and disabilities. Some stakeholders saw value in using the QALY for cross-disease comparisons in resource-constrained environments if the methodology was appropriate. All stakeholders recommended considering additional elements of value in decision-making around new DMD therapies. These elements reflect: economic and humanistic costs incurred by patients, caregivers, and families with Duchenne, such as indirect out-of-pocket costs, lost productivity, and family spillovers; meaningful attributes for individuals with disabilities and high unmet need, such as severity of disease, value of hope, and real option value; and factors that contribute to improvements in population health, such as insurance value, equity, and scientific spillovers.Conclusion: These findings highlight the need to expand traditional value assessment frameworks and take a holistic approach that incorporates the perspectives of individuals with Duchenne, caregivers, clinicians, and health economists when assessing the societal value of new DMD therapies. Broadening value assessment will prevent restricted or delayed access to therapies for individuals with Duchenne.Keywords: value assessment framework, qualitative research, rare diseases, ISPOR value flower
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.141 | 0.143 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.001 | 0.002 |
| Bibliometrics | 0.003 | 0.003 |
| Science and technology studies | 0.018 | 0.020 |
| Scholarly communication | 0.035 | 0.015 |
| Open science | 0.005 | 0.026 |
| Research integrity | 0.018 | 0.020 |
| Insufficient payload (model declined to judge) | 0.009 | 0.001 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".