A review of economic issues for <scp>gene‐targeted</scp> therapies: Value, affordability, and access

Garrison, Louis P.; Lo, Andrew W.; Finkel, Richard S.; Deverka, Patricia A.

doi:10.1002/ajmg.c.32037

Cited by 9 publications

(6 citation statements)

References 37 publications

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“…This initiative evaluated the importance of the elements of value from the ISPOR Value Flower [15,17] for assessing new DMD therapies through semi-structured interviews with patient advocates, clinicians, and health economists. The study was conducted in four phases: 1) selection, 2) preparation, 3) interviews, and 4) review and clarification.…”

Section: Methodsmentioning

confidence: 99%

“…In 2018, a special task force of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) created the ISPOR Value Flower, which includes 12 elements of value (petals) that can be considered within value assessments of healthcare technologies. The ISPOR Value Flower and its recent adaptations (Figure S1) highlight common but inconsistently used elements, as well as novel elements of value that have previously been underappreciated by many payers (Table S1) [15][16][17]. Including novel elements of value in value assessment frameworks may have particular relevance for individuals with rare diseases and transformative healthcare technologies such as cell and gene therapies [16].…”

Section: Introductionmentioning

confidence: 99%

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Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies

Fischer,

Furlong,

Kennedy

et al. 2023

Preprint

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BACKGROUND: 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). OBJECTIVE: To identify how value assessment frameworks may need to be adapted to measure the value to society of DMD therapies. METHODS: Three groups of stakeholders (patient advocates, clinicians, health economists) participated in semi-structured interviews around the International Society for Pharmacoeconomics and Outcomes Researchs Value Flower, which includes elements of value that can be considered 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 use of 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: the economic and humanistic costs incurred by patients, caregivers, and families with Duchenne, such as indirect out-of-pocket costs, lost productivity, and family spillovers; the attributes that are meaningful 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. CONCLUSIONS: 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 heath 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.

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Section: Methodsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies

Fischer,

Furlong,

Kennedy

et al. 2023

Preprint

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“…The results of that meeting series were published in a Special Issue of American Journal of Medical Genetics [18] . Individual publications included discussion of readiness for genetargeted therapies [19] , ethical and social implications [20] , newborn screening [21] , and economic implications [22] .…”

Section: Policy Implications Of Gene-targeted Therapy Platformsmentioning

confidence: 99%

The division of rare diseases research innovation at the national center for advancing translational sciences, NIH: mission, history, and current research activities

Brooks,

Grady,

Groft

et al. 2024

Rare Dis Orphan Drugs J

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The mission of the NCATS Division of Rare Diseases Research Innovation (DRDRI), formerly known as the Office of Rare Diseases Research, is to advance rare diseases research to benefit patients. DRDRI is part of the National Center for Advancing Translational Sciences, one of the 27 components of the US National Institutes of Health. DRDRI facilitates and coordinates NIH-wide activities involving rare diseases research, as well as directly supporting rare diseases research activities. These activities include the development and maintenance of a centralized database on rare diseases; collaboration and coordination with organizations focused on orphan products development and rare diseases research across the globe, advising the Office of the NIH Director on matters related to NIH-sponsored research involving rare diseases; and responding to information and policy requests about rare diseases within the NIH. DRDRI also supports various rare diseases research activities, including the Rare Diseases Clinical Research Network, rare disease-related conference grants, and assessment of the costs of untreated rare diseases. In addition, several of the projects DRDRI is supporting are “many diseases at a time” translational approaches for rare diseases, which emphasize leveraging commonalities across multiple rare diseases. These include the support of “basket trials” based on shared molecular etiologies across multiple rare diseases, as well as therapeutic platforms for the treatment of monogenic diseases, such as gene therapy and genome editing. This Perspective will provide an overview and summary of these various activities, noting where relevant our collaborative partnerships within the U.S. and internationally.

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“…Both private and public health insurers will need to develop innovative coverage policies that promote the development of these drugs by ensuring payment is ultimately provided. To be a sustainable model, a value‐based system needs to be developed which incorporates both individual health benefit and broader health economic considerations (please see Garrison et al, n.d., in this special issue). Otherwise, the current drug development model is likely to both propagate and widen disparities between rare disease families with more robust economic resources and those without.…”

Section: Expanding Notions Of Equity In Rare Disease Care Delivery An...mentioning

confidence: 99%

Gene‐targeted therapies: Towards equitable development, diagnosis, and access

Gaviglio

Skinner

Lou

et al. 2023

American J of Med Genetics Pt C

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Genomic and gene‐targeted therapies hold great promise in addressing the global issue of rare diseases. To achieve this promise, however, it is critical the twin goals of equity in access to testing and diagnosis, and equity in access to therapy be considered early in the life cycle of development and implementation. Rare disease researchers and clinicians must simultaneously recognize the life‐altering potential of early diagnosis and administration of gene‐targeted therapeutics while acknowledging that not everyone who experiences a rare disease and needs these therapies will be able to afford or access them. Achieving equity in the development of and access to gene‐targeted therapies will not only require innovations in research, clinical, regulatory, and reimbursement frameworks, but will also necessitate increased attention to the ethical, legal, and social implications when establishing research paradigms and the translation of research results into novel interventions for rare genetic diseases. This article highlights and discusses the growing importance and recognition of health equity across the spectrum of rare disease research and care delivery.

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A review of economic issues for gene‐targeted therapies: Value, affordability, and access

Cited by 9 publications

References 37 publications

Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies

Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies

The division of rare diseases research innovation at the national center for advancing translational sciences, NIH: mission, history, and current research activities

Gene‐targeted therapies: Towards equitable development, diagnosis, and access

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