Value in Health最新文献

Objectives: Recent scientific breakthroughs have propelled the development of disease-modifying and potentially curative cell and gene therapies (CGTs) for rare diseases, including those diseases previously considered untreatable. The unique characteristics of CGTs, however, pose challenges to the traditional methods of therapy value determination, reimbursement, and outcome evaluation used by regulatory and assessment agencies for product approval and market access. Notably, CGTs are one-time or short-course treatments, often first-in-class (precluding direct comparisons with effective alternatives), and have health benefits that are largely realized over time.

Methods: We summarized emerging health economics and outcomes research (HEOR) solutions in 5 areas pertaining to CGTs for rare diseases using examples from literature and recently approved therapies. These solutions include the collection of real-world data to generate real-world evidence, patient centricity and novel value assessment frameworks, evolution of economic evaluation methodologies, novel reimbursement models and management of affordability, and health equity and societal benefits.

Results: Advances in HEOR methods have informed the design and collection of long-term real-world data for CGT efficacy extrapolation, use of novel (including surrogate) endpoints, integration of the patient's voice and preferences, and application of sophisticated statistical methodology and artificial intelligence/machine learning techniques.

Conclusions: Continuing innovation in HEOR is expected to contribute to improved health outcomes, reduced costs, and enhanced access, ultimately enabling more efficient delivery of CGTs to patients living with rare diseases.

Objectives: Given that most informal caregivers providing help for patients with Alzheimer's disease are retired spouses or unemployed people, there is no market value for their time. Most articles that tried to estimate the cost of informal care for Alzheimer's disease rely on the so-called "replacement" methodology, which assumes that 1 hour of informal care has the same value as 1 hour of professional care. Little attention has been dedicated to exploring the validity of this assumption. In this article, we determine the relationship between the price of informal caregiving and professional care from the first-order condition of a theoretical model that maximizes informal caregivers' satisfaction with providing care.

Methods: This article formalizes the marginal substitution rate between informal and formal care. We assume that the caregiver's utility depends on the caregiver's burden and the patient's quality of life (QoL). After explaining the parameters of the marginal utility of caregivers, we estimate each of these parameters using PLASA data.

Results: Our results show how the value of informal care increases as the care contributes to improving patients' QoL but decreases as the burden on the caregiver increases and professionals contribute to patients' QoL.

Conclusions: The central assumption of the replacement cost method of perfect substitution between informal and formal care leads to a misestimation of the value of informal care. The effects of informal care must be considered (direct effect on the burden and indirect effects on the patient's QoL).

Objectives: To estimate the excess formal social costs or direct non-healthcare costs of dementia-related neuropsychiatric symptoms (NPS).

Methods: The presence of dementia, NPS, antipsychotic and antidepressant use, somatic and psychiatric comorbidities, and formal social benefits were studied in a regionwide cohort of all 60-year-old and older individuals. A random forest-based algorithm identified NPS, and 2-part regression models and entropy balance were used.

Results: Of the 215 859 individuals, 7553 (3.50%) had dementia, 74 845 (34.7%) had some NPS, and 20 787 (9.63%) received long-term care benefits. Notably, nearly two-thirds (63.9%) of people with dementia received benefits. The probability of having social costs varied markedly with age (odds ratio [OR] 12.28 [10.17-14.82] for >90-year-olds category), and the presence of dementia (OR 7.36 [6.13-8.84]) or NPS (OR 3.23 [2.69-3.88]). NPS (relative change [RC] 1.39 [1.31-1.49]) and dementia (RC 1.32 [1.24-1.41]) were associated with higher average benefit costs. Low socioeconomic status was significantly associated with both a higher probability of receiving benefits (OR 1.52 [1.38-1.68]) and higher costs of their provision (RC 1.18 [1.15-1.21]).

Conclusions: The burden of caring for NPS is greater than that indicated by the literature as these symptoms multiply the social costs of dementia by more than 3, owing to the greater use of residential care and formal coverage reaching more patients than that indicated by the literature. The greater presence of dementia and NPS in the population of lower socioeconomic status indicates an inequality in health attenuated by greater use of social benefits.