Challenges raised by the economic evaluation of CAR-T-cell therapies. The review by the French National Authority for Health.

Since 2013, the process of pricing of innovative drugs by the French National Health Insurance has considered both cost-effectiveness and budget impact. CAR-T cell therapies were first subject to economic evaluation from 2019 in France. We aim to describe the process and results of the economic evaluation of tisagenlecleucel and axicabtagene ciloleucel as well as the challenges these evaluations raised. Evaluations submitted by the firms were reviewed by HAS and submitted to the Committee of Economic Evaluation and Public Health (CEESP). The CEESP issued opinions related to: (1) the methodological quality of economic evidence and, (2) the cost-effectiveness and budget impact of the drugs. The CEESP invalidated the estimated incremental cost-utility ratios (ICUR) of tisagenlecleucel due to the insufficient clinical evidence and methodological quality to extrapolate the long-term progression of the disease after treatment and compare tisagenlecleucel with alternatives. The CEESP concluded that tisagenlecleucel was not proven cost-effective. The estimated ICUR of axicabtagene ciloleucel at €114,509/QALY vs. chemotherapies was associated with an acceptable level of methodological quality despite being based on a weak indirect comparison and limited data on quality of life. The CEESP considered axicabtagene ciloleucel ICUR to be "very high" and questioned the societal/community willingness-to-pay of the claimed price. The primary source of uncertainty surrounding the ICUR estimates of both drugs was the lack of hindsight on effectiveness. The economic evaluation of CAR-T cell therapies highlights the risk of inefficient resource allocation driven by limited clinical data. It calls for payment schemes accounting for this risk and effective collection of post-marketing data.

Coping with age-related hearing loss: patient-caregiver dyad effects on quality of life.

BACKGROUND AND AIMS: Patients with age -related hearing loss (ARHL) and their natural caregivers have to confront a disability that produces progressive lifestyle changes. There is an interest in studying the ability of patients and their caregivers to cope with the difficulties that affect quality of life (QoL). In a sample of patient-caregiver dyads in the specific context of ARHL, we examine whether the QoL of patients and caregivers is influenced by the coping processes they use from a specific actor-partner interdependence model (APIM). METHODS: This cross-sectional study involved dyads with patients having a diagnosis of ARHL. The self-reported data included QoL (WHOQoL-BREF) and coping strategies (BriefCope). The APIM was used to test the dyadic effects of coping strategies on QoL. RESULTS: A total of 448 dyads were included; the patients and caregivers were love partners for 59% of the dyads. Coping strategies, such as social support, avoidance, problem solving, and positive thinking, exhibited evidence of actor effects (degree to which the individual's coping strategies are associated with their own QoL). Effects on the partner (degree to which the individual's coping strategies are associated with the QoL of the other member of the dyad) were found, i.e., when the patients mobilized their coping strategy based on social support and problem-solving, their caregivers reported higher environmental QoL. CONCLUSION: This study emphasizes that the QoL for patients and their caregivers was directly related to the coping strategies they used. This finding suggests that targeted interventions should be offered to help patients and their relatives to implement more effective coping strategies.

Age-related hearing loss in individuals and their caregivers: effects of coping on the quality of life among the dyads.

OBJECTIVES: Age-related hearing loss (ARHL) impacts the daily living and quality of life (QoL) of affected individuals and the functioning of family caregivers. In the specific context of voluntary medical checkups, we examined sample dyads (ARHL individual and the caregiver) to determine whether QoL of patients and caregivers is influenced by coping strategies implemented either by themselves or their relatives. METHODS: This was a cross-sectional study with a descriptive/correlative design performed in a French preventive health center (Regional Institute for Prevention of Aging, Marseille, France) for the beneficiaries of pension funds of private sector employees. The samples included beneficiary-caregiver dyads. The beneficiaries had bilateral (mild to moderately severe) ARHL. Self-reported data were collected as follows: QoL using the World Health Organization Quality of Life questionnaire, coping strategies using the Brief Coping Orientation to Problems Experienced Scale, and anxiety and mood using visual analog scales. RESULTS: The final sample comprised 44 beneficiaries and 44 caregivers. The caregiver was the partner of the beneficiary in 73% of cases. The QoL scores of the social dimension were significantly lower for beneficiaries and caregivers compared with French age- and sex-matched controls. Among beneficiaries and caregivers, coping strategies based on problem solving were the most commonly used strategies. The use of positive thinking strategies was associated with higher QoL scores. The more one member of the dyad used an avoidance coping strategy, the more the other member used a positive thinking strategy. CONCLUSION: This study emphasizes that QoL of individuals with age-related hearing impairment and their natural caregivers is related to the coping strategies that they use. This finding suggests that targeted interventions should be offered to help individuals who experience emotional difficulties to implement more efficient coping strategies.