The RELIEF study assessed rates of hospitalization for acute diabetes complications in France before and after initiation of the FreeStyle Libre system.
A total of 74,011 patients with type 1 ...diabetes or type 2 diabetes who initiated the FreeStyle Libre system were identified from the French national claims database with use of ICD-10 codes, from hospitalizations with diabetes as a contributing diagnosis, or the prescription of insulin. Patients were subclassified based on self-monitoring of blood glucose (SMBG) strip acquisition prior to starting FreeStyle Libre. Hospitalizations for diabetic ketoacidosis (DKA), severe hypoglycemia, diabetes-related coma, and hyperglycemia were recorded for the 12 months before and after initiation.
Hospitalizations for acute diabetes complications fell in type 1 diabetes (-49.0%) and in type 2 diabetes (-39.4%) following FreeStyle Libre initiation. DKA fell in type 1 diabetes (-56.2%) and in type 2 diabetes (-52.1%), as did diabetes-related comas in type 1 diabetes (-39.6%) and in type 2 diabetes (-31.9%). Hospitalizations for hypoglycemia and hyperglycemia decreased in type 2 diabetes (-10.8% and -26.5%, respectively). Before initiation, hospitalizations were most marked for people noncompliant with SMBG and for those with highest acquisition of SMBG, which fell by 54.0% and 51.2%, respectively, following FreeStyle Libre initiation. Persistence with FreeStyle Libre at 12 months was at 98.1%.
This large retrospective study on hospitalizations for acute diabetes complications shows that a significantly lower incidence of admissions for DKA and for diabetes-related coma is associated with use of flash glucose monitoring. This study has significant implications for patient-centered diabetes care and potentially for long-term health economic outcomes.
GPP is a rare, chronic, neutrophilic skin disease, with limited real-world data characterizing patients with flares and the impact of flares on disease progression and morbidity.
Describe the ...clinical characteristics of patients with GPP, comorbidities, disease epidemiology and frequency and severity of flares, and compare patients with GPP with a matched severe psoriasis population.
In this population-based real-world cohort study an algorithm was developed to identify patients with GPP flares. Three cohorts were identified using the Système National des Données de Santé (SNDS) database covering almost the entire French population; a prevalent cohort (2010-2018), an incident cohort (2012-2015). A severe psoriasis cohort was compared with the GPP incident cohort using propensity score matching.
The prevalent and incident cohorts comprised 4195 and 1842 patients, respectively. In both cohorts, mean age was 58 years; 53% were male. Comorbidities were significantly more common in the incident cohort versus matched psoriasis cohort, respectively, including hypertension (44% vs. 26%), ischaemic heart disease (26% vs. 18%) and hyperlipidaemia (25% vs. 15%). In the incident cohort, the flare rate was 0.1 flares/person-year and 0.4 flares/person-year among the 569 out of 1842 patients hospitalized with flares. These patients had a mean (±SD) stay of 11.6 ± 10.4 days; 25% were admitted to the intensive care unit. In 2017, the cumulative incidence and cumulative GPP age-sex standardized prevalence were 7.1 and 45.2 per million, respectively.
Patients with GPP had a distinct comorbidity profile compared to patients with severe psoriasis, and GPP flares were associated with long hospitalizations.
Abstract There is a significant and growing interest among both payers and producers of medical products for agreements that involve a “pay-for-performance” or “risk-sharing” element. These payment ...schemes—called “performance-based risk-sharing arrangements” (PBRSAs)—involve a plan by which the performance of the product is tracked in a defined patient population over a specified period of time and the amount or level of reimbursement is based on the health and cost outcomes achieved. There has always been considerable uncertainty at product launch about the ultimate real-world clinical and economic performance of new products, but this appears to have increased in recent years. PBRSAs represent one mechanism for reducing this uncertainty through greater investment in evidence collection while a technology is used within a health care system. The objective of this Task Force report was to set out the standards that should be applied to “good practices”—both research and operational—in the use of a PBRSA, encompassing questions around the desirability, design, implementation, and evaluation of such an arrangement. This report provides practical recommendations for the development and application of state-of-the-art methods to be used when considering, using, or reviewing PBRSAs. Key findings and recommendations include the following. Additional evidence collection is costly, and there are numerous barriers to establishing viable and cost-effective PBRSAs: negotiation, monitoring, and evaluation costs can be substantial. For good research practice in PBRSAs, it is critical to match the appropriate study and research design to the uncertainties being addressed. Good governance processes are also essential. The information generated as part of PBRSAs has public good aspects, bringing ethical and professional obligations, which need to be considered from a policy perspective. The societal desirability of a particular PBRSA is fundamentally an issue as to whether the cost of additional data collection is justified by the benefits of improved resource allocation decisions afforded by the additional evidence generated and the accompanying reduction in uncertainty. The ex post evaluation of a PBRSA should, however, be a multidimensional exercise that assesses many aspects, including not only the impact on long-term cost-effectiveness and whether appropriate evidence was generated but also process indicators, such as whether and how the evidence was used in coverage or reimbursement decisions, whether budget and time were appropriate, and whether the governance arrangements worked well. There is an important gap in the literature of structured ex post evaluation of PBRSAs. As an innovation in and of themselves, PBRSAs should also be evaluated from a long-run societal perspective in terms of their impact on dynamic efficiency (eliciting the optimal amount of innovation).
Remote patient monitoring (RPM) has demonstrated numerous benefits in cancer care, including improved quality of life, overall survival, and reduced medical resource use. This study presents a budget ...impact analysis of a nurse navigator-led RPM program, based on the CAPRI trial, from the perspective of the French national health insurance (NHI). The study aimed to assess the impact of the program on medical resource utilization and costs.BACKGROUND/OBJECTIVESRemote patient monitoring (RPM) has demonstrated numerous benefits in cancer care, including improved quality of life, overall survival, and reduced medical resource use. This study presents a budget impact analysis of a nurse navigator-led RPM program, based on the CAPRI trial, from the perspective of the French national health insurance (NHI). The study aimed to assess the impact of the program on medical resource utilization and costs.Medical resource utilization data were collected from both medico-administrative sources and patient-reported questionnaires. Costs were calculated by applying unit costs to resource utilization and estimating the average monthly cost per patient. Sensitivity analyses were conducted to explore different perspectives and varying resource consumption.METHODSMedical resource utilization data were collected from both medico-administrative sources and patient-reported questionnaires. Costs were calculated by applying unit costs to resource utilization and estimating the average monthly cost per patient. Sensitivity analyses were conducted to explore different perspectives and varying resource consumption.The analysis included 559 cancer patients participating in the CAPRI program. From the NHI perspective, the program resulted in average savings of €377 per patient over the 4.58-month follow-up period, mainly due to reduced hospitalizations. The all-payers perspective yielded even greater savings of €504 per patient. Sensitivity analyses supported the robustness of the findings.RESULTSThe analysis included 559 cancer patients participating in the CAPRI program. From the NHI perspective, the program resulted in average savings of €377 per patient over the 4.58-month follow-up period, mainly due to reduced hospitalizations. The all-payers perspective yielded even greater savings of €504 per patient. Sensitivity analyses supported the robustness of the findings.The budget impact analysis demonstrated that the CAPRI RPM program was associated with cost savings from the perspective of the NHI. The program's positive impact on reducing hospitalizations outweighed the additional costs associated with remote monitoring. These findings highlight the potential economic benefits of implementing RPM programs in cancer care. Further research is warranted to assess the long-term cost-effectiveness and scalability of such programs in the real-world settings.CONCLUSIONThe budget impact analysis demonstrated that the CAPRI RPM program was associated with cost savings from the perspective of the NHI. The program's positive impact on reducing hospitalizations outweighed the additional costs associated with remote monitoring. These findings highlight the potential economic benefits of implementing RPM programs in cancer care. Further research is warranted to assess the long-term cost-effectiveness and scalability of such programs in the real-world settings.
Recurrent wheezing (RW) has a significant impact on infants, caregivers, and society, but morbidity and related medical resource utilization (MRU) have not been thoroughly explored. The burden of RW ...needs to be documented with population-based data. The objective was to assess the characteristics, medical management, and MRU of RW infants identified from national claims data. Infants aged from 6 to 24 months, receiving ≥2 dispensations of respiratory drugs within 3 months, and presenting a marker of poor control (index date), were selected. During the 6 months after index date, MRU was described in the cohort and among 3 subgroups with more severe RW, defined as ≥4 dispensations of respiratory drugs, ≥3 dispensations of oral corticosteroids (OCS), or ≥1 hospitalization for respiratory symptoms. A total of 115,489 infants had RW, corresponding to 8.2% of subjects in this age group. During follow-up, 68.7% of infants received inhaled corticosteroids, but only 1.8 U (unit) were dispensed over 6 months, suggesting discontinuous use. Control was mostly inadequate: 61.7% of subjects received OCS, 80.2% antibiotics, and 71.2% short-acting beta-agonists, and medical/paramedical visits were numerous, particularly for physiotherapy. Severe RW concerned 39.0% of the cohort; 32.8% and 11.7% of infants had repeated use of respiratory drugs and OCS, respectively, and 5.5% were hospitalized for respiratory symptoms. In this real-life nation-wide study, RW was common and infants had poor control and high MRU. Interventions are needed to support adequate use of controller therapy, and to improve medical care.
The aims of this research were to provide a better understanding of the specific evidence needs for assessment of clinical and cost-effectiveness of cell and gene therapies, and to explore the extent ...that the relevant categories of evidence are considered in health technology assessment (HTA) processes.
A targeted literature review was conducted to identify the specific categories of evidence relevant to the assessment of these therapies. Forty-six HTA reports for 9 products in 10 cell and gene therapy indications across 8 jurisdictions were analysed to determine the extent to which various items of evidence were considered.
The items to which the HTA bodies reacted positively were: treatment was for a rare disease or serious condition, lack of alternative therapies, evidence indicating substantial health gains, and when alternative payment models could be agreed. The items to which they reacted negatively were: use of unvalidated surrogate endpoints, single arm trials without an adequately matched alternative therapy, inadequate reporting of adverse consequences and risks, short length of follow-up in clinical trials, extrapolating to long-term outcomes, and uncertainty around the economic estimates.
The consideration by HTA bodies of evidence relating to the particular features of cell and gene therapies is variable. Several suggestions are made for addressing the assessment challenges posed by these therapies. Jurisdictions conducting HTAs of these therapies can consider whether these suggestions could be incorporated within their existing approach through strengthening deliberative decision-making or performing additional analyses.
Objectives While a French language version of the EQ-5D exists, to date, there has been no French value set to accompany it. The objective of our study was then to derive the French TTO value set of ...the EQ-5D. Methods A total of 452 respondents aged over 18 were recruited who were representative of the French population with regard to age, gender, and socio-professional group. The direct valuation of 24 health states was first obtained by Time Trade-Off (TTO), and the negative TTO values were bounded using the monotonic transformation. Several alternative model specifications were investigated to estimate the values for all 243 states in the EQ-5D descriptive system. Only the best fitting model is presented in this paper. The analysis was conducted at an individual level to make the maximum use of the available data, and we estimated mixed models with random intercept. Models were compared through the Akaike information criterion (AIC), the mean absolute error (MAE), and the Pearson correlation coefficient between the observed and the predicted values of each model. Results After exclusion, 443 respondents took part in the study. The best fitting model included the same variables as the N3-model used in UK. Conclusion This study provides the French value set of the EQ-5D based on the stated preferences of the French general public facilitating cost-effectiveness analysis.
Objective The goal of this study was to assess the differences between an ex ante and an ex post cost-eifectiveness analysis of Dabigatran etexilate vs VKAs for the prevention of thromboembolic ...events in non-valvular atrial fibrillation patients and to draw lessons on the design and use of real-world data for decision making. Methods The same model was used to calculate the cost-eifectiveness ratio using two sets of parameters. One set included the efficacy and safety outcomes data from RE-LY, the pivotal trial comparing Dabigatran to warfarin; cost data came from an ex ante publication. Outcomes data for the second set came from real-world data studies. Cost data were a mix of realworld data and other sources. Two treatment strategies were compared: treatment initiation by either Dabigatran or VKAs, followed by either VKAs or Dabigatran. A crude comparison of results was performed; the impact of data differences was then assessed. Probabilistic sensitivity results of the two analyses were compared. Results With real-world evidence, Dabigatran at both dosages was more effective for the prevention of ischemie strokes, intra-cranial haemorrhages, with less major extra-cranial haemorrhages and a similar risk of myocardial infarction. Using clinical trial data, Dabigatran 150 mg (resp. Dabigatran 110 mg) as a first-line treatment vs VKAs yielded an ICER of ϵ8077/QALY (resp. ϵ 13,116/QALY). Real-world evidence scenarios were cost-saving and more effective for both dosages. Conclusion The reassessment of outcomes and cost data had an impact on results, improving the efficiency of Dabigatran. We identify methodological issues which should be discussed if post-launch RWE based cost-effectiveness data become a standard in HTA decision making.
Objectives:
The objectives of this study were to describe the impact of systemic sclerosis associated interstitial lung disease, on quality of life, to estimate the correlation between quality of ...life and severity of lung disease and to assess the impact of interstitial lung disease on caregivers.
Methods:
Seven investigators included systemic sclerosis associated interstitial lung disease patients from December 2019 to April 2020. Sociodemographics and clinical data were collected. Patients reported outcomes and questionnaires were used with 1 generic patients reported outcome (EQ-5D-5L), 1 specific PRO (Brief Interstitial Lung Disease) and 2 self-reported questionnaires on impact of SSc complications and impact on caregivers. The correlation between forced vital capacity and EQ-5D-5L score was estimated with a multivariate linear regression model adjusted on several covariates.
Results:
In all, 89 patients were included. 26.4% were males, mean age was 58.2 ± 14.5 years. Mean EQ-5D-5L score = 0.79 ± 0.22 (median = 0.85). Mean EQ-5D-5L visual analog scale score = 60.8 ± 20.4 (median = 61.5). Mean King’s Brief Interstitial Lung Disease score = 58.4 ± 12.7 (median = 58.0). After adjustment on covariates, a significant correlation between forced vital capacity and EQ-5D-5L score was found with an increase of 0.003 of the EQ-5D-5L score for a 1% increase of FVC (p = 0.0096). No significant correlation between forced vital capacity and the EQ-VAS and King’s Brief Interstitial Lung Disease score were found. The impact of SSc on other organs was significantly correlated with EQ- 5D-5L score, respectively, for the impact scores on the lung system (p = 0.0003), heart system (p = 0.0182), Raynaud’s syndrome (p = 0.0015), digestive system (p = 0.0032), joints/muscles (p = 0.0003), skin (p < 0.0001), kidney (p = 0.0052) and gastro-oesophageal reflux (p = 0.0063). Significant correlations between King’s Brief Interstitial Lung Disease score and lung system (p < 0.0001), heart system (p < 0.0001), digital ulcers (p = 0.058), digestive system (p < 0.0001), kidney (p = 0.0004), skin (p = 0.0499) and gastro-oesophageal reflux (p = 0.0033) scores were found 68.5% of patients reported their need for a caregiver to help them in their daily life activities.
Conclusion:
Our study highlighted the strong burden of systemic sclerosis associated interstitial lung disease` for patients, especially with an impact on quality of life, on other organs manifestations and need for caregivers in their daily life.
A French Value Set for the EQ-5D-5L Andrade, Luiz Flavio; Ludwig, Kristina; Goni, Juan Manuel Ramos ...
PharmacoEconomics,
04/2020, Volume:
38, Issue:
4
Journal Article
Peer reviewed
Open access
Objective
The objective of this study was to develop a French value set for the EQ-5D-5L, for academic and clinical research, and for regulatory requirements for price-setting of drugs and medical ...devices.
Method
This study used the standardized valuation protocol developed by EuroQol, using computer-assisted personal interview software. A representative sample of 1048 French residents were interviewed by a market research company, under the supervision of the research team. Health states were valued using composite time trade-off and a discrete choice experiment. Modeling was used to create values for the 3125 possible health states. The composite time trade-off data were modeled using a Tobit model with censored observations at −1 and correcting for heteroscedasticity. A conditional logit model was used for the discrete choice results, and both models were combined using a hybrid model. An adjusted hybrid model was tested to correct for imbalance in the sample on age and sex compared with the general population. A comparison with the 3-level (3L) value set was performed.
Results
The adjusted model was preferred to comply with the representativeness of the general population. It provided a value set for which all coefficients were logically consistent. Values ranged from − 0.525 to 1. The distribution of values presented a shift towards higher values versus the 3L value set. Ranking of dimensions changed. Pain and discomfort and mobility were the dimensions with the highest potential for disutility compared with mobility and self-care for the 3L instrument.
Conclusions
This study provides a value set based on societal preferences of the French population, using an improved descriptive instrument of health-related quality-of-life health states. It will contribute to improve the quality of cost-effectiveness analysis in the French context and help stimulate disease-specific quality-of-life references for academic-, institutional-, and industry-promoted studies.
PDF
