Forecasting the potential impact of cell and gene therapies in France: projecting product launches and patients treated.

Objective: To evaluate the potential impact of cell and gene therapies (CGTs) in France by forecasting the number of patients that will be treated with CGTs over the period 2023-2030 by therapeutic area and region. Methods: A review of CGTs in clinical development and related disease epidemiology was conducted to forecast the number of CGT launches and patient population between 2023 and 2030. The number of expected launches was identified by filtering the clinical development pipeline with estimated time to launch and probability of success values from Project ALPHA. Disease prevalence and incidence in France were combined with projected adoption rates derived from historical data to forecast the patient population to be treated. Results: Up to 44 new CGTs are forecasted to launch in France in the period 2023-2030, which translates into more than 69,400 newly treated patients in 2030. Leading indications in terms of newly treated patients per year include cardiovascular disease, hematological cancers and solid tumors with 27,300, 15,200 and 13,000 newly treated patients in 2030, respectively. Discussion: The forecast suggests that the future landscape of CGTs will undergo a shift, moving from CGTs targeting (ultra) rare diseases to more prevalent diseases. In France, this will likely pose organizational challenges hindering patient access to these transformative therapies. Further research and planning around network organization and patient distribution are needed to assess and improve the readiness of the French healthcare system for ensuring access for this growing number of patients to be treated with CGTs.

Incidence and survival of transthyretin amyloid cardiomyopathy from a French nationwide study of in- and out-patient databases.

BACKGROUND: Precise data about ATTR-CM incidence rates at national level are scarce. Consequently, this study aimed to estimate the annual incidence and survival of transthyretin amyloid cardiomyopathy (ATTR-CM) in France between 2011 and 2019 using real world data. We used the French nationwide exhaustive data (SNDS database) gathering in- and out-patient claims. As there is no specific ICD-10 marker code for ATTR-CM, diagnosis required both amyloidosis (identified by E85. ICD-10 code or a tafamidis meglumine delivery) and a cardiovascular condition (identified by ICD-10 or medical procedure codes related to either heart failure, arrhythmias, conduction disorders or cardiomyopathies), not necessarily reported at the same visit. Patients with probable AL-form of amyloidosis or probable AA-form of amyloidosis were excluded. RESULTS: Between 2011 and 2019, 8,950 patients with incident ATTR-CM were identified. Incidence rates increased from 0.6 / 100,000 person-years in 2011 to 3.6 / 100,000 person-years in 2019 (p < 0.001), reaching 2377 new cases in 2019. Sex ratios (M/F) increased from 1.52 in 2011 to 2.23 in 2019. In 2019, median age at diagnosis was 84.0 years (85.5 for women and 83.5 for men). Median survival after diagnosis was 41.9 months (95% CI [39.6, 44.1]). CONCLUSIONS: This is the first estimate of nationwide ATTR-CM incidence in France using comprehensive real-world databases. We observed an increased incidence over the study period, consistent with an improvement in ATTR-CM diagnosis in recent years.

Choice of GPIIb/IIIa antagonist in percutaneous coronary intervention: how should economic criteria be factored in?

OBJECTIVE: Three GPIIb/IIIa antagonists are available in the market. In France, as in many countries, their acquisition costs strongly differ. The objective of this study was to analyze how economic criteria-beyond the acquisition cost-should be factored in, when choosing a GPIIb/IIIa antagonist. METHOD: Both clinical and economic papers on the use of GPIIb/IIIa antagonists in percutaneous coronary interventions published in peer-review journals from 1994 to 2002 were reviewed and analyzed. RESULTS: Cost differentials between products strongly vary from one 'cost concept' to another, i.e., acquisition cost, administration cost, hospital cost, net treatment cost. The comparison of efficacy is even more complicated, as most of the time only indirect comparisons are available, based on different clinical studies, with different durations and definitions of outcomes. Finally, cost-effectiveness ratios range from US dollar 10,695 per avoided event for eptifibatide (IMPACT II study) to US dollar 74,047 for tirofiban (RESTORE study). CONCLUSION: The concept of cost, inevitably entering into the choice of a medicinal strategy, must be used with caution. The amplitude of the difference between products, and the product favored by the difference, vary according to the cost concept retained.

Calcium-vitamin D3 supplementation is cost-effective in hip fractures prevention.

OBJECTIVE: To assess the cost implications for a preventive treatment strategy for institutionalised elderly women with a combined 1200 mg/day calcium and 800 IU/day vitamin D(3) supplementation in seven European countries. DESIGN: Retrospective cost effectiveness analysis based on a prospective placebo-controlled randomised clinical trial. DATA SOURCES: Recently published cost studies in seven European countries. Clinical results from Decalyos, a 3-year placebo-controlled study in elderly institutionalised women. TRIALS: Decalyos study, with 36 months follow-up of 3270 mobile elderly women living in 180 nursing homes, allocated to two groups. One group received 1200 mg/day elemental calcium in the form of tricalcium phosphate together with 800 IU/day (20 microg) of cholecalciferol (vitamin D(3)), the other placebo. RESULTS: In the 36 months analysis of the Decalyos study, 138 hip fractures occurred in the group of 1176 women, receiving supplementation and 184 hip fractures in the placebo group of 1127 women. The mean duration of treatment was 625.4 days. Adjusted to 1000 women, 46 hip fractures were avoided by the calcium and vitamin D(3) supplementation. For all countries, the total costs in the placebo group were higher than in the group receiving supplementation, resulting in a net benefit of 79000-711000 per 1000 women. CONCLUSION: This analysis suggests that the supplementation strategy is cost saving. The results may underestimate the net benefits, as this treatment has also shown to be effective in decreasing the incidence of other non-vertebral fractures in elderly institutionalised women.

Long-Term Cost Effectiveness of Interferon-beta-1a in the Treatment of Relapsing-Remitting Multiple Sclerosis : An Econometric Model.

OBJECTIVE: To evaluate the cost effectiveness of subcutaneous interferon-beta-1a (IFNbeta-1a) 44mug three times weekly in relapsing-remitting multiple sclerosis (RRMS) using an econometric model. METHODS: Data on RRMS patients treated with IFNbeta-1a 22 or 44mug subcutaneously three times weekly or placebo for up to 4 years were obtained from the Prevention of Relapses and disability by Interferon-beta-1a Subcutaneously in Multiple Sclerosis (PRISMS) study. The area under the Expanded Disability Status Scale (EDSS) score-time curve was used as a measure of disability and the effectiveness of therapy was expressed as EDSS-months of disability prevented. Costs were calculated for the UK and France, and results were projected to 10 and 20 years using a time series regression model. RESULTS: Over 10 years, treatment with IFNbeta-1a 44mug subcutaneously three times weekly prevented 121 EDSS-months of additional disability at a cost of Euros (euro)732 each (year of costing 2000). Over 20 years, 321 EDSS-months were saved at a cost of euro359 per month (year of costing 2000). CONCLUSION: This analysis indicated that IFNbeta-1a 44mug subcutaneously three times weekly is cost effective in RRMS and that treatment becomes increasingly cost effective over time.

[Cost of treatment and follow up of breast cancer: a retrospective assessment in a comprehensive cancer centre]. / Evaluation rétrospective du coût du cancer du sein dans un centre de lutte contre le cancer.

CONTEXT: Breast cancer is one of the major causes of premature death for women. Its cost management is important for both the national health insurance and the individual health care providers. OBJECTIVE: The objective of this study was to assess the global medical cost of breast cancer from diagnosis to follow up in one French medical centre: centre René-Huguenin, Saint-Cloud (92). METHOD: Duration of medical activities and other medical resources utilisations were collected from a retrospective cohort of 120 patients followed from January 1995 to February 2000. Unit costs were obtained from cost accounts of the Centre. RESULTS: The mean medical cost per patient was FF 66,067 [60,318-7,815] (USD $ 10,744 [9,809-11,679]). The mean cost varied from FF 41,875 (UDS $ 6,810) to FF 81,020 (UDS $ 13,175) depending on choice of type of therapy. The initial treatment phase was the most expensive, costing FF 48,397 [46,176-50,617] (USD $ 7,870 [7,509-8,231]) which represented 73.3% of the global cost. CONCLUSION: This study has provided an estimate of the real global cost of managing patients with breast cancer in a single French Comprehensive Cancer Centre (CLCC). The study method used is readily transposable to other treatment contexts and to other types of cancer.