Place of patient-reported outcomes & experiences measurements (PROMS/PREMS) in the assessment and pricing of Health technologies in France.

In the context of health technologies assessment, patient-reported outcome measures (PROMs) have become assessment criteria that are expected by evaluation agencies along with the other usual clinical criteria. PROMs instruments measure all aspects of patient experience in connection with their health: symptoms, activities of daily living (physical function, sleep, etc.), various aspects of health-related quality of life (QoL), compliance, global impression of change in wellbeing. PROMs are useful both as 1) a primary or secondary efficacy endpoints, and 2) a tolerability criterion to supplement vigilance data reported by clinicians. Measurement of PROMs must be subject to methodological rigor that is identical to that of other assessment criteria measured by an observer. Scales must be validated, suitable for the objective, and where possible specific to a disease. In addition to standard measures of quality of life, PROMs are taken into consideration in the assessments performed by the HAS, even if their impact on the conclusions is difficult to isolate, as assessments are multifactorial and take into account all data available with regard to the medical context. The CEPS will indirectly take into account PROMs in the fixing of the price or tariff only if they have contributed to the award of the ASA/ASMR by the ad hoc committee of the HAS. The working group has formulated three recommendations which aim to further the implementation of patient-reported outcome measures: (1) Better information for all parties involved in a dossier for technology assessment, (2) Systematization of the collection of PROMs for evaluation of health products, (3) Improved quality of dossiers thanks to the use of relevant and validated tools.

Early patient access to health technologies: Is innovation needed for early management?

The question of early patient access to innovative health technologies arises from the assumption that, once a certain level of effectiveness or efficiency is achieved, waiting for mainstream coverage would represent a loss of opportunity for patients or for the community. This was the premise on which the round table based its dialogue. Early access is understood as the funding of a technology that comes within this field and is CE-marked but has not yet attained "mainstream" coverage. There are several early access schemes in France ("forfait innovation", early coverage, exceptional coverage, RIHN). This round table was an opportunity to establish mapping, extended to devices not dedicated to early access but which could nevertheless provide some patients with access to non-mainstreamed technologies (Article 51, ETAPES experiments, DGOS call for projects, local schemes). It is an initial step that would need to be further developed and complemented by the dissemination of common communication materials available to all, including patients. The existing schemes are in fact still poorly known. Consideration would also have to be given to the advisability of developing these schemes in order to adapt them to the new European requirements. More generally, early access schemes must be integrated into an ecosystem that is conducive for their relevance: consideration of procedures associated with medical devices benefiting from early access; short time frames of examination; patient information. Finally, the round table proposes the creation of a new early access scheme, complementary to those that exist and that would be positioned, after CE marking, between the "forfait innovation" and mainstreaming: PRESTO (Prise En charge Sécurisée et Temporaire de technologies innOvantes) (secure and temporary coverage for innovative technologies).

Budget impact of intravenous ferric carboxymaltose in patients with chronic heart failure and iron deficiency in France.

AIMS: This analysis aims to evaluate the budget impact of intravenous iron therapy with ferric carboxymaltose for patients with systolic chronic heart failure and iron deficiency, from the perspective of the French public health insurance. METHODS AND RESULTS: A budget impact model was adapted to forecast the budget impact over 5 years, according to two scenarios: one where patients receive ferric carboxymaltose according to market share forecast and another where patients are not treated for iron deficiency. Clinical data were extrapolated from pooled data from four randomized controlled trials. The time horizon was extended to 5 years by applying transition probabilities estimated from the CONFIRM-HF trial. Epidemiological parameters for France were derived from the literature. Cost parameters were derived from national available databases. In the base case analysis, the modelled 5 year cost difference between the scenarios with ferric carboxymaltose vs. no iron deficiency treatment in a population of 189 334 prevalent and incident patients led to €0.8m savings. The cumulative savings resulted from a reduction in the hospitalization costs associated with worsening heart failure (€-35.8m) as well as a reduction in the follow-up costs (€-2.9m). These cost savings outweighed the costs of ferric carboxymaltose treatment (€37.7m). Sensitivity analyses showed that the budget impact varied from €-34m to €+146m. Parameters with the most impact on the budget were the hospitalization rate for patients not treated for iron deficiency, the number of ambulatory sessions needed, the absence of hospitalization cost differentiation between New York Heart Association classes, and administration settings costs. CONCLUSIONS: Iron deficiency treatment with ferric carboxymaltose in systolic chronic heart failure patients results in an improvement of New York Heart Association class and thereby increases the well-being of the patients, while providing an overall cost saving for the French national health insurance.

Cost-effectiveness of drug-eluting stents versus bare-metal stents in patients undergoing percutaneous coronary intervention.

OBJECTIVE: To determine the cost-effectiveness of drug-eluting stents (DES) compared with bare-metal stents (BMS) in patients requiring a percutaneous coronary intervention in France, using a recent meta-analysis including second-generation DES. METHODS: A cost-effectiveness analysis was performed in the French National Health Insurance setting. Effectiveness settings were taken from a meta-analysis of 117 762 patient-years with 76 randomised trials. The main effectiveness criterion was major cardiac event-free survival. Effectiveness and costs were modelled over a 5-year horizon using a three-state Markov model. Incremental cost-effectiveness ratios and a cost-effectiveness acceptability curve were calculated for a range of thresholds for willingness to pay per year without major cardiac event gain. Deterministic and probabilistic sensitivity analyses were performed. RESULTS: Base case results demonstrated that DES are dominant over BMS, with an increase in event-free survival and a cost-reduction of €184, primarily due to a diminution of second revascularisations, and an absence of myocardial infarction and stent thrombosis. These results are robust for uncertainty on one-way deterministic and probabilistic sensitivity analyses. Using a cost-effectiveness threshold of €7000 per major cardiac event-free year gained, DES has a >95% probability of being cost-effective versus BMS. CONCLUSIONS: Following DES price decrease, new-generation DES development and taking into account recent meta-analyses results, the DES can now be considered cost-effective regardless of selective indication in France, according to European recommendations.

MitraClip therapy in mitral regurgitation: a Markov model for the cost-effectiveness of a new therapeutic option.

Introduction Mitral regurgitation is a heart condition resulting from blood flowing from the left ventricle towards the left atrium, increasing the risk of heart failure and mortality. While surgery can greatly reduce these risks, some patients are not eligible, resulting in medication being their only therapeutic alternative. The MitraClip (Abbot Vascular) is a medical device that is percutaneously implanted and designed to eliminate leaking of the mitral valve. Methods The efficacy of the MitraClip strategy vs medical management was assessed using a 4-state Markov model based on the mitral regurgitation grade (mitral regurgitation grade 0, I/II, and III/IV, and death). At each 1-month cycle, patients were or were not hospitalized. The model analyzed a fictional population of 1000 patients over a 5-year period from a national Health Insurance perspective. The primary end-point was the number of deaths avoided. Data from the EVEREST II High Risk Study patients were used along with a literature review. Results At 5 years, among the 1000 patients, 276 deaths were found to be avoidable with the MitraClip strategy. The incremental cost-effectiveness ratio (ICER) was €93,363 per death avoided. The annual ICER was calculated to take into consideration excess costs resulting from the MitraClip over the first year (€29,984 vs €8557 for the reference strategy) and the reduction of costs in following years (€3122 for MitraClip vs €8557 for reference strategy). Thus, the mean ICER was calculated to be €20,720 per death avoided. Conclusion The MitraClip is a novel alternative therapy for mitral insufficiency in patients ineligible for surgery that may offer a medico-economic advantage.