Evaluation of digital medical devices: How to take into account the specificities of these solutions?

The beginning of the 21st century has seen an increasing number of digital medical devices (DMDs) arrive on the European market, bringing major benefits and changes for society. DMDs are unique in that they bring intelligence to the organisation of care, and generate and collect a wealth of real-life data with ultra-fast life cycles. They have specific requirements, particularly in terms of data security and interoperability. In France and Europe, the construction of evidence, the assessment process and evaluation methodologies with a view to purchase or reimbursement must adjust to these changes, given the specific features of these technologies. This digital leap has opened up new perspectives for healthcare, along with economic, ethical and regulatory issues. The challenge is to assess the clinical and organisational impact, reliability, safety, interoperability, efficiency and budgetary impact of DMDs in line with the requirements of new standards, guidelines and regulations. This should result in a coherent, pragmatic and proportionate evaluation, so that public decision-makers and buyers can take advantage of the potential opportunities that these digital devices offer to improve healthcare delivery. Thus, a fair and informed evaluation of DMDs would emerge, providing a solid basis to steer their inclusion into contemporary medical practices. This fundamental issue of evaluation, linked to the digital nature of these MDs, is what the round table, comprising experts from academia and/or hospitals, institutions and industry, sought to resolve. Discussions led to proposals on how DMDs should be evaluated, bearing in mind their complexity. The round table set out to identify the bottlenecks in the entire evaluation process, from the CE marking phase, compliance with French safety and interoperability requirements, through to national or local evaluation, in order to inform a purchasing policy and draw up proposals covering the entire spectrum. Ten concrete recommendations were put forward by the round table, aimed at improving the evaluation process by making it clearer and more adaptable, thus offering greater flexibility in the evaluation and decision-making stages. This well-thought-out approach is designed to facilitate a comprehensive and flexible evaluation of DMDs given the constantly evolving technological context.

CPAP resumption after a first termination and impact on all-cause mortality in France.

BACKGROUND: Continuation of continuous positive airway pressure (CPAP) therapy after initial prescription has been shown to reduce all-cause mortality versus therapy termination. However, there is a lack of data on the rates and impact of resuming CPAP in patients with obstructive sleep apnoea (OSA). This analysis determined the prevalence of CPAP resumption in the year after termination, characterised determinants of CPAP resumption, and examined the impact of CPAP resumption on all-cause mortality. METHODS: French national health insurance reimbursement system data for adults aged ≥18 years were used. CPAP prescription was identified by specific treatment codes. Patients who resumed CPAP after first therapy termination and continued to use CPAP for 1 year were matched with those who resumed CPAP then terminated therapy for a second time. RESULTS: Out of 103 091 individuals with a first CPAP termination, 26% resumed CPAP over the next 12 months, and 65% of these were still using CPAP 1 year later. Significant predictors of CPAP continuation after resumption included male sex, hypertension and CPAP prescription by a pulmonologist. In the matched population, the risk of all-cause death was 38% lower in individuals who continued using CPAP after therapy resumption versus those who had a second therapy discontinuation (hazard ratio 0.62, 95% CI 0.48-0.79; p=0.0001). CONCLUSION: These data suggest that individuals with OSA who fail initial therapy with CPAP should be offered a second trial with the device to ensure that effective therapy is not withheld from those who might benefit.

Health trajectories before initiation of non-invasive ventilation for chronic obstructive pulmonary disease: a French nationwide database analysis.

Background: Chronic obstructive pulmonary disease (COPD) is the most common indication for long-term domiciliary non-invasive ventilation (NIV) but there is uncertainty in data supporting current guidelines. This study described health trajectories before initiation of at-home NIV in people with COPD, and compared mortality outcomes between groups with different pre-NIV health trajectories. Methods: Data were from the French national health insurance reimbursement system database for individuals with COPD aged ≥40 years and ≥1 reimbursement for NIV between 1 January 2015 and 31 December 2019. Common health trajectories were determined using time sequence analysis through K-clustering (TAK analysis). Findings: Data from 54,545 individuals were analysed; the population was elderly (median age 70 years) with multiple comorbidities. Four clusters were generated. Cluster 1 (n = 35,975/54,545; 66%) had NIV initiated in ambulatory settings or after the first acute event/exacerbation. Cluster 2 (6653/54,545; 12%) started NIV after ≥2 severe exacerbations in the previous 6 months. Cluster 3 (11,375/54,545; 21%) started NIV after frequent severe COPD-related exacerbations in the previous year. Cluster 4 (652/54,545; 1%) started NIV after many long-lasting severe exacerbations. The four clusters differed in age, sex, comorbidities, pre-NIV investigations, and prescriber/location of NIV initiation. Mortality differed significantly between clusters: highest in Cluster 4 and lowest in Cluster 1. Interpretation: The significant heterogeneity in clinical initiation of NIV probably reflects the current lack of strong evidence and guideline recommendations. Knowledge about the characteristics and outcomes in different clusters should be used to address inequities and facilitate more consistent and personalised use domiciliary NIV in COPD. Funding: JLP and SB are supported by the French National Research Agency in the framework of the "Investissements d'avenir" program (ANR-15-IDEX-02) and the "e-health and integrated care and trajectories medicine and MIAI artificial intelligence (ANR-19-P3IA-0003)" Chairs of excellence from the Grenoble Alpes University Foundation. This work was supported by ResMed.

Why and how can we improve patient and caregiver information for the proper use of their medical devices throughout the care pathway?

Information is generally a major pillar in the relationship of trust between the patient and their healthcare team. In the case of medical devices, which are operator-dependent and for which the proper "handling" of the device will directly affect its performance, information on the proper use becomes a major therapeutic issue. To do this, patients must have access to quality information about health products. Twenty years after the Kouchner Act introduced a right to information for patients, our panel questioned the quality of information provided to patients about the medical devices they use during their care pathway. We mapped off the information process throughout the life of the medical device (manufacturer, prescription, dispensing, use and monitoring) to identify deficiencies and potential corrective solutions. Thus, the device could respond to the 5B as for the drug (right device, right patient, right indication, right time, and right use). Seven recommendations have emerged and are proposed in this article.

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.

Relationship Between CPAP Termination and All-Cause Mortality: A French Nationwide Database Analysis.

BACKGROUND: Randomized controlled trials have failed to demonstrate an effect of CPAP therapy on mortality. However, these studies have a number of important limitations, including low CPAP adherence, patient selection, and a small number of mortality events. RESEARCH QUESTION: What are the effects of CPAP therapy termination in the first year on all-cause mortality in patients with OSA from the Nationwide Claims Data Lake for Sleep Apnea study? STUDY DESIGN AND METHODS: Data from the Système National des Données de Santé (SNDS) database, the French national health insurance reimbursement system, for all new CPAP users ≥ 18 years of age were analyzed. The SNDS contains comprehensive, individualized, and anonymized data on health spending reimbursements for > 99% of all individuals living in France. OSA diagnosis was based on specific disease codes, whereas CPAP prescription was identified using specific treatment method codes. CPAP therapy termination was defined as the cessation of CPAP reimbursements triggered by the respiratory physician or sleep specialist in charge of follow-up. Patients who terminated therapy in the first year were propensity score matched with those who continued to use CPAP. The primary outcome was all-cause mortality. Three-year survival was visualized using Kaplan-Meier curves. Contributors to mortality also were determined. RESULTS: Data from two matched groups each including 88,007 patients were included (mean age, 60 years; 64% men). Continuation of CPAP therapy was associated with a significantly lower risk of all-cause death compared with CPAP therapy termination (hazard ratio [HR], 0.61; 95% CI, 0.57-0.65; P < .01, log-rank test). Incident heart failure also was less common in patients who continued vs terminated CPAP therapy (HR, 0.77; 95% CI, 0.71-0.82; P < .01). INTERPRETATION: These real-world data from a comprehensive, unbiased database highlight the potential for ongoing use of CPAP treatment to reduce all-cause mortality in patients with OSA.

CPAP Therapy Termination Rates by OSA Phenotype: A French Nationwide Database Analysis.

The nationwide claims data lake for sleep apnoea (ALASKA)-real-life data for understanding and increasing obstructive sleep apnea (OSA) quality of care study-investigated long-term continuous positive airway pressure (CPAP) termination rates, focusing on the contribution of comorbidities. The French national health insurance reimbursement system data for new CPAP users aged ≥18 years were analyzed. Innovative algorithms were used to determine the presence of specific comorbidities (hypertension, diabetes and chronic obstructive pulmonary disease (COPD)). Therapy termination was defined as cessation of CPAP reimbursements. A total of 480,000 patients were included (mean age 59.3 ± 13.6 years, 65.4% male). An amount of 50.7, 24.4 and 4.3% of patients, respectively, had hypertension, diabetes and COPD. Overall CPAP termination rates after 1, 2 and 3 years were 23.1, 37.1 and 47.7%, respectively. On multivariable analysis, age categories, female sex (1.09 (1.08-1.10) and COPD (1.12 (1.10-1.13)) and diabetes (1.18 (1.16-1.19)) were significantly associated with higher CPAP termination risk; patients with hypertension were more likely to continue using CPAP (hazard ratio 0.96 (95% confidence interval 0.95-0.97)). Therapy termination rates were highest in younger or older patients with ≥1 comorbidity. Comorbidities have an important influence on long-term CPAP continuation in patients with OSA.

Telemonitoring and experimentation in telemedicine for the improvement of healthcare pathways (ETAPES program). Sustainability beyond 2021: What type of organisational model and funding should be used?

Medical telemonitoring could be a significant lever for improving access to care and the cost-effectiveness of patient management, particularly for chronic conditions, by reducing hospital admissions. It also appears to be an opportunity to redesign the healthcare pathway and its organisation in a way that is suited to the demographic, economic, and technical challenges that our healthcare system must address. In order to prepare for the future of socialised financing of telemonitoring at the end of the ETAPES programme, participants at the round table worked on nine recommendations, based on an analysis of international literature and the French healthcare system. The main stance endorsed is the implementation of a fixed rate fee associated with the creation of generic guidelines for the diseases currently involved in the ETAPES trials. Similarly, the implementation of an evaluation that is appropriate for telemonitoring devices and of incentives for quality of care seems necessary for the sustainable deployment of telemonitoring in France. Further studies will be required in addition to the points discussed by the round table in order to delve deeper into certain topics, such as therapeutic support for patients.

New European medical device regulation: How the French ecosystem should seize the opportunity of the EUDAMED and the UDI system, while overcoming the constraints thereof.

The European regulation governing the placing on the market of medical devices (MD) provides for the gradual and mandatory phasing-in, within the European Union, of a unique device identification (UDI) system and a European database (EUDAMED) aimed at improving traceability and transparency of the market. The deployment of these tools will have a significant impact on the stakeholders of the medical device world (companies, health institutions, healthcare professionals, competent authorities, etc.). A strengths/weaknesses/opportunities/threats (SWOT) analysis conducted among the think tank participants shows real support for the objectives but also distinguishes a great deal of issues relating to the implementation of these tools. An inventory of the current traceability tools and databases used in France was then carried out to measure the gaps between the requirements of the European regulation and the current situation revealing that EUDAMED and the UDI system will ultimately have to slot into a complex and poorly interoperable ecosystem. An essential first step to facilitate this integration will be to increase the number of educational, awareness-raising and information initiatives for the stakeholders concerned. Several other recommendations were put forward to support the implementation of EUDAMED and the UDI system in France and thus enable their promises to crystallise in the future.

Care pathway, towards the establishment of tailored funding: Reasons and criteria for success.

Care pathways are often at the forefront of political thinking about health care practices in France without ever finding a durable means for their extension. Closely linked to funding of healthcare system, they have, once again, been the object of so many economical discussions in 2017, as part of a more optimistic climate of governance which is therefore more open to change. Our changing system, the development and increasingly chronic nature of diseases, the scale of technological breakthroughs, these are all factors driving this topic forward. The object of this work, after a necessary study of the semantics of the term "pathway" and even "funding", was to identify all prerequisites and good practices for the stakeholders to develop a pilot pathway and then its relevant implementation in France. To do so, the members of the Round Table have relied on the presentation of examples of care pathways in order to identify triggers to a progressive, adapted extension to the whole territory. The group has identified key elements and priorities for the establishment of public funding beyond existing funding to incentive team work, particularly in the case of treatment rupture points and/or when they have diverging interests. Finally, creating a climate of confidence among patients, professionals, hospitals, the ARS, payers and manufacturers in handling change management will become the key challenges of the implementation of future pathways.

What is the budget impact of a new treatment or new health technology arriving on the market?

In France, market access for innovative drugs (level I, II & III improvement of medical service rendered having significant impact on health insurance expenditure) involves medico-economic evaluation. In addition to cost-effectiveness analysis (CEA), budget impact analysis (BIA) can be performed, especially since the sustainability of the health insurance system has become a growing concern for all stakeholders. The members of the Giens 2016 round table discussed the contribution of BIA based on a review of the literature on distribution models, the participants' experiences including experience related to the modalities of hospital assessments, and examples from other countries. The round table established recommendations on four elements of interest: 1: the use of BIA from a contractual point of view - between manufacturers and the French Economic Committee for Health Products - during the price negotiations process, and from a prospective point of view within the framework of the annual review of budget expenditures; the definition of the target population and the rhythm of a health product's distribution are also major elements; 2: the interpretation of BIA results in light of CEA results: for what products and at what moment in the life cycle of these products; 3: the integration of the rhythm of a health product's distribution into the BIA; 4: preoccupations about how the level of health product implementation is integrated into the BIA in terms of opportunity cost and organisational quality for the promoter.

Assessment and non-clinical impact of medical devices.

Medical devices (MDs) cover a wide variety of products. They accompany changes in medical practice in step with technology innovations. Innovations in the field of MDs can improve the conditions of use of health technology and/or modify the organisation of care beyond the strict diagnostic or therapeutic benefit for the patients. However, these non purely clinical criteria seem to be only rarely documented or taken into account in the assessment of MDs during reimbursement decisions at national level or for formulary listing by hospitals even though multidimensional models for the assessment of health technologies have been developed that take into account the views of all stakeholders in the healthcare system In this article, after summarising the background concerning the assessment of health technologies in France, a definition of non-clinical criteria for the assessment of MDs is proposed and a decision tree for the assessment of MDs is described. Future lines of approach are proposed as a conclusion.