Acute otitis media (AOM) is one of the most common childhood infections. Recurrent AOM (rAOM) is defined as the presence of three or more AOM episodes in a period of six months. We describe the methodology used to update the recommendation of the 2018 Spanish National Antimicrobial Therapeutic Guide on the use of antibiotic treatments for rAOM in children.

We followed the GRADE-ADOLOPMENT approach to update the recommendation on antibiotic treatment for rAOM. Firstly, the research question was framed in a Population, Intervention, Comparison, and Outcome format. A comprehensive search strategy was developed, the results of which were screened according to the inclusion criteria. The selected studies were reviewed, and the quality of the evidence was assessed. Subsequently, an Evidence to Decision (EtD) framework was created and the new evidence was presented to the Guideline Development Group (GDG), which updated the recommendation on rAOM treatment in children.

Among the 1,934 references identified by the database searches, only one guideline from the National Institute for Health and Care Excellence (NICE91, updated in 2022) met our inclusion criteria. This CPG included five individual studies comparing antibiotic treatments for rAOM. None of the studies demonstrated a significant advantage for any treatment. The overall quality of the evidence for these comparisons was considered low. A GRADE EtD framework was elaborated using the NICE91 recommendations but contextualized to the Spanish National Health System. Based on the evidence, the GDG did not modify the current recommendation provided in 2018.

The overall quality of the available evidence regarding antibiotic use for rAOM in children was considered low. Further research is therefore needed to resolve the controversy and increase confidence in the appropriateness of using antibiotics in the treatment of rAOM, thereby improving the quality of life of children with this condition.

The 2021 reform of early access (EA) for medicinal products in France includes a mandatory data collection for each patient. The objective of this work was to perform a two-year review of the protocols for therapeutic use and data collection (PUT-RD) validated by the health technology assessment body (Haute Autorité de Santé [HAS]) in collaboration with the national regulatory body where applicable.

The minimum dataset defined in the PUT-RD varies depending on the regulatory status of the product and may include patients’ characteristics, conditions of use, efficacy, and safety data. A descriptive review of the PUT-RDs approved between 1 July 2021 and 30 June 2023 was carried out by HAS. The following information was retrieved in each published PUT-RD: type of EA (pre- or post-marketing authorization); collection of efficacy data (yes or no) (if yes, inclusion of a patient-reported outcome measure [PROM] or no); type of data source (registry or electronic platform provided by a contract research organization [CRO]).

During the review period, a total of 98 PUT-RDs were validated, corresponding to 98 EA favorable decisions. EAs were authorized prior to marketing authorization in 40 percent of the cases. A collection of follow-up efficacy data was planned in 52 PUT-RDs, including integration of patients’ perspectives through a PROM in 32 PUT-RDs. Data collection through a registry was planned in six PUT-RDs (using the DESCAR-T registry), otherwise data collection was mainly planned through private electronic platforms operated by CROs (80% of the PUT-RDs).

To optimize the generation of relevant clinical data to be used in the regular HTA process, EAs should be requested early, prior to marketing authorization. Additionally, the use of existing data sources, in particular disease registries, is to be developed to avoid duplicate data collection and alleviate the burden for healthcare professionals.

Preadoption assessments are the most frequent type of evaluation conducted by the Spanish Network of Agencies for Health Technology Assessment and Services of the National Health System (RedETS). RedETS aimed to develop a framework to utilize real-world data (RWD) to better adjust its assessments to the Spanish population’s context and provide live assessments throughout the lifecycle of health technologies.

A working group within RedETS was set up, which held several meetings to define the primary uses of RWD in the short term. Next, a manual review was conducted on national and international initiatives that provided guidance on the use of RWD in HTA. Common pathways for utilizing RWD in HTA were identified. The working group agreed to outline and explain the key overarching steps and provide general guidelines for working with RWD, developing as illustration a use case for an interventional technology. The Big Data project of Aragon (BIGAN) was chosen as the data source for the use case.

We formulated a case for leveraging RWD in the assessment of implantable cardiac defibrillators (ICDs) for the prevention of sudden cardiac death (SCD). Based on this scenario, we developed a methodological framework outlining a workflow consistent with RedETS practices. RWD complemented the usual process of systematic review of a technology. Crucial steps comprised the definition of data requirements through a data model specification, an exploratory data analysis, and the construction of a decision model. We presented solutions for dealing with unavailable data on essential variables and unstructured records. We discussed the main limitations to account for when working with RWD.

The task ahead holds great hope but requires overcoming some challenges to fully deploy RWD-driven methods. This entails fostering collaboration with health authorities and designated data holders to address data access challenges. In the short term, it is essential to include data scientists in assessment teams and provide appropriate capacity-building to encompass RWD tools and modeling techniques.

A program for integral, transversal, and multidisciplinary management of aortic stenosis (MITMEVA) is being implemented at the Clinic Barcelona University Hospital (CBUH) to provide adequate treatment for patients with aortic stenosis (AS). Eleven actions at different care points were implemented (e.g., awareness raising for the general population, a single entry path for patient referral, prehabilitation and rehabilitation, and a risk-sharing agreement). Preliminary results are presented.

A before-and-after implementation study was conducted with 131 patients under MITMEVA and 131 matched (for treatment, New York Heart Association classification, sex, age, and referral place) historical controls. Data were collected on resources used and quality of life and to calculate several key performance indicators (KPIs) (e.g., knowledge improvement in citizens, time from diagnosis to treatment, and patient involvement and satisfaction) for each implemented action. A descriptive analysis of KPIs and a Markov model were performed to simulate clinical and economic outcomes for patient health states over time after the first year until the tenth year after intervention.

The MITMEVA program increased quality-adjusted life-years by 1.78 (p=0.011) and reduced time from referral to first hospital visit by 24.7 percent (p=0.05), hospital complications by 19.7 percent (p=0.05), mean conventional ward stay from 12.8 to 8 days (p=0.01), and mean intensive care unit stay from 9.75 to 4.25 days, although the latter difference was not statistically significant (p=0.139). The mean cost per patient was reduced from EUR7,573.27 per patient to EUR6,024.61 per patient (p=0.01). The MITMEVA program was a dominant strategy. There was a 46 percent increase in correct AS symptom identification after delivering training on AS to the general population.

Integrated care approaches can potentially improve patient continuum of care if the strategies are deployed in a multidisciplinary and transversal way across healthcare actors. The MITMEVA program significantly improved clinical and economic outcomes and organization of care, benefiting patients and clinicians. Applying health technology assessment methods to such innovative projects can help prove the value of organizational innovations.

Comprehensive genomic profiling (CGP) identifies many targets at once. However, it is challenging for reimbursement decision-makers to incorporate all potential effects in their assessment. The aim of this study is twofold: first, to identify which factors, besides effectiveness and costs, might influence the choice for CGP in advanced cancer patients, and second, to identify the available evidence for these factors.

We performed a systematic literature review in MEDLINE, Embase, and Scopus with a two-step design. First, a scoping search was performed to identify relevant factors. Extracted factors were grouped with domains of the EUnetHTA core model and ISPOR (Professional Society for Health Economics and Outcomes Research) “value flower.” Two expert sessions were held to validate factors and construct definitions. Second, a systematic search was conducted to identify the available empirical evidence for these factors. Eligibility criteria for the systematic search were the use of CGP (≥200 genes), advanced cancer patients, and the presentation of empirical evidence on one of the factors.

Five factors were identified in the scoping search: “feasibility” (adopting tests in the health care system), “test journey” (pathway from requesting tests until reporting of results), “wider implications of diagnostic results” (impact of test beyond identifying on-label treatments), “organization of laboratories” (organization of tests and access to tests), and “scientific spillover” (learnings of testing). Eighty-three articles were included following the systematic search, and empirical evidence was identified for the factors “test journey” and “wider implications of diagnostic results”. Few studies had adequate comparative study designs. Heterogeneity was observed among studies in the definitions of outcomes and the reported evidence.

Comprehensive reimbursement decision-making for CGP can be supported by including the five identified factors. However, quantifiable evidence was only identified for the “patient test journey” and “wider implications of diagnostic results”. Current literature provides limited high-quality evidence to establish the added benefit of CGP, as adequately designed comparisons are lacking. For evidence-based decision-making, uniform outcome measurements are recommended.

Technical aids (TAs) are health devices (e.g., hearing aids, prostheses, wheelchairs) used to restore a body function or compensate for disability. In France, TAs are assessed based on clinical data, as are any medical devices (MDs). Considering that TAs are special MDs (no curative action), specific assessment methods might be proposed. The methods used in Europe were explored.

To learn about TA evaluation practices in Europe, the French National Authority for Health (HAS) sent a survey to nine identified European health technology assessment (HTA) agencies. The questionnaire was specific to TAs, and the questions asked were about: (i) the reimbursement of TAs (reimbursed or not, at national or local level), (ii) the HTA process (HTA process or not, HTA process description), and (iii) the methods used (any specific methods for assessing TAs other than HTA methods to assess “classic” MDs).

All the European HTA agencies contacted provided a response. All these countries have a healthcare system that allows TAs to be reimbursed. In Austria, Ireland, Italy, the Netherlands, Norway, Portugal, Spain, and the United Kingdom, TAs must be CE-marked and comply with technical standards to be reimbursed; no clinical data are required. However, the National Institute for Health and Care Excellence provides recommendations for certain TA categories. In Germany, the CE-marking and the respect of technical standards are mandatory, and certain TAs need to show a medical benefit (e.g., orthoses); nevertheless, there are no specific methods to assess TAs.

Overall, the feedback obtained does not reveal any specific evaluation method applied to TA assessment in the various European countries analyzed. France stands out from these European countries regarding the assessment of TAs because of its one-stop-shop system for health technology developers and the HAS requirements for technical and clinical data.

A program for integral, transversal, and multidisciplinary management of aortic stenosis (MITMEVA) was implemented at the Clinic Barcelona University Hospital to provide adequate and personalized treatment for patients with aortic stenosis (AS). MITMEVA includes 11 actions in the AS care pathway. This study aimed to test the scalability of MITMEVA by gathering the perspectives of relevant stakeholders in two other Catalan hospitals.

A mixed method study design was used to collect and analyze the views of relevant stakeholders on the scalability of the MITMEVA project. Qualitative and quantitative methods were used to answer the research question. All participants were interviewed individually using a semi-structured interview guide. The interviews were transcribed and analyzed by performing a content analysis with Atlas.ti software. Quantitative data were gathered and analyzed by adjusting the Intervention Scalability Assessment Tool (ISAT).

Nine participants were interviewed from two tertiary hospitals in Catalonia and eight ISAT questionnaires were completed. From the content analysis, 11 of the 15 themes identified related to actions implemented in the MITMEVA program. The results pointed toward a positive view of implementation of the MITMEVA program in general, showing a good scalability for all the ISAT domains. On a scale of zero to three, most domains were scored two or above.

The qualitative and quantitative results indicated that the MITMEVA program has potential for scaling up to other Catalan hospitals to improve the management of AS. Generally, stakeholders from the two participating hospitals were positive about the project. However, small adjustments will be needed to account for the culture and organizational characteristics of the hospitals when scaling up the MITMEVA program.