Challenges and Opportunities With Routinely Collected Data on the Utilization of Cancer Medicines. Perspectives From Health Authority Personnel Across 18 European Countries.

Background: Rising expenditure for new cancer medicines is accelerating concerns that their costs will become unsustainable for universal healthcare access. Moreover, early market access of new oncology medicines lacking appropriate clinical evaluation generates uncertainty over their cost-effectiveness and increases expenditure for unknown health gain. Patient-level data can complement clinical trials and generate better evidence on the effectiveness, safety and outcomes of these new medicines in routine care. This can support policy decisions including funding. Consequently, there is a need for improving datasets for establishing real-world outcomes of newly launched oncology medicines. Aim: To outline the types of available datasets for collecting patient-level data for oncology among different European countries. Additionally, to highlight concerns regarding the use and availability of such data from a health authority perspective as well as possibilities for cross-national collaboration to improve data collection and inform decision-making. Methods: A mixed methods approach was undertaken through a cross-sectional questionnaire followed-up by a focus group discussion. Participants were selected by purposive sampling to represent stakeholders across different European countries and healthcare settings. Descriptive statistics were used to analyze quantifiable questions, whilst content analysis was employed for open-ended questions. Results: 25 respondents across 18 European countries provided their insights on the types of datasets collecting oncology data, including hospital records, cancer, prescription and medicine registers. The most available is expenditure data whilst data concerning effectiveness, safety and outcomes is less available, and there are concerns with data validity. A major constraint to data collection is the lack of comprehensive registries and limited data on effectiveness, safety and outcomes of new medicines. Data ownership limits data accessibility as well as possibilities for linkage, and data collection is time-consuming, necessitating dedicated staff and better systems to facilitate the process. Cross-national collaboration is challenging but the engagement of multiple stakeholders is a key step to reach common goals through research. Conclusion: This study acts as a starting point for future research on patient-level databases for oncology across Europe. Future recommendations will require continued engagement in research, building on current initiatives and involving multiple stakeholders to establish guidelines and commitments for transparency and data sharing.

Dealing With Immunoglobulin Shortages: A Rationalization Plan From Evidence-Based and Data Collection.

Background: The demand and consumption of immunoglobulins (IgGs) are growing, and there are many difficulties in obtaining supplies. The aim of the study was to analyze the evolution of IgG consumption and cost over a decade, describe the measures implemented for clinical management in the context of regional public health system, and evaluate the initial impact of these measures. Methods: We performed a retrospective longitudinal study including patients of all public health systems in Catalonia. First, we analyzed data on consumption and cost of IgGs during a period between 1 January, 2010 and 31 December 2021. Second, we analyzed the impact of a set of regional measures in terms of annual consumption and cost of IgGs. Regional measures were based on rational evidence-based measures and computer registries. We compared the data of year before applying intervention measures (1 January and 31 December 2020) with data of year after applying clinical management interventions (1 January and 31 December 2021). In addition, detailed information on clinical indications of IgG use between 1 January and 31 December 2021 was collected. Results: Overall, in terms of population, the consumption of IgGs (g/1,000 inhabitants) increased from 40.4 in 2010 to 94.6 in 2021. The mean cost per patient increased from €10,930 in 2010 to €15,595 in 2021. After implementing the measures, the mean annual estimated consumption per patient in 2021 was statistically lower than the mean annual estimated consumption per patient in 2020 (mean difference -47 g, 95% CI -62.28 g, -31.72 g, p = 0.03). The mean annual estimated cost per patient in 2021 was also lower than the mean annual estimated cost per patient in 2020 (the mean difference was -€1,492, 95% CI -€2,132.12, -€851.88; p = 0.027). In 2021, according to evidence-based classification, 75.66% treatments were prescribed for a demonstrated therapeutic evidence-based indication, 12.17% for a developed therapeutic evidence-based indication, 4.66% for non-evidence-based therapeutic role indication, and 8.1% could not be classified because of lack of information. Conclusion: The annual consumption and cost of IgGs have grown steadily over the last decade in our regional public health system. After implementing a set of regional measures, the annual consumption of IgGs per patient and annual cost per patient decreased. However, the decrease has occurred in the context of the coronavirus disease 2019 (COVID-19) pandemic, which may have influenced their clinical use. Managing the use of IgGs through a rational plan with strategies including evidence-based and data collection may be useful in a shortage situation with growing demand. Registries play a key role in collection of systematic data to analyze, synthesize, and obtain valuable information for decision support. The action developed needs close monitoring in order to verify its effectiveness.

Characterization of the Pharmaceutical Risk-Sharing Arrangement Process in Catalonia.

Pharmaceutical risk-sharing arrangements have emerged as a reasonable tool to promote sustainable access to innovative medicines with uncertain clinical evidence and/or economic impact from the payer perspective. These funding mechanisms pose an alternative option to the traditional fixed-price methods and are intended to align the price of medication with the value delivered in treating patients, balancing clinical need with affordability in the face of increasing therapeutic innovation and ever-tight budgets. The Catalan Health Service (CatSalut) has set up a systematic, traceable, and transparent methodology for the design and implementation of risk-sharing arrangements and 15 of such access schemes have been successfully implemented until December 2019. Our experience has acknowledged the need for a robust study design, appropriate financial, technical, and administrative resources, and strong stakeholder commitment and communication as critical to the success of risk-sharing arrangements. While the experience in Catalonia has been positive and has served to highlight the potential of such schemes in tackling public health policy concerns, this exchange can often be undermined by the lack of transparency surrounding risk-sharing arrangements and the fact that the literature related to their methodology, implementation, and impact is scarce. Further studies should be conducted and shared to address this obstacle.

Registro de pacientes y tratamientos de medicamentos hospitalarios en Cataluña: 10 años de datos clínicos / Registry of patients and treatments of hospital medicines in Spain: 10 years of clinical data

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