Effectiveness of Montelukast on asthma control in infants: methodology of a French claims data study.

BACKGROUND: This pilot study, conducted on a 1/97th representative sample of French claims data, prepared a project to assess the effectiveness of Montelukast (MTL-4) as add-on therapy for asthma in infants (6-24 months) compared to inhaled corticosteroids (ICS), based on real-world data. Due to the very recent opening of French claims data for effectiveness research, and the complex structure of this data source, we first tested the feasibility of identifying infants with asthma and outcome criteria, and the ability to perform relevant comparisons. METHODS: We identified a cohort of infants with uncontrolled asthma and receiving ≥2 consecutive dispensations of any respiratory drug (R03 ATC classification) during a 6-month period. Uncontrolled asthma was identified either from exacerbations or from markers of acute loss of asthma control; date of occurrence of an event (exacerbation and/or acute loss of asthma control) was defined as index date. The study groups comprised infants receiving MTL-4 +/- ICS (MTL-4 group) or ICS without MTL-4 (ICS group) at index date. These two groups were matched on gender, age, quarter of index date, therapy before index date, past asthma-related hospitalization (ever), and were followed for 6 months. The outcome was the rate of infants with uncontrolled asthma, defined as above. RESULTS: This pilot cohort study included 1,149 infants with asthma (mean age 14.1 months, 64% boys). Of these, 51 and 768 were assigned to the MTL-4 and ICS groups, respectively. Uncontrolled asthma occurred in 78.8% and 78.4% of infants in these groups, respectively (oral corticosteroids were dispensed to 49% and 61%, respectively). Assessment of uncontrolled asthma, exposure to MTL-4 and ICS, and occurrence of outcomes were achieved. For the development of matching criteria, we defined a new marker of severity (therapeutic typologies). CONCLUSION: These data support the feasibility of the final project, to be conducted on claims data from the whole French population. We also showed that, with appropriate methodology and by using valid criteria, French claims data are an adequate resource for conducting comparative effectiveness studies in pediatric asthma. Finally, the algorithm used to identify infants with asthma could be applied to other studies using claims data.

Assessment of the comprehensiveness of paediatric national immunisation programmes in Europe: expert validation and future perspectives.

BACKGROUND: The breadth of protection of National Immunisation Programmes (NIPs) across Europe varies, however, this has not been assessed within published literature. Therefore, a framework was developed to assess the comprehensiveness of pediatric NIPs in Europe. This study aimed to validate and further develop criteria used to cluster countries into three tiers. RESEARCH DESIGN AND METHODS: Independent Europe-based experts (n = 23) in the field of pediatric vaccination were invited to participate in a double-blinded modified Delphi panel, with two online survey rounds and a virtual consensus meeting. Consensus was defined as ≥ 80% of experts rating their agreement/disagreement on a 9-point Likert scale. RESULTS: The number of preventable diseases covered by an NIP, simplification of the vaccination calendar, strengthened protection by increasing serotype, degree of funding and epidemiological factors were considered key concepts for consideration of the comprehensiveness of pediatric NIPs in Europe. Experts highlighted that the framework should be extended to include adolescent vaccines and populations up to 18 years of age. Consensus regarding further amendments to the framework was also reached. CONCLUSIONS: This Delphi panel validated a framework to assess the comprehensiveness of European NIPs. The framework can be used to facilitate discussions to help countries improve and expand the breadth of protection provided by their NIP.

Estimating the lifetime cost of vaccination in 23 European Countries: a modeling study.

BACKGROUND: All European countries have national immunization programs (NIPs) to protect gainst infectious diseases. We aimed to estimate the individual lifetime cost of vaccination in 23 European countries, assuming full compliance with NIP schedules. RESEARCH DESIGN AND METHODS: We used publicly available data to estimate the individual lifetime cost of vaccination with the vaccines that are currently recommended and funded in each country for healthy individuals and for individuals with underlying medical conditions. We included a scenario analysis for healthy individuals in which all currently recommended vaccines were universally funded, and compared the annual costs per person of vaccination to the annual per-capita costs of all-cause hospitalization and anti-infective medications. RESULTS: The individual lifetime cost of vaccination was €592-3,504 for healthy individuals (median: €1,663; 13-20 diseases), €744-9,081 for individuals with underlying conditions (median: €2,992; 13-21 diseases), and €1,225-4,832 (median: €2,565; 21-22 diseases) in the scenario analysis, with median values for vaccine acquisition of €1,203, €1,731, and €1,788, respectively. CONCLUSIONS: Our estimates show that the maximum potential cost of vaccination requires a relatively low level of investment assuming full compliance. These data could be useful for policymakers in future financial planning and evaluation of NIPs.

Addressing vaccine supply challenges in Europe: Expert industry perspective and recommendations.

Shortages of medicines are an increasing concern worldwide. In the European Union (EU), several initiatives have been launched by authorities to address this important public health issue. To contribute in finding solutions, Vaccines Europe (VE), representing 14 vaccine companies operating in Europe, conducted an analysis of the main root causes of vaccine shortages in Europe. Vaccines Europe has identified six main causes of vaccine shortages. Finding solutions will require a concerted effort and dialogue with the involvement of all key stakeholders. In this publication, Vaccines Europe is making a series of recommendations aiming at improving vaccine availability for Europe and beyond.

Vaccine market access pathways in the EU27 and the United Kingdom - analysis and recommendations for improvements.

BACKGROUND: Vaccine market access (VMA) pathways across the European Union (EU) and the United Kingdom (UK) are complex, lengthy, and heterogeneous, particularly when compared with pharmaceuticals. The knowledge base to inform recommendations for optimization of VMA is lacking. We therefore conducted a comprehensive evaluation of EU VMA pathways. METHODS: Research in two phases included: (1) mapping VMA pathways in each EU member state (including the UK) based on a literature review, expert interviews, and mathematical archetyping; and (2) interviews with vaccine experts to identify barriers, drivers, and recommendations for regional VMA alignments. RESULTS: Key steps in VMA across the EU include horizon scanning, early advice, National Immunization Technical Advisory Group (NITAG) recommendation for inclusion in national immunization programs, health technology assessment (HTA), final decision and procurement. We found significant complexity and heterogeneity, particularly for early advice, and in the roles, decision-making criteria, and transparency of NITAGs and HTA bodies. The most important drivers for rapid VMA included demonstration of disease burden and vaccine benefit (e.g., efficacy, safety, economic). Key barriers were budget limitations and complexity/clarity of VMA processes (e.g., need for national-regional consensus, clarity on process initiation, and clarity on the role of HTA). Recommendations for alignment at EU and member-state levels include information sharing, joint clinical assessment, initiatives to address funding and political barriers, and improved transparency by decision-making bodies. Early engagement with vaccine stakeholders was a key recommendation for manufacturers. CONCLUSIONS: There is significant potential for alignment, collaboration, and improvement of VMA across the EU. Roles, responsibilities, and transparency of key bodies can be clarified. The COVID-19 pandemic response should stimulate policies to improve access to all vaccines, including routine ones, and form the foundation upon which a consistent vaccine ecosystem can be created for the EU, one that is resilient, consistent between member states, and fit for purpose.

Recurrent Wheezing in Infants: A Population-Based Study.

Recurrent wheezing (RW) has a significant impact on infants, caregivers, and society, but morbidity and related medical resource utilization (MRU) have not been thoroughly explored. The burden of RW needs to be documented with population-based data. The objective was to assess the characteristics, medical management, and MRU of RW infants identified from national claims data. Infants aged from 6 to 24 months, receiving ≥2 dispensations of respiratory drugs within 3 months, and presenting a marker of poor control (index date), were selected. During the 6 months after index date, MRU was described in the cohort and among 3 subgroups with more severe RW, defined as ≥4 dispensations of respiratory drugs, ≥3 dispensations of oral corticosteroids (OCS), or ≥1 hospitalization for respiratory symptoms. A total of 115,489 infants had RW, corresponding to 8.2% of subjects in this age group. During follow-up, 68.7% of infants received inhaled corticosteroids, but only 1.8 U (unit) were dispensed over 6 months, suggesting discontinuous use. Control was mostly inadequate: 61.7% of subjects received OCS, 80.2% antibiotics, and 71.2% short-acting beta-agonists, and medical/paramedical visits were numerous, particularly for physiotherapy. Severe RW concerned 39.0% of the cohort; 32.8% and 11.7% of infants had repeated use of respiratory drugs and OCS, respectively, and 5.5% were hospitalized for respiratory symptoms. In this real-life nation-wide study, RW was common and infants had poor control and high MRU. Interventions are needed to support adequate use of controller therapy, and to improve medical care.