Real World Evidence on the Effectiveness of Nusinersen within the National Program to Treat Spinal Muscular Atrophy in Poland.

BACKGROUND: Spinal muscular atrophy (SMA) is a debilitating neuromuscular disease resulting in children's mortality and disability. Nusinersen is available to all SMA patients in Poland since 2019. AIM: To compare mortality or disease progression to mechanical ventilation in two patient cohorts before and after the program's introduction. Additionally, to describe the patient population treated with nusinersen and costs incurred by the public payer. METHODS: We used the National Health Fund (NHF) database to identify patients born in either 2014 or 2019, who received at least two health services with an ICD10 G12 diagnosis. Outcomes were time to event: death or first mechanical ventilation. We identified all benefits received by nusinersen-treated patients, between 1 January 2019 and 31 May 2022. RESULTS: Children with SMA born in 2019 had significantly lower mortality in the first years of their lives than children born in 2014. Approximately 875 patients (all age groups) were treated with nusinersen in the analysis period. The cost of causal drugs in this period amounted to €51.4 million. The cost of healthcare benefits amounted to €14.9 million. CONCLUSIONS: The drug program to treat SMA improved patient care in Poland. The NHF database was a reliable source to monitor resource-intensive therapies' costs, demography, and selected patient outcomes.

Impact of COVID-19 Pandemic on the Use of Antidepressant and Antianxiety Pharmaceuticals as Well as Sick Leave in Poland.

The COVID-19 pandemic caused a major upheaval to the lives of people and placed a strain on societal mental health. The aim of this research is to estimate the impact of the pandemic on the mental condition of the Polish population measured through the consumption of relevant medication and medical leave of absence from the workplace. METHODS: We analyzed national-level data on the consumption of pharmaceuticals used in clinical practice in Poland in the treatment of depression and anxiety alongside medical absence in the workplace using the Interrupted Time Series model to estimate the significance of the pandemic. RESULTS: We found no significant change regarding the consumption of pharmaceuticals with the development of the pandemic. Conversely, medical leaves of absence for psychiatric reasons increased significantly with the onset of COVID-19. The influence was strongest in the diagnosis of anxiety or reaction to severe stress and weakest in recurrent depression. CONCLUSION: The pandemic had a significant influence on the ability to work for psychiatric patients in Poland but did not change pharmaceutical use. Physicians should consider the mental health of patients impacted by the anti-epidemic measures. Further study is needed to fully understand the long-term impact of the pandemic on mental health in Poland.

The Potential to Reduce Patient Co-Payment and the Public Payer Spending in Poland through an Optimised Implementation of the Generic Substitution: The Win-Win Scenario Suggested by the Real-World Big Data Analysis.

High medication costs are one of the major barriers to patient adherence. Medication affordability might be improved by generic substitution. The aim of this study was to assess the effectiveness of the implementation of generic substitution mechanisms in Poland. This was a retrospective analysis of nationwide real-world big data corresponding to dispensation of metformin preparations in 2019 in Poland. Relevant prescription and dispensation data were compared to assess the prevalence of generic substitution and its economic consequences. Among the 1,135,863 e-prescriptions analysed, a generic substitution was found in only 4.81% of the packs dispensed, based on e-prescriptions issued for metformin under its originator version and 2.73% under generic drugs. It is estimated that if these values were applied to the total Polish drug market, patients could lose the opportunity to lower their co-payment by 15.91% and the national payer to reduce its reimbursement expenditures by 8.31%. Our results point at the suboptimal implementation of generic substitution in Poland. Therefore, relevant actions need to be taken in order to maximise the benefits provided by this mechanism. It could not only lead to the win-win scenario in which both patients and the national payer are secured substantial savings, but it could also have a positive impact on patient adherence.

Residual symptoms and disease burden among patients with rheumatoid arthritis in remission or low disease activity: a systematic literature review.

OBJECTIVES: To identify, describe and summarize evidence on residual symptoms and disease burdens in rheumatoid arthritis (RA) patients qualified as being in remission or low disease activity (LDA). METHODS: A systematic literature review (SLR) was conducted according to Cochrane collaboration guidelines. The population of interest was adult patients with RA in remission or LDA. The reported outcomes of interest were any symptoms or burdens. RESULTS: Fifty-one publications were identified through an eDatabase search. Together with 17 articles found through other sources, 68 were included for full text review. The most commonly reported residual symptoms were pain (number of studies = 25), fatigue (n = 21) and morning stiffness (n = 5). Reported disease burdens included mental health (n = 15), sleep disturbances (n = 7) and work productivity (n = 5), impairment in quality of life (n = 21), and functional disability (n = 34). Substantial residual symptoms and disease burdens were found to be present in patients in remission or LDA. CONCLUSION: This is the first SLR to investigate residual symptoms and disease burdens in RA patients in remission or LDA. The results indicate that despite achieving conventional clinical targets, the disease continues to affect patients, suggesting the existence of unmet need under the current treatment paradigm.