Access to biologics and Janus kinase inhibitors for treatment of rheumatic diseases in the biosimilars era in Poland: a nation-level study.

INTRODUCTION: By reducing treatment costs, biosimilars provide an opportunity to improve accessibility to highly effective drugs. OBJECTIVES: This study aimed to evaluate access to biologic disease-modifying antirheumatic drugs (bDMARDs) and Janus kinase inhibitors (JAKis) among patients with rheumatic musculoskeletal diseases within a 10 year timeframe in Poland. PATIENTS AND METHODS: We performed a retrospective analysis using a nationwide public payer database. RESULTS: By 2022, 11 102, 6602, and 4400 patients with rheumatoid arthritis (RA), psoriatic arthritis (PsA), and axial spondyloarthritis (axSpA) were treated with bDMARDs or JAKis. Peak drug utilization was observed for adalimumab, followed by etanercept and tocilizumab. Within the study timeframe, the estimated access to innovative drugs increased from 0.8%, 1.4%, and 0.8% to 3.2%, 8.7%, and 3.5% for RA, PsA, and axSpA patients, respectively. Affordable tumor necrosis factor inhibitors (TNFis) still predominate among innovative therapeutics, but their market share declined from 87% to 46%. The number of patients treated with other bDMARDs/JAKis almost doubled within the prespecified timeframe. Overall, the average annual treatment cost per patient decreased by 60%, from 7315 EUR to 2886 EUR. Despite recent safety warnings, JAKis appear to be increasingly utilized. Additional analyses regarding the COVID­19 pandemic showed impaired access to intravenous therapies, but not subcutaneous or oral formulations. CONCLUSIONS: In Poland, biosimilars­related savings improved availability of higher­priced innovative drugs rather than less costly TNFis. Data­driven resource allocation and dedicated policy solutions facilitating access to affordable biologics are recommended.

The perspective of Polish patients with rheumatoid arthritis - treatment expectations, patient-reported outcomes, and digital literacy (the SENSE study).

Introduction: A widely accepted treat-to-target strategy for rheumatoid arthritis (RA) requires the patient's perspective in making treatment decisions. However, data on treatment preferences and expectations of Polish patients with RA are scarce. The aim of the study was to determine the satisfaction with treatment and the nature of therapeutic preferences and expectations of Polish patients with moderate to severe RA. Material and methods: Fifty-two adult Polish patients with moderately to highly active RA were asked to complete patient-reported outcomes and patient-provided information questionnaires. Additionally, patient sociodemographic and clinical data and information on patient current and planned treatment strategies were collected. Results: The mean global assessment of patient satisfaction with treatment was 64.1 ±24.6, below the level of indicating satisfaction. Rheumatoid arthritis negatively impacted patients' lives, resulting in a 37.8% impairment of work efficiency and 45% impairment of total activity. Primary treatment expectations for patients were lasting relief of RA symptoms, reduced pain and swelling in joints, increased flexibility of joints, and general improvement of arthritis. The most acceptable potential side effect was weight gain and the least acceptable were increases in the risk of cardiovascular disease, infection, and malignancies. The rapid onset of the drug effect (up to 1 week) was a preference of 48.1% of patients. Access to internet health resources was important for 44.2% of patients, but the median total eHealth literacy score in the study population was 24.0 (interquartile range: 20.5-28.0, range 8-37), which means low digital health literacy (DHL). Conclusions: Understanding these treatment preferences and expectations of patients with RA is essential for clinical practitioners to facilitate shared treatment decision-making. Digital health literacy data suggest the need of further improvement.

Budget impact analysis and treatment availability with biosimilar TNF inhibitors in rheumatic diseases in Poland: real-world evidence using a nationwide database.

OBJECTIVES: Although several years have passed since biologic disease modifying antirheumatic drugs were introduced to the market, considerable disparities in access still remain. Tumour necrosis factor inhibitors (TNFi) have proven to be highly effective and safe for treating patients with rheumatic musculoskeletal diseases (RMDs). The emergence of biosimilars is promising for cost reduction and more equitable, widespread access. METHODS: A retrospective budget impact analysis based on final drug prices was conducted using 12 687 treatment courses for infliximab, etanercept and adalimumab. Estimated and real-life savings for public payer were calculated from an 8-year perspective of TNFi use. Data on the treatment cost and on the evolution in the number of patients treated was provided. RESULTS: From a public payer perspective, the estimated total savings amount to over €243 million for TNFi, with over €166 million attributed to treatment cost reduction in RMDs. Real-life savings were calculated as €133 million and €107 million, respectively. The rheumatology sector generated between 68% and 92% of total savings across models, depending on the adopted scenario. The overall decrease in mean annual cost of treatment ranged between 75% and 89% in the study frame. If all budget savings were spent on reimbursement of additional TNFi, a hypothetical total of almost 45 000 patients with RMDs could be treated in 2021. CONCLUSIONS: This is the first nation-level analysis that shows estimated and real-life direct cost-savings for TNFi biosimilars. Transparent criteria for reinvesting savings should be developed on both a local and an international levels.

Deep dive into achieving the therapeutic target: results from a prospective, 6­month, observational study nested in routine rheumatoid arthritis care.

INTRODUCTION: Achieving remission or lowdisease activity (LDA) is an integral principle of treat­to­target (T2T) strategy in rheumatoid arthritis (RA). Prior studies have reported that achieving T2T therapeutic goals may be realistic only for a fraction of patients. Prospective, real­world data on achieving target disease control in ambulatory care populations are limited for Central and Eastern European countries. OBJECTIVES: The aim of the study was to analyze the efficacy of treatment and determine simple predictors of achieving T2T therapy goals in daily RA practice. PATIENTS AND METHODS: This multicenter, 6­month study evaluated therapy outcomes and clinical characteristics of 791 consecutive RA outpatients, meeting the preset criteria of inadequate disease control. RESULTS: Only 9% of RA patients achieved remission or LAD after 3 months and 35% after 6 months. Achieving treatment targets after 6 months was associated with lower rates of pain, disability, presenteeism and absenteeism, which reflected improved quality of life. Provider views on adherence appeared discordant with patient claims, and did not predict target achievement. Never smoking, lower body mass index, and lower prednisone dose (<7.5 mg daily) were independently associated with a higher likelihood of achieving T2T therapeutic goals after 6 months. CONCLUSIONS: A combination of clinical characteristics and provider treatment decisions shapes the "profile" of a patient failing to achieve T2T goals. Low­dose steroid equivalent, never smoking, and lower body mass index appear as individual characteristics independently associated with achieving LDA / remission at 3 and 6 months.

Real-World Data from a Multi-Center Study: Insights to Psoriatic Arthritis Care.

INTRODUCTION: Real-world data indicate disparities in biologic access across Europe. OBJECTIVES: To describe the national structure of PsA care in Poland, with a particular focus on the population of inadequate responders (IRs) and difficulties associated with biologic therapy access. METHODS: A pool of rheumatologic and dermatologic care centers was created based on National Health Fund contract lists (n = 841), from which 29 rheumatologic and 10 dermatologic centers were sampled randomly and successfully met the inclusion criterium. Additionally, 33 tertiary care centers were recruited. For successful center recruitment, one provider had to recruit at least one patient that met the criteria for one of the four pre-defined clinical subgroups, in which all patients had to have active PsA and IR status to at least 2 conventional synthetic disease-modifying drugs (csDMARDs). Self-assessment questionnaires were distributed among physicians and their patients. RESULTS: Barriers to biologic DMARD (bDMARD) treatment are complex and include stringency of reimbursement criteria, health care system, logistic/organizational, and personal choice factors. For patients who are currently bDMARD users, the median waiting time from the visit, at which the reimbursement procedure was initiated, to the first day of bDMARD admission was 9 weeks (range 2-212; 32% < 4 weeks, 29% 5-12 weeks, 26% 13-28 weeks, 13% with >28 weeks delay). Out of all inadequate responder groups, bDMARD users are the only group with "good" therapeutic situation and satisfaction with therapy. Patient satisfaction with therapy is not always concordant with physician assessment of therapeutic status. CONCLUSIONS: Despite the fact that over a decade has passed since the introduction of biologic agents, in medium welfare countries such as Poland, considerable healthcare system barriers to biologic access are present. Out of different IR populations, patient satisfaction with treatment is often discordant with physician assessment of disease status.

[Bisphosphonates in the treatment of multiple myeloma]. / Bifosfoniany w leczeniu szpiczaka mnogiego.

Bisphosphonates are the most potent class of antiresorpitve drugs used in the treatment of bone diseases with enhanced resorption, like malignant osteolysis, osteoporosis and Paget's disease. Multiple myeloma is commonly associated with skeletal morbidity, including osteolysis, bone fractures, pain and hypercalcemia. These clinical complications result from increased osteoclast number and function which is due to release of osteoclast-stimulating factors by myeloma cells and the tumoral environment. By inhibition of osteoclast activity and inducing cell apoptosis, bisphosphonates can prevent development of bone destruction in myeloma patients. This article reviews efficacy of bisphosphonates in reducing skeletal events in patients with multiple myeloma and several in vitro studies which have shown that aminobisphosphonates may act as antimyeloma agents.