Higher Drug Costs Research Articles

Off-label use of intravenous immunoglobulin g in a highly complex pediatric hospital in Argentina. A prospective observational study

The World Health Organization recommends prioritizing safe and effective drugs proven by clinical or epidemiological studies. However, in population groups with little research, a drug can be used for an indication or pharmaceutical form different from that approved by the regulatory agency (off-label), extrapolating data from studies in adults and exposing pediatric patients. to develop an Adverse Drug Reaction (ADR) due to safety considerations that have not been systematically studied. Intravenous immunoglobulin (IVIg), a high-cost drug, is used with scant evidence in some low-prevalence pathologies. This paper describes and analyzes the off-label use of IVIg at the J. P. Garrahan Pediatric Hospital. Observational, descriptive, prospective study on off-label indications of IVIg. The sampling technique was non-probabilistic and for convenience during 7 months. 305 IVIg infusions were studied, corresponding to 111 patients. The indication classification showed that 22% (n=67) of the infusions were off-label. In neurology there was a higher percentage of off-label indications (46%) and within them 45% corresponded to the use in neurological disorders. 81% of the doses indicated off-label were in the range 0.8-1g/kg. The off-label infusions presented 61.5% (n=8) of the ADRs. Those from the Neurology service represented 87.5%; 75% being from the "Neurological disorders" group. In some cases, IVIg was indicated in an off-label manner, finding a statistically significant relationship with the appearance of ADR. This finding motivates the proposition of new hypotheses to carry out more studies.

P031 Experience from a UK dermatology regional virtual biologics and high-cost drugs multidisciplinary team

P031 Experience from a UK dermatology regional virtual biologics and high-cost drugs multidisciplinary team

Innovation, affordability, access: Alzheimer disease drugs and the Inflation Reduction Act.

This analysis examines the implications of new Alzheimer disease drugs in the era of the Inflation Reduction Act (IRA). It focuses on balancing innovation in Alzheimer disease treatment with affordability and access, assessing the impact on Medicare's budget, patient cost, and health care system readiness. A comprehensive review was conducted, synthesizing information from recent FDA drug approvals, drug pricing models, Medicare coverage policies, and the updated regulations under the IRA. This analysis reflects on the broader clinical and economic consequences of introducing new Alzheimer disease treatments. The study employs a qualitative review of existing literature, policy documents, and economic data. It explores the implications of Alzheimer disease drugs on health care policy, analyzing the economic and clinical impacts within the current health care landscape in the US. The study highlights the economic challenges posed by the high costs of new Alzheimer disease drugs, contrasting with their moderate clinical benefits and potential risks. It discusses the limitations of the IRA in regulating drug prices and the resulting implications for Medicare's budget. Additionally, it examines disparities in health care access and system preparedness for these newtreatments. The study findings underscore the need for a comprehensive approach to ensure fair pricing and equitable access to Alzheimer disease treatments. It suggests the application of frameworks such as the ISPOR Value Flower, focusing on diversity, equity, and comprehensive economic evaluations, to navigate the evolving landscape of Alzheimer disease treatment in the context of the IRA.

Sulfonated Bovine Serum Albumin Hydrogel for Anti-inflammatory Treatment of Rheumatoid Arthritis via Restoring Macrophage Phenotype

Rheumatoid arthritis (RA) is an autoimmune disease with high rates of late-stage disability. Unfortunately, effective treatments for RA are limited by side-effects and high cost of clinical drugs. Bovine serum albumin (BSA) with high biocompatibility and low cost is widely used as the carrier for drug delivery. Here, sulfonated BSA (sBSA) was first time synthesized and its hydrogel system with HA matrix (sBSA/HA) was prepared for RA treatment. In RAW 264.7 cells, sBSA, but not bare BSA, significantly reduced the generation of reactive oxygen species and pro-inflammatory cytokines induced by LPS to the normal levels or even below. The scavenger receptor on macrophages was identified responsible for the enhanced uptake of sulfonated BSA. In vivo, 3% sBSA/HA showed the abilities of anti-inflammation, bone protection and restoring clinical scores and claw thickness of healthy rats in collagen-induced arthritis (CIA). Compared with the positive control (methotrexate), sBSA/HA had significant advantages in terms of speed of action, treatment effectiveness, and duration. Notably, both sBSA and sBSA/HA promoted M2 polarization (P＜0.001) by reducing the M1(CD86)/M2(CD206) phenotype ratio, suggesting the possible immune-modulation mechanism of sBSA in treating RA. Conclusively, sBSA was verified as a promising candidate for RA treatment.

Modulation of Gut Microbial Biomarkers and Metabolites in Cancer Management by Tea Compounds.

Cancers are causing millions of deaths and leaving a huge clinical and economic burden. High costs of cancer drugs are limiting their access to the growing number of cancer cases. The development of more affordable alternative therapy could reach more patients. As gut microbiota plays a significant role in the development and treatment of cancer, microbiome-targeted therapy has gained more attention in recent years. Dietary and natural compounds can modulate gut microbiota composition while providing broader and more accessible access to medicine. Tea compounds have been shown to have anti-cancer properties as well as modulate the gut microbiota and their related metabolites. However, there is no comprehensive review that focuses on the gut modulatory effects of tea compounds and their impact on reshaping the metabolic profiles, particularly in cancer models. In this review, the effects of different tea compounds on gut microbiota in cancer settings are discussed. Furthermore, the relationship between these modulated bacteria and their related metabolites, along with the mechanisms of how these changes led to cancer intervention are summarized.

Non-receipt of targeted and immunotherapies in diverse patients with metastatic cancers.

e13563 Background: The rapid evolution of molecularly targeted kinase inhibitors (TKIs) and immune checkpoint inhibitors (ICIs) in the treatment of metastatic cancer, coupled with high drug costs, raises potential for unwarranted variation in patient management. We evaluated correlates of non-receipt of TKIs and ICIs in a diverse, population-based sample of patients with metastatic non-small cell lung cancer (NSCLC), kidney and bladder (GU) cancer, and melanoma. Methods: We identified 2240 patients diagnosed in 2019 with metastatic NSCLC, GU cancer, or melanoma and reported to the Georgia or Los Angeles SEER registries. Using SEER registry data, we linked all patients to a managing oncology physician. We collected data from the managing physician regarding receipt of TKIs and ICIs. We used bivariate analyses and multivariable logistic regression models to assess associations between non-receipt of TKIs and ICIs and patient-level factors. Results: We present results for a preliminary sample (N=894). About half (55%) of the patients had NSCLC, 31% had GU cancer, and 14% had melanoma. The mean age was 62 (SD 12); 59% were male; 65% were White, 16% were Black, 11% were Hispanic, and 7% were Asian; 52% had Medicare, 29% had private insurance, 9% had Medicaid, and 4% had no insurance; 89% resided in urban areas, 11% resided in rural areas. 22% lived in a census tract area with ≥20% of the population living below the poverty level. The Table shows that in bivariate analyses, non-receipt of TKIs was associated with NSCLC, older age, male sex, non-white race, and non-private or no insurance (all p<0.05). After adjustment, non-receipt of TKIs was associated with NSCLC, older age, male sex, and race. Asian and Hispanic patients had lower rates of non-receipt, and Black patients had higher rates. In bivariate analyses, non-receipt of ICIs was associated with NSCLC and GU cancer (p=0.004); this association remained significant after adjustment. Conclusions: Non-receipt of TKIs was associated with important non-clinical factors including race and sex. Non-receipt of ICIs was associated only with cancer type. These findings suggest that policy-relevant factors such as out-of-pocket costs for oral cancer therapies and differential access to molecular sequencing may contribute to unwarranted variation in receipt of TKIs. [Table: see text]

Adherence, health care utilization, and costs between long-acting injectable and oral antipsychotic medications in South Carolina Medicaid beneficiaries with schizophrenia.

Schizophrenia and schizoaffective disorder require long-term antipsychotic treatment with antipsychotic medications, but poor medication adherence can lead to increased health care utilization and costs. Long-acting injectable antipsychotics (LAIs) offer potential therapeutic advantages in that they require less frequent dosing and improved medication adherence. South Carolina has the highest adoption of LAIs among US states, making it an ideal population for comparing the effectiveness of LAIs vs oral antipsychotics (OAPs) in treating schizophrenia or schizoaffective disorder. To evaluate the effect of LAIs compared with OAPs on medication adherence, health care resource utilization, and costs among South Carolina Medicaid beneficiaries with schizophrenia or schizoaffective disorder. South Carolina Medicaid beneficiaries with at least 1 claim for an LAI or OAP between January 1, 2015, and December 31, 2018, aged 18 to 65, with at least 2 claims with diagnoses of schizophrenia or schizoaffective disorder were included. Propensity scores (PSs) were calculated using logistic regression adjusting for confounders and predictors of the outcome. We estimated the "average treatment effect on the treated" by employing PS-weighted t-tests and chi-square tests. A total of 3,531 patients met the inclusion criteria, with 1,537 (44.5%) treated with LAIs and 1,994 (56.5%) treated with OAPs. In PS-weighted analyses, the LAI cohort had a greater proportion of days covered than the OAP cohort with a 365-day fixed denominator (69% vs 64%; P < 0.0001), higher medication possession ratio with a variable denominator while on therapy (85% vs 80%; P < 0.0001), and higher persistence (82% vs 64%; P < 0.0001). The average number of inpatient visits and emergency department visits did not significantly differ between cohorts (0.28 hospitalizations, P = 0.90; 3.68 vs 2.96 emergency department visits, P = 0.19). The number of outpatient visits, including visits for medication administration, were greater in the LAI cohort (23.1 [SD 24.2]) vs OAP (16.9 [SD 21.2]; P < 0.0001); however, including the costs for medication administration visits, outpatient costs (per member) were approximately $2,500 lower in the LAI cohort (P < 0.0001). The number of pharmacy visits was greater in the OAP cohort (LAI 21.0 [SD 17.0] vs OAP 23.0 [SD 15.0]; P = 0.006). All-cause total costs were greater in the LAI cohort ($26,025 [SD $29,909]) vs the OAP cohort ($17,291 [SD $25,261]; P < 0.0001) and were driven by the difference in pharmaceutical costs (LAI $15,273 [SD $16,183] vs OAP $4,696 [SD $10,371]; P < 0.0001). Among South Carolina Medicaid beneficiaries, treatment with LAIs for schizophrenia or schizoaffective disorder was associated with greater medication adherence rates. Patients using LAIs had higher drug costs and total costs, but lower outpatient and total nondrug costs compared with those using OAPs.

Financial impact of fixed-dose versus weight-based dosing approaches for pembrolizumab and nivolumab in the Oncology Care Model for The US Oncology Network (The Network).

e13573 Background: PD-1/PD-L1 antagonists, pembrolizumab and nivolumab, are being used in an increasing number of cancer types. The FDA initially approved both agents based on a weight-based dosing (WBD) approach, but subsequently included a therapeutically equivalent fixed-dose (FD) approach for most indications. The Oncology Care Model (OCM) was a value-based care (VBC) program from Medicare that required total cost of care (TCOC) accountability from participating practices. We evaluated the impact of using the FD vs WBD on TCOC in the OCM for both pembrolizumab and nivolumab. Methods: Using Medicare claims data for 14 OCM-participating practices in The Network from July 2016 to June 2022, and corresponding weight data from the Electronic Health Record (EHR), we evaluated the distribution of patient weights, use of FD vs WBD, and the financial impact of the use of FD vs WBD vs weight-based dosing capped at the fixed dose (WBDc). Results: Summary statistics are shown (Table). In most cases, the FD was higher than the WBD for both nivolumab and pembrolizumab. FD led to an increase in drug cost of 8.4% for nivolumab and 27.2% for pembrolizumab, corresponding to a 11.3% increase in TCOC for the episodes where FD nivolumab and pembrolizumab were used. Conclusions: Fixed-dosing approaches for commonly used high-cost anti-neoplastic drugs can drive increases in TCOC. The use of WBD could lead to a decrease in drug cost and 11.3% savings of TCOC within OCM-like models, likely without impacting clinical outcomes. These findings encourage practices in risk-bearing models to reflect on the widespread adoption of FD. The withdrawal of varying sizes of vials can limit the use of WBD. Pharmaceutical manufacturers should assist with WBD by providing multi-use vials and varying sizes. The feasibility of using the WBD for pembrolizumab with an every 6-week regimen remains to be explored. [Table: see text]

Antioxidant, antidiabetic, and anti-inflammatory activities of flavonoid-rich fractions of Solanum anguivi Lam. fruit: In vitro and ex vivo studies

Diabetes mellitus is a major, rapidly growing endocrine disorder in most countries. The high cost and side effects of conventional drugs for the management of this disease have shifted attention to medicinal plants. Solanum anguivi (S. anguivi) fruits has been reported to be a very good and rich source of polyphenols such as flavonoids, that can be exploited. Flavonoids are plant secondary metabolites widely found in vegetables, fruits and seeds and are known to be of medicinal significance in different range of diseases like diabetes. This study involved in vitro and ex vivo assays on the antioxidant, anti-inflammatory, and antidiabetic properties of flavonoid-rich fractions of S. anguivi fruits. Healthy male Wistar rats (n = 5) weighing 150–180 g were used for ex vivo antioxidant and antidiabetic studies, their liver was exercised for the experiment. The percentage yields of the three flavonoid-rich fractions (Fr. A, B, and C) of S. anguivi fruits obtained from the column chromatographic technique were 15.53 ± 0.75, 11.53 ± 0.80, and 10.17 ± 0.49 mg/g quercetin equivalents. The three fractions (A, B, and C) of S. anguivi fruits significantly scavenged both 2,2-diphenyl-1-picrylhydrazyl (DPPH) with fraction A having the lowest IC50 value (26.14 ± 1.06 μg/ml) compared with fraction B (37.78 ± 5.12 μg/ml) and fraction C (38.24 ± 2.40 μg/ml) when compared with ascorbic acid with the least IC50 value (15.27 ± 0.34 μg/ml). While fraction A (19.61 ± 1.19 μg/ml) scavenged nitric oxide (NO) radicals better than fraction B (22.97 ± 0.55 μg/ml) and fraction C (49.95 ± 6.18 μg/ml). Although ascorbic acid had better scavenging ability than the three fractions (17.23 ± 0.16 μg/ml). The flavonoid-rich fraction A shows better result in inhibiting α-glucosidase with IC50 value of 16.24 μg/ml compared to fraction B (128.04 μg/ml) and fraction C (143.16 μg/ml). For α-amylase, flavonoid-rich fraction A had an IC50 of 31.50 μg/ml compared to B (84.32 μg/ml) and C (145.40 μg/ml). The various controls also showed promising results with acarbose having IC50 of 3.93 μg/mL and 15.66 μg/mL respectively for α-glucosidase and α-amylase. Our findings also showed that FeSO4-induced tissue damage decreased the levels of GSH, SOD, and CAT activities while increasing the levels of MDA. In contrast, following treatment with the three flavonoid fractions of S. anguivi fruits helped to restore these parameters to near-normal levels, by significantly increasing the potential of GSH, SOD, CAT and reducing the levels of MDA which signifies that flavonoid-rich fractions of S. anguivi have great potential to address complications arising from oxidative stress. In addition, the three flavonoid-rich fractions A, B, and C of S. anguivi fruits exhibited ex vivo anti-inflammatory properties via reduced nitric oxide levels in iron-induced oxidative damage. Data obtained from this study shows that the flavonoid-rich fraction of S. anguivi possess anti-diabetic property via inhibition of α-glucosidase and α-amylase and antioxidant property via free radical scavenging. Also, comparing all the fractions, flavonoid-rich fraction A appears to be more potent compared to the fractions B and C. Further research will be needed in isolating and as well applying the fractions in real life situations in the management of diabetes.

#2861 Real-life efficacy of tolvaptan for polycystic kidney disease (PKD) and association with patient adherence in London North-West specialist clinic

Abstract Background and Aims Tolvaptan is approved by NICE in the treatment of PKD afterrandomised controlled trials demonstrated a reduction in decline in eGFR. Aquaresis occurs as an effect, and this may limit tolerability as the second dose in a day causes nocturia and disturbed sleep. Thirty-three patients in our specialist PKD clinic were treated based on criteria of bipolar renal length &amp;gt;=15cm and decline in eGFR by &amp;gt; −5 ml/min/yr. We evaluated their real-life outcome regarding measures of adherence including an adapted Basel Assessment of Adherence to Immunosuppressive Medication Scale © (by permissions.nursing.unibas.ch/) originally validated for immunosuppression medications in transplant patients. A further 24 patients had finished treatment at our centre and were excluded as BAASIS could not be applied in hindsight. However, they illustrated a notably high level of intolerance leading to cessation in 16/24 (9 with nocturia, 4 transaminitis, 1 angioedema, 1 rash, 1 fatigue) emphasizing the importance of this work on adherence and response. Method BAASIS addresses recall of treatment adherence in the preceding 4 weeks, all 33 patients currently treated as of 16/1/24 were screened on telephone by two independent auditors with a standard non-judgemental introductory script designed to result in candid engagement. Investigators were blinded to renal outcomes. A decline in eGFR of less than 5 ml/min/yr was defined as treatment response. Results Of the 33 patients included, 14 males and 19 females, median age 49yrs (range 32-72), were on treatment for a mean of 2.7yr (SD 2.2, range 0.3-7.5). Mean eGFR (CKD-EPI) was 58 ml/min (SD 19, range 28-110) at start and 48 ml/mi (SD 23, range 17-102) at end of treatment. Median total daily dose was 120 mg/d (range 60-120). Attendance at clinic for monitoring was 100%. 18/33 patients had responded to treatment, with delta eGFR –0.1 ml/min/year (SD 3.4) versus non-responders’' delta eGFR –13 ml/min/year (SD 8.7) There was a total of 35 instances of non-implementation in 23/33 patients with missed (20 instances), delayed (13 instances) or self-alteration in doses (2 instances). Only 10/33 patient made no failure to implement Tolvaptan at any time. There were 9 patients with non-persistence of Tolvaptan treatment. Associative analysis of renal outcome with adherence was made for all 31 patients who had more than 6 months treatment, 2 patients being excluded because treatment had been too short-term. 25/31 had non-adherence defined by non-implementation or non-persistence or both, but this did not statistically associate with treatment failure ChiSq 1.951 p = 0.16. The element of non-implementation alone was however associated with treatment failure ChiSq 4.949 p = 0.03, this term includes patient behaviour of not taking and mistiming drug taking, and patient-initiated dose reduction and drug holidays. Conclusion Just over half (55%) of currently treated patients had made a clinical response to Tolvaptan. 23/33 patients had non-implementation within the 4 weeks preceding the validated adherence survey, and this was significantly associated with treatment failure. This study is limited to a small single-centre patient cohort but could easily be applied more widely. This study informs shared-decision making and we should encourage our patients to take this high-cost drug in the right amount and at the right times, and notably not to miss doses, thereby to achieve higher clinical response rates.

Role of reimbursement and Physicians' awareness in the survival of sorafenib-eligible advanced hepatocellular carcinoma patients.

In 2008, sorafenib became the first approved systemic therapeutic agent for advanced HCC. Although its pharmacological efficacy has been established, reimbursement for such a new, high-cost drug, as well as physicians' awareness and prescription practice, likewise contribute to its clinical effectiveness. We therefore conducted a retrospective study using 38 sorafenib-eligible, advanced HCC patients when sorafenib was approved but not yet reimbursed as a control and 216 patients during the reimbursed era. Study group showed longer survival at 8.2 months versus the control's 4.9 months (p = 0.0063 hazard ratio: 0.612 [0.431 ~ 0.868], p = 0.0059). Among the 42 (19.4%) patients who survived more than 2 years, 50% had tumor rupture, and all 32 patients with portal vein tumor thrombus and/or extrahepatic metastasis received sorafenib (p = 0.003). Furthermore, during their first 2 years of HCC management, sorafenib had been given in 29.1% of the treatment courses among survivors between 2 and 5 years while it was prescribed in 55.8% among the more than 5 years survivor group (p < 0.001). In conclusion, survival of sorafenib-eligible HCC patients significantly improved after reimbursement. Patients who underwent longer sorafenib treatment had a survival advantage, except for those with tumor rupture. Reimbursement and awareness of prescriptions for a newly introduced medication therefore improve clinical effectiveness.

Childhood Wilm’s tumour in Aba, South East Nigeria

Background: Wilm’s tumour is still a problem to paediatric oncologist in developing countries due to poverty, late presentation, high cost of chemotherapeutic drugs, ignorance and religious beliefs. Objective: To review the clinical presentation, management and outcome of Wilm’s tumour in Aba, South East Nigeria. Methods: The Demography, clinical presentation, investigation results, operative findings and outcome of patients less than 15 years old managed for Wilm’s tumour at the paediatric surgery unit of the Abia State University Aba, South East Nigeria from 2010 to 2020 were retrospectively reviewed. Results: There were 30 children (M: F 2 :1) with histological confirmed nephroblastoma over the 10 year period. Their median age was 5. Age range 4-15years. Palpable abdominal mass was the main presentation in all the patients. Treatment consisted of nephro-ureterectomy followed by adjuvant chemotherapy with Vincristine, Cyclophosphamide and Actinomycin D. Adriamycin was added for metastatic disease and anaplasia. Fifteen of the patients had stage III disease, 10 had stage II disease and 5 had stage IV disease. Stage I disease was not encountered. Five patients had inoperable tumour requiring preoperative chemotherapy. Five patients died from the complication of chemotherapy treatment. Seven relapsed with poor outcome, with a mean follow up of 20 months, 20months survival rate is 40%. Conclusion: There is high rate of morbidity and mortality from nephroblastoma in our environment due to late presentation, poverty, ignorance and poor compliance to treatment. The outcome will be improved through health enlightenment and healthcare funding.

P012 Health inequalities in the diagnosis and treatment of rheumatoid arthritis

Abstract Background/Aims Patients with chronic diseases, such as rheumatoid arthritis (RA), from socially deprived backgrounds are known to have more severe disease, comorbidity and higher mortality rates. This study used National Early Inflammatory Arthritis Audit (NEIAA) data submitted by Lancashire and South Cumbria Foundation Trust to ascertain if social deprivation influences time to diagnosis and treatment escalation onto a high-cost drug (HCD). Methods Patients diagnosed with an inflammatory arthritis were recruited to NEIAA over a five-year period (May 2018 to March 2023). Information on demographics, patient postcodes, duration of symptoms prior to referral, GP referral time and rheumatology waiting time were analysed. Index of Multiple Deprivation (IMD) was derived from patient postcodes and patients divided into three groups based on IMD deciles. Patients with deciles 1-3 were grouped as most deprived, deciles 4-6 as middle and deciles 7-10 as least deprived. A case note review of a subgroup of patients who had been on treatment for 2-5 years was undertaken to investigate whether IMD decile was associated with treatment escalation onto a HCD. Data were analysed using descriptive and inferential statistics on Stata. Results were adjusted for confounders including age and gender. Results A total of 265 patients with RA were included, mean age 60 years, 63% female and 10% from ethnic minority groups. There were 31%, 25% and 44% in the most, middle and least deprived groups respectively. There was a higher proportion of patients from minority ethnic backgrounds in the most deprived group (most 21% vs least 3%). Symptom duration at time of presentation to the GP was greater than 3 months in 57% vs 48% of patients in the most and least deprived groups, respectively. Median was used to compare the GP referral times and rheumatology waiting times as there were outliers in the dataset. The most deprived group had longer GP referral times (most 16 days vs least 7 days); however, median rheumatology waiting time was similar across the three groups (most, middle and least deprived 27, 24 and 25 days respectively). There was no difference in escalation onto a HCD across the three groups. Conclusion Patients from the most deprived areas presented later and took longer to be referred. However, time to being seen in the rheumatology clinic and escalation onto a HCD was not found to be associated with social deprivation. Exploring this further on a national level will help understand if social deprivation is influencing care elsewhere. This work supports the need to focus more resource on raising public awareness in more deprived areas. Disclosure S. Jeyaratnam: None. S. Fish: None. S. Horton: None. A. Madan: None. C. Rao: None. E. MacPhie: None.

P184 Geographical variations in delivery of intravenous treatments for ANCA-associated vasculitis

Abstract Background/Aims Timely access to intravenous immunosuppressant therapy is essential to effectively manage systemic vasculitis; however, significant variations in care and outcomes exists across centres. We systematically evaluated access to treatment for ANCA-associated vasculitis across the UK and Ireland. Methods This multi-method study included: a survey of 87 UK and Ireland Vasculitis Society sites and 11 Scottish Health Boards between Nov 2020 and June 2021; interviews with staff and patients with systemic vasculitis. Survey data was analysed descriptively. Interviews were audio or video recorded and analysed thematically. Results 59 responses from 51 Trusts/Health Boards across Scotland (n = 11), England (n = 33), Wales (n = 2), and Ireland (n = 5) included equal numbers of rheumatology and nephrology vasculitis services across diverse healthcare settings. 67 healthcare professionals across six case studies in Scotland (n = 3) and England (n = 3) and 32 patients with systemic vasculitis were interviewed. Services faced significant challenges in prescribing and delivering high-cost drugs to manage systemic vasculitis: balancing accountability versus bureaucracy, and tensions between different specialties looking after vasculitis patients when implementing access to high-cost drugs via multi-disciplinary networks. There was significant variation in how services delivered intravenous treatments: 10%(5/51) used inpatient beds; 35% (18/51) accessed another specialty unit or shared a daycase facility; 65% (33/51) used their own specialty daycase unit. Average wait time for urgent IV treatment was 2.67 days (range 1-10 days). 18% of services had waiting times &amp;gt;7 days, and of those 67% accessed intravenous treatments via another daycase facility. Specific challenges in accessing daycase facilities included availability of slots and staff, particularly in shared units, due to competition with other services and limited awareness of the importance of timely treatment for systemic vasculitis. This could impact on timing and treatment decisions e.g., prolonged steroid prescriptions. Staff training, competencies and governance to deliver cytotoxic drugs was problematic, particularly for smaller services, and 70% of services said national training modules would be helpful. Oncology units could provide workarounds but daycase nurses with vasculitis knowledge were a valuable source of advice and support for patients attending for treatments, identifying medical issues and arranging assessment if required. Conclusion There is significant variation in access to intravenous treatment for systemic vasculitis. Healthcare organisations should ensure timely access to urgent intravenous treatment for systemic vasculitis within 7 days and appropriately trained staff. National training modules for non-cancer chemotherapy could facilitate equitable access to training. Where treatment is delivered via other units, greater awareness of systemic vasculitis would help provision of information and support for patients. Disclosure W. James: None. A. Nicoll: None. L. Locock: None. L. Harper: None. M. Little: None. N. Basu: None. R.J. Hollick: None.

P018 A review of the use of anakinra in hyperinflammatory conditions at a large central London teaching hospital

Abstract Background/Aims The interleukin-1 receptor antagonist, anakinra, is increasingly used to treat inflammatory disorders. We recognized a need to provide patients with accurate information regarding the trajectory of their treatment. In this service evaluation project, we collected data on patients prescribed anakinra at a London teaching hospital over a 4-year period. Methods Patients included were started on anakinra to manage an inflammatory disorder between April 2019 and Feb 2023. Patients were identified by searching a high-cost drugs system. Data on indication for the drug, start and stop date(s) of treatment and any adverse effects were then collected using electronic patient records. Results 74 patients met inclusion criteria, 43 females and 31 males. Median age 38.5 years. 56 patients were treated for haemophagocytic lymphohisticytosis (HLH), 5 for Juvenile Idiopathic Arthritis (JIA) and 13 for Adult Onset Still’s Disease (AOSD). Median time on anakinra was 140.5 days (IQR 563.5), 1487.5 days (IQR 861) and 673 days (IQR 1017) for HLH, JIA and AOSD respectively. Of those treated for HLH, drivers and median duration of anakinra treatment for each respective driver were: infection (seven patients, 31 days), rheumatological (six patients, 785 days), haematological (26 patients, 36.5 days), iatrogenic (1 patient, 89 days), primary HLH (2 patients, 89 days), underlying rheumatological condition with infective/drug trigger (3 patients, 345 days), unknown (11 patients, 191 days). 9/56 patients with HLH remained on treatment at data collection - 4 with an unknown driver, two with AOSD, one with underlying Sjogren’s and a drug trigger, one with a haematological driver and one with primary HLH. 12/56 patients with HLH had died. Of those who died, eight had a haematological malignancy driving their condition, three had an infective driver and one an unknown driver. Five patients in our study had HLH driven by AOSD. Of these, 2/5 successfully stopped anakinra during the study period. Five patients (8.9%) with HLH required multiple courses of treatment with anakinra; three had lymphoma associated HLH, one primary HLH and one idiopathic HLH. Three patients were unable to continue with therapy due to side effects: neutropenia(1), rash (1), problems self-injecting (1). Conclusion From this data set we can begin to formulate answers to many commonly encountered questions regarding anakinra therapy. The length of time someone can expect to receive treatment with anakinra is significantly impacted by the indication for prescribing. Those with a haematological malignancy driving their HLH receive anakinra for the shortest time of all groups, usually while awaiting definitive treatment for their cancer, or before transition to palliative management. Some patients with AOSD driven HLH can safely stop treatment. Some patients may require multiple courses of treatment with anakinra for disease relapse. Adverse effects requiring treatment cessation are low in all groups. Disclosure I. Munro: None. R. Allen: None. A. Jones: None. J. Manson: None.

Comprehensive pharmacoepidemiological and clinical-economic analysis of antibacterial drugs consumed during the pandemic at the hospital level in Aktobe, Kazakhstan

&lt;b&gt;Aim: &lt;/b&gt;The study aimed to analyze the frequency and structure of antibacterial drug consumption during the COVID-19 pandemic at a dispensary hospital in Aktobe, Kazakhstan. It sought to identify the most frequently used and most costly antibacterial drugs, assessing their economic impact and usage patterns.&lt;br /&gt; &lt;b&gt;Methods:&lt;/b&gt; This descriptive, retrospective clinical, economic, and pharmacoepidemiological study was conducted using data from the dispensary hospital's pharmacy and patient records between March 13, 2020, and December 31, 2020. The ABC/VEN analysis and ATC/DDD methodology were applied to evaluate the consumption of antibacterial drugs. Antibiotics were classified according to WHO AWARe criteria.&lt;br /&gt; &lt;b&gt;Results:&lt;/b&gt; The study found that 27 different antibacterial drugs were used, representing 2.2% of all medications. The ABC/VEN analysis revealed that the majority of the budget was spent on a few high-cost drugs. Ceftriaxone was the most commonly used antibiotic, with a significant financial impact. According to the WHO AWaRe classification, most antibiotics fell into the Access and Watch categories.&lt;br /&gt; &lt;b&gt;Conclusion:&lt;/b&gt; The study highlights a high rate of antibacterial drug usage, with significant financial implications for the hospital. The predominance of certain high-cost antibiotics, like ceftriaxone, indicates a need for more rational and cost-effective use of these medications. The findings call for improved adherence to clinical guidelines, enhanced education for medical professionals, and optimized antibiotic utilization to prevent resistance development and ensure better patient outcomes, especially in pandemic situations.

Application of Decision Tree Method in ECG Signal Classification For Heart Disorder Detection

Cardiovascular Disease (CVD) is a group of diseases that affect the heart and blood vessels, and it is the leading cause of death globally. In Indonesia, Coronary Heart Disease (CHD) is one of the most prevalent CVDs. However, due to the high cost of drugs, lengthy treatment duration, and various supporting examinations required, treating CHD can be very expensive. An obstacle to treating heart disease in Indonesia is the insufficient number of cardiologists and experts experienced in interventional cardiology. Along with technological developments, the computer science community is encouraged to contribute to the medical field. For instance, using an electrocardiogram (ECG) can help prevent and minimize problems arising from heart disease. An Electrocardiogram (ECG) is a medical test that measures and records the heart’s electrical activity using a machine that detects electrical impulses. The use of Artificial Intelligence (AI) in ECG is rapidly increasing and has shown to have great potential in improving the diagnosis and treatment of cardiac patients. AI has become a valuable tool in helping doctors diagnose, predict risk, and manage heart disease with greater accuracy, speed, and precision. One of the machine learning methods used in this research is the decision tree method, which is often employed to make decisions. The decision tree method exhibited promising results, with an accuracy rate of 99% in identifying heart defects at an early stage. This method has significant potential to assist doctors in diagnosing heart defects at an early stage with high accuracy.

Patient Satisfaction With Public Pharmacy Services: Structural and Policy Implications From Greece.

Objectives This study aimed at investigating patient satisfaction with services offered by a certain type of public pharmacies in Greece (National Organisation for Healthcare Provision (EOPYY) pharmacies), tasked with dispensing mostly high-cost drugs, in an effort to highlight the aspects to be optimized. Methods The Patient Satisfaction with Pharmacist Services Questionnaire 2.0 (PSPSQ 2.0) questionnaire was the main instrument of our research. We received 201 full responses from patients themselves and patients' companions who had visited EOPYY pharmacies in Athens, the capital city of Greece, from October 2022 to January 2023. Results Patients seem satisfied with public pharmacies in general. In fact, the professionalism of the pharmacists, the respect that patients have received from them, and the information and explanations that were given by pharmacists, received a very high score. On the other hand, the parameters referred to the information that patients received from pharmacists for the overall improvement of their health had the lowest score, revealing an apparent lag in the field of medicinal advice. Conclusion Without any doubt, patients expect their pharmacists to be more guiding and to better communicate this role. This requires more time to be spent with patients, focused training, teamwork, layout, and other organizational interventions.

The increasing costs of medicines and their implications for patients, physicians and the health system.

Most new medicines entering the market are high-cost speciality drugs. These drugs can cost tens to hundreds of thousands of dollars per course of treatment and in some cases millions of dollars per dose. Approximately half of all spending on medicines is projected to target only 2-3% of patients, raising important questions about resource allocation. While there is no doubt that breakthrough innovations have transformed clinical care in some disciplines, it is also true that cost is becoming one of the primary barriers to treatment access and that many new medicines do not provide value commensurate with their prices. This article examines pricing trends, the reasons for high prices and their implications for access and clinical practice.

Is the national health insurance scheme a pathway to sustained access to medicines in Nigeria?

ObjectiveThe debate surrounding access to medicines in Nigeria has become increasingly necessary due to the high cost of essential medicine drugs and the prevalence of counterfeit medicines in the country. The Nigerian government has proposed the implementation of the National Health Insurance Scheme (NHIS) to address these issues and guarantee universal access to essential medicines. Access was investigated using the 3 A’s (accessibility, affordability, and availability). This paper investigates whether the NHIS is a viable pathway to sustained access to medicines in Nigeria.DesignThis was a cross-sectional study using a mixed-methods design. Both qualitative and quantitative methods were utilized for the study.SettingThis study was conducted at NHIS-accredited public and private facilities in Enugu State.Participants296 randomly selected enrollees took part in the quantitative component, while, 6 participants were purposively selected for the qualitative component, where in-depth interviews (IDIs) were conducted face-to-face with NHIS desk officers in selected public and private health facilities.ResultsThe quantitative findings showed that 94.9% of respondents sought medical help. Our data shows that 78.4% of the respondents indicated that the scheme improved their access to care (accessibility, affordability, and availability). The qualitative results from the NHIS desk officers showed that respondents across all the socio-economic groups reported that the NHIS had marginally improved access to medicine over the years. It was also observed that most of the staff in NHIS-accredited facilities were not adequately trained on the scheme’s requirements and that most times, essential drugs were not readily available at the accredited facilities.ConclusionThe study findings revealed that although the NHIS has successfully expanded access to medicines, there remain several challenges to its effective implementation and sustainability. Additionally, the scheme’s coverage of essential medicines is could be improved even more, leading to reduced access to needed drugs for many Nigerians. A focus on the 3As for the scheme means that all facility categories (private and public) and their interests (where necessary) must be considered in further planning of the scheme to ensure that things work out well.