High Cost Of Therapy Research Articles

Edaravone, a new therapeutic option in amyotrophic lateral sclerosis: Evaluation of challenges in the drug accessibility

Introduction: Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease, with about 3–4 years median survival from the onset of symptoms. Only two therapies for ALS have been proven effective in slowing down this condition, riluzole and edaravone. Recently approved edaravone, either original or generic, is not registered in Poland. It can only be used after special approval of Polish Ministry of Health. The costs of payment have to be covered by patient in full. Aim: In this article we will describe the public demand of edaravone in Poland for ALS treatment and problems related to prescribing the drug, like procedures and prices. Material and methods: This article is based on the available literature and on data obtained from Polish Ministry of Health. Results and discussion: The high cost of therapy affects the relatively widespread use of generic medicines not registered in Europe. We identify mechanisms of the demand and supply for various formulations of edaravone in Poland. These information should be of relevance to many other countries, especially within the European Union. Conclusions: Medicines available from licensed manufacturer are more expensive than offers available on-line. Although original chain of distribution presents with unrivaled warranty to avoid purchasing of adulterated drug we report the challenges of the edaravone treatment initiation in ALS patients in Poland.

Comparative study of different forms of Jellein antimicrobial peptide on Leishmania parasite

Typically, antimicrobial peptides (AMPs) are short positive charged peptides serving a key role in innate immunity as well as antimicrobial activity. Discovering novel therapeutic agents is considered as an undeniable demand due to increasing microbial species with antibiotic resistance. In this direction, the unique ability of AMPs to modulate immune responses highlighted them as novel drug candidates in the field of microbiology. Patients affected by leishmaniasis; a neglected tropical disease, confront serious problems for their treatment including resistance to common drugs as well as toxicity and high cost of therapy. So, there is a need for development of new drug candidates to control the diseases. Jellein, a peptide derived from royal jelly of honeybee has been shown to have promising effect against several bacterial and fungal species. In current study, anti-leishmanial effect of Jellein and its lauric acid conjugated form was investigated against two forms of Leishmania major (L. major) parasite. Moreover, cytotoxic effect of these peptides was studied in THP1 cell line and human Red Blood Cells (RBCs). Furthermore, the mechanism of action of peptides on L. major promastigotes was assessed through different methods. The results demonstrated that, conjugation of lauric acid to Jellein not only had no effect on the elevation of antimicrobial activity but also halted it completely. Moreover, Jellein caused a limitation in the number of L. major promastigotes by pore formation as well as changing the membrane potential rather than induction of apoptosis or activation of caspases.

377P - NGS in advanced NSCLC in a developing country: Ready for prime time?

BackgroundThe increasing availability of targeted therapies has made genomic testing an integral part of management of patients with lung cancer. The guidelines are moving towards upfront molecular profiling of all advanced non-small cell lung cancers (NSCLC) with Next Generation Sequencing (NGS). Whether this translates into a clinically meaningful benefit in these patients, especially in a resource constrained country is yet to be determined. MethodsPatients with advanced adenocarcinoma of lung who underwent tissue NGS testing after a negative EGFR, ALK and ROS1 report between April 2017 and March 2019 were included. We retrospectively analyzed the testing outcomes and any change in clinical decision making after the NGS report in such patients. ResultsOverall, NGS was done on tissue of 50 patients with advanced adenocarcinoma of lung. The median age was 58 years (range 27-82 years) and there were 31 males (58%). Most of the patients were never smokers 38(72%). The average turnaround time for the assay was 23 days (SD ± 15 days). One or more genomic alterations were found in 43 patients (86%) with most common genes affected being p53(43%), KRAS (19%), NOTCH1(13%), EGFR(6%), ROS1(4%), BRAF(8%) and HER2(8%). Seven (14%) of the patients were found to have mutations that were targetable by an FDA approved therapy and 9(18%) more patients had an off label available drug. So there was a potential for change of treatment in 16(32%) patients. Out of the 38(76%) patients who continued treatment at our institute, the NGS results led to a change in treatment in only 6(15%) patients. This was mostly due to limited availability of drugs, limited access to clinical trials and high cost of therapy. ConclusionsNGS is an important tool in the detection of targetable genetic alterations and results could potentially alter therapy. However, it is an expensive investigation and its clinical utility is questionable in a developing country like India. Single gene testing for common mutations is both rapid and cost-effective, and change in therapy after further NGS testing is possible in a very small percentage of patients. However, it may be considered after a detailed discussion with the patient in view of limited access to clinical trials or targeted therapies. Legal entity responsible for the studyNil. FundingHas not received any funding. DisclosureAll authors have declared no conflicts of interest.

PCN24 TYROSINE KINASE INHIBITORS: REAL WORLD EVIDENCE ABOUT HEALTH OUTCOMES IN A MIDDLE INCOME COUNTRY

The high cost of antineoplastic therapies generates unprecedented pressure on the health systems in middle income countries; aditionally, the Knowledge Discovery in Databases allows to extract considerable value to evaluated health outcomes. This study assessed the association between adherence and a specific payment model called PGP (Global Prospective Payment, between insurer and provider). Observational retrospective study. Patients with Chronic Myeloid Leukemia (CML) were identified with ICD-10-C921 diagnoses obtained from 2018 High Cost Account database of the institutional cancer cohort. Positive Philadelphia chromosome patients who took Dasatinib or Nilotinib during 2018 were included (n=66). Imatinib was excluded because this drug is included in the health benefit plan in Colombia. Patients who took 100% of the prescribed medication were considered adherent (Morinsky Scale 4/4). Results were analyzed using SPSS version 24, chi-square independence test was used to determine P-value and bivariate analysis was made using binary logistic regression. Dasatinib arm (n=29, median age 65y; 52% female; 62% private regimen), 50% adherent to therapy. Nilotinib arm (n=37, median age 50y; 43% female; 63% private regimen), 59% adherent to therapy. Regardless payment model and TKI, 55% were adherent and 49% had an optimal molecular response. Patients under the PGP model were 100% adherent and, based on the latest available PCR BCR/ABL (real time) report, 89% had an optimal molecular response according to European Leukemia Net recommendations (both TKIs). Significant association were established between Adherence and PGP model (p<0.004; OR=0,49 IC 95% 0,37-0,64) and between optimal molecular response to both TKIs and GPP model (p<0.001; OR=0,43 IC 95% 0,24-0,95). This study provides contemporary real-world evidence about association between adherence to TKIs, optimal molecular response and a prospective payment model in an institution that provides oncological health services. Ensuring adherence to high-cost therapies in middle-income countries through a payment model, could improve health outcomes.

PCN385 PAY-FOR-PERFORMANCE IN ONCOLOGY: A SUCCESSFUL STORY FROM BULGARIA

“Pay-for-performance” (P4P) is an agreement between payer and manufacturer where “the price level and/or revenue received is related to the future performance of the product in either a research or a real-world environment.” - A Towse, 2010. The objective of this study is to demonstrate a successful P4P solution for ensuring market access of a CDK4/6 inhibitor for the treatment of HR+/HER2− advanced breast cancer used in combination with aromatase Inhibitors (AI). Based on the results from the pivotal trial, Clinical benefit rate (CBR) at 6th month is used as a performance milestone in the calculation of the payment for outcomes. CBRs not met are translated into price reductions. As the new technology + AI had significantly better efficacy than AI, the manufacturer agrees to receive payments only for CBRs met, fixed at the level of 79.6%. Based on the expected treatments per year, the payer gets cost reduction of 208 298 BGN (152 512 €; 120 056 $) the first year, 559 000 BGN the second year (285 817 €; 322 190 $) and 698 465 BGN the third year (357 125 €; 402 574 $) or a cumulative saving of 1 465 763 BGN (749 444€; 844 820$) for total number of 200 patients treated. The model has been appraised and approved by the national HTA competent authority. P4P provides new approach to pay for innovative high-cost therapies, allows sharing the risk and limits total budget impact.

Modern Innovative Solutions to Improve Outcomes in Severe Asthma: Protocol for a Mixed Methods Observational Comparison of Clinical Outcomes in MISSION Versus Current Care Delivery.

BackgroundAsthma that is poorly controlled and undertreated can progress to more severe disease that is associated with high levels of unscheduled care that requires high-cost therapy, leading to a significant health economic burden. The identification and appropriate referral to a specialist asthma service is also often delayed by several months or years because of poor recognition and understanding of symptom severity. Current severe asthma services may take several months to provide a comprehensive multidisciplinary assessment, often necessitating multiple hospital visits and costing up to £5000 per patient.ObjectiveThis study aims to evaluate whether a new service model could identify poorly controlled and potentially severe asthma much earlier in the patient pathway, and then compare clinical outcomes between this new care model with standard care.MethodsModern Innovative Solutions to Improve Outcomes in (MISSION) Severe Asthma is a novel service model developed by asthma specialists from Portsmouth and Southampton severe asthma services. MISSION Severe Asthma identified patients with poorly controlled disease from general practice databases who had not been under secondary outpatient care in the last 12 months or who were not known to secondary care. In 1- or 2-stop assessments, a thorough review of diagnosis, disease phenotype, and control is undertaken, and clinical outcomes collected at baseline.ResultsA variety of clinical outcomes will be collected to assess the service model. The results will be reported in February 2020.ConclusionsThis protocol outlines a mixed methods study to assess the impact on disease control, unscheduled health care usage, and quality of life in patients seen in the MISSION clinic compared with a closely matched cohort who declined to attend.International Registered Report Identifier (IRRID)DERR1-10.2196/9585

Sulforaphane Inhibits Nonmuscle Invasive Bladder Cancer Cells Proliferation through Suppression of HIF-1α-Mediated Glycolysis in Hypoxia.

Bladder cancer is the fourth common cancer among men and more than 70% of the bladder cancer is nonmuscle invasive bladder cancer (NMIBC). Because of its high recurrence rate, NMIBC brings to patients physical agony and high therapy costs to the patients' family and society. It is imperative to seek a natural compound to inhibit bladder cancer cell growth and prevent bladder cancer recurrence. Cell proliferation is one of the main features of solid tumor development, and the rapid tumor cell growth usually leads to hypoxia due to the low oxygen environment. In this study we found that sulforaphane, a natural chemical which was abundant in cruciferous vegetables, could suppress bladder cancer cells proliferation in hypoxia significantly stronger than in normoxia (p < 0.05): 20 μM sulforaphane inhibited bladder cancer cell proliferation by 26.1 ± 4.1% in normoxia, while it inhibited cell proliferation by 39.7 ± 5.2% in hypoxia in RT112 cells. Consistently, sulforaphane inhibited cell proliferation by 29.7 ± 4.6% in normoxia, while it inhibited cell proliferation by 48.3 ± 5.2% in hypoxia in RT4 cells. Moreover, we revealed that sulforaphane decreased glycolytic metabolism in a hypoxia microenvironment by downregulating hypoxia-induced HIF-1α and blocking HIF-1α trans-localization to the nucleus in NMIBC cell lines. This study discovered a food sourced compound inhibiting bladder cancer cells proliferation and provided experimental evidence for developing a new bladder cancer preventive and therapeutic strategy.

Practical guide for the use of PCSK9 inhibitors in Portugal

Reducing low-density lipoprotein cholesterol (LDL-C) levels is one of the most important strategies for reducing the risk of cardiovascular events. However, in clinical practice, a high proportion of patients do not achieve recommended LDL-C levels through lifestyle and lipid-lowering therapy with statins and ezetimibe. PCSK9 inhibitors (PCSK9i) are a new therapeutic option that significantly (50-60%) reduces LDL-C levels, which in clinical trials translates into an additional reduction in risk for cardiovascular events, and has a good safety profile. However, it is a high-cost therapy, and therefore its use in clinical practice should take its cost-effectiveness into account. Priority should be given to use in patients at higher cardiovascular risk and those in whom high LDL-C levels persist despite optimal lipid-lowering therapy. This consensus document aims to summarize the main data on the clinical use of PCSK9i and to make recommendations for Portugal on the profile of patients who may benefit most from this therapy.

Cytotoxic effects of different aromatic plants essential oils on oral squamous cell carcinoma: An in vitro study

Background/Aim: Current approaches in therapy of head and neck cancers are surgery, radiotherapy and chemotherapy. However, recurrence, development of multidrug resistance, side effects, and high costs of therapy are significant problems which point to the need for more efficient and less toxic drugs and interventions. Material and Methods: Eight essential oils obtained from Thymus serpyllum, Mentha piperita, Juniperus communis, Rosmarinus officinalis, Melissa officinalis, Achillea millefolium, Zingiber officinale, and Helichrysum arenarium were tested for their anti-proliferative on oral squamous cell carcinoma (OSCC) culture and SCC-25 cell line. Cytotoxicity assays (MTT and Neutral red) were used to detect the effect of the mentioned essential oils. Results: T. serpyllum, M. piperita, J. communis, and R. officinalis essential oils exhibited the best anti-proliferative effect, on both types of cells. M. piperita had the greatest effect on SCC-25 cell line (4,5% of viable cells) and OSCC cells (7,2% of viable cells). Overall, cytotoxicity was higher in OSCC than in SCC-25 cell line. Conclusions: This study showed a clear anti-proliferative effect of four essential oils, in vitro making them novel potential antineoplastic agents.

Intravenous Immunoglobulin Utilization in a Pediatric Tertiary Care Teaching Hospital in Iran

Intravenous immunoglobulin (IVIG) is an expensive medicine which is widely used for unlabeled indications. We conducted this drug utilization review (DUR) to evaluate the appropriateness of IVIG utilization in Tehran, Iran. This cross sectional study was conducted in a referral pediatric tertiary care hospital in Tehran. During a three month period in 2015, medical records of inpatients with IVIG order were evaluated. Appropriate indications for IVIG were determined based on the evidenced based guidelines and literature. Medical records of patients were reviewed and demographic data as well as the diagnosis, previous treatments, IVIG indication, dose, duration and the adverse drug reactions (ADR) were documented. Additionally, cost of therapy was calculated. During the study, 115 patients received IVIG. In 51 cases (44.4%), a total of 1338 gram IVIG was administered inappropriately. We found that in 32 cases (27.8%), intractable epilepsy was the inappropriate indication. The most frequent prescribers of IVIG were clinical specialists of pediatric neurology, pediatric asthma and immunology followed by pediatric hematology. In terms of safety, we found that 64 (55.6%) patients experienced at least one ADR. The most prevalent ADRs were hypotension (27.8%), fever (26.9%) and chills (18.3%). Total cost of IVIG during the study period was 6,075,500,000 Rials (approximately 215,872 $). Irrational use of IVIG is still a considerable issue that costs a considerable amount. Due to the high cost of therapy, multifaceted interventions is necessary to be implemented to improve the prescribing practice.

Interdisciplinary and multiprofessional outpatient secondary individual prevention of work-related skin diseases in the metalworking industry: 1-year follow-up of a patient cohort

BackgroundIn Germany, work-related skin diseases are predominant within the spectrum of reported occupational diseases. Metal workers are among the high-risk professions. Offering effective prevention programs to affected patients is of utmost importance to avoid deterioration of the disease and job loss. We conducted a 1-year follow-up in patients who participated in a multidisciplinary, complex outpatient prevention program representing a standard procedure of patient care by the respective statutory accident insurance.MethodsThe multi-component prevention program consists of multiprofessional individual patient counseling, a structured skin protection seminar in a group, as well as workplace visits and on-site counseling in terms of appropriate skin protection (e.g. gloves). An observational study with a 1-year follow-up and four measurements (T1-T4, longitudinal pre/post-test design) including dermatological examinations and standardized written questionnaires was conducted between 2013 and 2016 to assess changes over time regarding job loss and disease severity.ResultsData from 94 patients (87 male, mean age: 45.4 years) were included in the analysis. One year after the skin protection seminar (T4), 83 patients (88.3%) remained in their original professional metalworking activity and four patients (4.3%) had given up their profession because of their skin disease. At baseline (T1), irritant contact dermatitis of the hands was the most frequent diagnosis (80.7%). Methods for self-reported disease severity showed good correlation with the clinical gold standard at T1 and T2 (dermatological examination with the Osnabrück Hand Eczema Severity Index / OHSI), and a significant decrease of the self-reported disease severity was found over time from T1 to T4 (p < 0.001). Further results indicate an improved self-perceived disease control and an overall satisfaction with the prevention program.ConclusionsThe results of this observational study demonstrate that the comprehensive prevention program positively influences the course of work-related skin diseases, increases the possibility to continue working in a “high-risk” profession and improves the disease management of metal workers. In the long term, the prevention program may lead to cost savings by preventing high therapy costs or professional retraining.

Hemophilia in the developing world: transforming lives through international collaboration

Hemophilia is an X-linked hereditary disorder. The worldwide incidence is 1 in 5000 (hemophilia A) and 1 in 30 000 (hemophilia B).1 Treatment for hemophilia includes deficient factor replacement with clotting factor concentrates (CFCs). CFCs are also used for acute hemophilic arthritis, along with rest, ice, compression, and elevation. There are many challenges for people with hemophilia, the most common of which are joint bleeds and deformity, which affect quality of life. Life-threatening visceral bleeding is not uncommon in hemophiliacs. Surgical intervention for hemophilia-related events that require it is always a demanding task, and the high cost of therapy is an obstacle to getting the necessary treatment. The following challenges are unique to the Indian population1-3: India has the second largest population of any country in the world. India has a high patient load: the total number of registered people with hemophilia is 18 353. Treatment for hemophilia is unaffordable for many. Less than 20% of the country’s population is covered by health insurance. India has a diverse geography. Many facilities that diagnose hemophilia are of poor quality. Care is suboptimal because many treatment facilities are of poor quality. Indians with hemophilia have a high rate of complications and disabilities.

Impact of Medicare Advantage Enrollment on Utilization of Intensity-Modulated Radiation Therapy and Cost of Care for Cancer Treatment

Intensity-modulated radiation therapy (IMRT) is an important driver of rising costs in oncology care, but the level of evidence supporting its routine use varies across disease sites, including breast, lung, and prostate. While Medicare Advantage (MA) plans have incentives to reduce health care spending, the effect of MA enrollment on utilization of high-cost medical services and quality of care is unclear, especially for cancer patients. We examined differences in IMRT utilization and health care spending between MA and traditional fee-for-service (FFS) Medicare for patients receiving radiation therapy (RT) across these disease sites. We performed a retrospective cohort study using 2009-2012 health insurance claims data from the Health Care Cost Institute (HCCI) and the Centers for Medicare and Medicaid Services (CMS). We identified 2,571 MA and 6,062 FFS Medicare beneficiaries from the HCCI and CMS databases, respectively, who were newly diagnosed with non-metastatic breast, lung, or prostate cancer in 2011 and were treated with external beam RT within 12 months of diagnosis. We measured utilization rate of IMRT, unit price of RT delivery, and RT-specific spending among patients within each cancer-type cohort. Outcomes were adjusted for differences in patient characteristics (age, sex, and Charlson co-morbidity index) and geography between MA and FFS beneficiaries. Statistical significance of the differences between groups was assessed using two-sample t tests. In both MA and FFS Medicare, the adjusted rate of IMRT utilization was highest among prostate patients and lowest among breast patients. Across all three disease sites, IMRT utilization was lower in MA than FFS Medicare, but the difference was statistically significant only for the prostate cohort (18.0 vs. 20.2%, p=0.149 for breast; 32.8 vs. 36.0%, p=0.385 for lung; 94.0 vs. 98.5%, p<0.001 for prostate). Average unit price of IMRT delivery was not significantly different in MA versus FFS Medicare. However, per patient RT-related costs were lower among MA patients in the breast and prostate cancer cohorts ($12,630 vs. $13,214 and $25,990 vs. $27,685, respectively; p=0.005 and p<0.001), but not significantly different among lung cancer patients ($15,653 vs. $15,024; p=0.301). IMRT utilization rates in both MA and FFS Medicare were consistent with the relative level of evidence supporting its use in each disease site. MA enrollment was associated with modestly lower IMRT utilization for all patients, with no evidence of a differential impact based on cancer type. The unit price of IMRT delivery was similar in MA versus FFS Medicare. We found no evidence that MA plans adopted utilization management tools that more effectively targeted high-cost therapies based on clinical evidence than in traditional Medicare.

Outcomes of Intensive Treatment of Adult Acute Myeloid Leukemia Patients: A Retrospective Study From a Single Centre.

Acute Myeloid Leukemia (AML) is a very aggressive cancer with difficult treatment and poor outcomes. The treatment of these patients is quite challenging due to various reasons including the need for extensive supportive care, and high cost of therapy. Reports on outcomes from India are few. We analyzed 93 adult patients (≥ 18years) with AML who were treated with curative intent between 2007 and 2014. Patients received daunorubicin at dose of 60-90mg/m2 and cytarabine 100mg/m2 during induction and consolidation with 3 courses of high dose cytarabine (1.5-3g/m2per dose for 6 doses per cycle). Only 4 patients underwent consolidation allogenic stem cell transplantation in first remission (CR1). The median age was 37 (18-66) years; males: 52%. Conventional cytogenetics (N = 63) showed 23% (N = 15), 56% (N = 35), 27% (N = 13) in good, intermediate risk and poor riskcategory respectively. FLT3-ITD was positive in 12/33 (36%) and NPM mutation in7/23 (30%). Daunorubicin dose was 60mg/m2 in 75% (N = 70) and 90mg/m2 in 25% (N = 23) patients. Induction mortality was 17% (16/93) [60mg/m2:19% (13/70), and 90mg/m2:13% (3/23); p = 0.39)]. Complete remission was achieved by 60% (56/93) [60mg/m2:53% (37/70), and 90mg/m2:83% (19/23); p = 0.09)]. The median overall survival was 9.2months and the actuarial survival at 2years was 30%. By univariate analysis, FLT3-ITD positivity, white cell counts higher than 100,000/mm3 at presentation, and use of lower dose of daunorubicin in induction were associated with poorer outcomes. Outcomes in adult AML are generally poor. Many patients with high risk disease don't receive allogenic transplantation in CR1. Increased availability of allogenic stem cell transplantation may help to improve outcomes.

THUR 122 Parkinson’s kinetigraph improves movement disorder service provision

IntroductionObjective evaluation of symptoms of Parkinson’s disease (PD) can be challenging. There is increasing interest in technological solutions to assess, monitor and manage people with PD.ObjectiveTo evaluate the effect of the Parkinson’s Kinetigraph (PKG) on management of patients with PD in a large tertiary movement disorder service.MethodsWe retrospectively reviewed the notes of 47 patients with PD (22 female, 25 male) who underwent PKG recording over a six month period. The indications and PKG findings, and the subsequent effect on clinical decision making and service provision were recorded.ResultsManagement was significantly altered in 25 patients (53%), while in 13 patients (28%) PKG confirmed the use of advanced therapies such as deep brain stimulation. Significant effects were seen with regard to service provision. Outpatient appointments could be deferred with advice following PKG in 15 (32%), advanced therapies assessment was improved in 16 (34%), while inpatient admission was avoided in six patients (13%).ConclusionThe use of PKG has enhanced service provision in our movement disorder service. In particular, it enhances our assessment of patients considered for high-cost advanced therapies, allows more efficient use of clinic appointments, and has the potential to reduce hospital admissions.

Drug utilization pattern in orthopaedic outpatient department of a tertiary care hospital in Kerala: a geriatric perspective

Background: Geriatric population due to the age related changes in pharmacokinetics and pharmacodynamics and the presence of comorbidities is vulnerable to drug interactions, adverse effects and high cost of therapy. This necessitates a periodic review of DU pattern in the geriatric population to ensure safe and effective treatment for them. The present study was undertaken to evaluate the DU pattern for medical conditions among the geriatric population in the Orthopaedic outpatient department (OPD) of a tertiary care hospital in Kerala.Methods: In this cross sectional observational study conducted in the Orthopaedics OPD of a tertiary care hospital, prescriptions were collected from patients attending the Orthopaedics OPD randomly over a period of 6 months. Out of these, prescriptions of male and female patients of age more than 60 years were sorted and analysed using World Health Organization drug prescribing indicators as well as additional parameters and the data was presented in the form of frequency and percentages using tables and charts.Results: A total of 800 prescriptions were collected and studied of which 76 (9.5%) belonged to patients from the geriatric population. Majority of the patients were in the age group of 61-70years (52.63%). Spondylosis (42.10%) was the most common indication for patients attending Orthopaedics OPD. Average number of drugs per prescription was 3.05 with a range between 1 and 5. Only 5.17% drugs were prescribed using generic name. Utilization from the essential drug list was 39.65%. The percentage of encounters in which an antibiotic and injection prescribed was 0% and 10.34% respectively. Of the total drugs prescribed 35% were FDCs. The most routinely prescribed drugs among the various classes were NSAIDs 34% followed by gastroprotectives (25%). The assessment of prescriptions with regard to completion and legibility was satisfactory.Conclusions: Current study pointed out deficiencies like polypharmacy, low prescribing of drugs by generic names, low prescribing of drugs from the essential drug list and higher use of FDCs. Use of antibiotics and injections was satisfactory and acceptable. Legibility and completion of prescription format was largely satisfactory. Proper strategies to rectify these deficiencies can ensure safe and effective treatment for geriatric patients.

Precision Medicine.

Every human being has twenty-three chromosomes and thousands of genes. Precision medicine aims to assess risk and customize treatment for specific genetic variants and disease characteristics associated with these human building blocks. Large databases of genetic information are needed to locate the best targets for specific therapies. This month's DataGraphic focuses on precision medicine's rapid growth in the past two decades in genetic tests and therapies and its successes in prolonging life and cutting some costs. It also highlights challenges ahead due to the high cost of therapies, the limited representation of racial and ethnic minorities in genetic databases, and uncertainty among professionals about how to apply genetic findings.

PCN222 - Potential Budget Impact Of High-Cost Therapies With Potential For Expanded Indications

PCN222 - Potential Budget Impact Of High-Cost Therapies With Potential For Expanded Indications

PCP2 - Nice-ly Paving the Way? Dynamic Pricing: The Future of Pricing and Reimbursement?

The National Institute of Health and Care Excellence (NICE) appraises the clinical and economic impact of health technologies to inform public reimbursement decision-making in England and Wales. Cost-effectiveness is the key criterion for informing recommendations. In April 2016, the Cancer Drugs Fund (CDF) was reformed to enable temporary reimbursement for oncology therapies where uncertainty exists, to collect real world evidence to inform subsequent NICE appraisals. In October 2016, osimertinib become the first drug to enter the newly-reformed CDF and 10 therapies have entered the CDF to 11/24/2017. Outside of oncology, three drugs (all orphan drugs appraised under the HST pathway) have been reimbursed under Managed Access Agreements (MAAs). Under MAAs, drugs are provided at a substantial discount while evidence generation activities are ongoing to inform future NICE appraisals. The first MAA was agreed in November 2015 for elosulfase alfa in Morquio A Syndrome with a second MAA in July 2016 for ataluren in Duchenne muscular dystrophy. Under a NICE consultation document published in October 2017 (on changes to increase technology appraisal process capacity), this ‘third option’ of temporary reimbursement under the CDF or an MAA was formally itemized for health technologies appraised via the single technology appraisal process, suggesting that these approaches may become more frequent in the future. As new technologies with transformational benefits are being developed for patients with severe unmet needs (e.g. CAR-T cell, gene therapies, and immuno-oncology), regulators are increasingly approving these at earlier stages of their clinical development process. This poses a challenge for reimbursement decision-makers who have to justify investment of limited economic resources for these high-cost therapies when significant uncertainty remains. Temporary reimbursement at a confidentially discounted level until the data package matures to inform a final reimbursement decision may represent a best-practice solution with applicability internationally.

Coordinated heart failure care in Poland: towards optimal organisation of the health care system

Heart failure has becoming an increasing medical, economic, and social problem globally. The prevalence of this syndrome is rising, and despite unequivocal positive effects of modern therapy, reduction of mortality has been achieved at the cost of more frequent hospitalisations. Unlike in many European countries, in Poland heart failure is usually recognised later, at a more advanced stage of the disease, leaving less time for ambulatory treatment and resulting in a high number of hospitalisations. The current paper presents the most important data regarding morbidity and mortality due to heart failure in Poland. The experts in the field focus on the key source of high costs of therapy and highlight several critical organisational deficits present in the Polish health care system. This background information builds a basis for a concept of coordinated care for patients with heart failure. The paper discusses the fundamental elements of the system of coordinated care for patients with heart failure necessary to enhance the diagnosis, improve therapeutic effects, and reduce medical, economic, and social costs.