High Cost Of Therapy Research Articles

CRRT in developing world.

Continuous renal replacement therapy (CRRT) has become a mainstay therapy in the intensive care unit (ICU) and its utilization continues to increase in developed countries. The wide variations of CRRT practice, however, are evident in developing countries while clinicians in these resource-limited countries encounter various barriers such as a limited number of nephrologists and trained staff, a gap of knowledge, machine unavailability, cultural and socioeconomic aspects, high-cost therapy without reimbursement, and administrative as well as governmental barriers. In this article, we demonstrate the situation of CRRT and discuss the barriers of CRRT in a resource-limited setting. We also discuss the strategies to improve CRRT practice. These recommendations can serve as a fundamental guideline for clinicians to implement CRRT in low-resource settings.

The economics of alternative payment models for pharmaceuticals.

Pharmaceuticals are priced uniformly by convention, but vary in their degree of effectiveness for different disease indications. As more high-cost therapies have launched, the demand for alternative payment models (APMs) has been increasing in many advanced markets, despite their well-documented limitations and challenges to implementation. Among policy justifications for such contracts is the maximization of value given scarce resources. We show that while uniform pricing rules can handle variable effectiveness in efficient markets, market inefficiencies of other kinds create a role for different value-based pricing structures. We first present a stylized theoretical model of efficient interaction among drug manufacturers, payers, and beneficiaries. In this stylized setting, uniform pricing works well, even when treatment effects are variable. We then use this framework to define market failures that result in obstacles to uniform pricing. The market failures we identify include: (1) uncertainty of patient distribution, (2) asymmetric beliefs, (3) agency imperfection by payer, (4) agency imperfection by provider, and (5) patient behavior and treatment adherence. We then apply our insights to real-world examples of alternative payment models, and highlight challenges related to contract implementation.

Prescription Behaviour and Barriers to Prescription of Biologicals for Treatment of Chronic Inflammatory Skin Diseases in Dermatological Practice in Two German Federal States.

The globally increasing prevalence of chronic inflammatory skin diseases has substantial costs. Biologicals have become available as therapeutic options, but are encumbered with barriers to prescription. The aim of this study was to evaluate the barriers to prescription of biologicals in the treatment of chronic dermatological diseases. Dermatologists working in private practices in the German federal states of Bavaria and Lower Saxony participated in a cross-sectional study. Economic and legal aspects, including “high therapy costs”, “low reimbursements”, and “fear of regress claims”, were identified as the most prevalent barriers. Significant differences between dermatologists from Bavaria and Lower Saxony were found only regarding the treatment of atopic dermatitis. This study demonstrates the prevalence of barriers to the prescription of biologicals in the treatment of chronic dermatological diseases. Overcoming these barriers could improve the usage of modern therapies and thereby expand patient-centred care for chronic skin diseases.

Aromatherapy: Prophylactic and Therapeutic Approach to Diseases of the Respiratory System

Infectious diseases are a significant public health challenge due to the increasing recognition of new infectious agents and the resurgence of infections until recently controlled. These infectious illnesses constitute many hospital admissions, mainly respiratory focus, which requires high-cost therapy. One of the strategies to prevent injuries leading to hospitalization is reducing the body’s inflammatory response. Thus, aromatherapy seems to be a useful adjuvant therapeutic tool due to the essential oils’ synergistic action with proven anti-inflammatory and antimicrobial properties. Hence, this study proposes an integrated review of the literature on aromatherapy as a tool to be used in prophylaxis and therapy in respiratory system disorders. After passing the criteria of exclusion and content analysis, a set of 124 articles narrowed to 44 articles that support the anti-inflammatory properties of the sesquiterpenes, such as the farnesol. This substance treats the respiratory system and is present in Cymbopogon nardus, Cymbopogon flexuosus, and Rosa damascena. Caryophyllene and its derivatives are present in Syzygium aromaticum, Rosmarinus officinalis, and Cannabis sativa. Besides, α-bisabolol and chamazulene present in Matricaria recutita and Achillea millefolium. Other oils have antibiotic properties that act against gram-positive and gram-negative bacterias according to their constitution’s complexity and interaction with these microorganisms. In this context, some species such as Cinnamomum zeylanicum, Citrus hystrix, Cymbopogon Citratus, Eucalyptus globulus, Eucalyptus odorata, Juniperus communis, Lavandula angustifólia, Lavandula intermedia, Melaleuca leucadendra, Mentha piperita, Pinus sylvestris, Syzygium aromaticum, and Thymus vulgaris are in the spotlight. Several studies, both in vitro and in vivo, have demonstrated the potential use of essential oils alone or combined with oils from other species as a strategy for the prevention of respiratory diseases and potentiation of standard therapies. This approach is possible due to the synergistic effects of substances with bioactive potential such as sesquiterpenes, monoterpenes, and phenylpropanoids.

Patient access to gene therapy medicinal products: a comprehensive review

BackgroundGene therapies have the potential to be a curative approach to a large number of genetic diseases. However, granting of a positive marketing authorisation does not equal patient access to...

PNS141 Deal or No Deal: Twenty Years of Pricing and Access Agreements in Products Assessed by NICE

Since their introduction, access arrangements such as patient access schemes (PAS) have become increasingly common in the UK. The objective of this analysis is to describe trends in uptake of commercial arrangements over time and to compare arrangements that have been implemented for National Institute for Health and Care Excellence (NICE) recommended medicines. A complete list of appraisals undertaken by NICE up to 30 June 2020 was extracted and analysed against published details of commercial arrangements. Products receiving negative recommendations were excluded from analysis. Decisions from multiple technology appraisals assessing >1 product or indication were counted as discrete outcomes. The proportion of appraisals utilising a commercial arrangement was calculated each year using the number of positive recommendations as the denominator. A total of 956 decisions were reviewed, of which 739 (77%) were positive. The proportion of approved products with any commercial arrangement generally increased from 2007 to 2020, with a peak of 91% (50 of 55 recommended products) in 2018. Over the last 5 years, approximately two thirds of positive recommendations were supported by a commercial arrangement of some form. The most common type of arrangement was the simple PAS, accounting for an average of 61% of all commercial arrangements from 2007 onwards. We identified a shift away from use of the complex PAS, towards more frequent use of commercial access agreements (CAA, with or without associated PAS) over the last 5 years (from 14% to 36%). There has been an increasing trend towards confidential agreements (such as simple PAS and CAA) since 2007. In part this is likely to reflect the challenges of achieving UK access in a wider context of global markets and price referencing. With the increasing prevalence of high cost therapies, this is a trend that is set to continue.

PBI49 A Comparative Analysis of Cost-Effectiveness Evaluations of Cell and GENE Therapies across HTA Organisations

Cell and gene therapies (CGTs) are novel, complex treatments that pose specific challenges for payers. With the increasing number of high-cost CGTs likely to strain budgets, multinational HTA collaborations to review methods or conduct joint assessments could become more important and common for these therapies. This research compared assessments of CGTs across HTA organisations to understand how assessment methodologies vary and how this impacts outcomes. Six HTA organisations were selected due to their use of cost-effectiveness and participation in international HTA collaborations (CADTH, ICER, NICE, SMC, TLV, ZIN). Information was sourced from official agency websites, peer reviewed publications and grey literature to understand each organisation’s methods and policies. HTA reports published by June 2020 from the scope organisations were retrieved for three CGTs case studies (tisagenlecleucel, axicabtagene ciloleucel, voretigene neparvovec) to investigate timelines, cost per QALY outcomes and recommendations. Although all organisations studied used cost-effectiveness, there were differences in methodologies and policies, including the use of explicit vs implicit cost per QALY thresholds and how thresholds are employed. Differences in assessment times for CGTs were observed across organisations, ranging from ∼4 months (CADTH) to 9-12 months (NICE). Significantly different cost per QALY estimates were reported across organisations for the three CGTs due to various factors, including: perception of long-term data, patient and clinician input, and weighting of societal impact. For voretigene neparvovec, cost per QALY estimates ranged from €426,000 (ICER) to €127,760 (NICE). Both the cost per QALY differences and the differences in thresholds resulted in varied reimbursement recommendations. While several organisations employ process modifications for high-cost therapies, few are CGT-specific. Organisations differ in how they handle data uncertainties and higher upfront treatment costs, resulting in varied cost per QALY outcomes. International collaborations provide opportunities to share learnings as more CGTs undergo HTA appraisal.

Clinical Determinants of Successful Omalizumab Therapy in Severe Allergic Asthma Patients: 4-Year-Long, Real-Life Observation.

IntroductionOmalizumab is a high-cost therapy recommended for the treatment of severe allergic asthma.ObjectiveTo find clinical parameters that are related to the sustained response to omalizumab.Patients and MethodsThis retrospective, real-life, 4-year follow-up was provided in Poland between March 2013 and May 2019. The success of omalizumab was assessed based on composed subjective and objective criteria. Simple/multiple regression analyses were performed to search for predictors of the response to omalizumab.ResultsA total of 989 severe allergic asthma patients were referred for omalizumab therapy, of whom 854 patients were considered eligible for treatment. At weeks 16 and 52, omalizumab was successful in 84% and 91% of patients, respectively. Treatment effectiveness was maintained up to the 4-year follow-up. Four predictors of the response to omalizumab were found at week 16 and two at week 52. The results at week 16 may be used as predictors of success at week 52 based on the model including baseline FEV1% and change in ACQ-7 and miniAQLQ score at week 16: the area under the ROC curve equals 0.746 [95% CI: 0.672–0.820].ConclusionOmalizumab therapy is very effective, with this efficacy sustained after 4 years of treatment. Success of the therapy can be predicted from the baseline FEV1% and clinical improvement (based on ACQ-7 and miniAQLQ scores) at week 16.

Abstract 14164: Trends in U.S. Spending on Diabetes Therapies by Insurance Payer From 2007 to 2017

Importance: Diabetes mellitus is an important cardiovascular risk factor, and diabetes therapies are among the most commonly prescribed medications in the U.S. While the introduction of new, high-cost therapies has increased costs, recent trends in national spending on diabetes medications by insurance payer have not been examined. Understanding the burden of costs amongst all payers may help inform strategies to optimize drug expenditures in the US. Objective: To produce nationally representative estimates of US spending on diabetes therapies from 2007 to 2017, stratified by insurance payers (Medicare, Medicaid, Private, VA, and TRICARE—a program for civilian health benefits for US Armed Forces). Trends in out-of-pocket costs were also examined. Design/Setting: Retrospective analysis of patient-level data from the Medical Expenditure Panel Survey, a nationally representative survey that is the most complete source of data on healthcare costs in the US, from 2007 to 2017. Participants: All patients receiving one or more classes of diabetes therapies. Outcomes: Inflation-adjusted total national expenditures on all diabetes therapies. Results: There were substantial increases in total expenditures on diabetes therapies for all payers during the study period, ranging from +165.6% for VA & TRICARE ($380.6 million to $1.0 billion) to +460.4% for Medicaid ($1.1 to $6.1 billion). Private insurers increased spending by +346.6% ($5.7 to $25.5 billion), and Medicare increased spending by +389.9% ($5.4 to $25.2 billion). Out-of-pocket spending increased more modestly (+15.7%, from $4.7 to $5.7 billion). Conclusions/Relevance: Spending on diabetes therapies increased dramatically between 2007-2017, with Medicare spending increasing by 389.9%. In contrast, increases in out-of-pocket costs have been more modest. Reasons for the rapid rate of rise in Medicare and private spending compared to out-of-pocket spending should be examined in future studies.

Descripción de un servicio de telerreumatología en una institución colombiana

IntroductionModels in health care have been static for a long time, but recently there has been a change in recognising that technology in the area of information and communication could lead to a change in improving health services. Telemedicine has been increasing and its use now extends to the entire process of health care. It is beginning to be implemented in the rheumatology area, in Colombia. The characteristics of a tele-rheumatology service are described, as well as a more detailed observation of a cohort of patients with rheumatoid arthritis (RA), in order to identify strengths and improvements. MethodsA descriptive observational cross-sectional study was conducted on the total population of patients who were treated by the tele-rheumatology service in the synchronous modality for a period of 30 months. As regards the follow-up of patients with RA, all patients were included who were treated exclusively by means of synchronous telemedicine for a minimum period of 6 months with at least three follow-ups, in which it was possible to calculate the clinimetry by Das28 with the use of C reactive protein (CRP). Measures of frequency, central tendency and dispersion according to type of variable will be used for the descriptive analysis. ResultsData was collected from 1905 patients during the period between August 2017 and March 2020. A total of 4864 consultations were made. Non-attendance of 368 (7.85%) consultations was registered. There were 1784 (83%) patients with a definitive diagnosis by the rheumatologist. A total of 284 patients (14.9%) were discharged by the rheumatology service, and 85 (4.46%) were referred for an exclusively face-to-face evaluation. Auxiliary medical care at the place of origin was provided by a general practitioner in 1,749 (91%) cases. There was no security during the care process as regards the physical examination in 46 (2.4%) cases. Of the total number of patients, 184 (9.6%) cases came from rural areas or municipalities far from the place of care. Biological therapies were prescribed in 139 patients, 56 new prescription during the 30 months. Of 479 RA patients, 200 met the criteria for follow-up. Of these, according to the activity measured by DAS28 with the use of ultrasensitive CRP, 54 patients (27%) were found on admission to the program in remission, 23 (11.5%) patients had low activity, 81 (40.5%) patients had moderate activity, and high activity was found in 42 (21%) patients. Regarding the start of follow-up, there was a 47% increase in the number of patients in remission, and low activity to 19.5%, in contrast to a reduction of 25% in patients with moderate activity and in 9% increase high activity in their last measurement, possible during monitoring. In the group of 200 patients from long-term follow-up, biological therapies were used 61 times. Of the 166 patients during follow-up without biological therapies on admission, these were required in 16%. DiscussionA detailed follow-up of the patients was carried out in the telemedicine service in a synchronous way for a period of two and a half years. The low percentage of absences shows a good adherence to the program. Physical examination, the main reason for medical care at the remission site, was not without difficulties, in this minority it was necessary to carry out diagnostic images. It would be important to assess this group of patients where there are doubts regarding the physical examination, those who have a certain number of appointments without a definitive diagnosis, or in whom biological or high-cost therapies would be used should necessarily be referred to face-to-face consultation. In the group of patients with RA, the percentage of biological use is considered high in relation to the expected standards. This may be for several reasons, such as the high number of patients with prior use of biological agents, the majority of patients with long-term disease, and difficulties in accessing follow-up due to rheumatology, as well as the small number of sero-negative patients in the study population, and practices related to non-presential medical practice may overestimate the activity of the disease. In conclusion, telemedicine has great advantages in the care of rheumatology patients, although it requires modifications to improve these services in favour of patients.

Hepatocellular carcinoma: considerations for managed care professionals.

Liver cancer is the most rapidly increasing cancer in the United States and is associated with a high cancer-related mortality. Seventy-five percent of liver cancers are hepatocellular carcinoma (HCC) resulting from cirrhosis. Patients are typically diagnosed late in the disease, with a relatively small percentage eligible for curative treatments. Despite the addition of several new therapies for advanced HCC, the 5-year survival rate is just 18%. The direct and indirect costs of HCC are substantial, and are expected to increase with the rise in disease incidence as well as a growing number of high-cost therapies entering the market. There are opportunities to improve the quality of care for patients with HCC through implementation of value-based reimbursement principles and pharmacist involvement in care.

FATORES DE RISCO ASSOCIADOS AO AGRAVAMENTO DE SEPSE EM PACIENTES EM UNIDADE DE TERAPIA INTENSIVA DE UM HOSPITAL DE ENSINO

The objective of the study is to outline the profile of patients admitted to an Adult Intensive Care Unit of a Teaching Hospital inthe Interior of São Paulo with sepsis and to evaluate the risk factors and clinical characteristics associated with the evolution to sepsis considering as outcomes of worsening septic shock and death. Medical records of patients in the Adult ICU were analyzed from July 2017 to July 2019, and a case-control study was carried out on these patients, considering 3 outcomes: sepsis, septic shock and death. The study included 154 patients admitted to the ICU during the study period. Of these, 105 patients with sepsis were included. The risk factors found were hyperthermia, anemia, renal failure, hyperkalaemia and the need for mechanical ventilation. Further reflection on sepsis is necessary, which is still one of the main causes of death in the ICU, causing high socioeconomic impact due to the high hospital cost and limited therapies.

Sidebar: CMS seeks to fix Medicaid best-price barriers in value-based contracting for high-cost therapies in Medicaid.

Sidebar: CMS seeks to fix Medicaid best-price barriers in value-based contracting for high-cost therapies in Medicaid.

ORPHAN DRUG REGULATION IN VARIOUS REGIONS OF THE WORLD

Orphan medications became a significant share of drug expenditures in every country. The high cost of therapy is caused by the low prevalence of diseases and the need to compensate the investment of producers in long-term development and registration procedures. The article highlights the main stages of the formation of legislation regulating the circulation of orphan medicines in the United States, covering state guarantees for the development of «orphan medicines», and imperfections of the existing system which influence the cost and availability of medicines. On the example of the EU, the author examines the unified approaches of EU countries to determining the orphan status of medicines, which is especially relevant in the context of convergence and harmonization of national legal systems regulating the circulation of medicines. The purpose of this article is to analyze various approaches to regulating orphan medicinal products in order to determine the most effective practices. The author identifies the main barriers in the provision of medicines to patients with orphan diseases in the Russian Federation, analyzes the main directions of the development of the drug supply system and outlines possible directions for expanding access to necessary treatment methods.

Efficacy and safety of statin therapy in multimorbid outpatients with very high cardiovascular risk

Aim. To analyze the efficacy and safety of statin therapy in multimorbid outpatients with very high cardiovascular risk in actual clinical practice.Material and methods. The study included 131 patients with an established very high cardiovascular risk. History and anthropometric data were collected. The Charlson Comorbidity Index (CCI) was calculated; patients were divided into groups of moderate (№ 1, ≤6 points) and high (№ 2,> 6 points) multimorbidity. The frequency of prescribing statins, the range of doses used, the achievement of lipid metabolism targets, and the incidence of adverse effects were evaluated.Results. The median of the CCI was 6 (5÷8) points. Group 1 included 72 patients, group 2 — 59 patients. Statins received 87 (66,4%) patients, more often in group 1 (n=54) than 2 (n=33), p=0,026. The minimum doses were taken by 17 patients, the mean — 66, the maximum — 4. Patients of group 2 received higher dosages (χ2 =9,3, p=0,010). The target level of total cholesterol was achieved in 8 (6,1%) patients, low-density lipoprotein cholesterol — no one (0,0%). Of the 106 patients ever taking statins, they were withdrawn in 19 (17,9%) patients. The reason for discontinuation in 7 patients were adverse effects, in 5 — the high cost of therapy; in 7 patients, the reason was identified. Adverse effects were recorded in 12 (11,3%) patients; there were no differences between groups (p=0,118).Conclusion. Patients with a very high cardiovascular risk are characterized by high multimorbidity. Statins are less commonly prescribed for patients with severe polymorbidity, but at higher doses. Despite the sufficient prescribing statins and the use of mean doses, target lipid levels were not achieved. The presence of multimorbidity was not associated with an increase in the incidence of statin adverse effects.

Essential Workflow and Performance Measures for Optimizing Acute Ischemic Stroke Treatment in India.

Essential Workflow and Performance Measures for Optimizing Acute Ischemic Stroke Treatment in India.

Multiple Myeloma Cost of Care Under the Oncology Care Model: The Influence of High-Cost Therapies.

As expenditures for cancer care continue to grow substantially, value-based payment models are being tested to control costs. The Oncology Care Model (OCM) is the largest value-based payment program in oncology. The primary objective of this analysis was to determine the impact of high-cost novel agents on total cost of care for multiple myeloma (MM) episodes of care in the OCM. This was a retrospective analysis using Medicare claims data for 258 MM OCM episodes initiated between July 1, 2016, and July 1, 2017. Patients were organized into 3 cohorts: those who received pomalidomide (cohort A), those who received lenalidomide (cohort B), and those who did not receive either drug but had received another chemotherapy agent (cohort C). We compared the actual episode expenditures and the Centers for Medicare and Medicaid target price to create an observed versus expected (O/E) ratio. The average O/E for cohort A (n = 73) was 1.73, compared with 1.31 for cohort B (n = 84) and 1.01 for cohort C (n = 101). The difference the in O/E ratio among the groups was statistically significant (P < .001). The average episode target price for cohorts A, B, and C was $66,149, $63,483, and $63,937, respectively. Despite the high cost of pomalidomide and lenalidomide, there was no significant difference in the average episode target prices of the cohorts. The O/E ratio and target prices of the cohorts demonstrate a lack of adequate adjustment to the OCM target price for episodes in which pomalidomide and lenalidomide were used to treat patients with MM.

Biosimilars: opportunities and risks

Biologic therapies are akey component of modern medicine, especially in the treatment of chronic conditions and in particular immune-mediated diseases. Biosimilars are molecularly highly similar variants of biologic therapies approved after patent expiration of the original product. To provide an overview of the emerging role of biosimilars and present data with respect to efficacy and safety. Since the approval of human insulin as the first biologic therapy, over 150 biologic therapeutics have been approved in the European Union (EU). Due to the high cost of development and production, biologic therapies place aheavy burden on healthcare systems and, at costs totaling 13.8 billion Euros annually, comprise one third of the annual drug expenditure in Germany. Biosimilars are highly similar versions of already approved biologic therapies that do not have clinically relevant differences with respect to efficacy, safety and immunogenicity, as far as can currently be ascertained. Through competition with the original product, biosimilars have been able to drive down prices and relieve the healthcare system without changing overall efficacy. The potential savings through biosimilars are estimated to be 500 million Euros in Germany alone. Currently, over 50biosimilars of 16 different biologic therapies are approved in the EU. Biosimilars are safe and economical alternatives to biooriginal drugs that can boost access to modern, high-cost therapies and relieve healthcare systems.

EP01 Audit of the monitoring and documentation of DAS28 score in patients prescribed high cost DMARDs for rheumatoid arthritis every 6 months according to British Society for Rheumatology guidelines

Abstract Background DAS28 is a measure of disease activity in rheumatoid arthritis (RA) which is comprised of the number of tender and swollen joints, inflammatory markers and visual analogue score. Both NICE and British Society of Rheumatology recommend DAS28 is recorded at least every 6 months in patients with RA, to monitor disease activity and ensure adequate response to treatment. CCG mandate 6 monthly DAS28 scores for patients on high cost DMARDs in order to secure funding. Absence of DAS28 scores results in CCGs refusing to fund treatment at the expense of the Trust. Methods Our aim was to review whether a DAS28 was documented in every consultation and if it meets the audit standard of a review every 6 months. If the 6 month target is not met, to understand the reason for this and to suggest ways to meet the standard. Blueteq is the software used to identify all patients with a diagnosis of RA between January 2014 and January 2019 at Royal Surrey County Hospital (RSCH). Clinic letters were reviewed to identify whether a DAS28 score was recorded, the date it was recorded and whether this was done within 6 months. Results In total, 207 patients are prescribed high cost DMARDs for RA at RSCH. 166 (80%) patients have a documented DAS28, but 41 (20%) patients did not have a documented DAS28. 14 (7%) patients have a documented DAS28 at least 6 monthly. 152 (73%) patients did not meet the audit standard. Conclusion Documentation of DAS28 in clinic letters at RSCH does not meet the audit standard. However, these scores have been recorded on Blueteq in order to satisfy CCG requirements. There needs to be consistency in where DAS28 is recorded. Factors contributing to an absence of a documented DAS28 in clinic letters include: no recent inflammatory markers, patients not receiving clinic appointments scheduled at the correct interval, patients not attending clinic, limited follow-up clinic capacity. DAS28 documentation can be improved by incorporating a table into each rheumatology clinic letter template. On initiation of a high cost therapy, the importance of regular blood monitoring and clinic attendance should be reiterated to patients as well as to the appointments booking team. An early arthritis pathway already exists, which can be extended to incorporate a high cost DMARD pathway detailing how to achieve target documentation. Disclosures: K.F.W. Tsoi: None. A. Sahni: None. B. Selvanayagam: None. M. Memon: None.

Request for an Andexxa Value Analysis.

This perspective is a formal request to the American College of Cardiology and American Heart Association (ACC/AHA) to perform a value analysis on andexanet (Andexxa) similar to what was completed for the PCSK9 inhibitors in the 2018 ACC/AHA Blood Cholesterol guidelines. Based on the safety and efficacy concerns of andexanet alfa, a value statement in and or as an addendum to society guidelines is vital considering the high cost of therapy. In this era of ever-increasing health care costs, every clinician, health system, national society, insurer, and pharmaceutical company should work to be good stewards of our society's resources.