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Health technology performance assessment : real-world evidence for public healthcare sustainability

Health technology performance assessment : real-world evidence for public healthcare sustainability

Two important aspects that can complicate HTpA adoption are the additional costs required and the need for more robust administrative and/or clinical data collection and storage. Costs involved in HTpA can be seen as an investment to achieve better health care; nevertheless, immediate financial resources need to be allocated to improve or create relevant IT systems. In Brazil, the law guarantees investment in research related to the public health system, and we believe that part of this resource can be allocated to HTpA. Another option valid across countries are arrangements with the companies and the creation of research funds. The publication of this Guideline, along with the creation of a specific program for HTpA, will allow the institutionalization and continuous improvement of the scientific methods to collect real world evidence towards optimization of available resources. Disinvestment per se is one of the consequences of HTpA. The
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Real-world evidence: How pragmatic are randomized controlled trials labeled as pragmatic?

Real-world evidence: How pragmatic are randomized controlled trials labeled as pragmatic?

When assessing new medicines (drugs or biologics) or new indications prior licensing, the typical RCT is highly explanatory (double-blind, placebo-controlled) [9]. Con- versely, the typical paradigm of a real-world, comparative effectiveness medicines RCT is highly pragmatic, and compares the effectiveness of two commercially available medicines that are prescribed in routine care but have not been previously compared to each other [10]. Pragmatic medicine RCTs help to inform decisions by end users of information such as clinicians and patients, and by deci- sion makers such as hospital, insurance and other policy makers. They are also used by industry in their price and reimbursement discussions with regulators. In an unsus- tainable health-cost environment, Health Technology As- sessment agencies and Managed Care Organizations want to have real-world evidence on comparative effectiveness of available interventions in clinical practice to inform their decisions. To this end, pragmatic RCTs could play a critical role in defining which interventions should be rec- ommended or prioritized.
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The inclusion of real world evidence in clinical development planning

The inclusion of real world evidence in clinical development planning

Background: When designing studies it is common to search the literature to investigate variability estimates to use in sample size calculations. Proprietary data of previously designed trials in a particular indication are also used to obtain estimates of variability. Estimates of treatment effects are typically obtained from randomised controlled clinical trials (RCTs). Based on the observed estimates of treatment effect, variability and the minimum clinical relevant difference to detect, the sample size for a subsequent trial is estimated. However, data from real world evidence (RWE) studies, such as observational studies and other interventional studies in patients in routine clinical practice, are not widely used in a systematic manner when designing studies. In this paper, we propose a framework for inclusion of RWE in planning of a clinical development programme.
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Riluzole: real-world evidence supports significant extension of median survival times in patients with amyotrophic lateral sclerosis

Riluzole: real-world evidence supports significant extension of median survival times in patients with amyotrophic lateral sclerosis

Abstract: Amyotrophic lateral sclerosis (ALS) is the commonest form of motor neuron disease and is a fatal, degenerative, multisystem disorder affecting upper and/or lower motor neurons in the motor cortex, brain stem, and spinal cord. ALS is characterized by progressive atrophy of associated bulbar, limb, thoracic, and abdominal muscles and supporting cells manifesting in a range of muscular symptoms such as weakness and wasting and eventual paralysis; the majority of patients will die from respiratory failure within 2–5 years of onset. Riluzole, a synthetic benzothiazole drug with glutamine antagonist activity, is indicated for the treatment of patients with ALS and is the only drug that has been shown to slow the course of the disease and extend survival in ALS patients. The original analyses, and subsequent meta-analyses, of data obtained from randomized controlled trials (RCTs) suggest that riluzole typically extends survival by 2–3 months and increases the chance of an additional year of survival by ~9%. However, published real-world evidence (RWE) from 10 clinical ALS databases indicates that riluzole therapy may afford much greater extension of survival, and improvements in median survival times of more than 19 months have been reported in the overall ALS patient population. This article will review the available data from RCTs and RWE on riluzole therapy.
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Creating and using real-world evidence to answer questions about clinical effectiveness

Creating and using real-world evidence to answer questions about clinical effectiveness

taken from hypothesis to creating evidence and uncertainty about the relevance of trial results to an ageing population with multimorbidity. RCTs in the therapeutic world are short – mainly because that is all that regulators require. As a result, rare complications of therapies may not be picked up in a trial that lasts 6–12 months. This is a very important function of RWE or post-marketing studies, i.e. the detection of unusual but significant drug-related issues –either on target or off tar- get. It is it not just the size of the numerator that is important but the possibility of tracking large numbers of patients over long periods that provides such useful information. Whilst RWE will most often not prove causality, it does enable a hypothesis to be raised and explored further. The potential advantages of using RWE are that costs are potentially much lower and retrospective RWE investigations can take place much faster, and the same may be true of some prospective investigations. Whilst RWD are inherently more messy, their advantage is that RWE studies will include people with multimorbidity, on usual prescribed doses, and standard patterns of adherence. Also, adverse effects of medication may not always be detected in the initial trials. 18,19
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Concordance to Guideline-recommended Statin Therapy: Real-world Evidence from India

Concordance to Guideline-recommended Statin Therapy: Real-world Evidence from India

Abstract: To evaluate concordance to the 2013 American College of Cardiology/American Heart Association (ACC/AHA) guideline on treatment of blood cholesterol for primary and secondary prevention of atherosclerotic cardiovascular disease (ASCVD) in India. Concordance to 2013 ACC/AHA guideline was assessed by retrospectively analyzing statin therapy prescribing practice as per ASCVD risk score in four statin-benefit groups in 23,295 patients aged 40-79 years from health facilities across India between 2017 and 2018. Mean (±SD) age of patients was 58.9 (±9.2) years; 62% were men; 60% (n=14,070) had clinical ASCVD. Among patients without ASCVD (n=7,122), 3.9% (n=278) had low-density lipoprotein- cholesterol (LDL-C) ≥190 mg/dL, 94.0% (n=6,694) had diabetes mellitus and 2.1% (n=150) patients had 10-year ASCVD risk ≥7.5%. Among 18,795 patients (81%) eligible for high-intensity statins, only 34% were concordant whereas 63% were treated with moderate-intensity statins. Among 2,290 patients eligible for moderate-intensity statins, 76% were concordant and 18% received high-intensity statins. Among patients with ASCVD (<75 years), 43% received high-intensity statins, 55% received moderate-intensity statins, while 2% did not receive statins. Among patients with diabetes and ASCVD risk <7.5%, 86% received moderate-intensity statins, but those with risk >7.5%, 83% remained under-treated. Most patients (82%) with LDL- C >190 mg/dL were prescribed with moderate-intensity statins. Most patients were receiving statins at dose non-concordant to 2013 ACC/AHA guideline, reflecting gaps in real-world practice of prescribing statins for primary and secondary prevention of ASCVD. Addressing care gaps and promoting compliance to optimize statin therapy will help reduce cardiovascular disease, especially in high-risk population among South Asians.
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<p>A real-world evidence of efficacy of palliative gastrectomy plus chemotherapy in metastatic gastric cancer patients</p>

<p>A real-world evidence of efficacy of palliative gastrectomy plus chemotherapy in metastatic gastric cancer patients</p>

commonly accepted fi rst-line chemotherapy regimen as the primary recommendation in China is fl uoropyrimidine (5-FU or capecitabine or S-1) plus platinum (cisplatin or oxaliplatin). 22–24 Taxane-based regimens are also adopted as fi rst-line chemotherapy; however, a high incidence of intolerance of taxane-based duplexes or triplexes has retarded their clinical application. 25–27 In the subgroup analysis, a survival advantage was derived from gastrect- omy in patients receiving taxane-based chemotherapy, whereas patients treated with fl uoropyrimidine plus plati- num might not gain a survival bene fi t from gastrectomy. The chemotherapy-regimen-dependent feature may be attributed to the negative conclusion of the REGATTA trial which used SP for postoperative chemotherapy. The present study preliminarily indicated that speci fi c che- motherapy regimens, such as taxane-based chemotherapy, were suitable for combination with palliative gastrectomy; however, further corroborative evidence is required.
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The real-world evidence on usage of bowel preparation agents before colonoscopy: a pan india study

The real-world evidence on usage of bowel preparation agents before colonoscopy: a pan india study

The key elements of the questionnaire were as follows: common indications for colonoscopy, medications before colonoscopy, commonly used agents for bowel preparation[r]

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Developing a multi-center clinical data mart of ACEI and ARB for real-world evidence (RWE)

Developing a multi-center clinical data mart of ACEI and ARB for real-world evidence (RWE)

We aimed to conduct various RWE studies on angiotensin-converting enzyme inhibitors (ACEI) and angiotensin II receptor blocker (ARB), commonly used as first- line therapy for blood press[r]

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Nalmefene: clinical and real world evidence in the treatment of alcohol dependence

Nalmefene: clinical and real world evidence in the treatment of alcohol dependence

Dopo avere informato la paziente circa l’opportunità di intraprendere un percorso di cura basato sulla ridu- zione del consumo alcolico come passaggio intermedio verso la completa aste[r]

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Creating and using real-world evidence to answer questions about clinical effectiveness

Creating and using real-world evidence to answer questions about clinical effectiveness

taken from hypothesis to creating evidence and uncertainty about the relevance of trial results to an ageing population with multimorbidity. RCTs in the therapeutic world are short – mainly because that is all that regulators require. As a result, rare complications of therapies may not be picked up in a trial that lasts 6–12 months. This is a very important function of RWE or post-marketing studies, i.e. the detection of unusual but significant drug-related issues –either on target or off tar- get. It is it not just the size of the numerator that is important but the possibility of tracking large numbers of patients over long periods that provides such useful information. Whilst RWE will most often not prove causality, it does enable a hypothesis to be raised and explored further. The potential advantages of using RWE are that costs are potentially much lower and retrospective RWE investigations can take place much faster, and the same may be true of some prospective investigations. Whilst RWD are inherently more messy, their advantage is that RWE studies will include people with multimorbidity, on usual prescribed doses, and standard patterns of adherence. Also, adverse effects of medication may not always be detected in the initial trials. 18,19
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<p>Nutrition Interventions Deliver Value in Healthcare: Real-World Evidence</p>

<p>Nutrition Interventions Deliver Value in Healthcare: Real-World Evidence</p>

Abstract: Value is a key guiding principle in healthcare, yet value is defined in varying ways by different stakeholders. In this paper, we review evidence of the health and financial tolls of malnutrition or poor nutrition, report positive results from recent nutrition-focused quality improvement programs in hospitals, and discuss clinical and policy implications of realizing best-practice nutrition care. Hospitalized patients with malnutrition diagnoses have up to two-fold greater hospital costs for care compared to inpatient stays for adequately nourished patients. By contrast, implementation of nutrition care programs for hospitalized adults (nutrition status screening, assessment and diagnosis of malnutrition, oral nutritional supplements provided when indicated) is associated with substantial per-patient, per-episode healthcare savings approaching $4,000. Improved nutrition care has also been associated with fewer complications and faster recovery (shortened lengths of stay, lower readmission rates). Nutrition care thus delivers value, which is evidenced by better patient outcomes at cost savings to healthcare systems.
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Real world data: an opportunity to supplement existing evidence for the use of long-established medicines in health care decision making

Real world data: an opportunity to supplement existing evidence for the use of long-established medicines in health care decision making

Abstract: Evidence from medication use in the real world setting can help to extrapolate and/ or augment data obtained in randomized controlled trials and establishes a broad picture of a medication’s place in everyday clinical practice. By supplementing and complementing safety and efficacy data obtained in a narrowly defined (and often optimized) patient population in the clinical trial setting, real world evidence (RWE) may provide stakeholders with valuable infor- mation about the safety and effectiveness of a medication in large, heterogeneous populations. RWE is emerging as a credible information source; however, there is scope for enhancements to real world data (RWD) sources by understanding their complexities and applying the most appropriate analytical tools in order to extract relevant information. In addition to providing information for clinicians, RWE has the potential to meet the burden of evidence for regula- tory considerations and may be used in approval of new indications for medications. Further understanding of RWD collection and analysis is needed if RWE is to achieve its full potential. Keywords: electronic health records, evidence-based medicine, real world data, real world evidence, randomized controlled trial
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<p>Real-World Adherence and Evidence of Subcutaneous and Sublingual Immunotherapy in Grass and Tree Pollen-Induced Allergic Rhinitis and Asthma</p>

<p>Real-World Adherence and Evidence of Subcutaneous and Sublingual Immunotherapy in Grass and Tree Pollen-Induced Allergic Rhinitis and Asthma</p>

Adherence to treatment is necessary for improving the ef fi cacy of treatment in patients with allergic respiratory dis- eases, reducing healthcare costs, and for minimizing the dis- ease ’ s burden on a patient ’ s life. 38 Overall, studies examining the pharmacoeconomics of AIT have been able to demonstrate cost savings. 45 However, if full treatment success is expected with only three continuous seasonal cycles, 27,28 early patient discontinuation considerably impacts the midterm cost- effectiveness of AIT, as the investment in year one or even year two without reaching year three could be considered a lost opportunity and non-recoverable cost. The discontinua- tion rate for SLIT treatments considerably depends on the number of visits to the physician per year. 46 Vita et al showed that SLIT discontinuation in the fi rst and second year was 15% and 18% for those with at least two annual visits, whereas patients with only one visit withdrew in 29% and 41% of all cases. 46 Based on the observed longer adherence of SCIT patients, cost-effectiveness comparisons based on real-world evidence probably favor SCIT. 22
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Clinical Utility of Advanced Microbiology Testing Tools

Clinical Utility of Advanced Microbiology Testing Tools

It is important that microbiologists conducting clinical utility studies consider which type of trial is most appropriate for the technology and endpoint they wish to study, as well as the ultimate stakeholder audience for the diagnostic technology. Whenever possible, studies designed to generate evidence of clinical utility should consider the needs of potential patient populations. For instance, hospital administrators often look for evidence generated in a system that closely resembles their organization, to provide confidence that the clinical utility demonstrated in a trial may translate to real-world experience. Also, many hospital systems may conduct their own trials with new diagnostic technology, to provide real-world evidence for improved workflow, decision making, and patient care resulting from the adoption of new technology. Published results are impactful for commercial payers when they can demonstrate clear changes in clinical decision making for patients that are representative of their plan members that were directly facilitated by the information provided by the test. Ideally, payers would like to see data demonstrating that these decisions correlated with positive clinical outcomes. However, payers are aware that many factors go into clinical out- comes beyond the diagnostic result, and therefore it may be sufficient to simply show a connection between the changes in decision making and potential clinical outcome. Once the audience is defined, the study designers also need to determine the appropriate level of evidence. The key considerations are the overall size of the trial and its representativeness. The size of the trial relates to the relative rarity of the events that occur. Statistical power calculations can be done to determine the number of patients needed relative to the delta in a change of endpoint. As the numbers of groups of patients and parameters to be measured increase, the number of patients needed to achieve a significant result will also increase. For instance, a study that measures only one patient group (e.g., high risk for respiratory conditions) with two diagnostic arms (e.g., SOC compared to multiplex panels) may be able to reach significance with 100 patients. A trial that was following three cohorts of patients (e.g., high risk, low risk, and the general population) and measuring three diagnostic arms (e.g., culture and phe-
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Calcipotriol/betamethasone dipropionate aerosol foam for the treatment of psoriasis vulgaris: case series and review of the literature

Calcipotriol/betamethasone dipropionate aerosol foam for the treatment of psoriasis vulgaris: case series and review of the literature

Abstract: An aerosol foam formulation of a once-daily, fixed-dose combination of a synthetic vitamin D 3 analog/synthetic corticosteroid (calcipotriol [Cal] 50 µg/g and betamethasone dipro- pionate [BD] 0.5 mg/g) has recently been introduced for the topical treatment of plaque psoriasis in adults. Data from several sources – randomized controlled trials, case reports (as highlighted in this review), and real-world evidence (RWE) – underscore the considerable and rapid clinical response, effectiveness, and favorable safety and tolerability of Cal/BD aerosol foam in mild-to- moderate psoriatic patients previously treated with class 3 or 4 topical corticosteroids, in patients unsatisfied with ongoing phototherapy in combination with topical therapy and in patients with moderate-to-severe psoriasis. In addition, our case series, considered together with other RWE, highlights that Cal/BD aerosol foam is more effective and with greater levels of patient prefer- ence and acceptability than comparator preparations. Thus, Cal/BD aerosol foam offers several treatment advantages, including relief of itch, and is an appropriate first-line topical therapy for consideration in patients with psoriasis of any severity.
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Science writing in the real world

Science writing in the real world

In the case of the ‘smaller’ organization, the marathon runner trains his body by stressing it. With a little stress the body responds and increases its capacity to tolerate more stress. If the stress is extreme and continued then the body fails – injury occurs. The athlete must walk the tightrope between over- and under-training. In life there are many applications of this principle of increasing our personal capacity by stressing the body thereby training it to improve performance. In the case of the ‘bigger’ organization, the African savanna and steppe have an amazing capacity to tolerate stress and disturbance, in the form of recurrent drought, fire, flood and herbivory. Earlier botanists working in South Africa considered that present grassland and savanna areas in the moist eastern regions were forest and scrub forest as little as 800 years ago, and it was only after the arrival of the Bantu that the woody vegetation got opened out by chopping and burning (Acocks 1975). However, it turns out that much of the savanna and steppe has been burnt every year or few by lightning or man and his predecessors, for millions of years – how else do we explain the biodiversity of the systems, the fire adaptations of many of the organisms, and the seeming need for the system to be burnt periodic- ally that its biodiversity not be lost (Ellery and Mentis 1992)? Of course it is common knowledge that forest does not ‘bounce back’ like steppe and savanna when defoliated, and at least in some parts of the world forest patches are refugia. But even here there are interesting contrasts. Along the northeast coast of South Africa, and going north into Mozambique, the dune forest has remarkable recovery capacity, converging on climax species composition after disturbance (bull-dozing or dune mining) in as little as 54 to 70 years (Mentis and Ellery 1998). Perhaps unlike most forest regions, along the southeast coast of Africa chronic disturbance has been a feature over recent evolutionary and ecological time. Sea level has risen and fallen as a result of monoclinal titling and global warming and cooling, and tropical cyclones and fire have occurred with short return periods. In consequence, dunes have been built, vegetated and destroyed frequently, and the dune forest would be expected to be resilient to disturbance of this nature.
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Use of evidence and knowledge translation approaches facilitating co creation of evidence in public health

Use of evidence and knowledge translation approaches facilitating co creation of evidence in public health

CHC is a new health technology to treat Dupuytren's contractures. There has been a growing interest to use it. However, its efficacy has been reported to vary in previously published studies. There has been considerable debate in funding CHC injections by the UK's National Health Service. The reason for this has been the weaknesses in the study designs, such as the efficacy measurements employed and the use of multiple injections and also the lack of analysis and reporting of data by disease severity, treatment cycle and joint type in some studies. All these factors combined together made it difficult to establish clinical efficacy and cost-effectiveness with fewer or single injections. Publication 7 and 8 are an example which demonstrates its use in establishing the benefit of CHC injections to inform development of local health policy and inform clinical practise in Herefordshire, UK. Given the uncertainties around the CHC injection, rather than deny coverage for it or grant unlimited coverage, the published works by using CED, attempts to ensure that patients' access is not prevented but is managed in a coordinated way, while also generating additional evidence to reduce any uncertainty about value.
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Nietzsche Contra the Real World

Nietzsche Contra the Real World

In her preface to Of Grammatology (1976), Spivak locates Derrida’s text as a thread of the social text of the ‘philosophers of difference’ that includes Deleuze, Heidegger, Nietzsche, Spinoza, and Heraclitus. To read Nietzsche geographically is to inherit this tradition’s critique of the real world. But it is also to undo the very thought that such a tradition is of or for the West, European. To say this differently: the problem of Europe – the fact that it is violently reproduced through an essentially imperial division of the world (Said 1978; Wainwright 2005) – means that our reading of Nietzsche (and Heraclitus) should be postcolonial.
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Real World Computer Forensics

Real World Computer Forensics

After the discovery process has finished, the hard drive or computer is sent back to the respective person from which it came. It is then determined to what degree the evidence found will support the client’s claim. Sometimes the evidence is not conclusive enough and the computer forensics expert must make this known. While the computer forensics expert is working for a client, it is not always a guarantee that the discovery will yield information that is helpful. In fact, some information can actually hurt the client or not be of any legal use at all. At the end of the investigation it is important to remember that it is not the job of the computer forensics expert to convict or acquit the client based on what they have found; there job is to only tell the truth about the data and information collected. From that point, the burden of conviction falls on the jury or judge to decide the outcome of the entire case.
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