Health economic modelling in Cystic Fibrosis:A systematic review

Introduction: Cystic Fibrosis (CF) is a heritable chronic condition. Due to the genetic and progressive nature of CF, a number of interventions are available for the condition. In the United Kingdom (U.K.) average annual cost of CF treatment is between €49,000 to €76,000 (2012) per patient [1]. A review of health economic modelling studies is warranted to provide decision makers and researchers with an in depth understanding of modelling practices in CF and guidance for future research. Methods: Online searches were performed in the 5 databases, studies were included if they were: a) Model based economic evaluation for management of Cystic Fibrosis. Articles were restricted to English language only, but no restriction was applied on publication year. Results: Nine studies were reviewed, most were Markov cohort models. Models evaluated pharmaceutical interventions and drug adherence. Modelling structure was consistent across most articles and a range of sources were used to populate the models. Cost and utility data were based on different sources and elicitation methods respectively. The majority of models failed to incorporate significant health events which impact both cost and disease progression. Conclusion: In our review we observed a lack of, application of European Medicines Agency (EMA) guidelines for clinical trial endpoints, model structure justifications and lastly, health-related quality of life derived utility information around important clinical events. Future work around conceptual modelling of CF progression, utility valuation of significant health events and meeting EMA guidelines for trial reporting is encouraged

Evidence summaries: the evolution of a rapid review approach

<p>Abstract</p> <p>Background</p> <p>Rapid reviews have emerged as a streamlined approach to synthesizing evidence - typically for informing emergent decisions faced by decision makers in health care settings. Although there is growing use of rapid review 'methods', and proliferation of rapid review products, there is a dearth of published literature on rapid review methodology. This paper outlines our experience with rapidly producing, publishing and disseminating evidence summaries in the context of our Knowledge to Action (KTA) research program.</p> <p>Methods</p> <p>The KTA research program is a two-year project designed to develop and assess the impact of a regional knowledge infrastructure that supports evidence-informed decision making by regional managers and stakeholders. As part of this program, we have developed evidence summaries - our form of rapid review - which have come to be a flagship component of this project. Our eight-step approach for producing evidence summaries has been developed iteratively, based on evidence (where available), experience and knowledge user feedback. The aim of our evidence summary approach is to deliver quality evidence that is both timely and user-friendly.</p> <p>Results</p> <p>From November 2009 to March 2011 we have produced 11 evidence summaries on a diverse range of questions identified by our knowledge users. Topic areas have included questions of clinical effectiveness to questions on health systems and/or health services. Knowledge users have reported evidence summaries to be of high value in informing their decisions and initiatives. We continue to experiment with incorporating more of the established methods of systematic reviews, while maintaining our capacity to deliver a final product in a timely manner.</p> <p>Conclusions</p> <p>The evolution of the KTA rapid review evidence summaries has been a positive one. We have developed an approach that appears to be addressing a need by knowledge users for timely, user-friendly, and trustworthy evidence and have transparently reported these methods here for the wider rapid review and scientific community.</p

Evaluating Quality of Decision-Making Processes in Medicines' Development, Regulatory Review, and Health Technology Assessment : A Systematic Review of the Literature.

Introduction: Although pharmaceutical companies, regulatory authorities, and health technology assessment (HTA) agencies have been increasingly using decision-making frameworks, it is not certain whether these enable better quality decision making. This could be addressed by formally evaluating the quality of decision-making process within those organizations. The aim of this literature review was to identify current techniques (tools, questionnaires, surveys, and studies) for measuring the quality of the decision-making process across the three stakeholders. Methods: Using MEDLINE, Web of Knowledge, and other Internet-based search engines, a literature review was performed to systematically identify techniques for assessing quality of decision making in medicines development, regulatory review, and HTA. A structured search was applied using key words and a secondary review was carried out. In addition, the measurement properties of each technique were assessed and compared. Ten Quality Decision-Making Practices (QDMPs) developed previously were then used as a framework for the evaluation of techniques identified in the review. Due to the variation in studies identified, meta-analysis was inappropriate. Results: This review identified 13 techniques, where 7 were developed specifically to assess decision making in medicines' development, regulatory review, or HTA; 2 examined corporate decision making, and 4 general decision making. Regarding how closely each technique conformed to the 10 QDMPs, the 13 techniques assessed a median of 6 QDMPs, with a mode of 3 QDMPs. Only 2 techniques evaluated all 10 QDMPs, namely the Organizational IQ and the Quality of Decision Making Orientation Scheme (QoDoS), of which only one technique, QoDoS could be applied to assess decision making of both individuals and organizations, and it possessed generalizability to capture issues relevant to companies as well as regulatory authorities. Conclusion: This review confirmed a general paucity of research in this area, particularly regarding the development and systematic application of techniques for evaluating quality decision making, with no consensus around a gold standard. This review has identified QoDoS as the most promising available technique for assessing decision making in the lifecycle of medicines and the next steps would be to further test its validity, sensitivity, and reliability.Peer reviewedFinal Published versio

Pharmacoeconomics of obesity in China: a scoping review

Background: With the growing rate of obesity and associated chronic conditions in China, there is a need to assess the health and economic burdens of obesity and examine the effectiveness of pharmaceutical, medical, and comprehensive weight-loss interventions. Areas covered: This article reviewed publications retrieved from PubMed and Google Scholar during 2010–2020 on pharmacoeconomic studies related to overweight and obesity in China. We identified five cost-of-illness studies and four cost-effectiveness analyses of weight-loss interventions, including bariatric surgeries and a comprehensive intervention program. Expert opinion: There is a lack of pharmacoeconomic analyses of obesity in China. Existing studies have often taken the health system perspective without accounting for productivity loss. Cohort studies and studies based on electronic health records or claims data are needed to provide the epidemiologic parameters required for homegrown economic evaluations of the health and economic burdens of obesity in China, as well as the cost-effectiveness of interventions to reduce obesity and its sequela

Developing a preference-based utility scoring algorithm for the Psoriasis Area Severity Index (PASI)

Introduction It is challenging to identify health state utilities associated with psoriasis because generic preference-based measures may not capture the impact of dermatological symptoms. The Psoriasis Area Severity Index (PASI) is one of the most commonly used psoriasis rating scales in clinical trials. The purpose of this study was to develop a utility scoring algorithm for the PASI. Methods Forty health states were developed based on PASI scores of 40 clinical trial patients. Health states were valued in time trade-off interviews with UK general population participants. Regression models were conducted to crosswalk from PASI scores to utilities (e.g., OLS linear, random effects, mean, robust, spline, quadratic). Results A total of 245 participants completed utility interviews (51.4% female; mean age =45.3y). Models predicting utility based on the four PASI location scores (head, upper limbs, trunk, lower limbs) had better fit/accuracy (e.g., R2, mean absolute error [MAE]) than models using the PASI total score. Head/upper limb scores were more strongly associated with utility than trunk/lower limb. The recommended model is the OLS linear model based on the four PASI location scores (R2 = 0.13; MAE =0.03). An alternative is recommended for situations when it is necessary to estimate utility based on the PASI total score. Conclusions The recommended scoring algorithm may be used to estimate utilities based on PASI scores of any treatment group with psoriasis. Because the PASI is commonly used in psoriasis clinical trials, this scoring algorithm greatly expands options for quantifying treatment outcomes in cost-effectiveness analyses of psoriasis therapies. Results indicate that psoriasis of the head/upper limbs could be more important than trunk/lower limbs, suggesting reconsideration of the standard PASI scoring approach

The use of multiple sclerosis condition-specific measures to inform health policy decision-making: mapping from the MSWS-12 to the EQ-5D

The final version of this paper has been published in Multiple Sclerosis, 18 (6), June 2012 by SAGE Publications Ltd, All rights reserved. © It is available at: http:// msj.sagepub.com/Walking impairment has a major influence on the quality of life of people with multiple sclerosis (MS). The Multiple Sclerosis Walking Scale (MSWS-12) assesses the impact of MS on walking ability from the patient's perspective, but in its current form, is not amenable for use in many policy decision-making settings

Valuing Health-Related Quality of Life: An EQ-5D-5L Value Set for England

Objectives: Measures of patient-reported health are increasingly used in clinical and health system decisions, and the EQ-5D is one of the most widely used questionnaires. It is recommended by NICE and is widely used in clinical trials, as well as in population health surveys and the NHS PROMs programme. A new version, the EQ-5D-5L, is now available. The objective of this study is to establish how important different sorts of health problems are to overall quality of life, and to produce the set of scores (‘value set’) required to use EQ-5D-5L data in decision-making and priority setting in the English NHS. Design: The study design followed an international research protocol. Each participant valued 10 health states using a time trade-off approach and completed seven discrete choice tasks. The data are used to model values for all 3,125 states described by the EQ-5D-5L. Setting: England general population. Participants: Data were collected in face-to-face interviews with 996 adult members of the general public, selected at random from residential postcodes. The sample is broadly representative of the general population. Results: The data obtained from participants had good face validity. Problems with pain/discomfort and anxiety/depression were the most important factor in overall quality of life. Values ranged from -0.281 (for extreme problems on all dimensions) to 0.951. Conclusions: The value set reported here will have important implications for public decisions made using EQ-5D-5L data. There are considerably fewer states judged to be ‘worse than dead’ compared to the current EQ-5D value set (4.93%, compared with over one-third) and the minimum value is higher (-0.281 compared to -0.594). The results imply that QALY gains for interventions seeking to improve very poor health will be smaller using the EQ-5D-5L tariff, and may previously have been overestimated

Effects of an evidence service on community-based AIDS service organizations' use of research evidence: A protocol for a randomized controlled trial

<p>Abstract</p> <p>Background</p> <p>To support the use of research evidence by community-based organizations (CBOs) we have developed 'Synthesized HIV/AIDS Research Evidence' (SHARE), which is an evidence service for those working in the HIV sector. SHARE consists of several components: an online searchable database of HIV-relevant systematic reviews (retrievable based on a taxonomy of topics related to HIV/AIDS and open text search); periodic email updates; access to user-friendly summaries; and peer relevance assessments. Our objective is to evaluate whether this 'full serve' evidence service increases the use of research evidence by CBOs as compared to a 'self-serve' evidence service.</p> <p>Methods/design</p> <p>We will conduct a two-arm randomized controlled trial (RCT), along with a follow-up qualitative process study to explore the findings in greater depth. All CBOs affiliated with Canadian AIDS Society (n = 120) will be invited to participate and will be randomized to receive either the 'full-serve' version of SHARE or the 'self-serve' version (a listing of relevant systematic reviews with links to records on PubMed and worksheets that help CBOs find and use research evidence) using a simple randomized design. All management and staff from each organization will be provided access to the version of SHARE that their organization is allocated to. The trial duration will be 10 months (two-month baseline period, six-month intervention period, and two month crossover period), the primary outcome measure will be the mean number of logins/month/organization (averaged across the number of users from each organization) between baseline and the end of the intervention period. The secondary outcome will be intention to use research evidence as measured by a survey administered to one key decision maker from each organization. For the qualitative study, one key organizational decision maker from 15 organizations in each trial arm (n = 30) will be purposively sampled. One-on-one semi-structured interviews will be conducted by telephone on their views about and their experiences with the evidence service they received, how helpful it was in their work, why it was helpful (or not helpful), what aspects were most and least helpful and why, and recommendations for next steps.</p> <p>Discussion</p> <p>To our knowledge, this will be the first RCT to evaluate the effects of an evidence service specifically designed to support CBOs in finding and using research evidence.</p> <p>Trial registration</p> <p>ClinicalTrials.gov: <a href="http://www.clinicaltrials.gov/ct2/show/NCT01257724">NCT01257724</a></p

What is the evidence for the effectiveness, appropriateness and feasibility of group clinics for patients with chronic conditions? A systematic review

Background Group clinics are a form of delivering specialist-led care in groups rather than in individual consultations. Objective To examine the evidence for the use of group clinics for patients with chronic health conditions. Design A systematic review of evidence from randomised controlled trials (RCTs) supplemented by qualitative studies, cost studies and UK initiatives. Data sources We searched MEDLINE, EMBASE, The Cochrane Library, Web of Science and Cumulative Index to Nursing and Allied Health Literature from 1999 to 2014. Systematic reviews and RCTs were eligible for inclusion. Additional searches were performed to identify qualitative studies, studies reporting costs and evidence specific to UK settings. Review methods Data were extracted for all included systematic reviews, RCTs and qualitative studies using a standardised form. Quality assessment was performed for systematic reviews, RCTs and qualitative studies. UK studies were included regardless of the quality or level of reporting. Tabulation of the extracted data informed a narrative synthesis. We did not attempt to synthesise quantitative data through formal meta-analysis. However, given the predominance of studies of group clinics for diabetes, using common biomedical outcomes, this subset was subject to quantitative analysis. Results Thirteen systematic reviews and 22 RCT studies met the inclusion criteria. These were supplemented by 12 qualitative papers (10 studies), four surveys and eight papers examining costs. Thirteen papers reported on 12 UK initiatives. With 82 papers covering 69 different studies, this constituted the most comprehensive coverage of the evidence base to date. Disease-specific outcomes – the large majority of RCTs examined group clinic approaches to diabetes. Other conditions included hypertension/heart failure and neuromuscular conditions. The most commonly measured outcomes for diabetes were glycated haemoglobin A1c (HbA1c), blood pressure and cholesterol. Group clinic approaches improved HbA1c and improved systolic blood pressure but did not improve low-density lipoprotein cholesterol. A significant effect was found for disease-specific quality of life in a few studies. No other outcome measure showed a consistent effect in favour of group clinics. Recent RCTs largely confirm previous findings. Health services outcomes – the evidence on costs and feasibility was equivocal. No rigorous evaluation of group clinics has been conducted in a UK setting. A good-quality qualitative study from the UK highlighted factors such as the physical space and a flexible appointment system as being important to patients. The views and attitudes of those who dislike group clinic provision are poorly represented. Little attention has been directed at the needs of people from ethnic minorities. The review team identified significant weaknesses in the included research. Potential selection bias limits the generalisability of the results. Many patients who could potentially be included do not consent to the group approach. Attendance is often interpreted liberally. Limitations This telescoped review, conducted within half the time period of a conventional systematic review, sought breadth in covering feasibility, appropriateness and meaningfulness in addition to effectiveness and cost-effectiveness and utilised several rapid-review methods. It focused on the contribution of recently published evidence from RCTs to the existing evidence base. It did not reanalyse trials covered in previous reviews. Following rapid review methods, we did not perform independent double data extraction and quality assessment. Conclusions Although there is consistent and promising evidence for an effect of group clinics for some biomedical measures, this effect does not extend across all outcomes. Much of the evidence was derived from the USA. It is important to engage with UK stakeholders to identify NHS considerations relating to the implementation of group clinic approaches. Future work The review team identified three research priorities: (1) more UK-centred evaluations using rigorous research designs and economic models with robust components; (2) clearer delineation of individual components within different models of group clinic delivery; and (3) clarification of the circumstances under which group clinics present an appropriate alternative to an individual consultation