Good Research Practice Research Articles

CC3 COST-EFFECTIVENESS ANALYSIS OF VORETIGENE NEPARVOVEC VERSUS BEST SUPPORTIVE CARE IN PATIENTS WITH RPE65-MEDIATED INHERITED RETINAL DYSTROPHY: A FRENCH HEALTHCARE SYSTEM PERSPECTIVE

An economic model was developed to evaluate the cost-effectiveness of voretigene neparvovec (VN) compared to best supportive care (BSC) in individuals with RPE65-mediated inherited retinal dystrophy (IRD), from a French healthcare system perspective. Methods were based on HAS guidelines and international good research practices for modelling. A Markov model was used and included six health states (HS) based on AMA visual deficiencies classification: Moderate Visual Impairment (VI), Severe VI, Profound VI, vision limited to “counting fingers”, vision limited to “hand motion” to “no light perception” and death. A lifetime horizon was used. Transition probabilities were calculated based on the results from the VN phase III trial and a natural history study. The economic endpoints used in the model were blindness-free years (BFY) and quality-adjusted life years (QALYs). Utility data from a vignette study were used. Resource utilization and costs included: disease-related costs (diagnosis, mutation testing, paramedical care and follow-up), drug costs (acquisition, administration and adverse events), costs of medical transport and accommodation and death. Patients treated with VN stayed longer in better HS while patients in the BSC arm progressed more quickly to worse HS. The treatment with VN results in a gain of 11.7 BFY and 4.5 QALY versus BSC. The ICER for VN versus BSC was €51, 552 per BFY and €132, 607 per QALY. Deterministic and probabilistic sensitivity analyses generally showed consistency with base case findings. When additional scenarios were explored, ICERs were most sensitive to variations in the multi-state model parameters and duration of treatment effect. VN is the first gene therapy approved in RPE65-mediated IRD and represents a clinically significant advancement in the management of this disease. VN was associated with an important gain of QALYs versus BSC and can be considered a cost-effective therapy in this orphan disease.

PCN149 COST-EFFECTIVENESS ANALYSIS OF DABRAFENIB+TRAMETINIB VERSUS WATCHFUL WAITING IN THE ADJUVANT TREATMENT OF PATIENTS WITH RESECTED BRAF V600 POSITIVE STAGE III MELANOMA : A FRENCH PERSPECTIVE

A cost-effectiveness model was developed to estimate the ICER of dabrafenib+trametinib (D+T) compared to watchful waiting (WW) in BRAF+ adjuvant melanoma, from a French healthcare perspective. Methods were based on HAS guidelines and international good research practices for modelling. A non-homogeneous semi-Markov cohort model was used with states defined on recurrence and death, including: Relapse-free survival (RFS), locoregional recurrence, distant recurrence (1st line and 2nd line) and death. A lifetime horizon was used, from treatment initiation. Marginal parametric distributions were fitted on individual data from the COMBI-AD trial to extrapolate time spent in each health state. Distributions were distinguished between therapeutic classes for 1st line distant melanoma. Overall survival was corrected with general population mortality for locoregional melanoma and long-term survivors with distant melanoma. Health-state utilities were estimated using EQ-5D data from COMBI-AD. Resource utilization and costs included drug acquisition and administration, routine surveillance and management of adverse events, melanoma recurrences and end-of-life. In the COMBI-AD trial and after extrapolation, D+T showed significant advantage in reducing the risk of recurrence vs. WW (respectively 47% and 56% of patients developed a distant melanoma over lifetime). D+T was associated with lower costs (Δ=-20,140€) and higher QALYs (Δ=+1.68) than WW. Deterministic and probabilistic sensitivity analyses showed consistency with base case findings. Results were mainly sensitive to variations in extrapolation of the RFS and therapeutic patterns defined for each arm in metastatic setting. D+T represents a clinically significant advancement for adjuvant treatment of resected BRAF V600 mutation-positive high-risk stage III melanoma. D+T dominates WW with gains in QALYs and a lower cost. New melanoma adjuvant treatments including immune checkpoint inhibitors have shown great results vs. WW by delaying recurrences and increasing the proportion of patients cured. The relative positioning of these new strategies still needs to be defined.

Feminism as an Evolving Culture of Inclusion and Affirmation: A Review of Contemporary Feminist Research from Theory to Practice

In Contemporary Feminist Research from Theory to Practice, Dr. Patricia Leavy and Dr. Anne Harris (2019) translate feminist principles into good research practice to offer learners of all career stages a concise and lively blueprint for bringing feminism out of the realm of theory and into that of application. Their constant critical thinking and consistent attention to detail orient readers to feminism as a dynamic, continuously evolving culture of inclusion and affirmation.

What do we know about R&D spillovers and productivity? Meta-analysis evidence on heterogeneity and statistical power

As Mohnen (1996: 40) has indicated, research and development (R&D) externalities is a two-sided theoretical issue. Its ‘dark’ side concerns the under-investment problem caused by non-appropriability of R&D benefits. On the ‘bright’ side, R&D spillovers are a source of productivity gains. Both aspects have been invoked to justify public support for R&D investment directly and indirectly. To establish whether public support can be justified due to productivity gains from spillovers, we meta-analyse 983 productivity estimates for spillovers and 501 estimates for own-R&D from 60 empirical studies. Our findings indicate that the average spillover effect is: (i) positive but heterogenous and smaller than what is reported in most narrative reviews; (ii) usually smaller than that of own-R&D capital; (iii) too small to be practically significant when evidence with adequate statistical power is considered. Controlling for observable sources of heterogeneity and best-practice research, the meta-effect is insignificant in the full sample but significant and large among OECD firms/industries/countries. We discuss the implications of these findings for future research and public support for R&D investment.

Duplicate and salami publication: a prevalence study of journal policies.

Duplicate and salami publication are unethical, but are common practices with substantial consequences for science and society at large. Scientific journals are the 'gatekeepers' of the publication process. We investigated journal policies on duplicate and salami publication. In 2018, we performed a content analysis of policies of journals in the disciplines of 'epidemiology and public health' and 'general and internal medicine'. Journal policies were searched, extracted, coded and cross-checked. The associations of disciplinary categories and journal impact factors with journal policies were examined using Poisson regression models with a robust error variance. A total of 209 journals, including 122 in epidemiology and public health and 87 in general and internal medicine, were sampled and their policies investigated. Overall, 18% of journals did not have any policies on either practice, 33% only referred to a generic guideline or checklist without explicit mention about either practice, 36% included policies on duplicate publication and only 13% included policies on both duplicate and salami publication. Having explicit journal policies did not differ by journal disciplinary categories (epidemiology and public health vs general and internal medicine) or impact factors. Further analysis of journals with explicit policies found that although duplicate publication is universally discouraged, policies on salami publication are inconsistent and lack specific definitions of inappropriate divisions of papers. Gaps exist in journal policies on duplicate and salami publication, characterized by an overall lack of explicit policies, inconsistency and confusion in definitions of bad practices, and lack of clearly defined consequences for non-compliance. Scientific publication and the academic reward systems must evolve to credit good research practice.

Guidelines for Safeguarding Good Research Practice. Code of Conduct

Guidelines for Safeguarding Good Research Practice. Code of Conduct

Political Realism as Anti-scholastic Practice: Methodological Lessons from Muckraking Journalism

What does the trend of “realism” in political theory portend, if anything, for how social and political scientists do their work? We can best see where realism’s rubber hits the road by re-examining the methodological comparison between political science and political journalism, according to which the academic field has long harbored assumptions of its own superiority. When the comparison between these two approaches to knowledge about politics is explicitly made, political science is typically justified by reference to distinctive (and higher) purposes and methods. Here, we reconsider conventional assumptions by reconstructing the journalistic practices and methodological reflections of two early figures in the American muckraking tradition, Lincoln Steffens and R. S. Baker. While their purposes were similar to those upheld by advocates of a publicly engaged political science, their methods, somewhat more surprisingly, are also applicable to the academic profession. Several anti-scholastic lessons on method—relevant to qualitative, quantitative, and interpretive approaches alike—emerge from the muckrakers’ example. The realist movement in political theory is congruent with the proposition that political science’s superiority complex is less easily defended and more obstructive to good research practice than even the most civically engaged researchers commonly assume.

Meta-analytic review of infants' preferential attention to familiar and unfamiliar face types based on gender and race

Infants’ earliest attention shows prioritization of social and communicative signals – faces. Not all faces receive equal attention; infants show early preferential attention to some face types over others. Although the pattern is not consistent across studies, potentially because infant experience varies, sample sizes and effect sizes are small, and methodological parameters are inconsistent, infants seem to show dynamic preferential attention first to familiar or caregiver-like (e.g., female, own-race) and developmentally later to unfamiliar (e.g., male, other-race) face types. This meta-analysis clarifies infants’ attention to familiar (i.e., caregiver-like) and unfamiliar (i.e., not caregiver-like) face types across the first year of life. The combined results from 19 studies (76 samples) of infant visual preferential attention reveal greater attention to familiar over unfamiliar face types (M = 2.69%, SE = 1.01), which declines over the first year of life. Face comparison type (race or gender) and infant age significantly predicted preferential attention: attention to familiar over unfamiliar race faces declined significantly with increasing infant age. There was no significant age-related decline in infants’ preferential attention to familiar gender faces. Task parameters did not significantly predict infants’ preferential attention. With the high variability in methodological parameters and reporting in the field, we suggest best practices for research to clarify our understanding of infants’ early visual attention to faces.

Turning the tables: A university league-table based on quality not quantity.

Background: Universities closely watch international league tables because thesetables influence governments, donors and students. Achieving a high ranking in atable, or an annual rise in ranking, allows universities to promote their achievementsusing an externally validated measure. However, league tables predominantly rewardmeasures of research output, such as publications and citations, and maytherefore be promoting poor research practices by encouraging the "publish or perish"mentality. Methods: We examined whether a league table could be created based on goodresearch practice. We rewarded researchers who cited a reporting guideline, whichhelp researchers report their research completely, accurately and transparently,and were created to reduce the waste of poorly described research. We used theEQUATOR guidelines, which means our tables are mostly relevant to health andmedical research. We used Scopus to identify the citations. Results: Our cross-sectional tables for the years 2016 and 2017 included 14,408papers with 47,876 author affiliations. We ranked universities and included a bootstrapmeasure of uncertainty. We clustered universities in five similar groups in aneffort to avoid over-interpreting small differences in ranks. Conclusions: We believe there is merit in considering more socially responsiblecriteria for ranking universities, and this could encourage better research practiceinternationally if such tables become as valued as the current quantity-focused tables.

Striving for State of the Art with Paradigm Interplay and Meta-Synthesis: Purpose-oriented Network Research Challenges and Good Research Practices as a Way Forward

AbstractWith the growing amount and increasing heterogeneity of research on purpose-oriented networks (PONs) in the public sector, it is imperative to find a way to synthesize this research. Drawing on the varied research perspectives on PONs, we advance the idea of paradigm interplay and meta-synthesis as aspirations for the field and argue this is especially key if we want the study of PONs to inform practice. However, we recognize several challenges in the current state of the PON research that prevent the field from making strides in paradigm interplay and meta-synthesis. We discuss six challenges which we consider the most critical: different labels, differences across research foci, variation in measurement, the nestedness of networks, the dynamism of networks, and variation in the network context. We suggest six good research practices that could contribute to overcoming the challenges now so as to make integration of the research field more of a possibility in the future.

Quality management in research: management process of the laboratories of Moroccan faculties of sciences and techniques

The implementation of a quality management system including management tools in the industrial sector has several advantages. In this perspective, can we use quality management tools to improve the performance of scientific research in Morocco and reduce the gap noticed in this field between the latter and the industrialized countries? To evaluate the level of application of good quality management practices related to scientific research in Morocco, a questionnaire was established and administered to the research laboratories managers of the faculties of sciences and techniques at the national level. Further to this study, it turns out that the good quality management practices related to the laboratory management and research management are applied by a percentage of the laboratories varies between 8 % and 75 %. Regarding the perspectives related to the quality management, 96 % of the laboratories managers declared important or very important the implementation of a quality management system in the laboratory. However, only 17 % have a certification (according to ISO 9001) and/or an accreditation (according to ISO/IEC 17025) project for the next 3 years, considering that the lack of financial means and staff involvement are the first two obstacles for the implementation of such projects. In short, the implementation of good quality management practices in research has several advantages and requires first of all training in the specific standards linked to this area. It also requires the sensitization of managers to the appropriate tools for managing research that can be used and whose purpose is the continuous improvement in the activities.

Teaching Good Research Practices: Protocol of a Research Master Course

The current crisis of confidence in psychological science has spurred on field-wide reforms to enhance transparency, reproducibility, and replicability. To solidify these reforms within the scientific community, student courses on open science practices are essential. Here we describe the content of our Research Master course “Good Research Practices” which we have designed and taught at the University of Amsterdam. Supported by Chambers’ recent book The 7 Deadly Sins of Psychology, the course covered topics such as QRPs, the importance of direct and conceptual replication studies, preregistration, and the public sharing of data, code, and analysis plans. We adopted a pedagogical approach that: (a) reduced teacher-centered lectures to a minimum; (b) emphasized practical training on open science practices; and (c) encouraged students to engage in the ongoing discussions in the open science community on social media platforms.

English as a Lingua Franca in the Academic Context: The Role of University Language Centres

English is now the dominant language in the higher education sector in Europe, as can be seen from objective indicators such as the growing number of degree programmes which use English as a medium of instruction. Teaching subjects through the medium of English (English Medium Instruction or EMI) may raise a series of questions and challenges for what concerns the accreditation and training of teaching staff. University language centres can play a fundamental role in helping the academic staff from feeling pushed towards teaching through English without a great deal of training and preparation. The present contribution reports on the activities implemented by the Language Centre in order to assist those members of the teaching staff (lecturers) who are confronted with the inherent challenges of teaching through English on a daily basis, and also highlights possible future avenues of research and instances of best practices.

Engaging Stakeholders, from Inception and Throughout the Study, is Good Research Practice to Promote use of Findings

The need for research-informed programming and policy making is well established. However, there is limited evidence that, when researchers actively promote utilization of research findings, stakeholders use such findings for decision making in low- and middle-income countries (LMIC). A common barrier for research uptake in LMIC is that researchers focus on passive dissemination of final findings as the primary vehicle to affect research uptake. A more active approach to facilitating research utilization (RU) is necessary. Project SOAR, a six-year USAID-funded operations research project, recognized this gap and developed an approach to include the end data users in the research process from inception to final results dissemination. In this commentary, we make recommendations for active facilitation of research uptake using emerging lessons from SOAR’s RU process that focuses on ongoing engagement of stakeholders throughout the life of the study.

Circular economy and real estate: the legal (im)possibilities of operational lease

Purpose A paradigm in circular economy (CE) is that suppliers retain ownership of their products and materials, and that the users “only” pay for services. In many legal systems, however, elements incorporated in a building are considered to be fixtures, and therefore legally part of the building. This means that ensuring multi-cyclic behaviour of individual building elements (e.g. the facade or a window) is not so evident. This paper explores, from the perspective of Dutch law, how to secure the ownership of the supplier or to find alternatives within the existing system of property law. Design/methodology/approach The authors performed a literature review of both CE and (Dutch) property law. The results of these reviews are discussed and illustrated by legal case studies. Findings The options principally advocated within CE to retain ownership of building parts leave legal uncertainties and do not offer a solid basis for the development of circular business models, especially considering immovables and fixtures. For these categories, buy-back and take-back contracts, and models for reuse and recycling seem more promising. Research limitations/implications The research is limited to a literature review. Although the legal principles discussed in this paper are valid for both civil and common law systems, and similar findings might, therefore, be expected internationally, this study focused on the specific Dutch legal context. Comparative legal research and research of best practices in the building industry is needed to test the applicability of the findings in an international context. Practical implications Following the findings, CE initiatives within real estate and the construction industry should focus on alternative implementations of the operational lease concept, taking into account CE’s ambitions to reduce the extraction of raw materials. Originality/value At the moment the challenges that property law poses CE, real estate and operational lease are hardly discussed within the literature. This paper explores this gap.

Measurement Equivalence of Patient-Reported Outcome Measures Migrated to Electronic Formats: A Review of Evidence and Recommendations for Clinical Trials and Bring Your Own Device.

A growing number of clinical trials employ electronic media, in particular smartphones and tablets, to collect patient-reported outcome data. This is driven by the ubiquity of the technology, and an increased awareness of associated improvements in data integrity, quality and timeliness. Despite this, there remains a lingering question relating to the measurement equivalence of an instrument when migrated from paper to a screen-based format. As a result, researchers often must provide evidence demonstrating the measurement equivalence of paper and electronic versions, such as that recommended by the ISPOR ePRO Good Research Practices Task Force. In the last decade, a considerable body of work has emerged that overwhelmingly supports the measurement equivalence of instruments using screen-based electronic formats. Our review of key works derives recommendations on evidence needed to support electronic implementation. We recommend application of best practice recommendations is sufficient to conclude measurement equivalence with paper PROMs. In addition, we recommend that previous usability evidence in a representative group is sufficient, as opposed to per-study testing. Further, we conclude that this also applies to studies using multiple screen-based devices, including bring-your-own-device, if a minimum device specification can be ensured and the instrument is composed of standard response scale types.

Privacy versus open science

Pervasive internet and sensor technologies promise to revolutionize psychological science. However, the data collected using these technologies are often very personal—indeed, the value of the data is often directly related to how personal they are. At the same time, driven by the replication crisis, there is a sustained push to publish data to open repositories. These movements are in fundamental conflict. In this article, we propose a way to navigate this issue. We argue that there are significant advantages to be gained by ceding the ownership of data to the participants who generate the data. We then provide desiderata for a privacy-preserving platform. In particular, we suggest that researchers should use an interface to perform experiments and run analyses, rather than observing the stimuli themselves. We argue that this method not only improves privacy but will also encourage greater compliance with good research practices than is possible through open repositories.

Neuro-assessment of leadership training

ABSTRACT The rapid growth of coaching practices has outpaced the current best-practice empirical research and created a difficult landscape to navigate for coaches and consumers alike. To overcome these challenges the present study proposed to employ the neuro-screening measure to develop and monitor an individually tailored coaching intervention for training senior-managers’ inspirational leadership. We have used the quantitative electroencephalogram (qEEG) screening to build the individual profiles of every coachee to assess their baseline (trait) characteristics in order to develop the coaching interventions to enhance effective and minimise ineffective behaviour. The qEEG-screening profile resulted in nine metrics characterising different traits and features of every coachee. Based on these profiles individualised 4-month coaching programmes were suggested to coachees. The results indicated that participation in the individually designed 4-month coaching programme was associated with significant improvement (70.7%) or optimisation (55.6%) of metrics in the 71.7% of coachees. This was paralleled by reduced scores in independent Beck Anxiety Inventory and Beck Depression Inventory scales. We concluded that the employment of qEEG-screening profiling allows coaches and trainers to assess deep dynamic neurological mechanisms that underpin baseline traits and features essential for effective, flexible and sustainable leadership, as well as track their development over time.

PSY1 CONCEPTUALIZING A FRAMEWORK FOR MODELING SYSTEMIC LUPUS ERYTHEMATOSUS

Systemic lupus erythematosus (SLE) has a complex and heterogeneous natural history. Our aim was to develop a conceptual model of SLE that characterizes the relationships between short- and long-term outcomes that could be applied in future economic evaluations and health technology assessments of new therapies. As per the ISPOR-SMDM Modeling Good Research Practices Task Force guidelines, a targeted literature review was conducted to identify any previously developed conceptual or economic models in SLE. A steering committee of clinical and economic experts was convened to review existing model frameworks and brainstorm a new conceptual model, building on revision and refinement of prior models. The relationships between patient characteristics, disease activity, commonly used treatments, organ damage, health-related quality of life (HRQoL), and mortality were all considered by the committee, as well as the likely data available to parameterize the model in the future. Based on the existing literature and consensus among committee members, the key components of the conceptual model included disease activity, corticosteroid use, and organ damage by individual system. Relationships not present in prior models included stratifying short-term disease activity by level (e.g., low, moderate, high)and corticosteroid use influencing subsequent disease activity. Higher disease activity levels (i.e., flare) would trigger higher short-term corticosteroid use and increased risk of long-term organ damage. Higher doses of corticosteroids should decrease disease activity in the short-term, while raising the risk of long-term organ damage. Relevant covariates identified included demographics, age at onset, cardiovascular comorbidities, baseline organ damage, history of lupus nephritis, and anti-malarial/other immunosuppressant use. Long-term outcomes include organ damage, mortality, HRQoL, and costs. By linking short- and long-term outcomes, this conceptual model provides a foundation on which to conduct future economic evaluations of new SLE therapies. The next step is to conduct a model validation using real world data.

Paper and electronic versions of HM-PRO, a novel patient-reported outcome measure for hematology: an equivalence study.

Aim: To determine measurement equivalence of paper and electronic application of the hematological malignancy-patient-reported outcome (HM-PRO), a specific measure for the evaluation of patient-reported outcomes in HMs. Patients & methods: Following International Society of Pharmacoeconomics and Outcomes Research ePRO Good Research Practice Task Force guidelines, a total of 193 adult patients with different HMs were recruited into a multicenter prospective study. The paper and the electronic version of the instrument were completed in the outpatient clinics in a randomized crossover design with a 30 min time interval to minimize the learning effect. Those who completed the paper version first, completed the electronic version after 30min and vice versa. Instrument version and order effects were tested on total score of the two parts of the HM-PRO (Part A: quality of life and Part B: signs & symptoms) in a two-way ANOVA with patients as random effects. Intraclass correlation coefficients (95% CI) and Spearman's rank correlation coefficients were used to evaluate test-retest reliability and reproducibility. The effects of instrument version and order were tested on total score of the two parts of HM-PRO. Results: The questionnaire version and administration order effects were not significant at the 5% level. There were no interactions found between these two factors for HM-PRO (Part A [quality of life]; p =0.95); and (part B [signs and symptoms]; p =0.72]. Spearman's rank correlation coefficients were greater than 0.9, and intraclass correlation coefficients ranged from 0.94 to 0.98; furthermore, the scores were not statistically different between the two versions, showing acceptable reliability indexes. Noteworthy, the difference between the completion time for both paper (mean = 6:38min) and electronic version (mean = 7:29min) was not statistically significant (n=100; p =0.11). Patients did not report any difficulty in completing the electronic version during cognitive interviews and were able to understand and respond spontaneously. Conclusion: Measurement equivalence has been demonstrated for the paper and electronic application of the HM-PRO.