1,721,178 research outputs found
Do European regulatory measures accelerate national market access in Belgium? A retrospective analysis of medicines centrally authorised between 2015 and 2020
No full textBackground At the European level, several regulatory measures (ie, priority medicines (PRIME) scheme, accelerated assessment, conditional marketing authorisation and authorisation under exceptional circumstances) are in place with the aim to expedite the marketing authorisation process for medicines targeting unmet medical needs (UMNs). However, the potential impact of these measures on subsequent decisions regarding market access at the national level, and ultimately if medicines making use of these supporting measures reach the patient earlier, remains unclear. Objectives This study seeks to (1) assess the impact of such European regulatory measures on the number of successful applications and time to reimbursement of this group of medicines in the national context of Belgium and (2) evaluate the association between the application of European regulatory measures and Belgian measures (ie, early access pathways and managed entry agreements). Design A total of 322 medicines granted a European centralised marketing authorisation between 2015 and 2020, excluding generic products/biosimilars, were included in the study. For this set of medicines, data on European and Belgian regulatory and market access measures were extracted from the websites of the responsible European and Belgian authorities and completed with requested information up to December 2022. Regression analysis was used to assess the association between the application of European regulations and Belgian measures. Survival and regression analysis was used to test the impact of such regulatory measures on the time to and rate of reimbursement in Belgium. Results From the total sample (n=322), 34% (n=108) received a European regulatory measure, and also 34% (n=108) had a Belgian measure applied. Overall, 63% (n=202) of the total sample was submitted for reimbursement in Belgium, and of these, 83% (n=167) were reimbursed at the time of assessment. The median regulatory assessment time at the European level was approximately 14 months, while the median Belgian reimbursement assessment time was approximately 11 months. The study found that regulatory measures did not significantly impact the European or national assessment times or status. A significant reduction in European regulatory assessment time was observed only in the cases of the PRIME scheme (p=0.0087) and accelerated assessment (p<0.0001). The study also indicated a positive association (p=0.0019) between the application of European measures and the application of Belgian measures. However, this significant association was not found for specific measures individually, with the exception of the accelerated assessment (p<0.0001). Medicines undergoing accelerated assessment were more likely to also receive a Belgian measure. Conclusion This study shows that while European regulatory measures targeting UMNs often trigger corresponding actions in Belgium, this alignment does not necessarily shorten the time from regulatory submission to reimbursement. Lacking submission for reimbursement by pharmaceutical companies appears to be the most frequent reason for absent reimbursement in Belgium. European policy initiatives promoting timely market entry across member states could be crucial for improving patient access.Funding for this research was provided by IMI CARE. The CARE project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking (JU) under grant agreement No 101005077. The JU receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA and BILL & MELINDA GATES FOUNDATION, GLOBAL HEALTH DRUG DISCOVERY INSTITUTE, UNIVERSITY OF DUNDEE. The content of this publication only reflects the author’s view and the JU is not responsible for any use that may be made of the information it contains. This study was supported by the KU Leuven. Researcher ZC is SB PhD fellow at FWO - 1S52125N
Going Beyond Counting First Authors in Author Co-citation Analysis
Full text linkThe present study examines one of the fundamental aspects of author co-citation analysis (ACA) - the way co-citation
counts are defined. Co-citation counting provides the data on which all subsequent statistical analyses and mappings
are based, and we compare ACA results based on two different types of co-citation counting - the traditional type that
only counts the first one among a cited work's authors on the one hand and a non-traditional type that takes into
account the first 5 authors of a cited work on the other hand. Results indicate that the picture produced through this non-traditional author co-citation counting contains more coherent author groups and is therefore considerably clearer. However, this picture represents fewer specialties in the research field being studied than that produced through the traditional first-author co-citation counting when the same number of top-ranked authors is selected and analyzed. Reasons for these effects are discussed
Variations on the Author
Full text link“Variations on the Author” discusses two of Eduardo Coutinho’s recent films (Um Dia na Vida, from 2010, and Últimas Conversas, posthumously released in 2015) and their contribution to the general question of documentary authorship. The director’s filmography is characterized by a consistent yet self-effacing form of authorial self-inscription: Coutinho often features as an interviewer that rather than express opinions propels discourses; an interviewer that is good at listening. This mode of self-inscription characterizes him as an author who is not expressive but who is nonetheless markedly present on the screen. In Um Dia na Vida, however, Coutinho is completely absent form the image, while Últimas Conversas, on the contrary, includes a confessional prologue that moves the director from the margins to the center of his films. This article examines the ways in which these works stand out in the filmography of a director who offers new insights into the notion of cinematic authorship
Appropriate Similarity Measures for Author Cocitation Analysis
Full text linkWe provide a number of new insights into the methodological discussion about author cocitation analysis. We first argue that the use of the Pearson correlation for measuring the similarity between authors’ cocitation profiles is not very satisfactory. We then discuss what kind of similarity measures may be used as an alternative to the Pearson correlation. We consider three similarity measures in particular. One is the well-known cosine. The other two similarity measures have not been used before in the bibliometric literature. Finally, we show by means of an example that our findings have a high practical relevance.information science;Pearson correlation;cosine;similarity measure;author cocitation analysis
Het ontwikkelen van een beslishulp voor patiënten om gedeelde besluitvorming in borstkanker te verbeteren.
No full textDuring the last two decades, shared decision making (SDM) has been increasingly applied in multiple health care decision contexts. Within the model of SDM, a patient and physician share information regarding the benefits and risks of potential options and personal preferences. They discuss the patient's preferences for each of the available options and discuss the patient's desire for involvement in decision making. Finally, they make or defer a decision and arrange follow-up if applicable. SDM is the preferred model for medical decision making in case of preference-sensitive decisions. Within these decisions, two or more options are available, none of which is clearly superior. To facilitate the process of SDM, patient decision aids (PtDAs) can be applied. The aim of a PtDA is threefold: they make the decision explicit and provide evidence-based information on the potential options, they help patients clarify their values and preferences for the different options and they support patients to communicate their preferences to their health care provider. PtDAs exist in various formats, ranging from paper-based brochures to interactive digital applications, using audio and video elements. The International Patient Decision Aids Standards Collaboration has established a checklist for the development of PtDAs, based on 12 quality domains, covering content, development process and effectiveness of the PtDA. Internationally developed PtDAs have proven to increase patients' knowledge and accuracy of risk perception, and decrease their decisional conflict. There are multiple PtDAs available in the disease areas of breast, prostate and lung cancer, osteoarthritis and osteoporosis, end of life decisions and cholesterol-related diseases. The variety in disease areas highlights that preference-sensitive decisions occur in many different health care decision contexts. The disease area of hormone-sensitive breast cancer is particularly suitable for the implementation of a PtDA. Patients receiving adjuvant endocrine therapy undergo a very long treatment that often causes a high impact on quality of life, resulting in suboptimal treatment adherence. The available treatments, tamoxifen or aromatase inhibitors, may cause adverse events such as increased risk of blood clots or joint and muscle pain, respectively. The efficacy of both treatments is comparable, although aromatase inhibitors are considered to be the most effective option. As treatment impact varies considerably between patients, a PtDA can help to tradeoff potential benefits and adverse events to determine the optimal therapy for individual patients. Although the practice of SDM and the use of PtDAs is internationally recognized, there are currently only very few PtDAs available in Belgium and there are no examples available for patients with breast cancer.
This PhD project aimed to improve SDM for Belgian patients with hormone-sensitive breast cancer by developing a PtDA for decisions regarding switching or continuing adjuvant endocrine therapy after 2-3 years. This PtDA should meet the needs of both patient and physician, by informing patients on the available decision options, eliciting their preferences for these options and supporting them to communicate their preferences to their treating physician. In order to meet this aim, four specific objectives were identified.
The first objective was to compare the process of decision making between patients and consumers and to identify innovative aspects of preference elicitation methods from the consumer research field. A literature review showed that the decision process between patients and consumers is highly comparable and identified five concepts from the consumer research field that might improve preference elicitation in healthcare. Preference elicitation methods that resemble real-life decision making as closely as possible, for example by providing time for self-reflection, are likely to generate the most accurate results.
The second objective was to assess the needs of both patients and physicians regarding a PtDA for breast cancer decision making. Four focus groups with 21 patients and five individual interviews with breast cancer specialists indicated that patients currently experience little involvement in their treatment decision making. Patients furthermore indicated a high need for information regarding treatment options, especially quantitative information on treatment benefits and risks. The breast cancer specialists acknowledged that SDM has become more important during the last decade but stated that it remains unclear how to implement this in clinical practice. Decisions regarding adjuvant endocrine therapy were deemed ideal for a PtDA intervention by both stakeholders as these treatments may have a high impact on quality of life and deliver limited benefits. Beneficial PtDA features were identified, such as the possibility to write down questions or to rate the impact of adverse events on daily life.
The third objective was to develop an interactive, online PtDA for patients with hormone-sensitive breast cancer switching adjuvant endocrine treatment. The combination of an in-depth literature review and stakeholder interviews were used to determine the content and design of the PtDA. Five attribute categories were identified using this approach: efficacy, adverse events, use, impact on quality of life and mechanism of action. Both patients and physicians rated potential attributes to determine the final selection, which included breast cancer mortality, risk of recurrence, treatment duration, joint and muscle pain, osteoporosis and increased thrombosis risk. The developed prototype PtDAs consists of three consecutive modules; an information module aiming to educate patients, a scenario-based module that will help patients to clarify potential impact on their everyday life and an adaptive conjoint analysis exercise to elicit patients' preferences for various treatment characteristics.
The final objective was to test the developed PtDA in a two-stage process. First, alpha testing in a research setting yielded an average usability score of 78.75 out of 100 using the System Usability Scale. Furthermore, content and lay-out were scored 8.9 and 8.5 out of 10 respectively by 11 patients; and quality, completeness and lay-out were scored 8.4, 8.4 and 8.2 out of 10 respectively by five health care professionals. Qualitative feedback was gathered by applying cognitive interviewing while using the PtDA and a short interview afterwards. Next, after implementing the feedback received during alpha-testing, beta testing in a clinical setting was performed using a pilot trial. Nine patients tested the PtDA in the week before their planned follow-up consultation at the university hospital of Leuven. The effect of the PtDA was assessed by determining the impact on the quality of the decision process and the decision itself using the following constructs: knowledge, values-choice agreement, feeling informed, feeling clear about values, discussing goals with health care providers, and being involved. Patient knowledge increased from 5.33 before using the PtDA to 7.78 afterwards. Decisional conflict was low after the consultation, with a score of 18.06. One month after the consultation, decisional conflict had increased significantly, with a score of 41,67. This might indicate the need for a broader support framework for patients by providing information and support them to discuss their preferences over a longer time period. Making the PtDA available after the consultation and planning follow-up consultation when needed could be potential solutions. The extent to which SDM was applied during the consultation was assessed using the SDM-9 item questionnaire. The average score of 20 out of a possibly 45 indicated that no real SDM took place, highlighting that patient directed interventions only, are not sufficient to implement SDM in clinical practice. Usability was again assessed using the System Usability Scale, resulting in a mean score of 71.25. Finally, patient preferences elicited for different treatment characteristics revealed high variability between patients, with a total of five different attributes being selected as 'the most important one' by seven patients. These results indicate the need to discuss individual treatment preferences during consultations.
Based on the information gained through the different chapters, recommendations are formulated for the future development of PtDAs in Belgium and the implementation of SDM in clinical practice.
The first set of recommendations is related to the development process of PtDAs. As the current use and development of PtDAs is very limited in Belgium, more research in other disease areas is needed to truly enable patient centered care. Stakeholder input has proven to be indispensable during the development process. Therefore, stakeholder opinions regarding PtDA design and content should be assessed using a combination of qualitative and quantitative approaches. Different formats or visual displays should be tested to meet the specific needs of the target group. Interactive, online applications offer a range of advantages regarding implementation and use. Video and audio materials can facilitate the learning process for users. Interactive features such as content control or the use of narratives may improve decision making. Furthermore, explicit preference elicitation methods that simulate real-life decision making may improve value clarification. The generated preferences weights can open up the discussion during a consultation. More research is required to facilitate optimal implementation in Belgium. The implementation strategy for a PtDA should already be considered during the development process. A centralized platform, either for the whole of Belgium or for Flanders, may provide easy access to PtDAs. However, not all patients are familiar with an online environment, nor does everyone have internet access. The needs of the target group should be assessed to identify the best implementation approach to allow for patient-centered care.
The second set of recommendations is developed to improve SDM in Belgium. The main recommendation here is to create awareness for SDM and to support implementation in clinical practice. A combination of clinical guidelines and practical measures such as logistic and financial support are needed. More research is needed to identify and address the barriers of Belgian health care professionals for SDM implementation. An example can be financial support to reimburse physicians for the time spent on the conduct of true SDM, for example in analogy to the compensation pharmacists can receive for performing counseling on the use of diabetes or asthma medicines. Moreover, financial support will be required to stimulate research on the development of PtDAs and the implementation of SDM. Furthermore, initiatives from various stakeholders and organizations should be harmonized in one collective approach. We should capitalize on the knowhow built up during international research, especially from countries with a comparable health care system such as the Netherlands. Care pathways in both primary and secondary care might need to be revised, to allow patients the necessary time to inform themselves and participate in decision making. More quality measures are needed to monitor the extent of SDM in clinical practice and to improve where needed. Another important recommendation is to apply trainings in SDM for health care professionals. If we truly want to implement SDM in routine clinical practice, a change of culture and mindset is needed. Only focusing on patient directed interventions will not suffice to accomplish this. Finally, public awareness for the right to engage in SDM should be raised. Patients should know where to find relevant information regarding their medical condition and available options at all times. More patient directed interventions such as PtDAs should be made available, either by developing new interventions or by translating and adapting PtDAs from the international scene.
By implementing these recommendations, we could truly start our journey towards patient-centered care.status: Publishe
Naar een aangepast regelgevend kader voor bacteriofaag therapie
No full textSummary
The worldwide emergence of antibiotic resistant bacteria and constraints to investment in potential solutions may eventually lead to a return to the pre-antibiotic era. As industries’ antibiotic pipeline is virtually dry and infectious diseases are steadily on the increase, the use of bacteriophages (bacteriophages are bacterio-specific viruses) to kill bacteria can be considered as a valuable option. Bacteriophages (meaning “bacteria-eaters”) are the bacteria’s natural enemies. In combination with or as substitute for antibiotics, bacteriophage therapy could be a therapeutic option in the eradication or control of bacterial colonization/infections. Bacteriophages can be considered as self-amplifying therapeutic products. By setting up a screening system for the circulating noxious bacteria and their respective bacteriophages it will be possible to obtain the right bacteriophage against any emerging pathogen.
Bacteriophages were discovered independently during World War I by the French-Canadian biologist Felix d’Herelle and by the English microbiologist Frederick Twort. d’Herelle developed a commercial laboratory in Paris that produced and distributed bacteriophage preparations against various bacterial infections. In the 1930s, therapeutic bacteriophages were also marketed in the United States by major pharmaceutical companies. The advent of antibiotics relegated bacteriophage therapy to complete obscurity in most of the Western world.
This PhD project aims at contributing to the creation of a dedicated European regulatory framework that makes the smooth re-introduction of bacteriophage therapy in the European Union possible. The research hypothesis is that final reflections and proposals, specifically designed in relation to bacteriophage therapy, will offer new and usable insights to all stakeholders involved. This research (11 studies) resulted in 14 international scientific publications that form the core of this thesis manuscript.
Chapter 1 of this manuscript is written as a general introduction and describes the natural bacteriophage, the problem of bacterial resistance development to antibiotics, the potential of natural bacteriophages in tackling this problem, the history of bacteriophage therapy and the actual European medicinal product regulatory setting relevant to the therapeutic use of bacteriophages. This chapter also contains an overview of the PhD project, including the research objectives.
Chapter 2 investigates the actual European regulatory and intellectual property hurdles relevant to the re-introduction of bacteriophage therapy into the European Union. This chapter also describes what a small-scale production of a qualitative bacteriophage cocktail, meant for use in e.g. a clinical trial, could look like. These investigations (and their results) intrigued regulators, politicians and medical ethical committees. The regulatory discussion on the subject of “bacteriophage therapy” was opened.
Chapter 3 explains what an optimal regulatory pathway, tailored to bacteriophage therapy, could look like. It explains why bacteriophage therapy is probably best served by a tailor-made and sustainable bacteriophage therapy concept.
Chapter 4 compares the bacteriophage therapy concept (explained in Chapter 3) with other medicinal products and assesses the compatibility of this bacteriophage therapy concept with current attitudes of national and European regulatory agencies towards bacteriophage products. Chapter 4 also analyses if the European Advanced Therapy Medicinal Products (ATMPs) Regulation, an example of an existing adapted medicinal product legislative framework, could be tailored to also cater for flexible bacteriophage therapy concepts.
Chapter 5 proposes a dedicated European regulatory frame for bacteriophage therapy, including quality and safety requirements for sustainable bacteriophage therapy products. These requirements are consensus-requirements defined by 33 bacteriophage experts from 11 different countries. The proposed regulatory framework was validated during an international workshop that took place at the Belgian Royal Military Academy (Viruses of Microbes II, Brussels, Belgium).
Chapter 6 investigates all stakeholders’ moral responsibility in relation to the large-scale re-introduction of bacteriophage therapy into the European Union. Moral principles are investigated and moral arguments are formulated in an effort to motivate all stakeholders to take bacteriophage therapy seriously. Although the efficacy of bacteriophage therapy has not been proven according to the actual standards of the European Union, bacteriophage therapy can be considered to be an Ethically Justified Medical Therapy (EJMT) within the European Union.
Chapter 7 is written in the format of a concluding discussion summarizing legislative proposals that could work for the conceptual re-introduction of natural bacteriophage therapy into the European Union.status: Publishe
Dispelling the Myths Behind First-author Citation Counts
Full text linkWe conducted a full-scale evaluative citation analysis study of scholars in the XML research field to explore just how different from each other author rankings resulting from different citation counting methods actually are, and to demonstrate the capability of emerging data and tools on the Web in supporting more realistic citation counting methods. Our results contest some common arguments for the continued
use of first-author citation counts in the evaluation of scholars, such as high correlations between author rankings by first-author citation counts and other citation
counting methods, and high costs of using more realistic citation counting methods that are not well-supported by the ISI databases. It is argued that increasingly available digital full text research papers make it possible for citation analysis studies to go beyond what the ISI databases have directly supported and to employ more
sophisticated methods
koamabayili/VECTRON-author-checklist: VECTRON author checklist
No full textWe have done our best to complete the author checklist relating to the use of animals in the hut study. Note that the objective for the hut study was to evaluate the IRS treatment applications for residual efficacy against Anopheles mosquitoes, including the local An. coluzzii mosquito population. Cows were only used to attract mosquitoes into the huts and no tests were carried out directly on the cows. The author checklist is intended for use with studies where experiments are carried out on animals, which is why we have had such difficulty in completing this for the hut study, as many of the questions do not relate to how the cows were used
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