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Simple Summary: Non-small cell lung cancer (NSCLC) is a leading cause of cancer-related deaths in the United Kingdom. Advances in treatments such as chemotherapy, targeted therapies, and immunotherapies have significantly improved outcomes for patients. However, these treatments have raised concerns about their potential heart-related side effects. This study investigated this issue using the UK's Yellow Card System, which collects data on drug side effects. The study examined adverse event reports for 56 NSCLC drugs, finding 128,214 adverse events, of which 6133 were cardiovascular issues. Alectinib emerged as the drug with the highest reported risk of cardiovascular problems, aligning with findings from other databases like the WHO's VigiBase. Although alectinib has been shown to significantly improve the survival rate of cancer patients, continuous monitoring is essential to ensure a benefit–risk balance can be maintained. Background: Non-small cell lung cancer (NSCLC) is a predominant cause of oncological mortality in the United Kingdom. There is a diverse spectrum of therapeutic options available, such as chemotherapies, targeted therapies and immunotherapies, which have significantly advanced patient prognoses. However, despite these advancements, there is an escalating concern regarding the potential cardiotoxic effects associated with these treatments. Objectives: This study aimed to explore the association between non-small cell lung cancer treatments and cardiotoxicity. Methods: A pharmacovigilance study was conducted utilising the UK Yellow Card System. The proportional reporting ratio (PRR) and reporting odds ratio (ROR) were calculated to detect signals. Results: Among the 56 shortlisted NSCLC drugs, the total number of adverse events reported was 128,214 with 6133 reports being cardiovascular adverse reactions. Among all the drugs analysed, alectinib demonstrated the highest ROR and PRR values, indicating the strongest signal for potential cardiovascular adverse events. Conclusions: This result was comparable to previous studies which also detected a signal of alectinib related to cardiovascular events using the WHO pharmacovigilance database, VigiBase. However, clinical studies demonstrated that alectinib largely improved progression-free survival (PFS) and overall survival in patients. Therefore, it is important to continue monitoring the real-world use of alectinib, so that a benefit–risk balance can be maintained. [ABSTRACT FROM AUTHOR]

In this study, the European Academy of Dermatology and Venereology (EADV) Task Forces on Quality of Life and Patient‐Oriented Outcomes and Urticaria and Angioedema has examined the Health‐Related Quality of Life (HRQoL) measurement in the treatment of urticaria. The Dermatology Life Quality Index was the most frequently used HRQoL instrument in clinical trials on urticaria. Many reports of clinical trials of urticaria gave no exact numeric results related to HRQoL changes, making clear conclusions and comparisons with other studies impossible. The interpretation of HRQoL impairment data is more difficult when assessed by instruments without severity stratification systems. The minimal clinically significant difference (MCID) is a more clinically oriented and relevant parameter than depending on statistically significant changes in HRQoL scores. Therefore, using HRQoL instruments with established MCID data in clinical trials and clinical practice is preferred. [ABSTRACT FROM AUTHOR]

Aim: To measure the impact of type 1 and 2 diabetes mellitus (T1D and T2D) on the QoL of a person's family members/partner and assess if there is any difference in family impact. Methods: A cross‐sectional study, recruited online through patient support groups, involved UK family members/partners of people with diabetes completing the Family Reported Outcome Measure‐16 (FROM‐16). Results: Two hundred and sixty‐one family members/partners (mean age = 57.9 years, SD = 13.8; females = 68.2%) of people with diabetes (mean age = 57.7, SD = 20.6; females = 38.3%; T1D n = 100; T2D n = 161) completed the FROM‐16. The overall FROM‐16 mean score was 10.47, SD = 7.8, suggesting a moderate effect on the QoL of family members of people with diabetes. A quarter (24.5%) of family members experienced a 'very large effect' or 'extremely large effect' on their QoL. The family impact of T1D (mean FROM‐16 = 12.61, SD = 7.9) was greater than that of T2D (mean = 9.15, SD = 7.5, p < 0.01), with being 'female' and 'parents of children and adolescents' rendered as significant predictors of greater impact. Family members of T2D had a lower risk of experiencing a high family impact (FROM‐16 score >16) compared with T1D (RR 0.561, 95% CI 0.371–0.849). Conclusions: Compared to T2D, family members of T1D experience a greater impact on their QoL, particularly those caring for children and adolescents. These findings have clinical and resource implications, indicating a need to assess this impact as a part of routine diabetes care to support impacted family members. The FROM‐16 could assess this impact in routine practice and further facilitate referral of family members to appropriate support services. [ABSTRACT FROM AUTHOR]

Background Primary endpoint measures in clinical trials are typically measures of disease severity, with patient-reported outcome measures (PROMs) relegated as secondary endpoints. However, validation of some PROMs may be more rigorous than that of disease severity measures, which could provide support for a primary role for PROMs. Objectives This study reports on 24 peer reviewed journal articles that used the Dermatology Life Quality Index (DLQI) as primary outcome, derived from a systematic review of randomized controlled trials (RCTs) utlizing DLQI, covering all diseases and interventions. Methods The study protocol was prospectively published on the PROSPERO database, and the study followed PRISMA guidelines. Searches were made using MEDLINE, The Cochrane Library, Embase, Web of Science, Scopus, CINAHL (EBSCO) and PsycINFO databases and records were combined into an Endnote database. Records were filtered for duplicates and selected based on study inclusion/exclusion criteria. Full-text articles were sourced and data were extracted by two reviewers into a bespoke REDCap database, with a third reviewer adjudicating disagreements. The Jadad scoring method was used to determine risk of bias. Results Of the 3220 publications retrieved from online searching, 457 articles met the eligibility criteria and included 198 587 patients. DLQI scores were used as primary outcomes in 24 (5.3%) of these studies comprising 15 different diseases and 3436 patients. Most study interventions (17 of 24 studies, 68%) were systemic drugs, with biologics (liraglutide, alefacept, secukinumab, ustekinumab, adalimumab) accounting for 5 of 25 pharmacological interventions (20%). Topical treatments comprised 32% (8 studies), whereas nonpharmacological interventions (n = 8) were 24% of the total interventions (N = 33). Three studies used nontraditional medicines. Eight studies were multicentred (33.3%), with trials conducted in at least 14 different countries, and four studies (16.7%) were conducted in multiple countries. The Jadad risk of bias scale showed that bias was uncertain or low, as 87.5% of studies had Jadad scores of ≥ 3. Conclusions This study provides evidence for use of the DLQI as a primary outcome in clinical trials. Researchers and clinicians can use this data to inform decisions about further use of the DLQI as a primary outcome. [ABSTRACT FROM AUTHOR]

Introduction: The field of Medicines Development faces a continuous need for educational evolution to match the interdisciplinary and global nature of the pharmaceutical industry. This paper discusses the outcomes of a 7-year collaboration between King's College London and the Global Medicines Development Professionals (GMDP) Academy, which aimed to address this need through a blended e-learning program. Methods: The collaboration developed a comprehensive curriculum based on the PharmaTrain syllabus, delivered through a combination of asynchronous and synchronous e-learning methods. The program targeted a diverse range of professionals serving in areas related to Medical Affairs. Results: Over seven annual cohorts, 682 participants from eighty-six countries were enrolled in the program. The program's effectiveness was assessed using Kirkpatrick's model, showing elevated levels of satisfaction (over 4.0 on a five-point scale), suggesting significant gains in competence at the cognitive level and leveraged performance. Notably, 70% of responding alumni reported significant improvement in their functions, corroborated by 30% of their supervisors. The further long-term impact of the program on their respective organization has not been established. Discussion: The GMDP Academy's program has significantly contributed to lifelong learning in Medicines Development, addressing educational gaps and fostering interdisciplinary collaboration. Its success highlights the importance of continuous education in keeping pace with the industry's evolving demands and underscores the potential of blended learning in achieving educational objectives in pharmaceutical medicine. [ABSTRACT FROM AUTHOR]

Background The Hyperhidrosis Quality of Life Index (HidroQoL©) is a measure of quality of life (QoL) impacts in hyperhidrosis (HH). Objectives We aimed to establish score banding systems for the HidroQoL total score for specific contexts representing different severity/impact categories by using the Dermatology Life Quality Index (DLQI) and the Hyperhidrosis Disease Severity Scale (HDSS) as anchors, including data from 357 patients from a phase III clinical trial. Methods We used the HDSS, the established DLQI score bands and two single items (items 5 and 7) of the DLQI as anchors for the creation of banding systems for the HidroQoL. These anchors were chosen via consensus among an expert group according to relevance to patient experience. Due to the distribution of the HDSS and the single DLQI item 7, receiver operating characteristic curves were computed in order to create an optimal cut-off value of the HidroQoL total score. For the DLQI banding system and the single DLQI item 5, we created a banding system for the HidroQoL based on the distribution of their different categories. Results A score of 30 and greater is proposed as the cut-off value for sweating that 'always interferes in daily activities', based on the HDSS as anchor. In terms of overall skin QoL effects, score bands of 0–6, 7–18, 19–25, 26–32 and 33–36 represent 'no effect', 'small effect', 'moderate effect', 'very large effect' and 'extremely large effect' on the patient's life, respectively. Conclusions In this study, we propose different banding systems for four different contexts: skin-specific QoL (DLQI banding), HH severity (HDSS), working and studying (single DLQI item 7) and social and leisure activities (single DLQI item 5). These banding systems and cut-off values can be used in clinical research and practice to place the patients in different severity categories. [ABSTRACT FROM AUTHOR]

Background: Lymphoma treatment can lead to long-term consequences such as fatigue, infertility and organ damage. In clinical trials, survival outcomes, clinical response and toxicity are extensively reported while the assessment of treatment on quality of life (QoL) and symptoms is often lacking. Objective: We evaluated the use and frequency of patient-reported outcome (PRO) instruments used in randomized controlled trials (RCTs) for Hodgkin lymphoma (HL) and their consistency of reporting. Methods: MEDLINE, CENTRAL and trial registries for RCTs investigating HL were systematically searched from 01/01/2016 to 31/05/2022. Following trial selection, trial, patient characteristics and outcome data on the use of PRO measures (PROMs) and reporting of PROs using a pre-defined extraction form were extracted. To assess reporting consistency, trial registries, protocols and publications were compared. Results: We identified 4,222 records. Following screening, a total of 317 reports were eligible for full-text evaluation. One hundred sixty-six reports of 51 ongoing/completed trials were included, of which 41% of trials were completed and 49% were ongoing based on registry entries. Full-text or abstract were available for 33 trials. Seventy percent of trials were conducted in the newly diagnosed disease setting, the majority with advanced HL. In 32 trials with published follow-up data, the median follow-up was 5.2 years. Eighteen (35%) completed/ongoing trials had mentioned PRO assessment in registry entries, protocol or publications. Twelve trials (67%) had published results and only 6 trials (50%) reported on PROs in part with the exception of 1 trial where PROs were evaluated as secondary/exploratory outcome. The most referenced global PROM was the EORTC-QLQ-C30 (12 studies), the EQ-5D (3 studies) and the FACT-Neurotoxicity (3 studies). FACT-Lymphoma, a disease-specific PROM for non-HL was mentioned in one ongoing trial. None of the trials referenced the EORTC QLQ-HL27, another disease-specific PROM developed specifically for HL patient's QoL assessment. Discussions: Only one-third of RCTs in HL report PROs as an outcome and only half present the outcome in subsequent publications, showcasing the underreporting of PROs in trials. Disease-specific PROMs are underutilized in the assessment of QoL in HL patients. Guidance on the assessment of PROs is needed to inform on comprehensive outcomes important to patients. [ABSTRACT FROM AUTHOR]

Background Over 29 years of clinical application, the Dermatology Life Quality Index (DLQI) has remained the most used patient-reported outcome (PRO) in dermatology due to its robustness, simplicity and ease of use. Objectives To generate further evidence of the DLQI's utility in randomized controlled trials (RCTs) and to cover all diseases and interventions. Methods The methodology followed PRISMA guidelines and included seven bibliographical databases, searching articles published from 1 January 1994 until 16 November 2021. Articles were reviewed independently by two assessors, and an adjudicator resolved any opinion differences. Results Of 3220 screened publications, 454 articles meeting the eligibility criteria for inclusion, describing research on 198 190 patients, were analysed. DLQI scores were primary endpoints in 24 (5.3%) of studies. Most studies were of psoriasis (54.1%), although 69 different diseases were studied. Most study drugs were systemic (85.1%), with biologics comprising 55.9% of all pharmacological interventions. Topical treatments comprised 17.0% of total pharmacological interventions. Nonpharmacological interventions, mainly laser therapy and ultraviolet radiation treatment, comprised 12.2% of the total number of interventions. The majority of studies (63.7%) were multicentric, with trials conducted in at least 42 different countries; 40.2% were conducted in multiple countries. The minimal clinically importance difference (MCID) was reported in the analysis of 15.0% of studies, but only 1.3% considered full score meaning banding of the DLQI. Forty-seven (10.4%) of the studies investigated statistical correlation of the DLQI with clinical severity assessment or other PRO/quality of life tools; and 61–86% of studies had within-group scores differences greater than the MCID in 'active treatment arms'. The Jadad risk-of-bias scale showed that bias was generally low, as 91.8% of the studies had Jadad scores of ≥ 3; only 0.4% of studies showed a high risk of bias from randomization. Thirteen per cent had a high risk of bias from blinding and 10.1% had a high risk of bias from unknown outcomes of all participants in the studies. In 18.5% of the studies the authors declared that they followed an intention-to-treat protocol; imputation for missing DLQI data was used in 34.4% of studies. Conclusions This systematic review provides a wealth of evidence of the use of the DLQI in clinical trials to inform researchers' and -clinicians' decisions for its further use. Recommendations are also made for improving the reporting of data from future RCTs using the DLQI. [ABSTRACT FROM AUTHOR]

Background: The African Medicines Regulatory Harmonisation (AMRH) Initiative was formed in 2009 and subsequently, three regional initiatives (East African Community Medicines Regulatory Harmonisation [MRH], Southern African Development Community [SADC]/ZaZiBoNa MRH, and the Economic Community of West Africa States MRH) were established. As these initiatives serve as a foundation for the African Medicines Agency (AMA), the aim of this study was to compare their operating models, successes and challenges to identify opportunities for improvement and alignment. Methods: A mixed method approach was used for the data collection using a questionnaire, the Process, Effectiveness and Efficiency Rating (PEER), developed by the authors specifically for this study and semi-structured interview techniques. There were 23 study participants (one from each agency of the member countries of the three regions). It was hoped that data generated from this study would lead to a series of recommendations, which would then be ratified by the regulatory authorities. Results: Most respondents stated that AMRH contributed to the strengthening of regulatory systems and harmonising regulatory requirements across economic regions of Africa, potentially resulting in improved access to quality-assured medicines. Although established at different times and at the discretion of each region, the marketing authorisation application review processes are largely similar, with a few differences noted in the eligibility and submission requirements, type of procedures employed, the timelines and fees payable. The challenges identified in the three regions are also similar, with the most noteworthy being the lack of a binding legal framework for regional approvals. Conclusion: In this study, we compared the process, successes and challenges of these three regional harmonisation initiatives in Africa addressing the areas of legal frameworks, information management systems, the accessibility and affordability of medicines and reliance that will bring greater alignment and efficiency in their operating models, thereby strengthening the foundation of the soon-to-be-operationalised AMA. [ABSTRACT FROM AUTHOR]

Simple Summary: It is recognized that patients with multiple myeloma (MM) experience a high burden of disease and treatment-related symptoms that impact upon their quality of life (QoL). In these patients, patient-reported outcome (PRO) measures are important in providing information on how treatment affects their QoL. In the past 10 years in the MM setting, the main focus has been to achieve the most durable remission with the best QoL as primary goals of therapy. Optimizing the QoL of patients with MM is an important treatment goal and the use of PROs in clinical trials has the potential to improve treatment outcomes. The present report, on behalf of the European Hematology Association (EHA), provides evidence-based guidelines for the use and reporting of PROs in patients with MM that have been developed according to the EHA's core Guidelines Development Methodology. Currently, there is considerable variation in the measurement of QoL in MM trials, thus underlining the importance of systematic measurements. These Guidelines will aid clinicians, regulatory agencies and the pharmaceutical industry in the measurement of QoL in patients with MM in clinical trials. In the era of personalized medicine there is an increasing need for the assessment of patient-reported outcomes (PROs) to become a standard of patient care. Patient-reported outcome measures (PROM) are important in assessing significant and meaningful changes as a result of an intervention based on a patient's own perspective. It is well established that active multiple myeloma (MM) can be characterized by a high burden of disease and treatment-related symptoms, with considerable worsening of quality of life (QoL). In general, and over the past decade, the focus has shifted to obtaining the most durable remissions with the best QoL as primary goals for MM treatment. Patients place considerable value on their QoL and communicating about QoL data prior to treatment decisions allows them to make informed treatment choices. Consequently, optimization of QoL of patients with MM is an important therapeutic goal and the incorporation of PROs into clinical trials has the potential of improving treatment outcomes. In this regard, guidance for the use and reporting of PROMs in MM in clinical trials is warranted. Under the auspices of the European Hematology Association, evidence-based guidelines for the use and reporting of PROs in patients with MM have been developed according to the EHA's core Guidelines Development Methodology. This document provides general considerations for the choice of PROMs in MM clinical trials as well as a series of recommendations covering a selection of PROMs in MM clinical trials; the mode of administration; timing of assessments; strategies to minimize missing data; sample size calculation; reporting of results; and interpretation of results. [ABSTRACT FROM AUTHOR]

Introduction: In the last few decades, there has been a rapid development in cancer therapies and improved detection strategies, hence the death rates caused by cancer have decreased. However, it has been reported that cardiovascular disease has become the second leading cause of long-term morbidity and fatality among cancer survivors. Cardiotoxicity from anticancer drugs affects the heart's function and structure and can occur during any stage of the cancer treatments, which leads to the development of cardiovascular disease. Objectives: To investigate the association between anticancer drugs for nonsmall cell lung cancer (NSCLC) and cardiotoxicity as to whether: different classes of anticancer drugs demonstrate different cardiotoxicity potentials; different dosages of the same drug in initial treatment affect the degree of cardiotoxicity; and accumulated dosage and/or duration of treatments affect the degree of cardiotoxicity. Methods: This systematic review included studies involving patients over 18 years old with NSCLC and excluded studies in which patients' treatments involve radiotherapy only. Electronic databases and registers including Cochrane Library, National Cancer Institute (NCI) Database, PubMed, Scopus, Web of Science, ClinicalTrials.gov and the European Union Clinical Trials Register were systematically searched from the earliest available date up until November 2020. A full version protocol of this systematic review (CRD42020191760) had been published on PROSPERO. Results: A total of 1785 records were identified using specific search terms through the databases and registers; 74 eligible studies were included for data extraction. Based on data extracted from the included studies, anticancer drugs for NSCLC that are associated with cardiovascular events include bevacizumab, carboplatin, cisplatin, crizotinib, docetaxel, erlotinib, gemcitabine and paclitaxel. Hypertension was the most reported cardiotoxicity as 30 studies documented this cardiovascular adverse event. Other reported treatment-related cardiotoxicities include arrhythmias, atrial fibrillation, bradycardia, cardiac arrest, cardiac failure, coronary artery disease, heart failure, ischemia, left ventricular dysfunction, myocardial infarction, palpitations, and tachycardia. Conclusion: The findings of this systematic review have provided a better understanding of the possible association between cardiotoxicities and anticancer drugs for NSCLC. Whilst variation is observed across different drug classes, the lack of information available on cardiac monitoring can result in underestimation of this association. [ABSTRACT FROM AUTHOR]

Aim: To investigate the epidemiology and clinical management of patients with type 2 diabetes (T2D) and established atherosclerotic cardiovascular disease (eASCVD) or high/very high ASCVD risk, defined by the 2021 European Society of Cardiology Guidelines, in seven countries in the Middle East and Africa (PACT‐MEA; NCT05317845), and to assess physicians' attitudes and the basis for their decision‐making in the management of these patients. Materials and Methods: PACT‐MEA is a cross‐sectional, observational study undertaken in Bahrain, Egypt, Jordan, Kuwait, Qatar, South Africa and the United Arab Emirates based on a medical chart review of approximately 3700 patients with T2D in primary and secondary care settings, and a survey of approximately 400 physicians treating patients with T2D. Results: The primary and secondary objectives are to determine the prevalence of eASCVD and high/very high ASCVD risk in patients with T2D. Current treatment with cardioprotective antidiabetic medication, the proportion of patients meeting the treatment criteria for reimbursement in the study countries where there is an applicable reimbursement guideline, and physician‐reported factors in clinical decision‐making in T2D management, will also be assessed. Conclusions: This large cross‐sectional study will establish the estimated prevalence and management of eASCVD and high/very high ASCVD risk in patients with type 2 diabetes across the Middle East and Africa. [ABSTRACT FROM AUTHOR]

Background: Following the establishment of Economic Community of West African States Medicines Regulatory Harmonization (ECOWAS-MRH) initiative in 2017, it was considered timely to carry out an evaluation of the current status of the initiative's operating model by the pharmaceutical industry users. This study examined the challenges being encountered and identified strategies that would strengthen the ECOWAS-MRH initiative moving forward. Methods: The Process Effectiveness and Efficiency Rating (PEER) questionnaire was used to collect data from manufacturers who have submitted applications to the joint assessment procedure and had identified recommendations for improving the performance of the ECOWAS-MRH initiative. Results: Ten pharmaceutical manufacturer participants (innovator, generic foreign, generic local) all reported that harmonisation of registration requirements was a major benefit, allowing submission of the same dossier to multiple countries, reducing the application burden and saving time and resources. Additionally, receipt of the same list of questions from several countries enables the compilation of a single response package, resulting in shorter timelines for approvals compared to the individualised country responses. Another benefit of a harmonised registration process was the simultaneous accessibility of medicines in various markets. Key challenges included a lack of centralised submission and tracking, differences in regulatory performance of the national medical regulatory authorities, a lack of detailed information for applicants and a low motivation to use the ECOWAS-MRH route with a preference for other regulatory pathways in the ECOWAS member states. Conclusion: This study identified several approaches to increase the effectiveness of this initiative: the implementation of risk-based approaches such as use of reliance pathways; establishment of a robust information technology systems, building assessor capacity to facilitate processing and monitoring applications; and priority review of ECOWAS-MRH products. [ABSTRACT FROM AUTHOR]