Search

Background Globally accepted roles of pharmacists are described in the Good Pharmacy Practice (GPP) standards, published by the World Health Organization (WHO) and the International Pharmaceutical Federation (FIP) in 2011. These standards provide a wide-ranging description of four main roles pharmacists fulfil. The global platform, where pertinent discussions around excellence and innovation in various pharmacy roles take place, is the annual congress of the pharmacy organisation representing the profession globally, FIP. Objectives Given the world pharmacy congresses present and reflect on the most topical and contemporary matters, this longitudinal study aimed at creating a historical overview of the frequency of appearance of the different GPP roles in the programmes of the past 17 congresses (2003–2019). This is to distinguish the dominance of different roles over time and thus their relevance for the profession. Methods The GPP standards served as a framework to create a set of keywords that were analysed for their frequencies of appearance in the programmes through text analysis. Trends in the four overarching GPP roles and at individual keyword level were analysed descriptively over time. Results The study found that all four GPP roles appeared in the programme each year and none of them was significantly missing, neither in the decade preceding the publication of the GPP standards nor in the decade thereafter. Role 3 “Maintain and improve professional performance” was most frequently represented, also demonstrating an upward trend in appearance, together with Role 4: “Contribute to improve effectiveness of the health-care system and public health”. Trends emerged towards patient-centred clinical focus and positioning pharmacy as an important player in the health-care system—observed also at individual keywords level in areas such as health promotion—away from the more traditional product-centred practice roles such as compounding. Conclusions GPP roles have been already covered by the FIP annual congresses (long) before 2011, when the GPP roles were formally adopted, and they stayed relevant in the decade after. The more pronounced dominance toward the roles related to improving professional performance and positioning pharmacy are in line with the trend that the rather technical topics in pharmacy are increasingly covered by specialised meetings and that the FIP annual congresses have moved toward more general, scholarly platforms for dialogue and conversation.

Background Medicine shortages are often described in plain numbers, suggesting all shortages have a uniform impact. However, some shortages have a direct and serious effect on patients and need a prompt reaction from stakeholders. This study aims to create a broad framework to assess the impact of a shortage. Method We identified high impact shortages and selected exemplary shortages which we considered our learning cases. From five learning cases, we identified elements that had a potentially profound impact on one or more of these cases. We tested data saturation on the elements with another five test cases. Based on these elements, we created a framework to assess impact of shortages on patients and presented practical examples how to rate these different elements. Subsequently, we visualised the impact of these five learning cases on patients in radar charts. Results The five elements which we identified as potentially having a large impact were 1) alternative product, 2) disease, 3) susceptibility, 4) costs and 5) number of patients affected. The five learning cases rated high on different elements, leading to diverse and sometimes even opposite patterns of impact. Conclusion We created a framework for assessing the impact of a medicine shortage on patients by means of five key elements. By rating these elements, an indication of the impact can be obtained.

Background: The target product profile (TPP) outlines the desired profile of a target product aimed at a particular disease and is used by companies to plan clinical development. Considering the increasing importance of health technology assessment (HTA) in informing reimbursement decisions, a robust TPP needs to be built to address HTA needs, to guide an integrated evidence generation plan that will support HTA submissions. This study assessed current practices and experiences of companies in building HTA considerations into TPP development.Methods: An opinion survey was designed and conducted in 2019, as a cross-sectional questionnaire consisting of multiple-choice questions. The questionnaire provided a qualitative assessment of companies’ strategies and experiences in building HTA considerations into the TPP. Eligible survey participants were the senior management of Global HTA/Market Access Departments at 18 top international pharmaceutical companies.Results: 11 companies responded to the survey. All companies included HTA requirements in TPP development, but the timing and process varied. The key focus of HTA input related to health problems and treatment pathways, clinical efficacy/effectiveness, and safety. Variance of HTA methods and different value frameworks were identified as a challenge for development plans. Stakeholder engagement, such as HTA scientific advice, was used to pressure test the TPP.Conclusion: This research provides insight into current practice and potential opportunities for value-based drug development. It demonstrates the evolution of the TPP to encompass HTA requirements and suggests that the TPP could have a role as an iterative communication tool for use with HTA agencies to enhance an integrated evidence generation plan.

BackgroundMany challenges in elderly pharmacotherapy are identified, including the use of Potentially Inappropriate Medications (PIMs) which may increase the odds of adverse events, especially in elderly patients with mental health disorders (e. g., behavioral, and psychological symptoms of dementia–BPSD, schizophrenia, bipolar disorder). However, information on the knowledge and practice of healthcare professionals (HCPs) about this topic is still scarce.MethodsA cross-sectional study was undertaken from July-October 2019. An online questionnaire was specifically designed and validated for this study. We sought HCPs (physicians, pharmacists, and nurses) worldwide, using (a) social media, via Facebook, Twitter, and LinkedIn; and (b) email contacts of the research team (convenience sample). Either way participants were asked to share on their social media or via e-mail the questionnaires with other HCPs (snowballing sample). The survey assessed two main domains: knowledge and practice. Knowledge was evaluated by self-report (perceived knowledge by a 5-item Likert confidence scale) and using three clinical cases, scored between 0 and 30 points (each one rated from 0 to 10 points; real knowledge). Barriers in clinical practice were evaluated using a 5-item Likert scale judging practitioners' opinion.ResultsA total of 165 questionnaires were collected. HCPs were mainly female (n = 114; 69.1%), with a mean age of 35.3 ± 11.3 years old. Seventy-two percent (n = 118) were pharmacists, 21.1% (n = 35) were physicians, and 7.3% (n = 12) nurses. There was a weak correlation, albeit significant, between perceived and real knowledge (r = 0.199; p = 0.001). The mean score of the clinical vignettes regarding elderly patients with dementia and bipolar disorder were 4.59 ± 4.08 and 4.86 ± 2.97 points, respectively. Most HCPs were classified as having an intermediate knowledge (n = 100; 60.6%) about medication complexities in the elderly with mental disorders. Most HCPs agreed that lack of time (81.6%; n = 138), lack of education and training on elderly pharmacotherapy (72.2%; n = 122), and lack of tools adapted to daily practice (61.8%; n = 105) were the main barriers.ConclusionsMost of the HCPs felt confident to manage medication complexities in elder patients with mental disorders, but only a minority obtained a good score in the knowledge assessment test. The main barriers identified included structural barriers (tools unfit for practice) and process barriers (time).

Background Over the past decade, a new class of drugs called CFTR (cystic fibrosis transmembrane conductance regulator) modulators have shown to be able to improve clinical outcomes in patient with Cystic Fibrosis. In this analysis, we have extensively reviewed the regulatory pathways and decisions adopted by FDA and EMA to speed up the development, the review and the approval of these drugs, with the aim of identifying possible clinical and public health implications associated with differences. Results CFTR modulators have been developed towards addressing three main genetic domains: (1) F508del homozygous (F508del/F508del), (2) F508del heterozygous, and (3) genotypes not carrying F508del mutation; and expanded from adult to paediatric population. Programs to expedite the reviewing and licensing of CFTR modulators were extensively adopted by FDA and EMA. All CFTR modulators have been licensed in the US as orphan drugs, but in the EU the orphan status for LUM/IVA was not confirmed at the time of marketing authorization as results from the pivotal trial were not considered clinically significant. While FDA and EMA approved CFTR modulators on the basis of results from phase III double-blind RCTs, main differences were found on the extension of indications: FDA accepted non-clinical evidence considering a recovery of the CFTR function ≥ 10% based on chloride transport, a reliable indicator to correlate with improvement in clinical outcomes. By contrast, EMA did not deem preclinical data sufficient to expand the label of CFTR modulators without confirmatory clinical data. Conclusions Regulators played an important role in fostering the development and approval of CFTR modulators. However, differences were found between FDA and EMA in the way of reviewing and licensing CFTR modulators, which extended beyond semantics affecting patients’ eligibility and access: FDA’s approach was more mechanistic/biology-driven while the EMA’s one was more oriented by clinical evidence. This might refer to the connection between the EMA and the Member States, which tends to base decisions on pricing and reimbursement on clinical data rather than pre-clinical ones. Here we have proposed a two-step personalized-based model to merge the ethical commitment of ensuring larger access to all potential eligible patients (including those harboring very rare mutations) with the one of ensuring access to clinically assessed and effective medicines through Real World Data.

Background and aims: There is an alarming delay in Europe for anticancer medicines becoming accessible for children. Following a paediatric European Union marketing authorisation, national Health Technology Assessment (HTA) agencies evaluate effectiveness, and safety of medicines to support decision on their cost and reimbursement. This study (a SIOPE Access to Medicines project) aimed to evaluate how these HTA evaluations take place for anticancer medicines indicated for paediatric use in Europe and to explore where the delays for market access originate. Methods: We obtained HTA reports from the public domain for nine European countries for blinatumomab, dinutuximab beta and tisagenlecleucel. We evaluated the time elapsed between marketing authorisation for a paediatric indication and a national HTA decision and the nature of the decision. Results: Out of 23 HTA decisions (four countries without blinatumomab report), 18 were positive, two with restrictions, three negative. For blinatumomab, tisagenlecleucel and dinutuximab beta, the median time to an HTA decision after regulatory approval for paediatric use was 353 days (range 193–751), 141 days (range 77–517) and 515 days (range 0–780), respectively, with variability between countries. Dinutuximab beta and tisagenlecleucel were first introduced in children, but did not result in shorter time to HTA decision. For blinatumomab, marketing authorisation followed 1008 days after the indication in adults, with HTA applications submitted a median of 167 days later, and a recommendation after 145 days. Conclusions: This study reveals ample variability in HTA decision making in nine European Union countries. Collaboration and alignment of required evidence is needed to facilitate robust scientific HTA assessments, also considering methodological challenges in paediatric oncology.