Journal of Pharmaceutical Policy and Practice最新文献

Background: The European Medicines Agency (EMA) issued regulatory recommendations and communications in 2021 about the risk of thrombosis with thrombocytopenia syndrome (TTS) associated with COVID-19 adenoviral vector vaccines Vaxzevria and Jcovden. Little is known about how such measures impacted on national vaccination strategies and how they influenced decision-making processes of policy-makers and experts. The aim of this study was to evaluate the impact of regulatory actions for Vaxzevria and Jcovden on national vaccination strategies following the EMA's 2021 review on safety measures on TTS.

Methods: A grey literature review was performed to gather information on EMA's actions as well as changes to national vaccination strategies in Denmark, Greece, Latvia, the Netherlands, Portugal and Slovenia. Semi-structured interviews were held with experts who had either advisory or decision roles in national vaccination programmes to contextualise the results of the grey literature review and discuss their experiences. Interviews were coded and analysed on national levels.

Results: EMA made various adaptations to product information and released safety warnings and other regulatory communications for both vaccines relating to TTS risk. Countries varied widely in changes made to national vaccination policies. Interviews revealed that experts relied mainly on their professional networks and information sources, other than the EMA, to inform their recommendations and/or decision-making.

Conclusion: EMA's regulatory actions were not the main source informing adaptations to national vaccination strategies. National decision-makers country reverted to their preferred sources. The use of varying sources explains some of the variations in the vaccination strategies.

Background: Drugs for rare diseases (DRDs) present unique challenges in evaluation and reimbursement due to high costs, limited clinical evidence, and complex healthcare decision-making. While international models exist for DRD reimbursement, Saudi Arabia faces distinct obstacles driven by high incidence of genetic diseases, healthcare system fragmentation, and evolving national policies. This study aims to identify key challenges in evaluating and reimbursing DRDs in Saudi Arabia.

Methods: A modified Delphi method was conducted between January and February 2025, involving healthcare policymakers, clinicians, health economists, and formulary decision-makers across multiple institutions. The process included three rounds: (1) open-ended questionnaires to identify challenges, (2) voting on structured statements using a Likert scale, and (3) consensus refinement through a roundtable discussion.

Results: Nineteen experts participated, with final consensus reached on 88 statements across eight themes: evaluation complexities, evidence limitations, economic and budgetary constraints, supply chain management, data generation and infrastructure, stakeholder concerns, patient and family barriers, and policy and collaboration gaps. The highest concern was economic constraints. Other challenges included inconsistencies in reimbursement decisions, and difficulties in managed entry agreements. Stakeholders emphasised the need for structured decision-making frameworks and national coordination to improve access and equity.

Conclusion: This study provides the first consensus on challenges in DRD evaluation and reimbursement in Saudi Arabia. Addressing these issues through policy reforms, stakeholder collaboration, and data infrastructure enhancement is crucial to optimising resource allocation and patient access. Further research should focus on implementing value-based agreement to ensure sustainable reimbursement strategies for DRDs.

Background: This study assessed how an educational intervention affected healthcare providers' knowledge and practice of pharmacovigilance (PV) principles, with an emphasis on enhancing the reporting ADRs.

Methods: In this cross-sectional study, a structured questionnaire was utilised. A pre- and post-educational intervention design was used to assess the influence of a PV workshop on ADRs reporting in Jordan. The PV educational workshop was a one-year interactive session that addressed core PV principles.

Results: A total of 250 healthcare providers participated in the study, including 14 general physicians (5.6%), 15 specialist physicians (6%), 93 pharmacists (37.2%), 51 clinical pharmacists (20.4%), 58 nurses (23.2%), 3 midwives (1.2%), and 16 others (6.4%). A positive trend in participants' familiarity with the PV term was shown, with 69.6% of respondents expressing improvement post-workshop. After the workshop, more than 70% of participants agreed that the reporting of ADRs increased. The utilisation of electronic forms for reporting ADRs was reported to be increased among 68.4% of participants. Regarding the improvement in the awareness of delayed ADRs, clinical pharmacists had higher knowledge scores (3.04) compared to general physicians (2.50; p = 0.041). Clinical pharmacists had a higher practice score (3.92) regarding the frequency of filling up a suspected ADR form compared to nurses (3.53; p = 0.042). When comparing the period before to after launching the workshop, the number of reported cases of ADRs increased from 546 to 1060, and the number of reported ADRs increased from 1216 to 1763.

Conclusion: The educational intervention improved healthcare providers' knowledge and practices related to PV and ADR reporting. These findings highlight the importance of targeted training initiatives in strengthening PV systems and promoting a culture of safety within healthcare settings.

Background: Ninety-one percent of 1500 patient groups surveyed across 78 countries perceive pharmaceutical firms' pricing policies as unfair. Despite this, there is little evidence of pharmaceutical companies adopting continuous process costing, a management accounting method that could help transparently track costs and determine fair pricing. This study investigates the link between unit costs, equivalent production units, and fair pharmaceutical pricing. To enhance transparency, fairness, and affordability, we propose mandating the disclosure of unit cost formulas, costing methods, and markup ceilings in annual reports, in alignment with UN SDG goals.

Methods: This review followed the Joanna Briggs Institute's (JBI) methodology for scoping reviews to frame the research question, identify relevant studies in databases, select studies, extract the data, report the results and guide consultation sessions with stakeholders with lived experience on potential implications. The search period was January 2022 to November 2023. We used Preferred Reporting Items for Systematic Reviews and Meta Analysis (PRISMA)-Scoping Review extension to present the results.

Results: 46 articles were eligible out of 1,281 initially screened. 8 articles addressed pharmaceutical unit production costs; 1 empirical study focused on Equivalent Units of Production (EUP) practices, revealing discrepancies between industry costing practices and academic models; and, the remaining 37 articles explored fair pricing frameworks, emphasising value-based pricing, ethics, and policy considerations. Three gaps emerged: no studies link pharmaceutical pricing to continuous process costing/EUP, despite extensive fair pricing research; absence of standardised methodologies for applying continuous costing in pharmaceutical contexts; and lack of state-regulated uniform costing systems or enforceable mark-up ceilings, impeding cost transparency and fair pricing.

Conclusions: No evidence linked pharmaceutical pricing models to continuous process costing/EUP. Addressing this gap requires mandatory disclosures of regulated uniform costing methods and mark-up ceilings in published annual financial reports. This will improve transparency, social accountability, fair pricing, and medicine affordability - aligning with UN SDGs 3 (Good Health) and 10 (Reduced Inequalities).

About eighty-eight percent of people worldwide rely on traditional and complementary medicine for their primary healthcare needs, with many pharmacies in developed countries offering plant-based over-the-counter medications. However, the increasing use of medicinal products, especially when combined with pharmaceuticals, poses health risks that are often underreported, particularly in low- and middle-income countries. Before 2012, Pakistan had no specific laws or regulatory bodies to oversee the manufacturing of herbal and alternative medicines. The establishment of the Drug Regulatory Authority of Pakistan (DRAP) in 2012 marked the beginning of regulatory oversight for herbal medicines for licensing, enlistment and Pharmacovigilance (PV). In 2015, DRAP created a national pharmacovigilance centre, and by 2018, it became a full member of the WHO's Program for International Drug Monitoring (PIDM). The Pharmacovigilance Rules of 2022 require all therapeutic product manufacturers to report adverse drug reactions (ADRs) and mandate the submission of Periodic Benefit Risk Evaluation Reports (PBRER) for various drugs. However, there are provisions allowing for PBRER submission waivers in certain cases. While Pakistan's pharmacovigilance system currently focuses on pharmaceutical and biological medicines, there is a pressing need to expand its scope to include herbal and traditional products. Strengthening the system involves setting robust quality and safety standards, conducting scientific research, regulating manufacturing practices, and ensuring proper labelling of herbal medications.

Background: Despite the alternatives available in metastatic colorectal cancer (mCRC), evaluating new therapies continues to be a challenge for clinicians and decision-makers. We aimed to generate a conceptual framework and establish the value criteria for evaluating treatments in mCRC and to apply this framework to assess the value of trifluridine/tipiracil (FTD/TPI) + bevacizumab (BEVA) in advanced lines of therapy for mCRC versus alternatives.

Methods: A multicriteria decision analysis (MCDA) was conducted according to the modified EVIDEM assessment framework and the following process: defining the decision problem, selecting and structuring the related criteria identified in a literature review, weighting criteria, measurement of performance, and scoring alternatives, to evaluate of mCRC therapies in Catalonia (Spain). An expert panel of five members with experience in cancer treatment in Catalonia was involved. A hierarchical and non-hierarchical weighting of the criteria and sub-criteria was performed. An evidence matrix of available treatments was developed, and experts assigned scores comparing FTD/TPI + BEVA versus alternatives.

Results: Five value criteria and 18 sub-criteria were selected to evaluate mCRC therapies. According to hierarchical method, efficacy had the highest weight, followed by safety, and lower weights for the rest: cost, guidelines/expert consensus, and epidemiology. In the efficacy dimension, overall survival (OS) had the highest weight, while severe adverse events received the highest score in terms of safety. Non-hierarchical weighting corroborated these results. Treatment with FTD/TPI + BEVA obtained a positive overall value contribution of 0.91, 0.43 points considering only comparative criteria. Comparative criteria were highly important, obtaining values contribution for efficacy, safety and cost of 0.19, 0.15 and 0.12 points, respectively. OS (0.20), progression-free survival (0.19), disease control rate (0.16) and health-related quality of life (0.16) were the most relevant sub-criteria.

Conclusion: Based on this MCDA, the high value contribution of FTD/TPI + BEVA suggests its substantial benefits over the most used alternatives.

Background: Polymyxin B is increasingly used to treat infections caused by multidrug-resistant gram-negative bacteria; however, its widespread clinical use is hindered by the high incidence of nephrotoxicity. Antioxidants, such as vitamin C, N-acetylcysteine (NAC), and methionine, have demonstrated renoprotective effects in preclinical models, although clinical evidence remains limited. This study aimed to investigate the potential protective effects of these antioxidants against polymyxin B-associated acute kidney injury (AKI) in real-world clinical practice.

Methods: This retrospective cohort study included adult in patients who received intravenous polymyxin B for ≥ 3 days between August 2018 and August 2020. The patients were classified into an antioxidant group (co-administered vitamin C, NAC, or methionine for ≥ 3 days) or a control group. Propensity score weighting was applied to balance the baseline covariates. The primary outcome was the incidence of polymyxin B-associated AKI and the secondary outcome was discharge mortality.

Results: A total of 321 patients were included, with 77 and 244 patients in the antioxidant and control groups, respectively. After propensity score adjustment, there were no statistically significant differences in AKI incidence (26.9% vs. 19.2%, P = 0.352) or discharge mortality (7.7% vs. 15.4%, P = 0.220) between the groups. Subgroup analyses of individual antioxidants also showed no significant differences in AKI or mortality, except for a lower unadjusted mortality in the methionine group (0% vs. 13.9%, P = 0.042), which lost significance after adjustment.

Conclusion: This exploratory study did not provide conclusive evidence that vitamin C, NAC, or methionine reduced the risk of polymyxin B-associated AKI. The potential association between antioxidant use and reduced mortality warrants further investigation. Large-scale prospective studies are required to confirm the clinical utility of antioxidant cotherapy in patients receiving polymyxin B.

The pharmacy systems have undergone fundamental changes in some Nordic and Baltic countries: Iceland, Norway, Sweden and Estonia. The political declared aims of the reforms have included increasing competition and/or effectiveness of the pharmacy market and the availability of medicines or pharmacies. The aim of this commentary is to describe the policy measures taken in these countries changing community pharmacy ownership, the arguments and rationales behind, and the evidence of the intended and unintended outcomes. Furthermore, we discuss the lessons learned for social pharmacy researchers. The aim of increasing the availability of pharmacies has been achieved, if interpreted as more pharmacies. However, the number of pharmacies has increased, mainly in urban areas, with a need to assure the availability of pharmacies in rural areas with regulations and/or subsidies in some countries. There were also unintended consequences. The aim to increase competition and diversity failed, as big domestic and foreign pharmacy chains conquered most of the pharmacy market. Learning for researchers in social pharmacy when studying pharmacy system changes includes considering the social, economic, and political reality in which the sector exists. The intended and unintended consequences of changes need a multi-method approach, often mixing quantitative (epidemiology and economics) and qualitative social science methods. Lastly, if we want evidence-based policymaking, we as researchers need to do better in communicating our research evidence to politicians and to the public.

Background: Substandard and falsified medicines (SFMs) continue to pose a significant threat to public health globally. However, there is limited evidence on use of SFMs for both humans and animals particularly in low- and middle-income countries such as Uganda. The study assessed knowledge, attitudes and practices on SFMs for human and animal use in Wakiso District, Uganda.

Methods: A cross-sectional survey that employed a structured questionnaire among 432 community members was conducted in Wakiso District. The questionnaire assessed knowledge, attitudes and practices on SFMs for human and animal use. Data was collected using the KoboCollect mobile application hosted on tablet computers. Univariate data analysis was conducted in Stata Version 14.

Results: The majority of respondents (83%) stated that they had heard about SFMs although only 31% could correctly define them. Only 7% of the respondents accurately identified a falsified medicine despite 24% stating that they believed they could recognise SFMs. Almost two-thirds (62% and 60%) of the respondents disagreed that most human and animal SFMs respectively were as good as genuine medicines. Most of the respondents strongly agreed or agreed that SFMs could be very dangerous for humans (96%) and for animals (95%). Respondents reported having bought products they suspected were SFMs for use in humans (14%) and animals (24%). Seeking health worker advice on the medicine brand (40%) / getting medicine from a trustworthy pharmacy (34%) for humans; and seeking a veterinary officer's advice for choosing the brand (43%) / getting medicine from a trustworthy veterinary pharmacist (29%) for animals were the most common measures respondents reported taking to ensure the medicine bought was genuine. Only 25% of the respondents mentioned informing a health worker and only 4% had reported suspicions of SFMs to the National Drug Authority.

Conclusion: Despite commendable attitudes, there was generally limited knowledge and related poor practices regarding SFMs for both humans and animals. There is a need for key stakeholder engagement involving health and regulatory authorities in both human and animal medicine to increase awareness on SFMs to minimise the potential risks to health among the community.

The healthcare system in Libya faces significant challenges due to political instability, fragmented infrastructure, and inconsistent medical practices. Clinical Practice Guidelines (CPGs) serve as essential tools for standardising care, ensuring evidence-based treatment, and optimising healthcare resources. In Libya, the lack of structured guidelines has contributed to disparities in disease management, affecting patient outcomes and overall healthcare efficiency. This commentary explores the critical need for CPGs in Libya, highlighting their potential to improve healthcare delivery, minimise variability in treatment, and enhance patient safety. While implementation poses challenges, including centralisation, limited research capacity, and resource constraints, integrating CPGs through a phased implementation framework could be a transformative step toward a more resilient and equitable healthcare system. By fostering collaboration among policymakers, healthcare professionals, and international organisations, Libya can lay the foundation for a systematic approach to disease management, ultimately improving the quality of care for its population. Healthcare reform in Libya is urgently needed, and strategic investments in CPG development and dissemination could drive the necessary transformation in Libyan healthcare.