Pharmaceutical Medicine最新文献

The Medical Affairs (MA) function in pharmaceutical companies creates a unique opportunity to ensure the internal linkage between Research & Development (R&D) and Commercial/Marketing functions, in addition to managing external scientific engagements with multiple stakeholders across life-science ecosystems. In recognition of the strategic value of MA, the objective of this paper is to share a comprehensive set of practical examples of the main deliverables within the MA function in the affiliate and align these with the two distinct phases; pre- and post-launch, respectively. We believe that an information gap currently exists in the available literature on these matters addressing practical aspects and examples beyond visionary, strategic thinking. Based on this contribution, further opportunities within MA can be discussed. In addition, we share our thoughts and considerations on future advancements in the role.

Like most private enterprises, the pharmaceutical industry has deeply rooted environmental, social, and governance (ESG) matters that challenge its long-term sustainability. Overcoming these external challenges requires collaborative and proactive steps as well as procedures guiding the adoption of ESG principles by all internal stakeholders. Environmental challenges such as climate change, and in addition the changes in society, have resulted in the need for governance addressing and coordinating efforts. The core function of medical affairs (MA) is connecting with stakeholders within a company and also between the company and external stakeholders. In this article, we describe the involvement of MA in several aspects of ESG, as a contributor, partner, and implementer. MA has a significant opportunity to emerge as a leading function involved in ESG strategies and their tactical implementation. Although the involvement of MA in the environment pillar of ESG is less, the function can implement changes relating to the conduct of meetings, clinical studies, and the digitalization of medical education via virtual platforms. Due to its patient centricity, MA is tasked to address social determinants of health to improve patients' outcomes. As a linking function within a company and with its external stakeholders, MA can provide proactive input in policy generation and enable effective governance by adherence to standards of accountability, ethics, and compliance, as well as transparency. Championing ESG is a collective responsibility that transcends any single department. It mandates a company-wide commitment. MA represents an essential pivot point in catalyzing the integration of ESG principles within industry, contributing to a healthcare ecosystem that is not merely more sustainable and ethical but also more conducive to patient health and public well-being.

Pharmacovigilance (PV) activities aim to identify potential risks of medicines and vaccines after they have been authorised in the market by collecting and analysing information on suspected adverse events from different stakeholders. These can be captured and transmitted electronically in the form of Individual Case Safety Reports (ICSRs). Hence, up-to-date ICSRs management systems, like VigiFlow and signal detection and management systems as VigiLyze, have an important role in the PV system of a country. In 2019, after various attempts to establish a PV database that could fulfil the needs of the country, Mexico's National Regulatory Authority, COFEPRIS (Federal Commission for the Prevention against Sanitary Risks) decided to implement these tools. This has been a successful project that is still ongoing, it has involved national and international organisations, and has required the participation and integration of different components of the national PV system. The implementation of these tools has allowed COFEPRIS to increase its reporting trends and quality of reporting, while contributing to make more efficient interactions and processes with PV stakeholders, even during the COVID-19 pandemic. It has also allowed them to strengthen their commitment to the WHO-Programme for International Drug Monitoring, while highlighting opportunities for improvement in the national PV scenario and in the PV tools themselves. The aim of this article is to describe the implementation process, give an overview of current results regarding ICSR data and processes, and highlight the achievements, challenges, and opportunities for improvement after the three years since the beginning of the project.

This brief paper aims to describe the Risk Evaluation and Mitigation Strategy (REMS) Public Dashboard launched by the US Food and Drug Administration (FDA) in December 2021. The FDA REMS Public Dashboard can be accessed through the REMS@FDA website. The dashboard was developed in Qlik Sense® to support a user-friendly interactive web-based tool that allows healthcare providers, patients, researchers, pharmaceutical companies, and regulators to readily access and visualize REMS information. The dashboard includes eight separate pages to capture information on all REMS, active REMS, REMS with elements to assure safe use, shared system REMS, REMS modifications, REMS revisions, released REMS, and REMS Summary; for REMS programs approved from 2008 to the present. Most of the pages allow users to choose different REMS characteristics to visualize and stratify the data by variables such as REMS approval time, application type, or REMS elements. This interactive platform is intended to allow users to quickly visualize trends over time and locate details of the REMS programs to inform emerging research and regulatory issues in the context of current drug safety. The FDA continues to explore ways to enhance public access of the REMS information in near real-time through the REMS Public Dashboard.

The centralised clinical trial authorisation process, introduced by European Regulation 536/2014, came into force on 31 January 2022. The Regulation is inflexible, both legally and in the technical detail of the authorisation process itself. The principal justification for moving away from the older European Directive 2001/20 seems to be limited to multinational trials: multiple applications to national competent authorities (NCAs), would theoretically be replaced by a single, internationally harmonised authorisation. In fact, the Regulation itself reserves many powers to the NCAs, and the latter, in any case, can lawfully impose further requirements even after that harmonised approval; the year's experience reflects these disadvantages. It would have been better if Regulation 536/2014 had been written to allow the European Medicines Agency greater flexibility, and offered an alternative, optional approach to clinical trial authorisation in the European Union.

Background: Clinical development paradigms for cell and gene therapies appear to be different to those of more conventional treatments: therefore, it is informative to explore this from the perspective of investments required to bring a new cell and/or gene therapy to the market. While there are a number of studies in the literature analyzing clinical-stage R&D costs for novel therapeutics, these are 'modality-agnostic' and thus do not elucidate costs specifically for the emerging class of cell and gene therapies.

Objectives: The objective of this study was to understand the research and development (R&D) costs associated with the clinical development of new cell and gene therapy assets METHODS: As part of our analysis of clinical-stage R&D costs for cell and gene therapies, we focused our efforts on cell and gene therapy assets recently approved by the US Food and Drug Administration (FDA) or expected to receive FDA approval by the end of 2024. A total of 25 therapies were identified for the study, 11 of which had sufficient level of detail for our clinical-stage R&D costing study. We calculated the clinical-stage R&D costs to bring a new cell and/or gene therapy to the market following a three-step approach, starting with (1) calculation of the out-of-pocket investment reported in US SEC reports; (2) we adjusted these figures for the risk of failure by applying a clinical trial phase-dependent attrition risk rate; (3) we accounted for the cost of capital of 10.5%.

Results: After accounting for R&D attrition rate (i.e., costs of failed programs) and applying a cost of capital at 10.5%, we estimate that the clinical-stage R&D investment required to bring a new cell and/or gene therapy to market is US$1943 M (95% CI US$1395 M, US$2490 M).

Conclusion: This knowledge can inform financial planning for biopharma companies looking to enter the space and inform policy makers within the context of the commercialization and pricing of such therapies.