WHO informal consultation on revision of guidelines on evaluation of similar biotherapeutic products, virtual meeting, 30 June – 2 July 2021

Publisher Copyright: © 2022The WHO informal consultation was held to promote the revision of WHO guidelines on evaluation of similar biotherapeutic products (SBPs) adopted by the Expert Committee on Biological Standardization (ECBS) in 2009. It was agreed in the past consultations that the evaluation principles in the guidelines are still valid, but a review was recommended to provide more clarity and case-by-case flexibility. The opportunity was therefore taken to review the experience and identify areas where the current guidance could be more permissive without compromising its basic principles, and where additional explanation could be provided regarding the possibility of reducing the amount of data needed for regulatory approval. The meeting participants applauded the leading role taken by the WHO in providing a much-needed streamlined approach for development and evaluation of SBPs which will provide efficient and cost-effective product development and increase patient access to treatments. It was recognized that the principles as currently described in the draft WHO guidelines are based on sound science and experience gained over the last fifteen years of biosimilar approvals. However, since these guidelines when finalised will constitute the global standard for biosimilar evaluation and assist national regulatory authorities in establishing revised guidance and regulatory practice in this complex area, it was felt that further revision and clarity on certain perspectives in specific areas was necessary to dispel uncertainties arising in the current revised version. This report describes the principles in the draft guidelines, including topics discussed and consensus reached.Peer reviewe

Consequences, Opportunities and Challenges of Modern Biotechnology for Europe

Modern biotechnology products and processes are an integral part of the EU economy, mainly in manufacturing, including pharmaceuticals, agro-food and healthcare. While some products are not recognizable to the broader public (e.g. the use of genetic markers in livestock breeding), others are used on a daily basis (detergents with enzymes, recombinant insulin) or have become subject of public discussion (e.g. genetically modified crops). Modern bioetchnology contributes significantly to the achievement of major EU polciy objectives.JRC.J.5-Agriculture and Life Sciences in the Econom

Marine Biotechnology: A New Vision and Strategy for Europe

Marine Board-ESF The Marine Board provides a pan-European platform for its member organisations to develop common priorities, to advance marine research, and to bridge the gap between science and policy in order to meet future marine science challenges and opportunities. The Marine Board was established in 1995 to facilitate enhanced cooperation between European marine science organisations (both research institutes and research funding agencies) towards the development of a common vision on the research priorities and strategies for marine science in Europe. In 2010, the Marine Board represents 30 Member Organisations from 19 countries. The Marine Board provides the essential components for transferring knowledge for leadership in marine research in Europe. Adopting a strategic role, the Marine Board serves its Member Organisations by providing a forum within which marine research policy advice to national agencies and to the European Commission is developed, with the objective of promoting the establishment of the European Marine Research Area

Consequences, Opportunities and Challenges of Modern Biotechnology for Europe - The Analysis Report

Biotechnology is generally considered one of the key technologies of the 21st century, with a potentially wide range of applications in e.g. healthcare, agriculture, and industrial production processes. However, this notion has not yet been substantiated, as the diversity of sectors in which biotechnology is applied makes it difficult to investigate its actual degree of diffusion. Against this background and following a request from the European Parliament, the European Commission initiated the Biotechnology for Europe Study (Bio4EU Study). The study's objectives are to assess the contributions of modern biotechnology to the achievement of major European policy goals, and to increase public awareness and understanding of modern biotechnology.This report presents an analysis of the collected data with a view to assessing the contributions of modern biotechnology to major EU policy goals such as economic growth and job creation (Lisbon Agenda), and environmental sustainability and public health (Sustainable Development Strategy). As such, the report can be considered a background document of the Bio4EU synthesis report, which sets out the main findings of the study.JRC.J.5-Agriculture and Life Sciences in the Econom

Surfactants, nanomedicines and nanocarriers: a critical evaluation on clinical trials

Advances, perspectives and innovation in drug delivery have increased in recent years; however, there is limited information available regarding the actual presence of surfactants, nanomed-icines and nanocarriers in investigational medicinal products submitted as part of a request for authorization of clinical trials, particularly for those authorized in the European Economic Area. We retrieve, analyze and report data available at the Clinical Trial Office of the Italian Medicines Agency (AIFA), increasing the transparency and availability of relevant information. An analysis of quality documentation submitted along with clinical trials authorized by the AIFA in 2018 was carried out, focusing on the key terms “surfactant”, “nanomedicine” and “nanocarrier”. Results suggest potential indications and inputs for further reflection and actions for regulators to actively and safely drive innovation from a regulatory perspective and to transpose upcoming evolution of clinical trials within a strong regulatory framework

The Nature and the Extent of the Market for Technology in Biopharmaceuticals

This paper explored an unusually comprehensive dataset of more than 2,000 drug R&D projects all over the world during the 1990s. This enabled us to characterise several features of the innovation process in pharmaceuticals, particularly the different role and comparative R&D performance of the large established drug companies vis-à-vis smaller high-tech specialist firms - the so-called New Biotechnology Firms (NBFs). Our results can be summarised as follows: a)The NBFs are largely an American phenomenon. More than half of the drug R&D projects originated in the US are by NBFs, while almost 90% of the drug R&D projects originated in Europe are from established pharmaceutical firms; b)Collaborative R&D projects are consistently more likely to occur in the US than in Europe. However, in-house projects are a significant majority of the drug R&D projects that entered the clinical stages. c)The established pharmaceutical companies have comparative advantages with respect to the NBFs in drug development (clinical trials). In drug discovery there is no advantage related to scale. Unlike clinical developments, where the large firms seem to have superior capabilities when compared to the NBFs, in discovery there is no inherent superiority (in terms of ultimate probability of success of the compounds) of either the NBFs or the large firms. d)The NBFs are not specialized in more risky R&D projects. In fact, more risky drug projects (i.e. drugs for which there is no or there are few existing remedies) are more likely to be undertaken by the larger pharmaceutical companies. This suggests that scale, market power, and the ability to moblise large amounts of resources are key factors in enabling the firms to sustain such higher risks. e)Other things being equal, the projects originated by the NBFs are more likely to fail in the earlier clinical stages. This suggests that the NBFs perform a good deal of exploration without incurring the higher costs of failing at later stages.Innovation, Markets for technology, Biopharmaceutical Industry, R&D Collaboration

Regul Toxicol Pharmacol

A history of safe use is a backbone of safety assessments for many current probiotic species, however, there is no global harmonization regarding requirements for establishing probiotic safety for use in foods and supplements. As probiotic manufacturers are increasingly seeking to use new strains, novel species, and next-generation probiotics, justification based on a significant history of use may be challenged. There are efforts underway by a variety of stakeholders, including the United States Pharmacopeia (USP), to develop best practices guidelines for assessing the quality and safety of probiotics. A current initiative of the USP seeks to provide expert advice specific to safety considerations for probiotics. Toward this goal, this review provides a helpful summary guide to global regulatory guidelines. We question the suitability of traditional animal toxicology studies designed for testing chemicals for relevance in assessing probiotic safety. This includes discussion of the use of excessive dose levels, the length of repeated dose toxicity studies needed, and the most suitable animal species used in toxicology studies. In addition, the importance of proper manufacturing practices with regard to final product safety are also included. Thus, an outline of essential parameters of a comprehensive safety assessment for a probiotic are provided.CC999999/ImCDC/Intramural CDC HHSUnited States

Personalized medicine : legal and ethical challenges

The printed version of this title will be available on Unigrafia online store: https://shop.unigrafia.fi/?lang=enThe paradigm of personalized medicine is an emerging topic, triggering some specific legal and ethical challenges as regards data collection, sharing and use, informed consent, privacy and public trust, and the changing status of patients and social equality. These legislative developments and challenges have been discussed in light of the Finnish and the common-European experiences. During recent years, the Finnish legislative processes aiming at generating ‘innovation-friendly’ legislation for scientific research purposes as well as integrating genomic research results into the clinical setting have been heavily challenged by rapid developments in technology and medicine. In particular, there is a need to pursue the right balance between scientific and commercial interests, public health, and individual rights. We aim at providing insights into the legislative processes surrounding personalized medicine with a special focus on how the freedom of science, equitable access to healthcare, public health, and commercial issues that must be balanced with individual rights as expressed in the EU Charter and the Council of Europe’s Oviedo Convention on Human Rights and Biomedicine. A wide spectrum of different types of challenges arises; among other things, there is a need to discuss the Finnish and international legislation of genome testing in terms of consent on behalf of a young child. Also the legal and ethical aspects of disruptive gene-editing technologies need to be analysed: How should we interpret the concept of human dignity in the bioethical discussion surrounding germline editing? Furthermore, an overview of ongoing initiatives to accelerate the market-entry of advanced therapy medicinal products will be provided. The European regulators are now taking measures to create a facilitative regulatory environment that encourages innovation, protects public health, and enables timely patient access to innovative, new therapies whilst ensuring patient safety. The role of risk-proportionate adaptations to clinical trials and GMP manufacture along with the European Medicines Agency’s early-access incentives and initiatives are presented as potential facilitators of market entry. Furthermore, in this context, the role of conditional reimbursement schemes and risk sharing-agreements is also discussed in light of the newest Finnish experiences. Furthermore, in terms of patent law, some specific challenges arise; in this anthology, attention is paid to the recent transatlantic legal dispute over a patent concerning the use of the CRISPR/Cas9 system in eukaryotic cells. Finally, some considerations beyond legal or ethical aspects of personalised medicine are presented. How can machine learning be used to support personalized care that addresses the patient’s needs

Genomic Research in Reproduction and Biobanking: An Analysis of International Legal Approaches

The article examines the regulatory issues of genomic research in human reproduction and biobanking. The approaches to legal regulation at the level of international law, integration organizations, and individual states are analyzed. Based on the analysis, proposals for legal regulation are formulated. Regarding the legal regulation of genomic research in the field of biobanking, the article discusses the issue of the legal standing of biobanks, approaches to the legal regulation of biobanks in various countries, and formulates proposals for the commercial use of the results of genomic research in biobanking. The article also provides an overview of some judicial decisions that had a certain impact on forming legal regulation of genomic research in the field of biobanking and human reproduction