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  1. Article: Regulatory Aspects for Approval of Advanced Therapy Medicinal Products in the EU.

    Fürst-Ladani, Shayesteh / Bührer, Anja / Fürst, Walter / Schober-Ladani, Nathalie

    Handbook of experimental pharmacology

    2023  Volume 284, Page(s) 367–387

    Abstract: In the European Union (EU), advanced therapy medicinal products (ATMPs) undergo evaluation by the European Medicines Agency's (EMA) Committee for Advanced Therapies (CAT) to obtain marketing authorization under the centralized procedure. Because of the ... ...

    Abstract In the European Union (EU), advanced therapy medicinal products (ATMPs) undergo evaluation by the European Medicines Agency's (EMA) Committee for Advanced Therapies (CAT) to obtain marketing authorization under the centralized procedure. Because of the diversity and complexity of ATMPs, a tailored approach to the regulatory process is required that needs to ensure the safety and efficacy of each product. Since ATMPs often target serious diseases with unmet medical need, the industry and authorities are interested in providing treatment to patients in a timely manner through optimized and expedited regulatory pathways. EU legislators and regulators have implemented various instruments to support the development and authorization of innovative medicines by offering scientific guidance at early stages, incentives for small developers and products for rare diseases, accelerated evaluation of marketing authorization applications, different types of marketing authorizations, and tailored programs for medicinal products with the orphan drug designation (ODD) and the Priority Medicines (PRIME) scheme. Since the regulatory framework for ATMPs was established, 20 products have been licenced, 15 with orphan drug designation, and 7 supported by PRIME. This chapter discusses the specific regulatory framework for ATMPs in the EU and highlights previous successes and remaining challenges.
    MeSH term(s) Humans ; European Union ; Rare Diseases ; Drug Approval
    Language English
    Publishing date 2023-04-05
    Publishing country Germany
    Document type Journal Article
    ISSN 0171-2004
    ISSN 0171-2004
    DOI 10.1007/164_2023_648
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article ; Online: Development and Regulation of Connected Combined Products: Reflections From the Medtech & Pharma Platform Association.

    Kühler, Thomas C / Schoenmakers, Marc / Shergold, Oliver / Affolter, Stephan / Bolislis, Winona Rei / Foster, Ruth / Gardner, Paul / Hruschka, Svenja / Jomini, Thierry / Kaveripakam, Sathish / Mayerhofer, Karl / Scherini, Tomaso / Swierczynska, Marta / Vandal, Gretchen / Fürst-Ladani, Shayesteh

    Clinical therapeutics

    2022  Volume 44, Issue 5, Page(s) 768–782

    Abstract: Purpose: Patients taking a medicinal product in a homecare setting typically use a medical device to facilitate the injection process. Reductions in wireless connectivity costs, combined with the rapid adoption of smartphones with connectivity to cloud- ... ...

    Abstract Purpose: Patients taking a medicinal product in a homecare setting typically use a medical device to facilitate the injection process. Reductions in wireless connectivity costs, combined with the rapid adoption of smartphones with connectivity to cloud-based services, are enabling these drug delivery devices to now be connected to a digital ecosystem as connected combined products (CCPs). The purposes of this article are to identify the challenges in developing and releasing these products when they straddle different regulatory frameworks and standards and to highlight gaps in the European Union regulations.
    Methods: Industry subject matter experts from pharmaceutical, medical device, and consultancy companies, who are members of the Medtech & Pharma Platform Association, formed 4 working groups to address current best practice for developing and releasing CCPs and the different relevant regulatory frameworks. The 4 areas studied were clinical and regulatory, usability and human factors engineering, development and life cycle management, and cybersecurity.
    Findings: Development teams require new skills to create innovative products that have a good safety profile and are simple to use, such as design thinking to understand user needs and systems engineering to manage complexity and ensure interoperability. Risk management process should integrate cybersecurity, data privacy, and data integrity, whereas design control processes should enable asynchronous development cycles for hardware and software components. Regulatory frameworks exist for individual components within the CCP. However, for a complex product, regulatory guidance is needed when combining components with different risk and safety profiles and to ensure that the responsibilities and liabilities of companies contributing components are clear. The efficient management of software changes and product updates, as well as dealing with end-of-life hardware and backward compatibility to older software versions, needs agile approaches when it comes to regulatory updates.
    Implications: The regulatory uncertainties and development processes outlined in this article need to be addressed. We call for joint discussions among the various stakeholders in the fields of medicinal products, medical devices, and in vitro diagnostics, as well as standalone software, data protection, and cybersecurity experts, together with regulators and lawmakers in the European Union to meet in focused discussion groups with the aim of devising pragmatic solutions and regulations for the benefit of the sector and hence the patients it serves.
    MeSH term(s) Ecosystem ; Humans ; Pharmaceutical Preparations
    Chemical Substances Pharmaceutical Preparations
    Language English
    Publishing date 2022-04-04
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 603113-4
    ISSN 1879-114X ; 0149-2918
    ISSN (online) 1879-114X
    ISSN 0149-2918
    DOI 10.1016/j.clinthera.2022.03.009
    Database MEDical Literature Analysis and Retrieval System OnLINE

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