Regulatory requirements for clinical trial and marketing authorisation application for cell-based medicinal products

2009 ◽  
Vol 53 (1) ◽  
pp. 24-29 ◽  
Author(s):  
P. Salmikangas ◽  
E. Flory ◽  
J. Reinhardt ◽  
T. Hinz ◽  
R. Maciulaitis
Keyword(s):  
Clinical Trial ◽  
Marketing Authorisation ◽  
Medicinal Products ◽  
Regulatory Requirements ◽  
Marketing Authorisation Application

Clinical trial supplies: investigational medicinal products (IMPs)

2016 ◽  
Keyword(s):  
Clinical Trial ◽  
Study Drug ◽  
Storage Conditions ◽  
Medicinal Products ◽  
Regulatory Requirements ◽  
Expiry Date ◽  
Clinical Trial Directive ◽  
Qualified Person ◽  
Protocol Compliance

This chapter describes the procedures and records associated with accountability of investigational and non-investigational medicinal products (IMP and NIMP) used in clinical trials, to show that the drug has been labelled according to the regulations, stored in conditions to keep it stable, prepared and administered to the correct subjects in accordance with the protocol, has been fully accounted for and destroyed if unused. Manufacture of IMP is discussed together with methods of blinding. The role of the Qualified person (QP) is reviewed. The need for study drug accountability is discussed in context with the regulatory requirements (Clinical Trial Directive 2001/20/EC and in particular, GMP Directive 2003/94/EC Annex 13). The chapter explains what needs to be accounted for and describes the types of records including: labelling records, delivery and transportation, receipt, storage, preparation, dispensing and administration, unblinding records, reconciliation, returns and destruction. Discussions are included on protocol compliance, management of excursions resulting from incorrect storage conditions, management of dosing errors and documentation errors, expiry date re-labelling, drug recall and re-supply. Sections are included on considerations for non-commercial studies, GMP requirements for UK Phase I clinics and sites requiring a MIA (IMP) license.

scholarly journals Regulatory Requirements for the Quality of Allergen Products for Allergen Immunotherapy of Food Allergy

2021 ◽  
Vol 21 (5) ◽  
Author(s):  
Lisa Englert ◽  
Vera Mahler ◽  
Andreas Bonertz
Keyword(s):  
Allergen Immunotherapy ◽  
Major Allergen ◽  
Food Allergies ◽  
Manufacturing Processes ◽  
Critical Element ◽  
Medicinal Products ◽  
Regulatory Requirements ◽  
Quality Aspects ◽  
Allergenic Activity ◽  
Recent Developments

Abstract Purpose of Review Medicinal products for allergen immunotherapy (AIT) of food allergies have gained enormous momentum in recent years. With this new class of products entering marketing authorization procedures, compliance to regulatory requirements becomes a critical element. Here, an overview is provided on specific requirements and aspects concerning the quality control and manufacturing of these products. Recent Findings Recent developments in the field of AIT for food allergies are divers, including products for oral, epicutaneous, and subcutaneous application, most notably targeting egg, milk, and peanut allergy. As the source materials for food AIT product are typically produced for food consumption and not for medicinal purposes, unique challenges arise in the manufacturing processes and controls of these medicinal products. Individual approaches are needed to assure acceptable quality, including control of relevant quantitative and qualitative characteristics. Major characteristics for quality verification include determination of protein content, total allergenic activity, and major allergen content. The applied manufacturing processes need to be established such that relevant process parameters are kept within justified limits and consistency of produced batches is assured. Summary Allergen products for food AIT present specific challenges with respect to quality aspects that differentiate them from other commonly available AIT products. While established regulation is available and provides clear guidance for most aspects, other issues require consideration of new and individual settings relevant here. Consequently, as experience grows, respective amendments to currently available guidance may be needed.

The Magistral Preparation of Advanced Therapy Medicinal Products (ATMPs)

2020 ◽  
pp. 1-7
Keyword(s):  
Marketing Authorisation ◽  
Health Care Systems ◽  
Human Keratinocytes ◽  
Public Health Care ◽  
Medicinal Products ◽  
Bacteriophage Therapy ◽  
Access To Treatment ◽  
Advanced Therapy Medicinal Products ◽  
Advanced Therapy ◽  
Advanced Therapies

Advanced Therapy Medicinal Products (ATMPs) embody innovative therapies that have created great hope for patients suffering from previously untreatable diseases. Unfortunately, the pharaonic cost to produce and authorise ATMPs is a challenge for both patients and public health care systems, ultimately reducing patients’ access to treatment. Over the last 11 years, only 15 ATMP marketing authorisation applications received a positive draft opinion from the European Medicines Agency’s (EMA’s) Committee for Advanced Therapies (CAT). Moreover, due to poor return on investment, several ATMPs have already been removed from the market. In addition to the centralised procedure to obtain a marketing authorisation, the legislator foresees an alternative route for authorising ATMPs, the so-called “ATMP Hospital Exemption”. However, such ATMPs must be produced on a limited scale, on a non-routine basis. As a result, valuable ATMP therapies that have been used for years in hospitals may disappear. To avoid this, we propose, in this paper, an additional possibility to regularise ATMPs: the “Magistral Preparation of ATMPs”. It is a feasible pathway, which was already proposed for bacteriophage therapy, and which is particularly suitable for personalised therapies and considerably decreases the cost of the final products. We also discuss the practical impact of the ATMP regulation for (for-profit) industries and for (non-profit) hospitals. Two practical examples, the cultured human chondrocytes and the cultured human keratinocytes, are discussed.

scholarly journals Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs

BMJ Open ◽  
2017 ◽  
Vol 7 (9) ◽  
pp. e017358 ◽  
Author(s):  
Viviana Giannuzzi ◽  
Annalisa Landi ◽  
Enrico Bosone ◽  
Floriana Giannuzzi ◽  
Stefano Nicotri ◽  
...  
Keyword(s):  
Marketing Authorisation ◽  
Development Process ◽  
Developmental Process ◽  
Clinical Stage ◽  
Phase Iii ◽  
Marketing Approval ◽  
Medicinal Products ◽  
Safety Issues ◽  
Stage Of Development ◽  
Orphan Medicinal Products

ObjectivesThe research and development process in the field of rare diseases is characterised by many well-known difficulties, and a large percentage of orphan medicinal products do not reach the marketing approval.This work aims at identifying orphan medicinal products that failed the developmental process and investigating reasons for and possible factors influencing failures.DesignDrugs designated in Europe under Regulation (European Commission) 141/2000 in the period 2000–2012 were investigated in terms of the following failures: (1) marketing authorisation failures (refused or withdrawn) and (2) drugs abandoned by sponsors during development.Possible risk factors for failure were analysed using statistically validated methods.ResultsThis study points out that 437 out of 788 designations are still under development, while 219 failed the developmental process. Among the latter, 34 failed the marketing authorisation process and 185 were abandoned during the developmental process. In the first group of drugs (marketing authorisation failures), 50% reached phase II, 47% reached phase III and 3% reached phase I, while in the second group (abandoned drugs), the majority of orphan medicinal products apparently never started the development process, since no data on 48.1% of them were published and the 3.2% did not progress beyond the non-clinical stage.The reasons for failures of marketing authorisation were: efficacy/safety issues (26), insufficient data (12), quality issues (7), regulatory issues on trials (4) and commercial reasons (1). The main causes for abandoned drugs were efficacy/safety issues (reported in 54 cases), inactive companies (25.4%), change of company strategy (8.1%) and drug competition (10.8%). No information concerning reasons for failure was available for 23.2% of the analysed products.ConclusionsThis analysis shows that failures occurred in 27.8% of all designations granted in Europe, the main reasons being safety and efficacy issues. Moreover, the stage of development reached by drugs represents a specific risk factor for failures.

scholarly journals New European Commission Regulation on Variations to the Terms of Marketing Authorisation for Medicinal Products and its Impact on Croatian Legislation

2010 ◽  
Vol 61 (3) ◽  
pp. 311-322
Author(s):  
Adrijana Martinac ◽  
Siniša Tomić ◽  
Mirna Šimičić
Keyword(s):  
European Commission ◽  
Medicinal Product ◽  
Marketing Authorisation ◽  
Regulatory System ◽  
Efficacy And Safety ◽  
Medicinal Products ◽  
Commission Regulation ◽  
Approval Procedure ◽  
Definition Of ◽  
The Eu

New European Commission Regulation on Variations to the Terms of Marketing Authorisation for Medicinal Products and its Impact on Croatian LegislationVariations introduced to medicinal product documentation must not affect the quality, efficacy, and safety of the product. Croatian Medicinal Products Act and accompanying ordinances are largely aligned with the EU regulations. The EU has now tried to simplify the issue of variations with a new Regulation, creating differences in the definition of and approach to resolving certain types of variations between Croatia and the EU. These differences could hinder the approval procedure for variations in Croatia, particularly for medicines already approved in the EU. Amending the Croatian Ordinance on medicines already authorised in the EU would be one way of maintaining the efficiency of the Croatian regulatory system.

scholarly journals Should Anthroposophic Medicinal Products Be Regulated in Europe?

2017 ◽  
Vol 24 (1) ◽  
pp. 46-66
Author(s):  
Geneviève Michaux
Keyword(s):  
Marketing Authorisation ◽  
Holistic Approach ◽  
Anthroposophic Medicine ◽  
The European Union ◽  
Medicinal Products ◽  
Herbal Medicinal Products ◽  
European Tradition ◽  
The Status ◽  
Herbal Medicinal ◽  
Traditional Herbal Medicinal Products

European Commission’s reports suggest that the European Union should address the status of anthroposophic products, i.e. products that are developed, manufactured and prescribed in accordance with the holistic approach on which anthroposophic medicine is based. Anthroposophic products cannot be placed as such on the European market because they cannot meet the marketing authorisation or even registration requirements set out by European or national pharmaceutical law. Yet, the 95-year European tradition and good safety profile of anthroposophic products justify giving them an easier access to market. Such access can result from specific rules on anthroposophic products, but can be more efficiently achieved by encouraging the Member States to better apply the existing rules on marketing authorisation procedures or on registration of homeopathic and traditional herbal medicinal products, or by including anthroposophic substances, manufacturing methods or uses in monographs.

scholarly journals Clinical Investigator Responsibilities

2011 ◽  
Vol 7 (2) ◽  
pp. 124-128 ◽  
Author(s):  
Allison R. Baer ◽  
Susan Devine ◽  
Chris David Beardmore ◽  
Robert Catalano
Keyword(s):  
Clinical Trial ◽  
Clinical Practice ◽  
Good Clinical Practice ◽  
Regulatory Requirements ◽  
Clinical Investigator

When conducting a clinical trial, it is important that clinical investigators successfully meet all research expectations, including regulatory requirements and the Guidelines for Good Clinical Practice.

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