scholarly journals Quality Assessment of Investigational Medicinal Products in COVID-19 Clinical Trials: One Year of Activity at the Clinical Trials Office

2021 ◽  
Vol 14 (12) ◽  
pp. 1321
Author(s):  
Diego Alejandro Dri ◽  
Giulia Praticò ◽  
Elisa Gaucci ◽  
Carlotta Marianecci ◽  
Donatella Gramaglia
Keyword(s):  
Clinical Trials ◽  
Regulatory Network ◽  
Regulatory Framework ◽  
Lessons Learned ◽  
Emergency Setting ◽  
The European Union ◽  
Medicinal Products ◽  
One Year ◽  
Assessment Results

One year after the spread of the pandemic, we analyzed the assessment results of the quality documentation submitted to the Clinical Trials Office of the Italian Medicines Agency as part of the request for authorization of clinical trials with a COVID-19 indication. In this article, we report the classification of the documentation type, an overview of the assessment results, and the related issues focusing on the most frequently detected ones. Relevant data regarding the Investigational Medicinal Products (IMPs) tested in COVID-19 clinical trials and their quality profiles are provided in the perspective of increasing transparency and availability of information. Some criticalities that have been exacerbated by the management of clinical trials during the emergency period are highlighted. Results confirm that IMPs tested in authorized COVID-19 clinical trials are developed in agreement with the same legal requirements for quality, safety, and efficacy as for any other medicinal product in the European Union (EU). The same strong regulatory framework applies, and there is no lowering in the safety profile due to the pandemic; authorized IMPs meet the highest standards of quality. The regulatory network should capitalize on lessons learned from the emergency setting. Some take-home messages are provided that could support the regulatory framework to expand its boundaries by innovating and evolving even though remaining strong and effective.

scholarly journals EMEA and Gene Therapy Medicinal Products Development in the European Union

2003 ◽  
Vol 2003 (1) ◽  
pp. 3-8 ◽  
Author(s):  
Marisa Papaluca Amati ◽  
Francesco Pignatti ◽  
Alexis Nolte ◽  
Nirosha Amerasinghe ◽  
Daniel Gustafsson ◽  
...  
Keyword(s):  
Gene Therapy ◽  
European Union ◽  
Clinical Trials ◽  
Good Clinical Practice ◽  
Evaluation Agency ◽  
The European Union ◽  
Medicinal Products ◽  
Regulatory Processes ◽  
National Competent Authorities ◽  
Practice Implementation

The evaluation of quality, safety, and efficacy of medicinal products by the European Medicines Evaluation Agency (EMEA) via the centralized procedure is the only available regulatory procedure for obtaining marketing authorization for gene therapy (GT) medicinal products in the European Union. The responsibility for the authorization of clinical trials remains with the national competent authorities (NCA) acting in a harmonized framework from the scientific viewpoint. With the entry into force of a new directive on good clinical practice implementation in clinical trials as of 1 May 2004, procedural aspects will also be harmonized at EU level. Scientifically sound development of medicinal products is the key for the successful registration of dossiers and for contributing to the promotion and protection of public health. The objective of this paper is to introduce the EMEA regulatory processes and scientific activities relevant to GT medicinal products.

The Proposed EU-regulation on Clinical Trials on Medicinal Products: An Unethical Proposal?

2013 ◽  
Vol 20 (4) ◽  
pp. 347-362 ◽  
Author(s):  
Jilles Heringa ◽  
Joseph Dute
Keyword(s):  
Clinical Trials ◽  
Human Subjects ◽  
The European Union ◽  
Medicinal Products ◽  
Member States ◽  
Legal Documents ◽  
Human Use ◽  
Proposed Regulation ◽  
High Level ◽  
Force Member

Abstract The Commission has proposed a regulation ‘on clinical trials on medicinal products for human use’ to introduce one regulatory framework for clinical trials in the European Union. This regulation should replace the current clinical trials directive (2001/20/EC). In this article we describe and critically review the main provisions of the proposed regulation. We assess the consequences for a sound authorisation procedure of clinical trials and the level of protection for human subjects. We note that the proposed regulation is inconsistent with applicable international legal documents, such as the Biomedicine Convention and the Declaration of Helsinki. We conclude that the proposed regulation does not ensure a “high level of human health protection” — required by its legal basis in the TFEU — because it may force Member States concerned to accept a reporting Member States’ approval of — in their estimation — an unethical clinical trial.

scholarly journals Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan Designation

2021 ◽  
Vol 11 ◽  
Author(s):  
Jorn Mulder ◽  
Tobias van Rossum ◽  
Segundo Mariz ◽  
Armando Magrelli ◽  
Anthonius de Boer ◽  
...  
Keyword(s):  
Pancreatic Cancer ◽  
Medicinal Product ◽  
Marketing Authorisation ◽  
Clinical Stage ◽  
Treatment Options ◽  
Lessons Learned ◽  
The European Union ◽  
Medicinal Products ◽  
Stage Of Development ◽  
Dismal Prognosis

Pancreatic cancer has a dismal prognosis and only a few treatment options are available. In the European Union, pancreatic cancer classifies as a rare disease, allowing drug developers to apply for orphan medicinal product (OMP) designation. The aim of this study was to provide more detail on OMPs for pancreatic cancer. All applications for OMP designation submitted to the EMA between 2000 and 2019 were identified. For each medicinal product that received an OMP designation, the mode of drug action, use of protocol assistance, and current life cycle status was determined. Fifty-two medicinal products received an OMP designation. At the time of submission, eighteen OMPs were at the non-clinical and 34 OMPs were at the clinical stage of development. At least fourteen kinds of mode of action were explored in the condition. For eighteen out of 52 OMPs protocol assistance was sought. At the time of data analysis, one OMP received marketing authorisation and 24 OMPs were ongoing in development. Many medicinal products for pancreatic cancer received an OMP designation and the majority of these products was already in the clinical stage of development. Nonetheless, the success rate of OMPs for pancreatic cancer that reach the market is low, and increasing this rate is something to aspire. Fortunately, development is still ongoing for a part of the OMPs, and a few developers are planning to submit a marketing authorisation application in the near future. This however does not guarantee success, as pancreatic cancer remains a difficult disease to treat. Developers are advised to make optimal use of incentives such as protocol assistance, establishing (early) dialogue between regulators and drug developers and to agree on important topics such as clinical trial design.

scholarly journals The Regulation of electronic money institutions in the SADC region: Some lessons from the EU

2014 ◽  
Vol 17 (6) ◽  
pp. 2269
Author(s):  
MD Tuba
Keyword(s):  
South African ◽  
Regulatory Framework ◽  
Lessons Learned ◽  
The European Union ◽  
Payment Systems ◽  
Electronic Money ◽  
Regulatory Approach ◽  
Technological Developments ◽  
The Eu ◽  
Regulatory Challenges

This article analyses the different approaches adopted for the regulation of payment systems in a variety of legislative instruments by the European Union (EU). It looks in particular at how the institutions that issue new electronic money products are regulated and supervised by the relevant authorities in the EU, in comparison with existing institutions such as banks. It analyses some of the lessons that may be learned by the South African Development Corporation (SADC) from the regulatory approaches for electronic money institutions adopted by the EU. The article asks if the approach adopted by the EU may be useful for the future regulation of electronic money institutions in the SADC. The proliferation of electronic devices that arrived with the invention of the Internet has sparked some regulatory challenges. This development has become global and involves both developed and developing countries, including regions such as the SADC. It is asked if these technological developments should be  addressed by means of a concrete regulatory framework while they  continue to develop, instead of the regulators waiting to observe and acquaint themselves with the relevant regulatory challenges that underpin the innovations. The EU has attempted to address the anticipated  regulatory challenges that came about with the development of electronic money and to align its regulatory approach with other payment systems. This article discusses the regulatory approaches adopted in the EU and provides an overview that the SADC may use in order to adopt an effective regulatory framework for electronic money and the institutions that issue these methods of payment. It analyses both the achievements and the challenges that the EU faced (and continues to face) in developing the regulation of e-money, and recommends some possible approaches derived from the lessons learned.

scholarly journals Biosimilars: the science of extrapolation

Blood ◽  
2014 ◽  
Vol 124 (22) ◽  
pp. 3191-3196 ◽  
Author(s):  
Martina Weise ◽  
Pekka Kurki ◽  
Elena Wolff-Holz ◽  
Marie-Christine Bielsky ◽  
Christian K. Schneider
Keyword(s):  
Decision Making ◽  
European Union ◽  
Clinical Trials ◽  
Safety Data ◽  
Real Life ◽  
Medical Community ◽  
The European Union ◽  
Efficacy And Safety ◽  
Medicinal Products ◽  
Regulatory Decision

Abstract Despite the establishment of a specific approval pathway, the issuance of detailed scientific guidelines for the development of similar biological medicinal products (so-called “biosimilars”) and the approval of several biosimilars in the European Union, acceptance of biosimilars in the medical community continues to be low. This is especially true in therapeutic indications for which no specific clinical trials with the biosimilar have been performed and that have been licensed based on extrapolation of efficacy and safety data from other indications. This article addresses the concerns frequently raised in the medical community about the use of biosimilars in such extrapolated indications and explains the underlying scientific and regulatory decision making including some real-life examples from recently licensed biosimilars.

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