Call for More Effective Regulation of Clinical Trials with Advanced Therapy Medicinal Products Consisting of or Containing Genetically Modified Organisms in the European Union.

2021 ◽  
Author(s):  
Stuart Gavin Beattie
Keyword(s):  
European Union ◽  
Clinical Trials ◽  
Genetically Modified Organisms ◽  
Genetically Modified ◽  
The European Union ◽  
Medicinal Products ◽  
Advanced Therapy Medicinal Products ◽  
Advanced Therapy

scholarly journals Decision-making on marketing authorization of Advanced Therapy Medicinal Products in the European Union

Cytotherapy ◽  
2017 ◽  
Vol 19 (5) ◽  
pp. S85
Author(s):  
S. de Wilde ◽  
D. Coppens ◽  
M.L. de Bruin ◽  
H.G. Leufkens ◽  
H. Guchelaar ◽  
...  
Keyword(s):  
Decision Making ◽  
European Union ◽  
Marketing Authorization ◽  
The European Union ◽  
Medicinal Products ◽  
Advanced Therapy Medicinal Products ◽  
Advanced Therapy

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.

scholarly journals Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union

2021 ◽  
Vol 12 ◽  
Author(s):  
Carolina Iglesias-Lopez ◽  
Antònia Agustí ◽  
Antonio Vallano ◽  
Merce Obach
Keyword(s):  
European Union ◽  
Clinical Trials ◽  
Added Value ◽  
Life Cycle Approach ◽  
The European Union ◽  
Open Label ◽  
Advanced Therapy ◽  
Advanced Therapies ◽  
Number Of Patients ◽  
The Eu

Several advanced therapy medicinal products (ATMPs) have been approved in the European Union (EU). The aim of this study is to analyse the methodological features of the clinical trials (CT) that supported the marketing authorization (MA) of the approved ATMPs in the EU. A systematic review of the characteristics of pivotal CT of ATMPs approved in the EU until January 31st, 2021 was carried out. A total of 17 ATMPs were approved and 23 CT were conducted to support the MA (median, 1, range, 1–3). Of those studies, 8 (34.78%) were non-controlled and 7 (30.43%) used historical controls. Only 7 (30.4%) were placebo or active-controlled studies. Among all CT, 21 (91.3%) were open-label and 13 (56.52%) had a single-arm design. To evaluate the primary endpoint, 18 (78.26%) studies used an intermediate and single variable. The median (IQR) number of patients enrolled in the studies was 75 (22–118). To date, ATMPs’ approval in the EU is mainly supported by uncontrolled, single-arm pivotal CT. Although there is a trend toward an adaptive or a life cycle approach, a switch to more robust clinical trial designs is expected to better define the benefit and the therapeutic added value of ATMPs.

Sign in / Sign up

Export Citation Format

Share Document