EC Publishes New Guidelines on Good Clinical Practices for ATMPs

Regulatory NewsRegulatory News | 16 October 2019 |  By 

Thanks to a 2007 regulation on advanced therapy medicinal products (ATMPs), the European Commission (EC) on Wednesday released new guidelines on the good clinical practice (GCP) requirements for these complex and innovative products that can include gene and cell therapies.

The 15-page guidelines discuss clinical trial design, non-clinical studies, quality of the investigational ATMPs, the safe conduct of the clinical trial, upstream interventions on subjects and administration procedures, traceability, retention of samples, protection of trial subjects, safety reporting and monitoring.

“While the general principles of GCP set out in ICH Guidelines are applicable to clinical trials with ATMPs, in some cases, it may be necessary to adapt those to the specific characteristics of ATMPs (e.g. regarding retention of samples). The implementation of additional measures may also be necessary (e.g. traceability requirements for ATMPs that contain cells or tissues of human origin, follow-up of patients after end of the clinical trial, training on upstream intervention of subjects and/or administration procedures),” the guidelines explain.

On the use of placebo in ATMP clinical trials, the guidelines note that when invasive procedures are required to administer the ATMP or for the collection or extraction of cells or tissues, control groups receiving placebo should not be subjected to a procedure if it presents more than minimal risk and minimal burden.

The guidelines also discuss the potential for unknown long-term effects with some ATMPs, explaining that the “duration of the biological activity of a given ATMP should be taken into consideration when determining the need of subject follow-up.”

But the guidelines also note, “If the risk of delayed adverse events is low, long-term follow-up is not required. Where long-term follow-up is necessary, it is recommended that the sponsor considers discussing the duration of the monitoring scheme with the concerned national competent authority.”

As far as capturing adverse events in a trial, the guidelines say that, “Where appropriate, reporting forms and data capture systems (serious adverse events forms, case report forms for recording of adverse events) should be adapted to reflect a differentiated causality assessment for each component of the ATMP (e.g. the cell-based part and the medical device part in the case of combined ATMPs), the application process and, where applicable, any required concomitant medication.”

The guidelines further discuss how the use of each investigational medicinal product should be traceable from delivery to the clinical trial site to the administration to the clinical trial subject.

And when the investigational ATMP contains cells or tissues of human origin, the traceability from the recipient of the product to the donor of the cells or tissues should be ensured. “The traceability system should be bidirectional (from donor to subject and from subject to donor) and data should be kept for 30 years after the expiry date of the product, unless a longer time period is required in the clinical trial authorization,” the guidelines say.

The release of the new ATMP GCP guidelines for ATMPs coincides with a new consultation on a manufacturing guide for ATMPs.

Guidelines on Good Clinical Practice specific to Advanced Therapy Medicinal Products


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