EC Releases New Advanced Therapy Guidelines, Site Suitability Template

October 21, 2019

The European Commission has finalized guidelines for trials of cell- and tissue-based therapies as part of the transition to its new clinical trials regulations expected in 2020.

The guidelines, designed to supplement the more general ICH E6 good clinical practices guideline, make recommendations for handling, storage and use of human tissues and cells, which the EC refers to as advanced therapy medicinal products (ATMP).

They also present recommendations for tracing ATMPs from the donor through delivery to the trial site and ultimately to the recipient.

Additional safeguards should be applied for children and pregnant women, or in the case where a subject has an immediate need for the life-saving therapy, the guidelines say. They also recommend patient follow-up after the end of a trial and staff training on the process of taking biopsies/extracting cells before manufacturing or administering the investigational product.

The guidelines also say control groups receiving placebo only should not be subjected to a procedure if it presents more than minimal risk and minimal burden. Any invasive procedures should be outlined in the protocol or investigator’s brochure if they are part of the trial.

The EC also released a new Site Suitability Template for sponsors of all trials to use in their applications. The template, which must be completed for all sites in a trial, describes the site, facilities, equipment and trial procedures.

The new clinical trials regulation took effect in May 2016, but the EC has allowed trials to follow guidelines of the former clinical trials directive until the commission develops or revises all compliance documents. The commission still is working on guidelines on the content of the trial master file and a question-and-answer document on the new regulations.

Read the new guidelines here:

View the template here:

McKesson and Aetion Build RWE Platform for Cancer Trials

McKesson and Aetion have entered into a collaboration to use real-world evidence to support clinical trials in oncology, targeting tumors, breast, lung and melanoma cancers.

The collaboration combines Aetion’s evidence platform with data from McKession’s IKnowMed electronic health record system.

The platform will be available to researchers at Brigham and Women’s Hospital in Boston, which is leading the FDA’s RWE pilot project RCT Duplicate (CenterWatch Weekly, April 15, 2019).

The two companies are both members of the Friends of Cancer Research RWE pilot project. The project focuses on assessing frontline treatments in patients with advanced non-small cell lung cancer.

NSF International Acquires CRO AMAREX

NSF International has acquired a majority interest in Amarex Clinical Research, taking over the CRO’s current phase 1 trials and adding trial management to its suite of life science consulting services.

Maryland-based CRO Amarex will be absorbed by NSF’s global health and science consulting business and will be called Amarex Clinical Research. It will retain its chief executive officer, Kazem Kezempour.

The merger will allow NSF to add clinical trial management and operations expertise, biostatistics, data management, pharmacovigilance and data safety monitoring committee capabilities to its medical writing and consulting services.

Amarex will gain access to NSF’s international expert network. Financial terms were not disclosed.

Real-World Data Only Partially Valid, Study Shows

The ability of real-world data (RWD) to fully replicate clinical trial results is limited, a new study says, but RWD can be used to support some of a trial’s data needs.

In a study published in the Journal of the American Medical Association, a group of university researchers examined the data from 220 clinical trials conducted in the U.S. in 2017, matching them to information gleaned from electronic health records, insurance claims, disease registries and patient-reported data.

Only 15 percent of the trials’ data could be fully replicated using the RWD. In 72 percent of cases examined, however, conclusions reached using RWD matched at least one aspect of a trial’s data, such as inclusion/exclusion criteria or primary endpoints.

“Although the increasing use of real-world evidence in medical research presents opportunities to supplement or even replace some clinical trials,” the researchers conclude, “observational methods are not likely to obviate the need for traditional clinical trials.”