EU CRO Group Seeks to Widen Act on Post-Authorisation Efficacy Studies

By Zachary Brennan

- Last updated on GMT

EU CRO Group Seeks to Widen Act on Post-Authorisation Efficacy Studies

Related tags: European union, European commission

The European CRO Federation is calling to widen the scope of a proposed delegated act related to pharmacovigilance legislation to include scenarios when observational PAES (post-authorisation efficacy studies) will be required.

The comments come as the European Commission is deciding whether to adopt the act to amend the 2010 legislation and provide more clarity on situations in which PAES may be required to complement efficacy data. The majority of respondents -- including the federation, EMA and UK’s MHRA -- support the delegated act because it would “provide legal certainty, increase predictability and contribute to a focused dialogue​” between regulators and MAHs.

The federation, meanwhile, seeks clarity from the Commission on scenarios when PAES would be voluntarily conducted and when they would be imposed on MAHs. The federation takes particular issue with observational or non-interventional studies and how the delegated act would respond to them, noting that German regulators claim this type of PAES does not exist because they are considered PASS (post-authorisation safety studies).

There is more confusion around non-interventional or observational studies than around clinical trials​,” the federation says. “Therefore, the scope of this delegated act should be widened and all possible scenarios should be covered​.” The group also says the Commission should call studies “experimental” and “observational," rather than “interventional” and “not interventional” because the latter can be “strongly misleading​.”

Non-interventional / observational studies are fundamental in evaluating a drug’s safety profile and identifying “hypotheses to be later validated by interventional / experimental studies​,” the federation says, noting the example of comparing the collection of data “under real life conditions which subsequently will be compared with historic data from pivotal clinical trials​.”

The EMA agreed with the federation, adding, “It is in the interest of public health to fully explore the potential added value of observational studies and pragmatic trials in the context of regulatory decisions​.”

The EFPIA (European Federation of Pharmaceutical Industries and Associations) also called for the expansion of the delegated act’s scope, noting that it “considers the notion that PAES should always equal randomized clinical trials (RCT) is outdated and does not correspond to current expectations​.”

Other Points of Concern

In addition, a separate section in the delegated act on data quality “may be a crucial point in the delineation between pre authorisation studies and PAES​,” the CRO federation notes. “We also would welcome some discussion on the prevention of centre selection bias (e.g. whether centres should be identified by a random procedure)​.”

More details on the design and prerequisites for PAES -- such as protocol, sample size determination and data quality -- are also sought by the federation.

Related news

Related products

RACE Act Prompts Pediatric Oncology Trials

RACE Act Prompts Pediatric Oncology Trials

PRA Health Sciences | 04-May-2020 | Technical / White Paper

Many providers prescribe drugs off-label to pediatric patients, even though there have been few pediatric trials for many of these drugs. In response,...

Manufacturing Cyto and Non-Cyto Drugs in One Facility

Manufacturing Cyto and Non-Cyto Drugs in One Facility

Baxter BioPharma Solutions | 01-Mar-2020 | Technical / White Paper

Recent market reports suggest increasing product niches, which may lead to decreasing numbers of units per product, making dedicated facilities less practical....

What do big pharma companies spend on R&D?

What do big pharma companies spend on R&D?

Zymewire | 15-Aug-2019 | Technical / White Paper

The free Big Pharma R&D Spend report examines the financial performance and research & development investments of the top 25 pharmaceutical companies...

Related suppliers

Follow us


View more