Euro Roundup: EMA seeks feedback on streamlining drug development for life-threatening diseases
The European Medicines Agency (EMA) is running a consultation into plans to streamline the nonclinical development of treatments for severely debilitating or life-threatening diseases.
EMA outlined its plans in a draft concept paper, in which it explained the problems created by the lack of guidance on streamlining nonclinical development outside of oncology. The International Conference on Harmonization’s (ICH) S9 guideline accelerates R&D in cancer. Lacking an equivalent framework for other therapeutic areas, EMA allows deviations from standard requirements on a case-by-case basis.
Deviations are supported by ICH M3, which “mentions the potential for flexibilities in the toxicological evaluation for pharmaceuticals intended for life-threatening or serious diseases,” EMA said. However, the lack of guidance on implementing the flexibilities has resulted in an ad hoc approach, which EMA said can cause “inconsistency in evaluations and lack of harmonization” about the need for nonclinical tests.
Building on its experience of enabling rapid nonclinical safety evaluation during the COVID-19 pandemic, EMA plans to “consolidate the regulatory view” and describe a rationale for tailored testing programs in a reflection paper. The agency is collecting feedback on the plans until 30 September to inform work on the reflection paper, a draft version of which is scheduled for publication in the third quarter in 2027.
EMA’s reflection paper will provide examples of where streamlined development could be possible, as well as details of when safety testing in line with ICH M4 and S6 may remain appropriate. Chronic toxicity studies may be warranted for some treatments of severely debilitating or life-threatening diseases, EMA said.
The agency also intends to cover the potential to defer, and otherwise change the timing and duration of, nonclinical studies. EMA is proposing to use weight of evidence (WoE) approaches to evaluate when to offer a streamlined pathway, for example by using data from “similar in-class products or platform(s)” to inform safety assessments.
EMA sees new approach methodologies (NAMs) as complementary to WoE strategies. NAMs provide alternatives to animal testing in nonclinical development. EMA’s incorporation of NAMs into its advice on streamlining nonclinical development reflects its plan for the reflection paper to put a “strong emphasis” on replacing, reducing, and refining the use of animals in drug development.
EDQM starts consultation into new safeguards against EG and DEG contaminants
The European Directorate for the Quality of Medicines and HealthCare (EDQM) is seeking feedback on stronger safeguards against ethylene glycol (EG) and diethylene glycol (DEG) contamination.
EG and DEG are toxic substances linked to hundreds of deaths. Seeking to protect patient safety, EDQM is running a consultation into an analytical procedure intended to make detection of the impurities more robust. The proposed procedure will form part of the European Pharmacopoeia’s macrogols monograph. The monograph covers 12 grades of macrogol, a substance also known as polyethylene glycol.
The proposed procedure is a gas chromatography technique that uses a capillary column. Using the technique, drugmakers can “quantify the impurities via a derivatization step and the use of an internal standard,” EDQM said.
Other proposed changes include revised limits for EG and DEG. The current monograph sets a combined limit for the substances. EDQM is seeking feedback on plans to set individual limits for the contaminants: 620 ppm for EG and 0.1% for DEG. As well as being used as adulterants, both substances are impurities, especially in low-molecular mass grades of macrogols.
The consultation, which closes 30 September, is part of a broader effort to improve the control of EG and DEG. The work includes recently adopted propylene glycol and glycerol monographs, as well as ongoing efforts to prepare dedicated EG and DEG tests for liquid sorbitol and maltitol.
Swissmedic clarifies QR code requirements after rise in queries about regulations
The Swiss Agency for Therapeutic Products (Swissmedic) has updated its mobile technologies guidance to clarify the rules on using QR codes on medicinal product packaging.
Swissmedic has seen an uptick in questions about QR code captions and placement, as well as requests for clarification on the details and placement of URLs. In response, the agency has updated its guidance on mobile technologies and added two questions to an associated Q&A document.
The revised guidance took effect on 1 August. Through the update, Swissmedic has clarified its advice on the placement and captions for QR codes. QR codes can be printed on the medicinal product information or the packaging.
Swissmedic recommends manufacturers voluntarily provide a short URL in addition to the QR code and label the QR code with a short, meaningful caption such as “medicinal product information.” The agency sees the voluntary actions as ways to make QR codes more user-friendly.
The new Q&A questions cover the acceptability of QR codes linking to official repositories of information on drug products. Swissmedic clarified that linking QR codes to the official repositories for patients and healthcare professionals is its preferred method of implementation, “given the legally established duty of publication of medicinal product information via” the platforms.
Companies can link to other platforms as long as “the relevant approved medicinal product information is provided there in complete and up-to-date form,” Swissmedic said.
EMA posts guidance on submitting annual safety reports via new safety module
EMA has published guidance on submitting annual safety reports (ASRs) via the new safety module in compliance with the Clinical Trials Regulation (CTR).
Under CTR, sponsors must file an annual report on the safety of each investigational medicinal product used in a clinical trial, other than placebo. ASRs evaluate the evolving safety profile and benefit-risk balance of a drug candidate, including active or proposed actions to mitigate potential risks and protect clinical trial participants.
On 28 September, EMA plans to integrate the new ASR safety module into the Clinical Trial Information System (CTIS). The safety module is currently implemented in the CTIS training environment, giving drug developers and authorities an opportunity to train themselves on the new workflow.
All new ASRs must be submitted through the new safety module starting 28 September. EMA will leave the old system in place for two months to allow Member States to finalize ASRs submitted before the new module goes live. During that two-month period, sponsors will be able to access relevant requests for information via the old system.
European Commission releases tool to check joint clinical assessment eligibility
The European Commission has created a tool to help health technology developers determine if their medicinal products are eligible for joint clinical assessment (JCA).
Under the Health Technology Assessment Regulation (HTAR), new medicines for the treatment of cancer, as well as advanced therapy medicinal products, have been subject to JCAs since January 2025. Officials will expand the program to cover orphan drugs in 2028 and to cover all medicinal products within the scope of the HTAR in 2030.
The Commission’s eligibility tool poses a series of questions. After answering whether they are filing for a marketing authorization or for a variation, users are sent down branching paths to determine whether their products are subject to JCAs. The questions cover the timing of the planned application and details of the product.
Other News:
Swissmedic updated guidance on providing, distributing, and publishing officially mandated information to reflect changes that took effect on 1 July. Swissmedic Notice
