Euro Roundup: EMA launches new PRIME tools to accelerate treatments for unmet medical needs

The European Medicines Agency (EMA) has introduced three features to improve its PRIME scheme for supporting the development of medicines targeting unmet medical needs.

EMA launched the initiative in 2016 and, building on its analysis of the first five years of the program, piloted three features from 2023 to 2025. The pilot found the features enhance regulatory agility and improve support to developers, EMA said, leading the agency to make them permanent parts of PRIME.

One feature is a regulatory roadmap and product development tracker. EMA designed the tracker to provide a framework for monitoring product development and replace the previous annual update. The pilot found the tool effectively tracks critical development issues, but also revealed challenges related to usability and administrative burdens.

The feedback led EMA to identify a need to refine the structure and formatting to enhance usability for developers and streamline interpretation for assessors. Other recommendations include strengthening guidance on update frequency, level of detail, and impact categorization.

EMA also piloted an expedited scientific advice procedure. The pilot found the procedure was faster than the standard scientific advice pathway. Yet the pilot also revealed a tension, with drug developers calling for more agility and broader access criteria while regulators stressed the importance of maintaining a focused scope.

Key recommendations include the expansion of scenarios where the expedited procedure can be sought, including issues arising from kick-off and submission readiness meetings. EMA will also explore ways to increase flexibility outside Scientific Advice Working Party plenary cycles, which constrained the speed of the procedure during the pilot.

Submission readiness meetings are the third new feature. The meetings provide an opportunity for the drug developer, rapporteur, and EMA and national regulatory experts to talk 9 to 12 months before a filing for approval. Although the pilot suggested the meetings are worthwhile, developers called for more flexible timing while regulators noted variability in discussions of critical operational topics.

EMA will consider more flexible timing, which could enable meetings to happen when more data are available to discuss. Other recommendations include the creation of clear routes for timely follow-up of outstanding risks, including expedited scientific advice for issues requiring formal feedback.

Press Release

EU watchdogs call for data protection clarity amid push to streamline trial regulation

European Union watchdogs have warned that planned changes to processing personal data under the Clinical Trials Regulation (CTR) need clarifying and limiting.

The European Biotech Act proposed in December includes changes to a section of CTR dealing with data protection. Independent EU body the European Data Protection Board (EDPB) commented on the CTR changes last week. EDPB published a joint opinion with the European Data Protection Supervisor (EDPS), the EU’s independent data protection authority.

While noting that the changes are intended to simplify regulations, EDPB and EDPS said work to achieve that goal should not lower the level of protection applied to clinical trial data. The watchdogs want the final legislation to reflect the “particularly sensitive” nature of the personal data processed during clinical trials.

EDPB and EDPS support the goal of harmonizing how sponsors and investigators process personal clinical trial data. The watchdogs said establishing a single legal basis for processing personal data will help to address the existing fragmentation of the regulatory framework and improve legal clarity.

However, EDPB and EDPS also recommended that changes be made before the update takes effect. The watchdogs called on the Commission to clarify the roles of sponsors and investigators as sole or joint controllers and confirm that the 25-year minimum retention period applies only to personal data in the clinical trial master file.

EDPB and EDPS also discussed proposals intended to provide a legal basis for further processing of data in other clinical trials or for scientific research. The watchdogs understand that one provision of the CTR proposals is intended to provide the legal basis and recommend that the Commission more clearly state that objective in the revised legislation.

Other recommendations affect the introduction of obligations on sponsors that plan to use AI in clinical trials. EDPB and EDPS advised the Commission to clarify that the obligations under CTR are in addition to requirements imposed by the AI Act.

Joint Opinion

PRAC recommends chikungunya vaccine label update to reflect aseptic meningitis risk

EMA’s safety committee has recommended Valneva updates the label of its chikungunya vaccine Ixchiq to reflect the latest evidence on the risk of aseptic meningitis.

The Pharmacovigilance Risk Assessment Committee (PRAC) assessed the risk of aseptic meningitis and other nervous system disorders in Ixchiq recipients last year, concluding that the vaccine should only be given when there is a significant risk of chikungunya infection. At that time, most reported serious side effects were in people 65 and older.

A case of aseptic meningitis in a healthy young adult following vaccination with Ixchiq led PRAC to review the risks. Aseptic meningitis is an inflammation of membranes surrounding the brain and spinal cord.

The Ixchiq label already lists aseptic meningitis as a side effect with an unknown frequency. Following the update, the vaccine label will state that serious side effects, such as aseptic meningitis, have been seen in healthy young adults.

PRAC is assessing whether new information on the risk of aseptic meningitis and other emerging safety evidence affects the balance of benefits and risks of Ixchiq. The committee is evaluating the impact of the information in a six-monthly periodic safety update report assessment that will end in June.

Press Release

EMA updates advice on determining therapeutic equivalence of antibiotic tablets

EMA has provided guidance on determining the therapeutic equivalence of tablet formulations of the antibiotic rifaximin.

Discussing an oral 200 mg tablet formulation, EMA said the antibiotic acts locally in the intestinal lumen and has low but measurable absorption in the gastrointestinal tract. Rifaximin’s properties prompted a question about whether fasting and fed study results can be paired with comparative dissolution data to determine therapeutic equivalence.

EMA said registration may be based on the demonstration of bioequivalence both under fasting and fed conditions. When bioequivalence has been shown, studies with clinical endpoints are not needed. The conclusion reflects the fact that rifaximin does not fulfill the requirements for a biowaver and has some degree of systemic bioavailability.

The agency provided the information in an update to its question-and-answer document about clinical pharmacology and pharmacokinetics.

EMA Q&A

Other News:

An EFPIA analysis has found declining regulatory performance is affecting Europe’s competitiveness as a global life science location. While EFPIA found regulatory performance is improving in China and the US, the pharma trade group said the relative decline in new drug approvals and low use of expedited reviews in Europe indicate declining performance in the region. EFPIA Report

MedTech Europe, Swiss Medtech, and SVD have called a recent deal between the EU and Switzerland a crucial step toward reducing regulatory duplication and improving the efficiency of supply chains. The medical devices chapter of a mutual recognition agreement lapsed in 2021, forcing Switzerland to set up its own pathways. Switzerland could accept EU-certified devices under the new deal. Press Release

EMA said the war in the Middle East is causing “various levels of disruptions” to drug supply. While there have been no reports of critical shortages, companies have told EMA that interruptions to air freight and maritime routes, plus rising costs, are affecting operations. EMA Notice

Euro Roundup: EMA launches new PRIME tools to accelerate treatments for unmet medical needs

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