Euro Roundup: Committee warns ‘overly expansive’ MDR interpretation could undermine access to cell and tissue products
A Council of Europe committee has warned that “an overly expansive interpretation” of the Medical Device Regulation (MDR) could undermine access to therapies based on human blood, tissues, and cells.
The European Committee on Organs, Tissues and Cells (CD-P-TO) report looked at the interface between MDR and legislation on substances of human origin (SoHO). SoHO legislation came into force in 2024 and will apply from August 2027, creating a new framework for the use of human blood, tissues, and cells. CD-P-TO wants authorities to urgently clarify aspects of the regulations.
“Without clarification of the interface between the MDR and the SoHO Regulation before [7 August 2027], SoHO entities risk entering the post-2027 period simultaneously subject to two overlapping regulatory regimes with separate competent authorities, inspection frameworks, and documentation requirements,” the committee said.
SoHO entities depend on highly specialized, low-volume medical devices that are increasingly being withdrawn because of MDR compliance requirements, CD-P-TO said. Some companies have reintroduced devices as “research use only” (RUO) products. In other cases, SoHO entities are using MDR-compliant devices outside of their intended purposes.
Medical Device Coordination Group (MDCG) guidance covers both scenarios. The guidance goes beyond the requirements supported by MDR and creates practical difficulties for SoHO entities, CD-P-TO said. The committee’s concerns include MDCG’s conclusion that the rules on in-house medical devices apply when a health institution assigns an intended purpose to an RUO product.
When a SoHO entity uses an RUO product, it makes a clinical decision about how to deploy the device, CD-P-TO said. The committee argued that national law and the SoHO Regulation, not MDR, should govern such decisions.
CD-P-TO has called for MDCG to revise the guidance to address the concerns. The committee sees no need for the European Commission to change its MDR proposals, reflecting a belief that they provide a “structurally sound basis” that is being undermined by interpretation of the legislation.
EMA picks pancreatic cancer program for phased review to showcase reformed pharma law
The European Medicines Agency (EMA) has begun a phased review of Revolution Medicines’ metastatic pancreatic cancer drug candidate, daraxonrasib.
In a late-phase trial, Revolution found daraxonrasib nearly doubled overall survival versus chemotherapy in metastatic pancreatic cancer patients. The readout raised hopes that the biotech’s pan-RAS inhibitor can improve outcomes in a hard-to-treat tumor type. As evidence on daraxonrasib accumulated, EMA added the asset to the Cancer Medicines Pathfinder it uses to accelerate assessment of critical drugs.
Targeting a speedy review, EMA’s Committee for Medicinal Products for Human Use (CHMP) has agreed to carry out a phased assessment of daraxonrasib. The committee will review the quality, nonclinical, and clinical data as they become available and in advance of Revolution’s submission of a full approval application.
The US Food and Drug Administration already offers such rolling reviews to accelerate access to drugs. EMA could bolster its use of phased reviews through planned reforms to the European Union’s pharma legislation.
“The review of daraxonrasib will serve as an example for some of the provisions of the reformed EU pharmaceutical legislation which is expected to strengthen the use of phased reviews,” EMA said. “This approach is foreseen as a tool to allow for a more agile, streamlined evaluation of medicines which would ultimately lead to earlier patient access to promising treatments.”
The overall timeline for reviewing daraxonrasib cannot be predicted, EMA said. However, the agency expects the process to be shorter than a regular review because its committee will have assessed some of the data by the time Revolution applies for approval.
EMA and CHMP plan to “consider further medicines under development for phased review when this approach is feasible and expected to accelerate assessment.” Officials will decide whether a phased review is suitable on a case-by-case basis, considering whether the drug is likely to address an unmet medical need.
EMA schedules workshop amid push to embed women’s health considerations in regulation
EMA has arranged a women’s health workshop to further efforts to tackle issues such as representation in clinical trials and the assessment of sex-specific differences in the use and effects of medicines.
The agency framed the workshop, which it will broadcast live on 28 and 29 September, as part of its work to better embed women’s health considerations in medicines development and regulation. Women live longer than men, on average, but spend more years in poorer health. The disparity reflects gaps in disease prevention, diagnosis, and treatment, EMA said.
At the workshop, EMA will examine women’s health research gaps and identify priorities to strengthen the evidence base. Input from patients, healthcare professionals, academia, industry, and global partners will inform explorations of further opportunities.
EMA’s current activities center on representation in clinical trials, drug assessment and labeling, drugs in pregnancy and breastfeeding, and real-world evidence. The agency’s initial analysis shows that women are appropriately represented in clinical trials, but officials plan to perform more detailed assessments of the situation at different stages of development.
Upcoming areas of focus include reducing regulatory uncertainty by encouraging developers to make full use of early development support mechanisms. Through mechanisms such as its Innovation Task Force, EMA wants to help drug developers find early solutions for methodological challenges they face when developing new medicines related to women’s health.
European Commission publishes JCA reports for Amgen and PharmaMar lung cancer drugs
The European Commission has published joint clinical assessment (JCA) reports on small cell lung cancer (SCLC) drugs developed by Amgen and PharmaMar.
Weeks after publishing its first JCA report, the Commission released assessments of Amgen’s Imdylltra and PharmaMar’s Zepzelca. The first report only assessed one of the eight contexts, referred to by the acronym PICO, in which the drug could be used because of a lack of evidence. Amgen and PharmaMar provided evidence to inform assessments of all their PICOs, although there was only one for Zepzelca.
Amgen’s pivotal trial of Imdylltra in extensive-stage SCLC informed all seven PICOs and provided data for direct comparisons in three. Assessors relied on indirect comparisons between the pivotal trial and other studies to inform the other four PICOs.
Yet the assessors also noted uncertainties in Amgen’s pivotal data, including missing information on the first subsequent therapy and the treatment of patients beyond radiographic progression of the disease. Other issues include missing post-baseline scans that could bias results. Issues with PharmaMar’s data included differences between the study population and the claimed indication.
EMA recently approved Imdylltra and Zepzelca. The JCA reports will inform Member States’ assessments of whether to make the medicines available to their populations.
EMA inks deal with New Zealand’s Medsafe to improve collaboration and information sharing
EMA and the European Commission’s health directorate have signed a working arrangement with New Zealand’s drug and device regulatory agency.
The arrangement is intended to strengthen cooperation between European health authorities and the New Zealand Medicines and Medical Devices Safety Authority (Medsafe). Objectives include enabling the exchange of information on the quality, safety, and efficacy of medicinal products, including evidence from the collection, monitoring, and analysis of adverse reactions.
EMA and Medsafe could cooperate on developing guidance, training activities, and the promotion of mutual learning. Representatives of one agency could participate as observers or experts in procedures run by the other agency, where frameworks permit.
The working arrangement took effect on 6 July. Periodic reviews of the interaction are planned.
