Euro Roundup: EMA seeks feedback on framework designed to drive uptake of data standards
The European Medicines Agency (EMA) is holding a consultation about a draft framework intended to cut the time it takes to implement data standards.
Working with the Heads of Medicines Agencies (HMA), EMA has developed the draft framework for use across the European medicines regulatory network (EMRN). Last year, EMRN released a strategy for maximizing the value of the data it manages. The strategy included the goal that data should be good quality, well documented, standardized, and accessible to maximize its value.
Supporting that objective, EMA and HMA have developed a framework aligned with the Network Data Steering Group’s (NDSG) mandate to use standards to drive data interoperability and exchange. Standardization entails putting data in a common format to simplify exchange, analysis, and the use of AI.
The draft framework covers the adoption, creation, updating, and implementation of data standards for use by EMRN. As well as enabling the network to manage and govern the process for regulatory data standardization use cases, the framework will allow external stakeholders to highlight their use cases for consideration by EMRN.
“A new streamlined process will be achieved by defining specific roles, responsibilities, and objectives for individuals and governance groups involved in the standardization activities,” EMA and HMA said. “These together should provide clear governance, oversight, and transparency, and resolve the previous siloed ad hoc approach to standardization.”
The old approach led to long implementation timeframes, as well as to a disconnect between standards projects, IT systems development, and business process change management, EMA and HMA said. The framework should tackle those issues by enabling people with limited knowledge of data standardization to understand the process for creating, adopting, and implementing standards.
EMA and HMA have established seven principles for the framework. The principles include supporting open data standards, avoiding overlapping or competing standards, and using terminologies already in the EMRN.
If successful, the framework will lead to the implementation of more standards over “a much shorter timeframe.” EMA and HMA will assess the effectiveness of the framework, providing NDSG with insights to improve the document.
The draft framework is open for consultation until 18 September.
MHRA posts position paper on regulating microbiome-based medicinal products
The Medicines and Healthcare products Regulatory Agency (MHRA) has clarified the pathway for microbiome-based medicinal products (MBMPs) to encourage the development and licensing of the medicines in the UK.
MHRA defines MBMPs as products that prevent, treat, or diagnose disease through the modulation, restoration, replacement, or functional activity of the human microbiome. MBMPs achieve those effects using live microorganisms, a defined consortium of microorganisms, a complex microbial ecosystem, or non-viable microorganisms or their derived components.
The agency encourages companies to submit marketing authorization applications for MBMPs, which it may regulate as biological medicinal products or advanced therapy medicinal products. As of July, MHRA was yet to authorize an MBMP. Two donor-derived microbiota products have been licensed in the US.
“MHRA considers MBMPs acceptable in principle and capable of meeting UK regulatory standards for marketing authorization,” the agency said. “Progress toward licensure will depend on resolving challenges which may include product characterization, [chemistry, manufacturing, and controls] standardization, safety (including antimicrobial resistance), and clinical evidence generation.”
The agency covered the challenges in the position paper. Inherent variability, which complicates characterization, is a central challenge for MBMP development, MHRA said. Thorough characterization of the MBMP is essential, the agency explained, as these may vary from fixed product compositions to tailored microbial consortia. The composition may change over time, forcing updates to characterization.
Safety assessments must pay particular attention to the potential for contamination with pathological microorganisms or toxins. Vulnerable populations may be at risk of infection, and MBMP developers should assess the likelihood of horizontal gene transfer, especially the potential transfer of antimicrobial resistance. MHRA accepts that MBMPs require risk-based, product-specific safety strategies.
The UK has yet to publish overarching guidance on MBMPs, leaving the class of drugs reliant on broader legislation and guidance, European and international documents, and product-specific MHRA advice. Aspects of the rules are in flux, with the UK currently reviewing the European Union Regulation on Substances of Human Origin (SoHO). SoHO may apply in Northern Ireland but not Great Britain.
MHRA is encouraging MBMP developers to engage early with its staff. The recommendation reflects the scientific novelty, regulatory complexity, and potential international divergence in expectations faced by MBMP developers.
MHRA releases guidance on 14-day assessment timeline for Phase 1 clinical trials
MHRA has published guidance on a stepwise pilot program designed to establish a 14-day assessment timeline for Phase 1 healthy volunteer clinical trial applications in the UK.
The agency launched the pilot at the end of March but, having initially constrained the scale and speed of the program, only published guidance this week. In the first three months of the pilot, MHRA carried out 28-day reviews, compared to its standard 30-day assessments, and only offered one slot per week. The agency cut its deadline to 21 days and increased its workload to two slots per week on 29 June.
MHRA’s guidance sets out the next steps for the program. The agency will cut its deadline to 18 days on 28 September and reach its 14-day goal on 28 December. Throughput will scale up as the timeline shortens, with MHRA offering three slots from 28 September and four slots from 28 December.
The pilot operates on a first-come, first-served basis that requires sponsors to book slots to secure an accelerated review. If the pilot slots are full, MHRA will review applications under its standard 30-day process. MHRA’s guidance outlines the process for booking a slot in the pilot.
EMA staffers write paper on regulatory research priorities for AI in the drug lifecycle
There is strong convergence on the importance of AI accuracy, reliability, data governance, and ethical considerations, EMA employees wrote in a paper published in Clinical Pharmacology & Therapeutics.
The paper features data from a survey of 273 regulators, pharmaceutical industry professionals, patients and consumers, academics, and healthcare professionals. Ranking the challenges identified in the survey, EMA outlined priorities, most of which related to accuracy, governance, ethics, fairness, and bias. The list could inform research and funding priorities to address knowledge gaps.
While the survey revealed broad consensus on some priorities, the researchers reported “heterogeneous expectations regarding regulation and oversight beyond existing legal frameworks.” Regulation and oversight ranked fourth in the overall prioritization of domains, but there was no clear top-ranked query within the topic.
Other News:
EMA has posted preliminary findings from a pilot program that allows drugmakers to send its approvals of post-authorization changes to national regulatory authorities (NRAs) outside the EU. By the end of last year, 77 NRAs had participated in the pilot and 75% of them had reported saving resources. Four-fifths of post-authorization changes were approved in half the usual time. Pilot Findings
EMA has updated multiple question and answer documents to add information on IRIS, the platform that the agency uses to exchange regulatory and scientific information with companies. The agency updated documents on pre-authorization procedural advice, notified bodies, companion diagnostics, and medicinal products intended exclusively for markets outside the EU.
