Regulatory Focus™ > News Articles > 2021 > 10 > Euro Roundup: EMA publishes guideline on running registry-based studies

Euro Roundup: EMA publishes guideline on running registry-based studies

Posted 28 October 2021 | By Nick Paul Taylor 

Euro Roundup: EMA publishes guideline on running registry-based studies

The European Medicines Agency (EMA) has released recommendations on key methodological aspects specific to the running of registry-based studies by marketing authorization holders and applicants.
 
EMA, working through its Patient Registry Initiative and the Cross-Committee Task Force on Registries, has explored ways to improve the use of patient registries to support regulatory decision-making. The project led to a series of workshops that generated recommendations and later to a discussion paper. Now, EMA has collated the information, and recommendations from other groups in the European Union and overseas, into a guideline on registry-based studies.
 
The guideline draws a distinction between registries and registry-based studies, explaining that in the latter a specific research question is probed “using the data collection infrastructure or patient population of one or more patient registries.” The duration of follow-up, patient enrollment and data collection are all dictated by the research question.
 
Elsewhere in the guideline, EMA explains how marketing authorization holders and applicants can use the findings of registry-based studies. The acceptability of evidence from the studies depends on the product, the registry and the study, leading EMA to advise drug developers to consult with it or a national competent authority early.
 
EMA identifies a range of potential uses of the findings of registry-based studies. Pre-authorization, the agency expects preclinical studies and clinical trials to form the core of the evidence on a drug. Yet, there can be a role for registry-based studies, for example in providing evidence of how a disease is treated in the real world or in contextualizing the results of uncontrolled clinical trials. When using the evidence pre-approval, EMA recommends discussing the acceptability of the proposed approach with regulators, health technology assessment bodies and payers.
 
A different set of use cases applies once a product is on the market. “Patient registries can be the basis for recruitment and randomization for RCTs and non-interventional studies, post-authorization efficacy studies and post-authorization safety studies performed after marketing authorization. They may allow linkage of patient records with other data sources such as biobank data, census data, or demographic data,” the guideline states.
 
The next section of the 35-page guideline describes how to plan a registry-based study, design the protocol, select the patient population, and gather, analyze and report the data. Those topics take up around the first half of the document. The rest of the guideline is given over to legal matters, considerations on patient registries and appendices such as a checklist for evaluating the suitability of registries for registry-based studies.
 
EMA Guideline
 
EU Parliament votes for more transparent COVID-19 vaccine policies  
 
The European Parliament has called for legislation to make COVID-19 vaccine research, purchasing and distribution more transparent to help counter vaccine hesitancy and disinformation.
 
Many of the requests relate to current and future contracts between the Commission and COVID-19 vaccine manufacturers. The resolution, which was adopted by 458 votes to 149, with 86 abstentions, calls on the Commission to publish non-redacted versions of purchase agreements without delay. The parliamentarians want the disclosures to cover the amount of public investment that has gone into the development of the vaccines, pricing and the number of doses sent to each country.
 
Other requests relate to clinical trial data. The politicians want the Commission to “ensure that the contracted companies make full clinical trial results and protocols of potential COVID-19 vaccine research available for evaluation, as soon as possible, by independent scientific experts, beyond experts at the EMA.” The request covers statistical analysis plans, clinical studies and reports.
 
Dolors Montserrat, chair of the petitions committee, framed the requests as part of a push to address vaccine hesitancy. “Petitions show that some citizens are reluctant about vaccinations. This is why we need to be transparent about how COVID-19 vaccines are developed, purchased and distributed. For the strategy to be successful, the public must be provided with more information,” Montserrat said.
 
Press Release, Adopted Resolution
 
EMA answers questions about sending data to EudraVigilance dictionary
 
EMA has answered tens of frequently asked questions about the electronic submission of investigational medicinal product data to the Extended EudraVigilance medicinal product dictionary (XEVMPD).
 
The document spans fundamental questions, such as the legal obligation to submit the data, to more specific queries. The more specific questions include: “In our clinical trial, we are using a medicinal product which is already authorized. It is however studied for a new indication not listed in the SmPC. How should we submit such product to the XEVMPD?”
 
EMA has divided the start of the document up into questions about the submission of investigational medicinal products, development medicinal products and sponsor information. The rest of the text covers topics including pharmaceutical product information, printed product information and the Anatomical Therapeutic Chemical code.
 
One question addresses whether sponsors need to submit information on placebos to XEVMPD. EMA said only information on the active ingredients of the investigational medicinal product needs to be submitted, quoting other documents to make its case.
 
EMA FAQ
 
MHRA calls for sponsors to use combined review pathway ahead of January switch
 
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has asked sponsors to start using its combined review pathway for clinical trial applications. Combined review will become the way all filings are processed from January but MHRA wants sponsors to use the pathway before then.
 
In an update to its guidance on the authorization of clinical trials, MHRA said its combined review service, formerly known as Combined Ways of Working, will become how all new Clinical Trials of Investigational Medicinal Products (CTIMPs) applications are prepared, submitted and reviewed from 1 January. Combined review allows sponsors to make one filing and receive a single UK decision.
 
While the mandatory use of the pathway is still two months away, MHRA is asking sponsors that “have any new CTIMP applications planned over the coming weeks or months” to file for combined review.
 
Combined review applications “should be started and submitted using the new part of Integrated Research Application System (IRAS) and not in the standard part of IRAS,” MHRA said. The regulatory requirements and fees are the same as for the old non-combined review. However, MHRA is advising applicants to refer to the Health Research Authority website, rather than its guidance, for information about the application submission, processing and assessment steps.    
 
MHRA Guidance
 
Commission conducts unannounced antitrust inspections at animal health company
 
The European Commission is conducting unannounced inspections of “a pharmaceutical company active in animal health in Belgium.”
 
Officials launched the inspections because of “concerns that the inspected company may have infringed the EU antitrust rules that prohibit the abuse of a dominant position.” The Commission is working with the Belgian competition authority on the inspections, which are a preliminary investigatory step into suspected anti-competitive practices.
 
At this stage, little else is known for sure. The company has not been found guilty of anti-competitive behavior, and the duration of the investigation will depend on a range of factors, such as the complexity of the case and the extent to which the business cooperates with the Commission.
 
Press Release
 
Other News:
 
EMA’s human medicines committee has ruled a booster dose of Moderna’s COVID-19 vaccine Spikevax may be considered in people aged 18 years and up. EMA Notice

 

© 2021 Regulatory Affairs Professionals Society.

Regulatory Focus newsletters

All the biggest regulatory news and happenings.

Subscribe