Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news.
EMA Seeks Feedback on the Use of Disease Registries for Regulatory Purposes
The European Medicines Agency (EMA) is seeking feedback on the use of patient disease registries for regulatory purposes. EMA dedicates most of the draft discussion paper to an overview of good registry practices and registry studies.
Data housed in registries can be useful in the regulatory evaluation of medicines, for example by providing insights into the safety and efficacy of drugs in the real world. In the past, EMA has asked companies to create registries covering specific products and made use of the data for post-approval purposes. However, EMA is yet to make extensive use of the registries public organizations create to track patients with a particular disease.
The draft discussion paper is part of EMA’s effort to make better use of the wealth of clinical data in patient disease registries. EMA drafted the paper in light of the findings of an effort to identify barriers to regulatory use of the data and a series of workshops focused on diseases including cystic fibrosis and multiple sclerosis. EMA undertook these activities after starting to focus on the topic in 2015.
EMA has emerged from the workshops with a set of good registry practices. The good practices discuss all the considerations that go into designing a high-quality registry, from the enrollment of patients through to measures to improve data quality and governance principles.
The discussion paper also sketches out the roles pharmaceutical companies and regulators can play in disease registries. EMA thinks pharmaceutical companies and regulators have complementary roles in the context of disease registries as both groups share the goal of using the resources in the evaluation of medicines.
Even so, EMA lists distinct activities related to each group. EMA envisions pharmaceutical companies using disease registries to understand the regulatory data requirements that are likely to arise when they file for approval of a drug, and to guide the planning of registry-based post-approval studies. To support these and other activities, EMA thinks companies should talk to the coordinators of disease registries and, if needed, provide funding or assistance to increase data quality control.
The paper also presents information about registry-based studies, including an overview of how they differ from registries. While registries are data collection systems, registry studies are investigations set up to answer research questions. These investigations use data collected in registries.
In the section on registry studies, EMA explains the regulatory context for such research, discusses study protocols and populations and provides advice on how to collect and analyze data.
EMA is accepting feedback on the discussion paper until the end of June. The agency will consider the responses as it works to finalize the text in the fourth quarter of 2019.
EMA Adopts Guidelines on Biological Quality Documents, Therapeutic Equivalence
EMA has adopted guidelines on the quality of experimental biologicals and how to show the therapeutic equivalence of locally acting products in the gastrointestinal tract.
The Biologics Working Party began work on the biologicals draft in 2016 to facilitate implementation of the European Union Clinical Trials Regulation. Two years later, the guideline is finalized and in force. EMA is advising people to read the guideline in connection with the regulations on clinical trials.
In the guideline, EMA sets out what information sponsors need to provide to demonstrate the quality of proteins and polypeptides used in clinical trials. The requested information covers points such as a description of the manufacturing process, an overview of process development and details of efforts to characterize the biological substance.
The second guideline covers the requirements companies need to fulfill before being freed from the need to run clinical trials to demonstrate the therapeutic equivalence of locally applied, locally acting gastrointestinal products. EMA changed its stance on the requirements in response to an increase in its understanding of the sensitivity of in vitro
and in vivo
methods. The new understanding led EMA to conclude such alternative methods may be more sensitive than traditional clinical endpoints.
EMA sought feedback on its new position last year. The European Federation of Pharmaceutical Industries and Associations (EFPIA) was among the organizations to provide feedback, but the trade group found EMA unwilling to accept some of its more significant proposals. EFPIA asked EMA to consider allowing the use of in silico
models. EMA rejected the idea, stating that, “These tools are recognized as valuable tools for the sponsors’ decisions, but not yet for regulatory decisions.”
The therapeutic equivalence guideline is due to come into force in May.
, Equivalence Guideline
, Industry Feedback
EMA Cancels November Meeting About Brexit, Delaying Next Industry Talk Until 2019
EMA has canceled its November industry stakeholder meeting on Brexit. The agency was tentatively scheduled to meet with the industry to discuss the current Brexit situation next week, but canceled the event and thereby pushed back the next gathering until January.
Earlier this year, EMA scheduled, in some cases on a tentative basis, a series of meetings with industry stakeholders to discuss Brexit. The last event of the year was pencilled in for 22 November, but EMA has decided not to go ahead with the meeting. The decision means there will be no meetings between the gathering in September and an event tentatively scheduled for January.
EMA should have a clearer idea of the near-term regulatory consequences of Brexit by January. As it stands, the UK and EU have reached an agreement that would maintain something approaching the status quo from a regulatory standpoint through to the end of 2020.
However, there are doubts about whether the British Prime Minister has enough support for the deal in Parliament. It is unclear what will happen if parliament votes against the deal. The sort of no-deal Brexit feared by the pharma and medical device industries is one possibility.
, EMA Statement
Notified Body BSI Gets Dutch Designation to Offer Device Firms Brexit Escape Route
BSI has received full designation as a medical device notified body from Dutch and EU authorities. The designation clears BSI’s office in Amsterdam to support medical device companies that want to move their CE mark certificates out of the UK.
Following the UK referendum on EU membership, BSI, a large notified body, identified its existing base in Amsterdam as a platform for helping to adapt to Brexit and the incoming rules on medical devices. Around 18 months after disclosing the plan, BSI has completed the application process and got full designation for its Dutch office.
The designation means companies can follow a “straightforward ... primarily administrative” process to migrate existing CE mark certificates between the UK and the Netherlands. Companies that take up this option will free themselves from some uncertainty about the impact of Brexit.
BSI will share information freely between the UK and Dutch notified bodies to maintain the full traceability and linkages of the certificates. Companies will need to change the notified body number on the labels of their products. BSI said companies can make these changes during normal product lifecycle plans, in the move to the new device regulations or when they introduce new products.
While some companies may welcome the certainty of working with an ex-UK notified body, BSI plans to grow both its British and Dutch businesses.