Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news.
EMA Updates Data Quality Control, IT Papers as Introduction of New Process Nears
The European Medicines Agency (EMA) has updated documents on data quality control and electronic filings ahead of the introduction of a new process in November. EMA will start using the new process on 4 November, at which time the regulator will start sending out alerts every time it validates the data submitted in support of an authorized product.
Adoption of the new process continues an initiative that began in July 2014 with the start of an EMA review of the quality and integrity of data submitted to its EudraVigilance system. EMA told the industry of its expectations in June and gave companies until the end of 2014 to improve the quality of their data and update their information. From next month, EMA will send out an alert detailing what, if any, changes it has made whenever it performs data quality control on the filing for a drug.
For marketing authorization holders (MAH) running their own pharmacovigilance systems, the new process will necessitate an update to their operations to ensure they are capable of receiving the new alert. WEB Trader, the system provided by EMA, is already prepared for the new process. EMA has tried to help users through this process — it has been possible to receive test alerts since July — and with the go-live date now nearing the regulator has provided more details of what will change.
The revised data quality control and EudraVigilance documents include further details of the format and naming conventions that will be used by EMA. Officials have also used the updates to the papers as an opportunity to extend the amount of time they have to perform data quality checks. EMA had originally said it would perform quality control checks on each submission made after 1 February within four months. In the latest revision, EMA changed the self-imposed deadline to six months.
EMA Document, More
Breast Cancer Guidelines Updated After Roche, Teva Back Draft Text
The Committee for Medicinal Products for Human Use (CHMP) has added a section on neoadjuvant breast cancer study endpoints to its guidelines on evaluating oncology drugs in human trials. CHMP made the change after its draft endpoint text received the support of Roche, Teva and others.
Officials at EMA drafted the text on the use of pathologic complete response as a surrogate endpoint in breast cancer trials to give drug developers a way to assess efficacy sooner. CHMP felt the improvement in disease-free and overall survival in patients with breast cancer — and subsequent lengthening of clinical trials — made acceptance of such an early endpoint necessary. Pathological complete response is the absence of residual invasive cancer in breast specimens and lymph nodes.
CHMP put its proposal to allow the use of pathological complete response as an endpoint in neoadjuvant breast cancer studies to the industry in a draft paper last year. Roche and Teva were among the organizations to respond favorably to the draft, describing it respectively as “important” and “very useful”. While others, notably the European Federation of Pharmaceutical Industries and Associations, found fault with sections of the text, CHMP has now adopted a tweaked version.
The new version of CHMP’s guideline on the evaluation of anticancer medicinal products in humans, which includes the pathological complete response section, is due to come into force in February 2016. For oncology drug developers, the change creates an opportunity to design trials that show quickly whether a drug is working. Instead of waiting for the disease to progress, sponsors can look for the absence of residual invasive cancer when evaluating haematoxylin and eosin.
EMA to Host Workshop on Extrapolating Efficacy to Discuss Reflection Paper
EMA is holding a workshop next year on the scientific validity of approaches to extrapolating efficacy. The workshop is being timed to coincide with the availability of a reflection paper, giving EMA an opportunity to collect face-to-face feedback before advancing its regulatory strategy.
Officials at EMA have already spent more than three years discussing the topic and still have lots more work to do before guidelines are in place. The overall goal is to eliminate unnecessary studies by applying the conclusions reached in one patient population or disease to another. Such processes are already used to turn data from trials in adults into judgements about the safety and efficacy of drugs in children. EMA thinks there is scope to expand and standardize the use of these processes.
The industry will have a chance to comment on the idea at a two-day workshop in May 2016, registration for which opens on 10 December. EMA is aiming to have a reflection paper ready by the time of the workshop. The paper, which will be based on discussions that took place at a meeting in September, is part of EMA’s attempts to establish whether a structured extrapolation approach is needed to improve interactions with the industry and standardize decisions by its committees.
EMA last put its thoughts on the topic down on paper in 2012, when it released a concept paper for consultation. At that time, EMA was aiming to have a reflection paper ready within 12 months of the finalization of the concept paper. While that timeline has slipped considerably, EMA is now once again stepping up its plans to establish a regulatory framework for the extrapolation of efficacy.
EMA Workshop, Concept Paper
Strong Demand for Orphan Drug Session Forces EMA to Close Registration Early
EMA has closed registration for a workshop on orphan drugs early in response to strong demand. The workshop is being held to discuss how drug developers can show a new orphan medicine offers significant benefits over existing treatments.
There are still more than seven weeks to go until the event takes place, but EMA is now no longer accepting registrations. Interested parties who have missed out on the event will be able to view a live webcast of the workshop. The level of interest in the session is a reflection of the importance of orphan drugs to the development plans of small and large companies, alike, plus their significance to the payers and health-technology assessment (HTAs) bodies that need to weigh their value.
EMA will use the one-day event to discuss how such companies can use comparative efficacy and effectiveness methodologies to show significant benefits over alternative treatments in marketing authorizations. The session will go one step further in the process by discussing how showing significant benefits affects HTA evaluations, pricing decisions and patient access.
The Pharmacovigilance Risk Assessment Committee (PRAC) held an uneventful meeting, at which it neither initiated nor concluded a safety assessment. PRAC is currently evaluating the safety of the class of SGLT2 inhibitors used to treat Type II diabetes, Biogen’s multiple sclerosis drug Tysabri and human papillomavirus (HPV) vaccines. PRAC Notice
The Committee for Medicinal Products for Veterinary Use (CVMP) has recommended the approval of new drugs from Eli Lilly and CEVA Santé Animale. CVMP reached a majority positive opinion on the Eli Lilly drug, which is designed to cut the risk of clinical mastitis in dairy cows. CEVA’s drug, which cuts the risk of intramammary infections, was backed by a consensus opinion. CVMP Notice