The recommendations on eligibility to the PRIME scheme adopted at the CHMP meeting of 24-27 February 2020 has been posted on the EMA website, along with the cumulative overview of recommendations on PRIME eligibility requests adopted since the scheme began in 2016. In keeping with the usual proportion of grants and denials, of 5 applications for the month, 1 was granted whilst 4 were denied. The one granted is a gene therapy for “treatment of X-linked Retinitis Pigmentosa owing to defects in Retinitis Pigmentosa GTPase Regulator” supported by clinical exploratory data. The four that were denied are chemical substances, all of which also included clinical exploratory data, in the therapeutic areas of cardiovascular diseases, gynaecology, endocrinology, and neurology. For further details, please click: https://lnkd.in/ePz256f
ICH Q12 covers technical and regulatory c onsiderations for pharmaceutical product lifecycle management. It has been in the works for quite a while and is intended to globally harmonise the management of post-approval changes to chemistry, manufacturing and controls (CMC). However, there are some conceptual differences between ICH Q12 and the current EU legal framework, meaning that there are limitations on how fully ICH Q12 can currently be implemented within the EU.
Incompatible sections are those on scientific risk-based approaches to defining established conditions and associated reporting categories (described in Chapter 3.2.3 of the new guideline) and on the product lifecycle management document (PLCM, described in Chapter 5). Within the EU, regardless of the current text within ICH Q12, the definition of established conditions and their reporting categories must follow the requirements laid down in the current EU Variations Regulation and associated EU guidelines. The PLCM cannot currently be recognized if submitted. https://lnkd.in/g2vefsg
EMA has just published the adopted Qualification Opinion on the Multiple Sclerosis clinical outcome assessment (MSCOA) and an overview of comments received. The intent is for this COA instrument to serve as a primary, co-primary, or secondary endpoint to assess efficacy in clinical trials at various stages of drug development, including proof of concept, dose-ranging, confirmatory and registration trials. There are four specified performance outcome measures assessing important dimensions of multiple sclerosis (MS), which are considered as a battery of tests, some or all of which could be used as a dysconjugate composite endpoint by sponsors in a clinical trial. Full details are available here https://lnkd.in/d_WeSAN
An important incentive offered by the legislation is the possibility for sponsors of orphan medicinal products to receive reductions in the regulatory fees payable to the Agency. A special contribution is allocated annually to the Agency by the European Union (EU) for fee reductions for orphan medicinal products. Since the year 2000, over 2,233 orphan designations have been issued by the European Commission, of which so far 169 have resulted in authorised medicinal products. This link https://lnkd.in/ddDfHfV presents a nice table detailing the EMA policy on the level of fee reductions reflects the priority given to ‘protocol assistance’ and the support to small and medium-sized enterprises (SMEs) and other information on the scheme. EMA has issued this updated presentation on the statistics for Orphans https://lnkd.in/gt-xChE
The purpose of the re-organisation has been stated as to ensure that the Agency operates as efficiently as possible, taking into account the rapidly evolving landscape for pharmaceutical research and development, and driven by the need to recalibrate to a lower head count following the relocation of the Agency to Amsterdam in 2019. Several org charts have been issued. A new EMA org chart https://lnkd.in/ejYBSyJ, where operations in the area of human medicines have been integrated into one Human Medicines Division, which will be led by Alexis Nolte. In addition, four mission-critical task forces have been established to support the human and veterinary medicines divisions. Here is a link to the new task forces org chart https://lnkd.in/esvDkNe. Updates to the existing org charts for Stakeholders & Communication Division, Information Management and Advisory functions have also been issued and can be found on the site.