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Regulatory News | 10 May 2017 | By Zachary Brennan
With the European Medicines Agency’s (EMA) departure from London and loss of UK expertise (about 60 of the 897 EMA staffers are from the UK) looking more likely, the agency on Wednesday released a report outlining its successes from 2016, when 27 new medicines were authorized and several new programs took shape.
Like in the US, EMA is increasingly approving new drugs more quickly for those with unmet needs. In 2016, more than one in three new medicines was recommended for approval using at least one of EMA's early access tools.
Also, similarly to the US, the EU is seeing a booming number of applications for orphan medicines. Orphan designations from the EMA reached a peak in 2016, with 329 compared to 258 in 2015.
EMA is also working with its US and Japanese regulatory partners on orphan medicines, and almost a third of applications for orphan designation were submitted to EMA and to another regulatory authority in parallel in 2016.
Christa Wirthumer-Hoche, chair of EMA’s management board, said: "In 2016, a lot of work was also done behind the scenes to prepare for the implementation of the new European Clinical Trial Regulation which opens up a new era for carrying out clinical trials in the EU. The Board has closely monitored the ongoing development of the EU’s clinical trial portal and database, a system that will provide a single platform for the submission and maintenance of clinical trial applications and authorisations, and supports their coordinated assessment and supervision."
In 2016, EMA received 84 PRIME applications – one in two from small and medium-sized companies. Of those, 15 medicines were granted a designation, including six for oncology treatments (out of 17 requested). And 10 of these medicines granted the designation are also considered by EMA to be advanced therapies.
Guido Rasi, executive director of EMA, added: "The key initiative in 2016 was the launch of PRIME (PRIority MEdicines), a new scheme through which we give early, proactive and enhanced support to those developing medicines that target an unmet medical need. In order to get breakthroughs in medicines to patients more quickly, PRIME aims to foster better planning of medicine development. This will help companies to generate the high-quality data we need to assess the quality, safety and efficacy of medicines."
Advanced therapies were another highlight for EMA, as two were approved in 2016: GlaxoSmithKline’s Strimvelis, a gene therapy manufactured from a patient’s own immature bone marrow cells that improves their ability to fight infection, and which the company says has already been used twice; and Italy-based Molmed’s Zalmoxis, a cell-based therapy which contains genetically modified T cells for patients receiving a haploidentical haematopoietic stem cell transplant.
In a first for any regulator worldwide, EMA launched a new clinical data website in 2016, which since its inception has seen 1,820 users, including 350 academics, 6,474 documents viewed, 23,443 documents downloaded and 330 downloads per day.
"Access to comprehensive data will lead to a dramatic increase in accessible knowledge about individual medicines. The new database will allow researchers and academics to use clinical study reports to ask new questions about a medicine, pursue new lines of enquiry and research, and also provide regulators with a more robust evidence base. Our initiative will ultimately benefit the practice of medicines as a whole and will help us to achieve our aim of making available the best-possible medicines to address the medical needs of EU patients," Rasi added.
The number of requests for access to EMA documents continued to rise in 2016, with double the requests granted as in 2014 and a steady decline in requests refused.
EMA currently has a maximum of 210 days to carry out an assessment of a marketing authorization (which compares with FDA's standard review of 10 months and priority review of six months).
During the EMA assessment, concerns with an application may be identified, requiring further information or clarification from the company, which requires the clock to be stopped to give the company time to reply. EMA’s Committee for Medicinal Products for Human Use (CHMP) opinion is then transmitted to the European Commission which decides on granting the marketing authorization within 67 days after receipt of the CHMP opinion.
The actual overall time required for the assessment of initial marketing authorization applications in 2016 remained stable in 2016 when compared to other recent years. The average number of days for a medicine authorized via EMA’s centralized procedure in 2016 were: 199 days for the CHMP assessment phase, 11 days for EMA post-opinion phase, 156 days for company clock-stops and 52 days for the final EC decision process.
The number of notifications of withdrawn products for safety reasons also saw a slight decline from 132 in 2015 to 160 in 2015 to 118 in 2016.
But the number of good manufacturing practice (GMP) inspection requests increased by 18.5% in 2016, which EMA linked to the growing number of centrally authorized products. Meanwhile, EMA in 2016 conducted almost twice as many GMP inspections as in 2012. The agency also received 181 suspected quality defects which led to a total of 16 recalls, or one more than in 2015 and two more than 2014.
In September 2016, EMA, Japan's Pharmaceuticals and Medical Devices Agency (PMDA) and the US Food and Drug Administration (FDA) discussed regulatory approaches for the evaluation of new antibacterial agents.
Conclusions from this meeting were presented at the G7 Health Ministers’ meeting in Japan that same month, with the three agencies agreeing "that some flexibility should be applied to the requirements for clinical development programmes for antibacterial agents, in particular where treatment options for patients are limited due to antimicrobial resistance."
The group also reiterated that it may be appropriate to accept abbreviated development programs for new antibiotics addressing an unmet need related to antimicrobial resistance, and they agreed that alignment of data requirements by regulators worldwide can contribute to stimulating new antibiotics development.
Later, at the end of 2016, EMA and FDA discussed the establishment of a joint working group to consider in more detail clinical development and data requirement aspects in the context of concrete applications for new antibiotics.
In addition, thanks to a data-sharing initiative launched by EMA with regulators from Canada, Japan and the US, EMA is revising a guideline on Alzheimer’s disease medicines and the treatment of other types of dementia.
Also in 2016, EMA started offering parallel scientific advice with health technology assessment (HTA) bodies on a routine basis as part of scientific advice activities.
The offering follows a five-year pilot project where drug developers received simultaneous feedback on their development plans from both EMA and HTA bodies.
Sixty-three parallel scientific advice procedures were included in the pilot and a report showed that such procedures achieved a high level of alignment between the data requirements of both the regulators and HTAs.
"Parallel scientific advice is one of the Agency’s key initiatives to improve patient access to important new medicines because it ensures that medicine development programmes generate appropriate data for regulators and HTA bodies and allow for the assessment of both benefit-risk balance and added value. This can reduce delays between a medicine’s marketing authorisation for the European market and decisions on reimbursement that are taken at the national level," EMA noted.
EMA press release
Tags: PRIME, Brexit, EMA in 2016