EMA in 2014: A Landmark Year for Approvals, With Eyes on the Future

Posted 30 April 2015 By Michael Mezher

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In the European Medicines Agency's (EMA) Annual Report 2014, top regulatory officials describe the agency's role as both a "gatekeeper" and "enabler." This dual role means that the agency must not only work to ensure the safety of patients in the EU, but also to facilitate the development of new and innovative therapies.

By the Numbers

2014 was a landmark year for EMA in many ways. The agency recommended record numbers of products to treat rare diseases, launched its adaptive pathways pilot project and adopted a new clinical data policy.

In total, EMA recommended 102 products—82 human medicines and 20 veterinary medicines—for approval in 2014. Among these products were the first authorized treatments for Duchenne muscular dystrophy, erythropoietic protoporphyria and the first-ever stem cell-based therapy.

The number of products submitted to receive orphan product designation skyrocketed in 2014. In 2013, 201 products were submitted for orphan product designation, up only 2% over 2012. In 2014, the agency received 329 of these submissions, a 63% increase over 2013.

Even more significant is the fact that EMA granted orphan designation to 196 products in 2014, a nearly 550% increase from 2013.

EMA also observed increased interest in its tools intended to assist companies during product development. In 2014, EMA received 551 requests for scientific and protocol advice, up 16% over the previous year. The agency offers its advice, intended to answer scientific questions and clarify regulatory requirements, to companies for a fee. Additionally, 22 of these requests were to qualify novel methodologies, a nearly 50% increase over 2013.

Recent studies by EMA staff and industry groups have found that seeking, and complying with, EMA's scientific advice leads to fewer challenges and a greater likelihood of a positive recommendation.

Looking Forward

Many of EMA's initiatives in 2014 were forward-looking. During 2014, the agency began a sweeping review of the use of antimicrobials to develop a strategy to head off the threat of antimicrobial resistance.

The new EU clinical trial regulations, which mandate the creation of a clinical trails database and submission portal, were also approved in 2014. Once the database is online, sometime after 28 May 2016, the public will have greater access to information on authorized clinical trials within the EU.

Additionally, EMA has increased its collaborative efforts with health technology assessment (HTA) bodies, as the agency sees early engagement and feedback from these entities as an important factor for pharmaceutical companies developing new medicines.

 

EMA Annual Report 2014

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Categories: Drugs, Orphan products, Clinical, Postmarket surveillance, Reimbursement, Regulatory strategy, Submission and registration, News, Europe, EMA

Tags: Orphan product designation, adaptive pathways pilot project, scientific advice, protocol assistance

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