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Posted 21 May 2015 | By Nick Paul Taylor
Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news.
EMA has posted guidance on the eligibility criteria and process for organizations representing patients, consumers and healthcare organizations that want to be involved with the regulator’s activities. The document is a response to last year’s revision of the criteria for involvement.
Organizations coming up for re-assessment, as well as those applying for the first time, will need to follow the new guidance document and meet the revised criteria it explains. The document is a step-by-step overview of the assessment process, from the initial task of checking if the applicant is a nonprofit through to the analysis of the organization’s finances.
A large portion of the document is taken up with an overview of what organizations need to do to meet EMA’s transparency requirements. Organizations need to open up their finances to EMA, notably by listing all funding sources, the proportion that comes from industry and the percentage accounted for by the largest donor. Sharing of a full financial statement is strongly recommended.
EMA has added Bluebird Bio and Pluristem Therapeutics to its adaptive pathways pilot project. Bluebird and Pluristem are the first companies to reveal they are participating in the pilot, which EMA set up to assess shifting some of the burden of data gathering from pre- to post-approval.
Companies following the adaptive pathway have two options. The first model is to file for approval in a subpopulation and then upgrade to a broader indication once additional clinical trial and real-world data is available. Alternatively, firms can file for a conditional approval, the receipt of which is tied to postmarket data collection requirements. Both approaches offers drugmakers an abbreviated route to market and allow patients with major unmet needs to receive new therapies.
EMA began seeking participants in the pilot project in March 2014 and trimmed the initial pool of 34 applicants down to the six most suitable candidates late last year. The drugs in development at the first two companies to go public about their participation are indicative of the types of therapies EMA sees benefiting from the program. Bluebird is developing a gene therapy for beta-thalassemia major, while Pluristem is working on placenta-based cell therapies.
Such treatments pose new challenges for regulators, a fact demonstrated by the drawn-out approval process for uniQure’s gene therapy Glybera. Bluebird is hoping its gene therapy will follow a more direct route to market. The company is aiming to win a conditional approval on the basis of interim data from two clinical trials, after which it will seek a conversion to full approval using final results from the two studies, long-term follow-up data and real-world outcomes.
Pluristem Release, Bluebird Release
The European Commission has published an operational manual to inform inspections of tissue and cell procurement operations. The 86-page document is intended to guide countries that are establishing their first inspection processes, while also encouraging standardization among member states with more advanced regulatory programs.
Under European Union law, member states have obligations regarding the safety and quality of human tissues and cells destined for transplantation or use in assisted conception. Some of the laws date back more than 10 years but the legislation — and the commission’s guidance on how to comply with it — is continuing to evolve. The operational manual published this week is the first version to be drafted since April’s Commission Directive on imported cell and tissue quality.
The passing of the directive has necessitated a reworking of an annex covering the technical requirements for import and exports. In the new, expanded annex, the commission gives national regulators a checklist of topics that should be covered in inspections, from ascertaining the types of tissues and cells being imported to confirmation that there is a written agreement between the supplier and buyer.
Swissmedic has started searching for new software for its adverse event database after deciding its current platform lacks some important features. The regulator has asked software suppliers to send details of potentially suitable platforms.
Uppsala Monitoring Centre (UMC) in Sweden is currently hosting and running the system using its VigiFlow platform. However, with Swissmedic concluding that the current version of VigiFlow falls short of its expectations of a modern pharmacovigilance system, the regulator needs to find an alternative. The task for Swissmedic now is to assess whether an off-the-shelf product will meet its needs or if it needs to commission custom software.
Swissmedic is seeking feedback from software developers before making a decision, which it will base upon the affordability and effectiveness of the various options. The regulator has more than two years to transfer to a new system, having set a deadline for the end of 2017. Respondents to the request for information on potential pharmacovigilance software systems have until May 26 to submit their feedback.
The European Medicines Agency (EMA) has published draft guidelines on the quality, nonclinical and clinical data developers of gene therapies should include in regulatory filings. EMA has placed particular emphasis on the need to reach high quality standards before starting nonclinical and clinical tests, something multiple applications to date have failed to achieve.
Guidelines on quality take up more than one third of the 42-page document and almost as many pages as the nonclinical and clinical sections combined. Members of the Committee for Advanced Therapies (CAT) divided up the document in this way after reviewing the issues they have seen in gene therapy submissions to date. The submission model proposed by CAT follows the common technical document headings, but the nature of gene therapies means the content is unusual.
EMA is requesting full information on the vector system, including explanations to show the gene therapy developer has considered the risks posed by their chosen approach. If the applicant is using an integrating vector, they must consider the risk of insertional mutagenesis. Applicants also need to provide a full breakdown of the history and biological characteristics of the vector’s parent virus or bacterium.
The guidelines also provide extensive advice on process controls, starting materials, impurities and other factors of relevance to the manufacture and quality control of gene therapies. EMA wants companies to be confident these issues will not affect the validity of nonclinical and clinical studies before they start running tests. The guidelines go on to explain the general principles that should inform nonclinical and clinical research, as well as detail the specific tests developers must run.
EMA is accepting comments on the draft until August 31. When the text is finalized and comes into force, it will replace a document EMA drafted between 1999 and 2001.
Draft Guidelines, Press Release
Abbott, Arterial Remodeling Technologies (ART), Implanet and Mauna Kea Technologies each received a CE Mark. Abbott received the status for the latest iteration of its Absorb stent system, while ART was recognized for its bioresorbable scaffold. Implanet and Mauna Kea won clearance to sell different diameters of orthopedic implants and a laser endomicroscopy platform, respectively, Abbott, ART, Implanet, Mauna Kea
Tags: EU Regulatory Roundup, Regulatory Roundup, European Regulatory Roundup