Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news authored by RAPS staff and members of RAPS' European Advisory Committee.

Know of news we might have missed? Let us known by emailing us at news@raps.org

GVK Calls EMA Suspension Recommendation ‘Highly Disproportional’

GVK Biosciences has called the European Medicines Agency’s (EMA) proposal to suspend 700 drugs “unprecedented and highly disproportional.” While describing itself as “respectful” of EMA’s decision, GVK BIO pointed out perceived flaws in the regulator’s reasoning.

The Indian contract research organization (CRO) questioned the wisdom of extrapolating the issues French regulators found with nine trials to apply to all studies conducted at its Hyderabad clinical site. GVK BIO also noted French regulators downplayed the importance of the alleged electrocardiogram data manipulation to the demonstration of bioequivalence.

Nonetheless, EMA felt the concerns raised by the French inspectors were serious enough to deem data from GVK BIO as insufficient to support continued use of a product. Of the 1,000 products EMA reviewed, only the 300 that have enough supporting data from other sources are unaffected by the suspension recommendation.

GVK Statement I Wall Street Journal I Regulatory Focus

NICE Recommends Soliris in First Test of Ultra-Orphan Evaluations

The United Kingdom’s National Institute for Health and Care Excellence (NICE) has published the first guidance document to pass through its highly specialised technologies (HST) program. NICE introduced the program to improve its assessment of treatments for ultra-rare diseases.

In the first guidance document produced by the program, NICE recommended funding Alexion’s Soliris in certain conditions. Soliris treats atypical hemolytic uremic syndrome (aHUS), a disease with a global prevalence of fewer than six people per million. NICE called Soliris a “step change” that delivers “[quality-adjusted life year gains] of a magnitude that is rarely seen for any new drug treatment.”

However, the cost of the drug caused NICE to add caveats to its recommendation. The official forecast of the budget impact is confidential, but an illustrative estimate released by NICE put the cost over the next five years at £346 million ($524 million). NICE has asked for research into the dosing and duration of treatment with Soliris to see if the cost can be lowered.

The recommendation nonetheless offers some encouragement to developers of drugs for ultra-rare diseases. Soliris was recommended by NICE’s ultra-rare disease drug predecessor the Advisory Group for National Specialised Services (AGNSS) in 2012, but the government wanted more evidence of affordability. AGNSS was scrapped in 2013 and its responsibilities passed to NICE’s HST team.

Press Release I NICE Guidance

EU Court Upholds Exclusivity of Similar Orphan Drugs

The European Union General Court has ruled the 10-year period of market exclusivity for orphan drugs applies to the second product to win approval in a particular indication. Teva had petitioned the court to overturn EMA’s decision to refuse to validate an application for its generic copy of Glivec.

Lexology broke down the details of the case. Novartis won approval and orphan drug status for Glivec as a treatment for chronic myeloid leukaemia (CML) in 2001, giving it a 10-year window in which no similar therapies could come to market in that indication. In 2006, Novartis waived its right to exclusivity so it could bring Tasigna to market.

Teva applied for approval of a generic version of Glivec in 2012, more than 10 years after the drug was given a window of exclusivity. However, as Tasigna was granted its own period of exclusivity, EMA ruled the orphan drug legislation barred the introduction of a competitive product. Teva took the case to the EU Central Court, which dismissed the action. The firm can appeal to the Court of Justice.  

Lexology

EMA Adds Safety Report Repository as Pharmacovigilance Rollout Continues

EMA has introduced another piece of its pharmacovigilance strategy: an electronic repository for periodic safety update reports (PSURs). The resource will allow member states to view all PSURs on an active ingredient in one place, potentially making it easier to perform ongoing assessments of the risks and benefits of products.

Marketing authorization holders must submit PSURs every six months for the first two years a drug is on sale and once a year for the following two years. The reports, the assessments of them and any comments or final outcomes will now be housed in a single repository that is accessible by the European Commission, EMA, agency committees and certain people in member states.

The repository should simplify the reporting process for drug companies, too. EMA said the system eliminates the need to use manual file naming conventions and enables the attachment of an XML delivery file to submissions. The regulator is hosting separate educational webinars for new and existing eSubmission Gateway users, as well as national competent authorities, next month.

EMA Statement 

French Regulator Begins Building Medical Device QC Committee

France’s Agence Nationale de Sécurité du Médicament et des Produits de Santé (ANSM) has put out a call for people to join its medical device quality control committee. The regulator is seeking people with expertise in ionizing radiation and other fields relevant to medical devices.

ANSM is trying to build a panel with a broad range of expertise. France’s public and independent nuclear safety bodies will each send a representative. ANSM has also given three seats on the panel to a medical technology trade group and another three to a consortium of independent third-party inspection bodies.

The regulator is particularly interested in experts in dosimetry, biomedical engineering, radiology and radiotherapy. Experts appointed to the committee will serve three-year, renewable terms and be expected to attend up to four meetings a year.

ANSM Request

Other News:

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has moved Nexium Control 20mg onto the general sale list (GSL). Drugs on the GSL can be sold by someone with no pharmacy training to a person without a prescription. MHRA made the decision despite four of the 10 responses to its public consultation opposing the change. MHRA Statement

EMA has recommended seven new drugs for approval. The batch of recommendations includes three drugs from The Medicines Company to treat bacterial skin infections, thrombotic cardiovascular events and haemostasis. Cubist Pharmaceuticals, Merck & Co and Novo Nordisk accounted for the rest of the recommendations, which included an antibiotic, HIV therapy and obesity drug. Reuters

The controversial Medical Innovation Bill introduced by Lord Saatchi moved a step closer to passing into law. Members of the UK House of Lords voted in favor of the bill, which now passes to the House of Commons. However, with parliament due to shut down on March 30 ahead of the upcoming election, the bill — which has been heavily criticized — may run out of time. The Law Society Gazette

EMA’s effort to create a market for orphan-style veterinary drugs has started to gain traction. Last year, EMA approved three drugs for minor species or rare diseases in major species — such as sheep — and received 29 requests for products to be awarded the classification. The minor use minor species (MUMS) policy came into force in September 2009. Press Release I Regulatory Focus

European authorities have awarded a CE Mark to AliveCor for an algorithm used in its app to detect atrial fibrillation. Having received the certification, AliveCor will begin selling the revised version of its AliveECG app in the UK and Ireland. MassDevice

MHRA has redesigned its website. The regulator’s online presence is now part of the general government services page gov.uk and mirrors its style. MHRA said it has also rewritten its content so it is easier to understand. Press Release