Regulatory Focus™ > News Articles > 2021 > 7 > Euro Roundup: MHRA misses multiple performance targets due to COVID-19, CAP grandfathering

Posted 22 July 2021 | By Nick Paul Taylor 

Euro Roundup: MHRA misses multiple performance targets due to COVID-19, CAP grandfathering

3023 The UK Medicines and Healthcare products Regulatory Agency (MHRA) missed several performance targets last year as it contended with challenges including COVID-19. A target for the production of validation reports for marketing authorization applications was among the missed objectives.
MHRA aims to produce a validation report for a new marketing authorization application within 14 days of case creation 97% of the time. In its 2020 to 2021 fiscal year, MHRA hit that deadline for 91% of cases. MHRA said it missed the target because new marketing authorization applications were “significantly impacted” in the fourth quarter by operational pressures in centrally authorized product (CAP) grandfathering.
“Various technical issues surrounding the CAP grandfathering processing necessitated the short-term redeployment of experienced staff from the business as usual new MA area into supporting CAP grandfathering. This resulted in some limited slippage in performance. This area is now beginning to return to normal in-target performance, but a residual impact will be seen in Q1 2021-22,” MHRA said.
MHRA also missed its target of resolving 95% of information cybersecurity incidents within 15 days of them being reported. In the 2020 to 2021 financial year, MHRA resolved 81% of incidents within that timeframe. MHRA said the underperformance happened because the information security team had to deal with an increased number of cases that required investigation and responses from users. It took the team longer than 15 days to resolve some of those larger investigations.
The agency also more narrowly missed its performance targets for responding to requests under the Freedom of Information Act and to Parliamentary Questions. In both cases, MHRA said it missed the targets “for reasons including resourcing issues and increased workload due to COVID-19.”
MHRA Report
Dutch regulator issues $23M fine over ‘excessive’ price of rare disease drug
The Netherlands Authority for Consumers and Markets (ACM) has fined Leadiant €19.6 million ($23.1 million) for charging “far too high a price” for a rare disease drug. ACM accused Leadiant of taking an old drug that once sold for less than €1 a capsule and using the regulatory system to gain exclusivity.
News of Leadiant’s activities emerged in 2018, when the nonprofit medicine price watchdog Dutch Pharmaceutical Accountability Foundation revealed plans to file a claim with ACM. The foundation got involved after seeing the price of a year’s supply of chenodeoxycholic acid (CDCA) rise quickly from €300 to €150,000.
ACM set out the sequence of events in its statement. The competition regulator said Leadiant, then called Sigma-Tau Pharmaceuticals, acquired a CDCA-based drug from another manufacturer in 2008. At that time, the drug had been supplied for years under various trade names in the Netherlands for off-label treatment of the rare hereditary metabolic disorder cerebrotendineous xanthomatosis (CTX) at a maximum price of €46 per 100 capsules. Leadiant bought another CDCA product from Solvay Pharmaceuticals in 2009 and raised the price to €885 late that year.
Leadiant received European Medicines Agency (EMA) orphan drug designation for CTX in 2014. The following year, Leadiant removed its CDCA product, then sold as Chenofalk, from the market. Leadiant went on to win EMA approval for CDCA in CTX in 2017 on the strength of two retrospective cohort studies. With orphan drug status giving it 10 years of market exclusivity in CTX, Leadiant then raised the price of a pack of CDCA to €14,000. The price of a year’s supply rose to €153,000.
“After a small, low-risk investment, Leadiant implemented a huge price increase for a drug that had already existed for years. In this case, there was no innovation at all. We consider this to be a very serious violation. The price increase offers Leadiant a very high return, but it offers patients very few additional benefits, and it drives up the costs to society,” Martijn Snoep, chairman of the ACM board, said in a statement.
The fine relates to the price Leadiant charged in the Netherlands from June 2017 to December 2019. Over that period, ACM said no alternatives to Leadiant’s product were available, so health insurers had to keep paying for the medicine despite the company charging an “exorbitantly high and unfair” price. A hospital began making CDCA at its pharmacy in January 2020, ending Leadiant’s monopoly.
ACM Notice
Finland updates instructions for submitting drug shortage notifications
The Finnish Medicines Agency (Fimea) has updated its instructions for submitting notifications of drug shortages. Fimea made the changes after detecting “a particularly large number of notifications and appendices with inaccurate or incomplete information.”
Officials have seen errors in the notification dates and Nordic Article Numbers. Fimea has responded to the errors by telling drugmakers, “The notification date must correspond to the date when the notification is submitted.” The requirement applies to new and update notifications. Fimea registers submissions sent after 4 PM the next day,
The new advice also states date and Nordic Article Number fields “must not contain any extra text or spaces before the requested information.” The notifications are processed by machine, so incorrect information prevents the details from being updated on the Fimea website.
Fimea shared the updated instructions alongside a revised appendix. The new appendix contains a field for the purchase order number.
Fimea Notice
MHRA revises guidance on renewing marketing authorizations for medicines
MHRA has updated its guidance on renewing marketing authorizations for medicines. The revised guidance features a new section on using Committee for Medicinal Products for Human Use (CHMP) opinions to support renewals in the UK.
Under the recognition route, the holder of a full or conditional marketing authorization can use the renewal application filed for the centralized product to seek renewal in Great Britain. MHRA is asking applicants that use the route to clearly indicate they are doing so in the cover letter and provide the CHMP opinion as evidence of acceptance. Applications pending a CHMP opinion will be put on hold.
MHRA is asking applicants to submit the positive opinion and other documents, such as a copy of the final assessment report, via its portal. A copy of the Commission Decision is needed to conclude the renewal. MHRA is charging applicants that use the recognition route for the first five-year renewal of a marketing authorization of a new active substance a reduced fee of £747 ($1,027).
MHRA Guidance
EMA shares opinion on diethanolamine as excipient in topical products
EMA’s Safety Working Party (SWP) has issued its opinion on the use of diethanolamine and coconut oil diethanolamine condensate as excipients in topically applied human medicinal products.
SWP began looking into the safety of the excipients after the Committee for Medicinal Products for Veterinary Use raised concerns relating to carcinogenicity and genotoxicity. Based on preclinical data, the safety group calculated the maximum daily limit of diethanolamine for lifetime use, as well as a far higher limit for products that are used for up to 12 months.
The opinion also features details of the safety of medicinal products that are rinsed off and advice on avoiding the formation of nitrosamines. SWP said the reaction that causes nitrosamine formation is “very unlikely” for dermal products “since as a rule they do not possess an acidic pH as present in the stomach.”
SWP Opinion


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