Euro Roundup: UK sets up group to involve patients in ILAP pathway

RoundupsRoundups | 26 August 2021 |  By 

UK sets up group to involve patients in Innovative Licensing and Access Pathway
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has formed a patient group to support its Innovative Licensing and Access Pathway (ILAP). MHRA set up the group to ensure that “patient views are an integral part of the ILAP ambition.”
UK officials shared details of ILAP late last year, describing how the pathway will accelerate time to market by enabling drug developers to access input from MHRA and NICE. MHRA updated its ILAP guidance this week with a new section on the governance of the pathway and the involvement of patients in the process.
The agency said the patient reference group will play a particularly important role at three points in the process, starting with the Innovation Passport designation stage. Drug developers need to show their medicines could benefit patients and meet other criteria to obtain a passport and thereby gain access to the pathway.
MHRA also sees a role for patients at the Target Development Profile stage, which is designed to create a road map for early access, and in the development of the patient engagement tool. MHRA has previously said the engagement tool will “consider the patient’s voice and experience from clinical design, scientific advice and all the way through to the generation of meaningful real-world data post-licensing and pharmacovigilance.”
The agency disclosed the creation of the patient reference group alongside details of a steering group it has set up to oversee ILAP activities. The steering group will make recommendations for Innovation Passport applications and take responsibility for the strategic development and implementation of ILAP. 
MHRA Guidance
NICE seeks feedback on plans to create faster, more flexible HTA processes
The UK's National Institute for Health and Care Excellence (NICE) is seeking feedback on changes that could make it easier for developers of treatments of severe diseases and gene therapies to access the UK market.
NICE, which determines whether new treatments are good value for money, is aiming to cut the time it takes patients to receive novel therapies and offer more flexibility with its cost effectiveness decisions. Those overarching goals are supported by specific proposals, including a plan to put more emphasis on treatments for severe diseases.
Currently, NICE can recommend treatments that breach its typical value threshold if they are for use in the last months of a patient’s life. The modifier, which mainly benefits cancer drugs, exists despite there being no evidence society puts more value on end-of-life care. There is evidence that society puts a higher value on treatment for severe diseases, though, leading NICE to propose the extension of the modifier beyond end-of-life therapies. The size of the modifier is still being discussed.
NICE is also reconsidering the evidence companies need to provide, noting that research challenges can make it hard to show the long-term benefits of treatments for rare and pediatric diseases. Giving committees the flexibility to recommend therapies despite uncertainty could increase patient access. Similarly, NICE is proposing to “use more evidence from patients’ lived experience,” rather than rely so heavily on data from randomized controlled trials.
The proposals also feature specific positives for developers of gene therapies. NICE uses a discount rate to adjust costs and benefits that will happen in the future. The rate is set to 3.5%, but NICE wants to reduce it to 1.5%. NICE said the change could make a “big difference to some treatments, like gene therapies,” which are one-time interventions with multi-year benefits. However, cutting the rate would impact healthcare spending, meaning NICE needs to talk to other groups before making the change.  
NICE is accepting feedback on the proposals until 13 October.
NICE Consultation
European Commission hits back after Illumina closes Grail deal mid-probe
The European Commission has opened an investigation into Illumina’s decision to close its $8 billion takeover of liquid biopsy player Grail before obtaining regulatory approval.
Despite pending trials and investigations on both sides of the Atlantic, Illumina closed the takeover earlier this month. The action prompted a swift response from the Commission, which is now looking into whether the closing of the deal before the conclusion of its investigation constitutes a breach of merger regulations. 
“We deeply regret Illumina's decision to complete its acquisition of Grail,” Margrethe Vestager, executive vice president of the Commission, said. “Companies must wait for our approval before a transaction can go ahead. This obligation, that we call standstill obligation, is at the heart of our merger control system and we take its possible breaches very seriously.”
Illumina was aware of the risks when it closed the deal, warning investors the Commission is likely to try to fine it 10% of its annual turnover. Yet, with the Grail offer due to expire before the Commission makes a decision, Illumina has gambled that it will be able to get the takeover past authorities in the European Union, either because they support the deal or a court rules they lack jurisdiction over it.
Commission Notice
MHRA recalls metformin batch after discovering nitrosamine impurity
MHRA is recalling more than 10,000 units of metformin oral solution after discovering low levels of a nitrosamine impurity. The presence of N-nitrosodimethylamine (NDMA) above the acceptable limit was discovered during routine monitoring.  
Regulators around the world have found NMDA in some metformin products in recent years. Unlike in the heartburn drug ranitidine, the impurity is only seen in a fraction of metformin products, and as such regulators think it should be possible to make the medicines without the contaminant. Not all manufacturers are managing to eliminate the impurity, though.
The latest recall affects a single batch of metformin oral solution sold by Rosemont Pharmaceuticals in the UK. MHRA responded to the discovery of the potentially genotoxic and carcinogenic impurity with a Class 2 recall that requires pharmacies and wholesalers to quarantine all stock immediately.
While the recall will stop more of the contaminated metformin from reaching the public, MHRA is recommending that patients continue taking treatments as prescribed until they talk to their relevant healthcare professional. The recommendation reflects a belief the risk of suddenly stopping taking the treatment for type 2 diabetes is higher than the potential threat posed by the impurity.
MHRA Notice
France forms committee to set technical specifications for medicinal cannabis
The French National Agency for Medicines and Health Products Safety (ANSM) has set up a temporary scientific committee focused on technical specifications for the cultivation and supply of medicinal cannabis.
The committee will define methods for the traceability of the plants, pharmaceutical quality criteria, controls for cultivation in France and assessments of the THC and CBD content of the plants. ANSM set the committee up in response to a 2020 decree that permits the medicinal use of cannabis in some circumstances and a pending decree that will allow cultivation of the plant in France.
ANSM’s committee is set to meet for the first time next month. After four months, the committee, which will feature representatives of multiple government ministries, will issue an opinion.
ANSM Notice (French)
Other News:
MHRA has approved Regeneron’s anti-SARS-CoV-2 antibody cocktail for use in the treatment and prevention of acute COVID-19 infection. The government is yet to say which patients will receive the treatment. MHRA Notice


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