Euro Roundup: MHRA shares guidance on planning approval submissions and seeking support

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has shared guidance on accessing pre-submission advice and support, as well as on providing information about future submissions for approval.

In the pre-submission advice and support guidance, MHRA explains that it offers a service for companies that are planning to seek approval for a medicine in the UK. The service “provides stakeholders with the means to understand how the process of compiling and submitting applications and supporting evidence applies for their product,” MHRA said. The service is distinct from MHRA’s scientific advice offering.

Companies must request a pre-submission meeting at least three months before they plan to submit an application for approval of a new active substance or biological product. The guidance includes an email form for requesting a meeting.

Applicants intending to seek approval via the international recognition procedure also have the option of requesting pre-submission advice from MHRA. Again, the agency has provided a form to request advice and support. MHRA will assess the requests and either direct the company to relevant guidance, provide written advice or offer a pre-submission meeting.

MHRA also published a document on how marketing authorization holders can provide information on planned or potential future submissions.

“Having advanced knowledge of expected submissions of all types and requests for advice is key for the MHRA to effectively deliver our services to the highest quality and to be predictable with our assessment and advice timelines,” MHRA wrote.

Collecting the data will enable the agency to assess the short- and medium-term demand for its services. MHRA said this will allow it to ensure consistent and predictable delivery of timelines for assessments and advice.

The agency has provided a template spreadsheet to help companies submit the requested information. MHRA said it is trying to “build a picture of all incoming applications and new indications up to five years in advance of submission.” The requested information includes the product name, active ingredient, services required, and timing of the expected submission.

MHRA said, “Any pipeline data submitted is not intended to be concrete,” adding that it “will not be holding stakeholders to account based on this data.” The agency knows circumstances change over time and, as such, its pre-submission team “will keep records of incoming pipeline data and request updates on an approximate six-monthly basis.”

The request for information is aimed at all marketing authorization holders, regardless of whether they have 1 or 100 candidates in their pipelines. MHRA will process the information into “a restricted master spreadsheet which has defined access across relevant parts of the organization.” The agency will put the individual spreadsheets in locked secure folders until an update is requested from the company.

MHRA Guidance, More

ABPI calls for NICE to immediately review disease severity drug value modifier

The Association of the British Pharmaceutical Industry (ABPI) has said the UK National Institute for Health and Care Excellence (NICE) should immediately review the severity modifier it uses to weight the value of medicines that treat more severe conditions.

NICE introduced the severity modifier in 2022. Before that, the institute applied an end-of-life modifier to improve access to treatments for cancers and other end-of-life conditions. The new modifier could improve access to certain medicines by shifting the focus from end-of-life care to other drugs. As a cost-neutral change, it could also reduce access to other drugs.

ABPI said the severity modifier is a “step forward” but its implementation has raised access concerns. The concerns reflect the potential for the cost-neutral model to “inadvertently limit access to late-stage treatments for some patients with severe diseases,” the trade group said.

“The recent analysis suggests that the way in which the severity modifier has been applied so far is on a more conservative basis than is needed, even to deliver opportunity cost neutrality,” ABPI said. “Some medicines previously eligible for the end-of-life modifier are now ineligible for the highest severity weighting under the new system.”

ABPI’s analysis of feedback from companies that have been through the NICE process found the severity modifier has resulted in an average quality-adjusted life year (QALY) weighting of 1.073. That is lower than the QALY weighting NICE used to design the modifier, 1.119, and lower than the average QALY weighting, 1.125, which was calculated to be “opportunity cost neutral” compared to the previous modifier.

The trade group wants NICE to “adjust downwards the cut-off levels used to determine the degree of severity so that more medicines can benefit.” It is also calling for the replacement of the cost-neutral approach with a model that “better reflects societal preferences for helping people with severe disease to access innovative treatments.”

“There is room for much more flexibility in how the severity modifier is applied,” ABPI said. “By allowing for deviation from opportunity cost neutrality, the NHS could potentially deliver broader access to newer, more effective treatments for severe conditions, better reflecting the value that patients place on these treatments.”

ABPI Statement, Modifier Report

Swissmedic updates information on some non-standardized medicinal products

The Swiss Agency for Therapeutic Products (Swissmedic) has provided an update on non-standardized medicinal products, revising an information sheet and list of products affected by a manufacturing rule. Swissmedic previously created the product category to cover patient-specific preparations.

The update replaces the preparations “platelet-rich plasma” and “platelet-rich fibrinogen” with the preparation group “platelet products for autologous or allogeneic use.” The agency also added details about establishment licenses for manufacturing and distributing non-standardized medicinal products. The other changes affect the legal provisions for preparations with a purely cosmetic purpose.

The updates affect an annex on medicinal products and medicinal product groups with a manufacturing process subject to mandatory authorization. Swissmedic’s Agency Council agreed to update the annex and other documents earlier this year after talking to subject-matter experts and official bodies.

Swissmedic Notice

Other news:

A German advisory panel has recommended everyone aged 75 years or older is vaccinated against the respiratory syncytial virus. The STIKO panel also recommended physicians vaccinate people aged 60 to 74 years who have a severe form of an underlying disease or live in a care facility. STIKO’s advice positions German doctors to prescribe recently launched GSK and Pfizer vaccines this fall. STIKO Notice

MHRA has approved Astellas Pharma’s zolbetuximab. The approval covers patients with gastric or gastro-esophageal junction cancer that is positive for Claudin18.2 and negative for HER2. Zolbetuximab, which Astellas will sell as Vyloy, targets Claudin18.2. HER2 is the target of AstraZeneca and Daiichi Sankyo’s Enhertu, another drug used to treat gastro-esophageal junction cancer. MHRA Notice

EMA has published a direct healthcare professional communication about the potential for patients to experience anaphylactic reactions months to years after starting treatment on the multiple sclerosis drug glatiramer acetate. The drug, which is sold under brands including Copaxone, was the subject of a review across the European Union that triggered the letter to healthcare professionals. EMA Notice

Euro Roundup: MHRA shares guidance on planning approval submissions and seeking support

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