GovWire

1. Overview

TheMHRAoffers a national Conditional Marketing Authorisation (CMA) scheme for new medicinal products. This is available in specific circumstances where comprehensive clinical data are not yet complete but it is judged that such data will become available soon.

2. Guidance for UK Conditional Marketing Authorisation Applications

Until implementation of the Windsor Framework on 1 January 2025[1] CMAs can only be granted by MHRA for Great Britain (England, Wales and Scotland).

In Northern Ireland, until that date, a CMA is only available through the EMAs Centrally Authorised Procedure as laid down in Regulation (EC) No 726/2004.

From 1 January 2025, the MHRA will grant UK-wide CMAs for products in line with the conditions set out in Regulation 50I of the Human Medicines Regulations 2012 (as amended) and explained in this guidance.

These products will be authorised as Windsor Framework Category 1 products. Please refer to further guidance on these categories: UK-wide licensing for human medicines - GOV.UK (www.gov.uk)

In cases where a medicinal product fulfils an unmet medical need, the MHRA may grant a CMA where comprehensive clinical data which are normally required, are not yet complete, but it is judged that such data will become available soon.

The scheme is aimed at products that fulfil an unmet medical need:

1. for the treatment, prevention or diagnosis of seriously debilitating or life-threatening diseases; or
2. for a product to be used in emergency situations, in response to public health threats.

The criteria for a CMA are that the applicant can demonstrate:

• the balance of benefit and risk of the medicine is positive;
• it is likely that the applicant will be able to provide comprehensive clinical data following authorisation;
• the medicine fulfils an unmet medical need;
• the benefit of the medicines immediate availability to patients is greater than the risk inherent in the fact that additional data are still required.

Unmet medical need means medical need in relation to a condition for which there is currently no satisfactory method of diagnosis, prevention or treatment authorised in the UK, or, if one exists, the medicinal product will offer a major therapeutic advantage to those affected.

The Marketing Authorisation Application (MAA) must still contain adequate evidence of safety and efficacy to enable the MHRA to conclude that the risk-benefit balance of the medicinal product is positive. Once a conditional marketing authorisation has been granted, the marketing authorisation holder must fulfil specific obligations within defined timelines. These obligations could include completing ongoing or new studies or collecting additional data to confirm the medicines benefit-risk balance remains positive.

There is no specific application route for a CMA; applicants should submit their MAA dossier as for a full Marketing Authorisation at: https://www.gov.uk/government/collections/licencing-how-to-apply

Applicants wishing to submit an application for a CMA to the MHRA should state their justification for a CMA and indicate clearly what clinical studies are underway and when comprehensive clinical data will become available.

Eligibility for a CMA will be determined by the MHRA at the time of MAA assessment. At the completion of the assessment, the MHRA will determine whether to approve the application and grant a CMA or whether the risk benefit ratio is negative and reject the application.

The designation of a product as being eligible for a CMA by the EMA or another jurisdiction may be taken into account by the MHRA, but the final decision on eligibility of the product for the UK scheme will rest with the MHRA.

CMAs will be valid for one year and will be renewable annually, upon application by the marketing authorisation holder (MAH).

The marketing authorisation can be converted into a standard (full) marketing authorisation (no longer subject to specific obligations) once the MAH fulfils the obligations imposed and the complete data confirm that the medicines benefits continue to outweigh its risks.

3. Guidance for UK Marketing Authorisations under exceptional circumstances

The MHRAs scheme for applications under exceptional circumstances is available for medicines where a comprehensive data package cannot be provided, because the condition to be treated is rare or because collection of full information is not possible or is unethical. Until 1 January 2025 applications for Marketing Authorisations under Exceptional Circumstances in Northern Ireland must be submitted to the EMA. From that date the MHRA can issue them as UK-wide MAs.

The scheme is covered under Human Medicines Regulations 2012, Regulation 60, Conditions of UK marketing authorisation: exceptional circumstances.

Approvals will only be granted under this scheme where there are exceptional circumstances and where the applicant can demonstrate that it is not possible to provide comprehensive data on the efficacy and safety under normal conditions of use.

The designation of a product as being eligible for an exceptional circumstances scheme by the EMA or another jurisdiction may be taken into account by the MHRA, but the final decision on eligibility of the product for the UK scheme will rest with the MHRA.

This authorisation route normally does not lead to a standard marketing authorisation.

3.1 Actions for those applying for UK marketing authorisations under exceptional circumstances

The MHRA is likely to impose specific obligations on the holder of a Marketing Authorisation that is approved under exceptional circumstances. These will be communicated to the applicant during the review and will be aimed at the provision of information on the safe and effective use of the product.

Applicants are required to discuss their submissions with the MHRA prior to submitting their Marketing Authorisation Application.

4. Guidance for national scientific advice

The MHRA offers a national scientific advice service. This service is available for developers of medicinal products and marketing authorisation holders and can be requested at any stage of the products lifecycle. The procedure for requesting scientific advice is described in further guidance.

Applications for scientific advice submitted by UK-based Small and Medium-sized Enterprises (SME<

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