On 18th October, 2024 the UK Medicines and Healthcare products Regulatory Agency (MHRA) updated their guidance on the processes related to "Conditional Marketing Authorisations (CMAs), Marketing Authorisations under Exceptional Circumstances, and National Scientific Advice" for medicines. These pathways are designed to support timely access to medicines, especially in urgent public health scenarios or for conditions where complete clinical data may not be available at the time of application.
CONDITIONAL MARKETING AUTHORISATIONS (CMA)
A Conditional Marketing Authorisation allows for the early approval of medicines based on less comprehensive data than typically required, provided the medicine's benefits outweigh the risks, and there is a pressing need for the product. CMAs are granted when:
The medicine targets serious diseases or unmet medical needs.
There is sufficient evidence to indicate a positive benefit-risk balance.
The applicant agrees to provide comprehensive clinical data post-authorisation.
This option is particularly useful for conditions such as rare diseases, emergencies like pandemics, or medications that address critical public health concerns. A CMA is granted by the MHRA following completion of the MAA assessment, at which point it will decide whether or not to approve the application and grant a CMA, or if the risk benefit ratio is negative and the application should be rejected.
MARKETING AUTHORISATION UNDER EXCEPTIONAL CIRCUMSTANCES
A Marketing Authorisation under Exceptional Circumstances applies when comprehensive data on efficacy and safety cannot be obtained, even after marketing. This is typically due to the rarity of the disease, ethical considerations, or because the disease in question evolves rapidly.
The applicant is not required to provide the standard level of clinical data upfront.
The authorisation is given on the basis of the data that is scientifically available at the time of application.
The MHRA may impose additional requirements, such as specific pharmacovigilance obligations or a commitment to complete additional studies.
The MHRA may consider the EMA's or another jurisdiction's designation of an exceptional circumstances scheme when determining whether the product is eligible for the UK scheme.
NATIONAL SCIENTIFIC ADVICE
To support applicants in navigating the regulatory framework, the MHRA offers National Scientific Advice. This allows sponsors to engage in discussions with the MHRA at various stages of product development, particularly around the planning of clinical trials and the design of studies.
Study design: The agency provides advice on the adequacy of the clinical trial design to generate robust data on the medicine's efficacy and safety.
Data requirements: Guidance on the type of data required for specific types of authorisations, including conditional or exceptional circumstances.
Regulatory requirements: Assistance with understanding the regulatory standards applicable in the UK and how they align with international guidelines.
Applications for scientific advice submitted by UK-based Small and Medium-sized Enterprises (SME) will be exempt from the scientific advice fee. Applicants must submit evidence of their SME status along with the scientific advice form
For further information, the full guidance can be accessed here.
Also, check out this updated guidance about "Rolling review for marketing authorisation applications" which details using the rolling review route when you want to submit your eCTD dossier in separate parts for pre-assessment instead of making a full consolidated document submission.
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