The MHRA ensures that any new medicine or treatment is safe and effective, and that the benefits outweigh possible side effects. The licence will confirm what condition a medicine should be used for and what dose is best.
For any prospective treatment for Huntington’s disease the MHRA’s role is to:
- the safety and quality standards of medicines and medical equipment
- the safety of the supply chain for medicine and medical equipment
- promoting international standards to ensure biological medicines are effective and safe
- educating the public and healthcare professionals about the risks and benefits of medicines and medical equipment
- supporting research and development that benefit public health
- influencing UK and international regulations so that they protect public health
When a company has carried out a successful clinical trial and believe they have sufficient data to demonstrate its safety and effectiveness they can choose to submit a Marketing Authorisation Application (MAA) to the MHRA.
This includes:
- All preclinical and clinical data
- Manufacturing and quality information
- Risk–benefit analysis
The MHRA reviews:
- Safety
- Efficacy
- Quality of production
Approval Decision
The MHRA may:
- Approve the medicine → it can be licensed in the UK
- Request more data for further consideration
- Reject if standards aren’t met
If a drug is licensed then doctors are able to prescribe them. However, there is an additional process to decide if the NHS will pay for the drug.
These organisations decide which drugs work well and are cost effective to use.
They are:
- The National Institute for Health and Care Excellence (NICE) in England
- The Scottish Medicines Consortium (SMC) in Scotland
- The All Wales Medicine Strategy Group (AWMSG) in Wales
After licensing, the MHRA continues to collect information. This information is about how well the drug works and about the side effects, meaning they continue to check the safety and effectiveness of the drug.
