Developing a new treatment can take many years, from the early stages of laboratory research through the essential stages of clinical trials and regulatory checks to the point where it can be prescribed for people living with conditions such as EB.  

Timeline of stages in drug approval process (England)Drug approval process.

NICE advises the NHS in England and Wales but the process in Scotland is slightly different. In Northern Ireland, marketing authorisation comes from the European Medicines Agency (EMA).


Licensed medicines

After a clinical trial has shown a new medicine is safe and effective, it must be granted a license before it can be made available for widespread use. The license confirms the health condition it can treat and the recommended dosage.

Licenses are granted by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK, by the European Commission, acting on advice from the European Medicines Agency (EMA) in Europe and by the Food and Drug Administration (FDA) in America.  These bodies consider all the evidence to ensure that the medicine is safe and effective. The license they then grant means we can trust that the medicines we are offered will help rather than harm us.

Because different countries have different processes, some medicines can be available in some countries but not in others.

When a medication has a license from the MHRA that confirms it is safe and effective, it can be made available in the UK to buy privately at a price set by the pharmaceutical company that manufactures it.

Once the price of the medication is set, the National Institute for Health and Care Excellence (NICE) assesses the evidence for the effectiveness of the treatment and its cost. NICE recommends whether it should be funded by the National Health Service (NHS) and for which conditions and symptoms. The medicine should be available through the NHS within three months after NICE has made the funding decision.


Unlicensed medicines

A medicine is considered ‘unlicensed’ if it is used to treat a condition not specified in its license. Unlicensed use means that there is a recommended dosage and known safety and side effects of a medicine but that it may not have been fully evaluated in a new condition it is used ‘off-label’ to treat.

A doctor may recommend an unlicensed medication if they think the potential benefits are greater than any risks. 

Drug repurposing means using an existing medicine that already has a license for one condition to treat another condition. It can also apply to using an existing licensed medicine in a different dose or form to that specified in the license.


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