Early Access to Medicines scheme

Last month the UK Government’s Department of Health announced an Early Access to Medicines Scheme. But, what is this new scheme? and what does it mean for people living with MND?

The drug trial process

The only proven drug to slow progression in MND is riluzole. This is the only drug that has passed all the stages of drug testing – known as clinical trials – and has been shown to be beneficial for people living with MND. Clinical trials are the ‘gold standard’ and are put in place to ensure that a drug is safe and beneficial before it is given to patients.

At present, there are a number of potential drugs for MND undergoing the clinical trial process. The first stage of testing is known as a Phase I clinical trial. Phase I clinical trials are where a drug is tested in humans for the first time, which means the researchers are looking at safety only.

Phase II clinical trials are carried out to find out if the drug is safe and well-tolerated in people living with MND, during this stage of testing the researchers will also determine the correct dose of the drug. If the results are positive after Phase II, the drug would then enter Phase III testing, to find out whether it is beneficial or not in MND.

After Phase III testing the drug, and all of its clinical trials data, is reviewed by the appropriate licensing body before the drug can be licensed for use (in Europe this is the European Medicines Agency – EMA). After a license is given, the data will be continuously collected over a longer period of time, which is known as Phase IV testing.

Early Access to Medicines

Last April, we wrote a blog post about adaptive licensing. Adaptive licensing is another proposed way to get drugs to patients more quickly, but it’s not the same as the Early Access to Medicines Scheme. The Association welcomes the idea of more drugs being made available to more people more quickly, however more detail is needed on this current scheme.

The Early Access to Medicines scheme will allow early access to drugs that have been proven to be safe and beneficial – we are still unsure as to what stage of clinical trial testing this would be, but statements by the Medicines and Healthcare Regulatory Agency (MHRA) have suggested that this could be several years before a drug is licensed.

Before a drug is given early access the drug company will have to complete the following steps:

• Step I – Promising innovative medicine (PIM) designation The PIM designation will give an indication that a product may be eligible for the Early Access to Medicines Scheme (based on early clinical data). The PIM designation will be issued after a MHRA scientific meeting with the drug company.
• Step II – Early access to medicines scientific opinion The scientific opinion will describe the benefits and risks of the medicine, based on the information submitted to MHRA by the drug company after sufficient data have been gathered from the patients who will benefit from the medicine. At this stage the scientific opinion will then support the prescriber and patient to make a decision on whether or not to use the medicine before its licence is approved.

In MND, and many other neurological diseases, there is a lack of drugs passing the Phase II stage of testing, with drugs often showing no potential benefit or highlighting safety concerns. This is possibly due to the exact cause of MND not being known. Once researchers identify the causes it is more likely that we will be able to develop successful treatments that pass this stage of testing.

Find out more:

Our MND Campaigns post explains more about the Early Access to Medicines Scheme, including what our next steps are. You can read the blog post here.

Following the announcement of the scheme, the Association of the British Pharmaceutical Industry (ABPI) and the UK Bioindustry Association have organised a meeting to better understand it. The Association of Medical Research Charities (AMRC) has invited member charities, including the Association, to attend this meeting later this month to find out more about the scheme.

Research Information Co-ordinator, Dr Samantha Price, comments: “Riluzole is the only drug that has been through the rigorous clinical trial process, proving to both the scientific and medical community that it is beneficial in MND.

“This scheme and speeding up access to new drugs is not the main problem in MND, it’s developing effective therapies in the lab that is. This is why it is essential for us to identify the causes of MND. Once we have passed this step, we can further understand the disease and really go on to develop successful treatments.

“Hopefully this meeting later this month will give the Association a better understanding of the scheme and what it means for people living with MND.”

• MND Campaigns blog post
• Research Information Sheet D – Clinical trials