"'Early access' drugs scheme launched for severely ill," BBC News reports.
The government has launched the Early Access to Medicines Scheme designed to help patients with life threatening or debilitating conditions get access to unlicensed medications that could potentially be of benefit.
How are drugs currently regulated?
In the UK, drugs need to gain a marketing authorisation, sometimes called a licence, before they can be used to treat patients. This licence specifies what condition(s) the drugs can be used for. To get this licence, the manufacturers must provide evidence that their drug has been shown to be beneficial in clinical trials (usually phase III clinical trials) and is sufficiently safe to allow wider use among people with that particular condition. This process of testing drugs and then applying for a licence takes a long time – sometimes up to a decade – and not all drugs are successful.
Healthcare professionals can already prescribe unlicensed drugs if there is no suitable licensed alternative and they judge that the drug is likely to benefit the patient. They need to follow professional guidance for this type of prescribing, and there are regulations guiding how the drugs are made and imported. Drugs prescribed in this way may have already received a licence for use in another country (for example the US), but not in the UK.
What role does the MHRA play?
The Medicines and Healthcare Products Regulatory Agency (MHRA) is a Department of Health agency that assesses whether drugs are sufficiently effective and safe to be granted a licence for use in the UK. Drugs can also be granted a licence for use in all European Union (EU) and European Economic Area (EEA) countries through a similar assessment performed by a centralised organisation called the European Medicines Agency (EMA).
What changes have been announced?
A new scheme – the Early Access to Medicines Scheme (EAMS) has been set up to allow patients who have life threatening or seriously debilitating conditions to get access to drugs at an earlier stage in their development than they would normally be available, and before they have been granted a licence. There also needs to be a medical need for these drugs, either because there are no other treatments available or the available treatments are not suitable (for example if the person has not responded to them).
How will the Early Access to Medicines Scheme work?
A drug will have to pass through two stages to be part of the EAMS scheme:
- being designated as a “promising innovative medicine” (PIM)
- being given an official opinion on whether it can be provided under EAMS
Companies can submit an initial application to the MHRA if they think their drug meets the requirements for the scheme, with evidence to support this from the early stages of their clinical trials (for example, from phase II trials). The MHRA will assess the application and decide if they agree that the drug may be eligible for the scheme. If so, the medicine will be given a PIM designation.
The MHRA will assess the balance of benefits and risks, and issue a summary of its findings and its scientific opinion on these. This will allow healthcare professionals and patients to make more informed decisions about whether the drug is appropriate for them. Drugs being prescribed under this scheme have to be provided free of charge by the manufacturer.
The scheme does not replace the normal licencing process, but the early access scheme could allow access several years before the formal licence is granted. For example, the drug could be made available after phase II trials while phase III trials are still ongoing.
When are the changes come into effect?
The Early Access to Medicines Scheme is scheduled to launch in April 2014. At this point, companies can submit applications to the MHRA for their drugs to be considered for the scheme.
What has been the reaction to the news?
Drug companies and patient groups have mainly reacted positively to the launch of the scheme. The BBC quotes the chief executive of the BioIndustry Association, Steve Bates, who said: “(the launch) shows the UK is committed to an ‘all hands on deck approach’ to speedily progress promising innovative therapies to the patients that need them.”
Echoing this, Harpal Kumar, chief executive of Cancer Research UK, said: "Time is of the essence for many cancer patients, particularly those with more advanced disease.
"It can mean the difference between life and death. Therefore this scheme, which has at its heart the potential to bring promising new medicines to patients faster, is to be warmly welcomed."
However, reaction has not been universally positive. The Faculty of Public Health at the Royal College of Physicians has apparently warned that the scheme could expose vulnerable patients to untested medicines that could shorten, rather than lengthen, their life.