New fast track drug scheme to help Pharma get more data

#MSResearch fast-tracking drugs to MS

The UK government has launched the Early Access to Medicines Scheme designed to help people 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 MHRA is a Department of Health agency which 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 people 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)mbeing 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.

This allows NHSers to join studies when they may not full-fill the criteria to enter trials. This allows pharma to collect data provided they pay for it, which will help get them a licence. 

Will MSers get access to this scheme? 

Will Pharma Buy into this as they are supplying free drug?

Rare side effects often only appear once a drug is licenced, if they appear before licencing is this an extra risk for the company.

Would you sign up for a trial with the risk you get placebo or something you don’t want, or will you wait and be sure you get drug. This was one percieved problem with the Alemtuzumab trial, you know what you got, if you were put in the beta interferon arm you were more likely to drop out.

What will ProfG make of this?

Is the Early Access Scheme a first arm to the Big Pharma Alternative (BPA) have the Government listened (click here)? 

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