'Early access' drugs scheme launched for severely ill


The BBC's Fergus Walsh describes how the scheme will work

Severely ill patients and those with rare debilitating conditions could be given new medicines years before they are licensed, under new regulations.

The Early Access to Medicines scheme would enable a small number of promising medicines to be fast-tracked.

The idea is to help patients in England with severe illnesses who have no other treatment options.

The Medicines and Healthcare Products Regulatory Agency (MHRA) will oversee the scheme, being launched in April.

Health Secretary Jeremy Hunt said: "What patients want is sometimes to try medicines that may not be clinically proven to be effective but are clinically safe.

"We are streamlining the process so these medicines can be used much earlier - particularly if they have early promise - and that is something which will bring hope to a lot of patients."

Hope for Liam

Liam Triggs

One patient who could potentially benefit is Liam Triggs, aged nine, from Liverpool, who has Duchenne muscular dystrophy.

The muscle-wasting condition is progressive and Liam's condition will continue to deteriorate unless an effective treatment is found. There are several potential medicines in late-stage clinical trials.

Liam's mother, Joanne Ashton, said: "We are very hopeful that there might be an opportunity for him to take part in clinical trials in the future.

"This step forward has given us the hope we so desperately need. I would not put my son's life in danger, but we will do anything we can to help him."

The MHRA stressed that the early access scheme would in no way replace the system of bringing drugs to market via clinical trials.

But that can typically take a decade of patient trials and assessments before a licence is granted.

For unlicensed drugs to be used under the early access scheme, sufficient data from several years of clinical trials would be needed to show that it looks like a promising and innovative treatment.

The MHRA will also carry out an assessment of the benefits and risks, which will be available for doctors and patients.

The MHRA said seriously ill patients - with conditions like cancer or muscular dystrophy - might be allowed access to a drug before it is licensed, and outside a clinical trial, where it could be shown there were no other treatment options.

Life and death

The MHRA has estimated that one or two medicines a year might qualify under the scheme, although some in the pharmaceutical industry think it could be higher.

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."

Department of Health officials said the scheme would benefit pharmaceutical companies by enabling them to gain experience of their medicines being used in the NHS.

How the scheme will work

The Early Access to Medicines scheme will work alongside and not replace the current system of clinical trials.

When the MHRA has enough data to reach an opinion that a new drug works and is sufficiently safe, it can issue a promising innovative medicines (PIM) designation.

This could occur years before a medicine gets licensed while large scale Phase III clinical trials are still continuing or while drugs are waiting for authorisation.

Before it could be prescribed there would need to be a scientific opinion from the MHRA describing the risks and benefits of the medicine, which would enable doctors and patients to make an informed decision about whether to use the drug.

This might enable companies to attract investment and help ensure new drugs are tested in the UK.

Steve Bates, of the BioIndustry Association, said the move showed that the UK was committed to an "all-hands-on-deck approach" to speedily progress promising innovative therapies to patients.

But there was concern from industry that the scheme is currently unfunded by government, so companies will not be reimbursed for the cost of unlicensed drugs.

Patients 'vulnerable'

Mr Bates said: "Without centrally funded reimbursement, the scheme runs the risk of being underutilised."

The plans have drawn criticism from the Faculty of Public Health (FPH) at the Royal College of Physicians.

In its response to a consultation it said the scheme would not be of overall value to patients.

The FPH said the current licensing system had worked well, and patients with serious illnesses might be vulnerable to taking risks with medicines that had not been fully tested and which might shorten rather than prolong their lives.

Fergus Walsh Article written by Fergus Walsh Fergus Walsh Medical correspondent

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  • rate this

    Comment number 24.

    I wonder - just wonder a bit how much the pharma companies will be charging for these drugs?

  • rate this

    Comment number 23.

    Yeah! Great! This is how fascists do medicine! Ignore clinical trials (making sure it's not toxic to humans) just give it to those desperate enough to give it a go! Hang on a min - isn't this how thalidomide was tested???

  • Comment number 22.

    This comment was removed because the moderators found it broke the house rules. Explain.

  • rate this

    Comment number 21.

    Nothing is ever perfect, there are problems in NHS & also serious supply shortage problems of various drugs in UK but hey, would you rather be in 1980 or 1970 when so many of todays treatments, technology & drugs did not exist.


    As a live kidney donor for my partner I know about desperation, what would YOU do for a son/daughter/wife, just belittle them would you!!

  • rate this

    Comment number 20.

    It certainly makes sense to give patients who are definitely dying of their disease drugs that haven't fully gone through the testing process and may even be dangerous. There isn't much to lose and everything to gain. The results of giving the drugs to these patients will speed up the path to the drug being licensed on the drugs that do prove to be good, so saving many lives in the long run.

  • rate this

    Comment number 19.

    It smacks of asking the desperate to do desperate things! - not the most inspiring notion I've ever heard of

  • rate this

    Comment number 18.

    Believe it when I see it.

    Currently patients are denied treatment, on the NHS, for various things that others in the next postcode are getting treatment for.

    It boils down to NICE and the local heath trusts deciding what is the "best bang for their buck", rather than what is in the patients interests.

    Will hat change with this new "early access" initiative, I doubt it.

  • rate this

    Comment number 17.

    This only benefits Big Pharma, their drugs generally don't work and have serious side effects, there's a good reason they are not allowed on the market; most don't even beat placebos.

    Look what happened with the Swine Flu scandal Fergus (which you pushed hard), now there are sick children all around the world who were harmed from the untested jab which was rushed out.

  • rate this

    Comment number 16.

    Ahhh, the drug licensing rules.

    I beautiful little con where the government and pharmaceutical companies withold life saving medicine and drive prices up to crazy levels.

    As the model was made for the U.S healthcare 'system' (Market) it doesn't really hold much relevance in a social healthcare model such as the NHS, so not much money will be lost, which is why the Tories aren't bothered too much

  • rate this

    Comment number 15.

    Best news ever....
    I cant believe that people living with a death sentence are not already able to have access to drugs that could possibly save there lives. This is not before time, if its a terminal illness they have nothing to loose and everything to gain.
    Best news I've heard for a very long time.

  • rate this

    Comment number 14.

    This sounds really good, however I would advise caution! To me it sounds like a very right-wing way of performing medicine. I can see an area of opportunism for unethical treatments; whereby standard (tried & tested) treatments are discarded in favour of "experimentation" - and it'll be the less-educated that go for these ideas. Safeguards are in place for a reason, why mess with the system?

  • rate this

    Comment number 13.

    Let's face it, if diagnosed with a terminal illness, it's always going to be worth taking a punt on any drugs that might prolong your life.

  • rate this

    Comment number 12.

    Medical Marijuana should be the first 'untested' drug to be rolled out to the multitude of patients that want it and need it.

  • rate this

    Comment number 11.

    It has taken far too long for plain common sense to prevail with consequence of more people suffering needlessly.

    It is attrocious when laws/regulations designed to safeguard people actually inhibit progress in saving & safeguarding peoples lives.

    There are many areas of society where laws are morally & downright wrong because they are not flexible to meet variables & prevent injustices

  • rate this

    Comment number 10.

    @ 6.

    Read the article before commenting, it states "What patients want is sometimes to try medicines that may not be clinically proven to be effective but are clinically safe."

    They are not going to fast track something that will clearly kill people.

  • rate this

    Comment number 9.

    Human guinea pigs. Behind all the nice sounding words it is merely a cheap way of testing new drugs on humans; with people who are very ill.
    Who will be in charge - Dr Mengele?

  • rate this

    Comment number 8.

    This is the most heartening piece of news I have heard in a long time. It seems like that, for once, common sense is prevailing. I hope this scheme goes on to relieve pain and save lives!

  • rate this

    Comment number 7.

    Now if this idea is paired with the 'stratified' treatment being pioneered in Glasgow, (http://www.bbc.co.uk/news/uk-scotland-26558971) perhaps the efficacy of the new drugs will be discovered faster and benefit more patients more quickly.

  • rate this

    Comment number 6.

    It is very difficult question indeed. If it will be an a success, then a lot of people will be happy. But what if the 'speed up' drugs will happens to be not safe? All those trial and procedures are for the reason...

  • rate this

    Comment number 5.

    This should have been brought in an age ago.I am on a drug trial as in my 20's I was struck (can happen to any1 at ANYTIME) with an untreatable condition that caused swelling in my brain. WITHOUT being on a drug trial I wouldn't be typing this message, know what BBC is or who I am. There will be cynics who spout the dangers but it should be our risk 2 take & might cut an even faster path 4 others.


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