'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
    0

    Comment number 324.

    323. magictricks
    Big companies often ignore real treatments because of patents, patents for medical supplies should be banned."

    If there weren't medical patents then no one would spend millions on researching new treatments. If they did, then the next day someone would just copy it and sell it a lot cheaper because they didn't have the research costs.

  • rate this
    -1

    Comment number 323.

    Big companies often ignore real treatments because of patents, patents for medical supplies should be banned.

  • rate this
    0

    Comment number 322.

    Or is this an acceptance that most of the clinical trials are flawed anyway- ie the BMJ recently declared that there was no evidence for a third of drugs commonly used, and that adverse events were hidden away from reports.. so might as well scrap them?

  • rate this
    0

    Comment number 321.

    I'm confused,only last week NICE did a u_turn on providing me with a drug that would give me several extra months of life. They have denied it because of cost,I've no idea where l stand now.

  • rate this
    0

    Comment number 320.

    Will this apply to Sativex?

  • rate this
    0

    Comment number 319.

    Shame on you all; Liam's mother may be reading this with hope of positive feedback and so many unconstructive comments.

    Very disappointing to read.

    The drugs being fast tracked have been tested but simply not licenced, due to that its simply cutting out the paperwork and bureaucracy.

    As long as its done with the upmost safety and concern of the patient then its a good thing

  • rate this
    0

    Comment number 318.

    When you have nothing else going for you anything will do but it's rather selfdeceptive to imagine cheating death for very long it makes acceptance of fatal outcome all the more difficult. Still hope is what all such patients have left hope that they will be in the statistical group that receive some benefit from an experimental drug with no guaranteed efficacy. A roll of the dice.

  • rate this
    0

    Comment number 317.

    @316 Clarice
    get to bed
    ++

    AwWwW, I like it when we argue in public then you go to bed and realise you need me there. Won't be long dear.

  • rate this
    -2

    Comment number 316.

    315 Hamish
    To whom? You or the government or the majority of people?

    More like the majority of people

    get to bed

  • rate this
    +1

    Comment number 315.

    314 clarice
    Well yes, that's fairly obvious
    ++
    To whom? You or the government or the majority of people?

  • rate this
    -2

    Comment number 314.

    313 Hamish

    perhaps people should be responsible for the choices they make, manufacturer/supplier should inform you of possible risks & user should bear consequences, extended to drugs currently illegal. If a drunk/stoned person commits a crime punish the crime but don't make intoxication a crime in itself.

    Well yes, that's fairly obvious

  • rate this
    +1

    Comment number 313.

    312.Clarice
    the drugs available should be used for people who need them, if requested by them
    ++

    perhaps people should be responsible for the choices they make, manufacturer/supplier should inform you of possible risks & user should bear consequences, extended to drugs currently illegal. If a drunk/stoned person commits a crime punish the crime but don't make intoxication a crime in itself.

  • rate this
    +1

    Comment number 312.

    This has kept me from my beloved bbc4 music... but the drugs available should be used for people who need them now, according to need, not cost, and if requested by them

  • rate this
    +2

    Comment number 311.

    Dying is the last thing I´ll do.

  • rate this
    +1

    Comment number 310.

    309.speed_of_dark
    1 Hour ago
    @299

    The fact that the word "nearly" exists suggests it has meaning to the human mind. I've certainly been nearly run over by vehicles a few times, it didn't actually happen but it was close enough to be seriously scary and make me take stock. Surely nearly dying is much the same?
    +++

    You've nearly got a point.

  • rate this
    +1

    Comment number 309.

    @299

    The fact that the word "nearly" exists suggests it has meaning to the human mind. I've certainly been nearly run over by vehicles a few times, it didn't actually happen but it was close enough to be seriously scary and make me take stock. Surely nearly dying is much the same? Death is close enough that there is relief and often thoughtfulness at how close it was or could have been.

  • rate this
    +1

    Comment number 308.

    NHS Hospital STILL don't get the basics right!

    Records are Write Once Read NEVER.

    Hearing Aid users are multiply failed every other day hospitals makes each one deaf by mishandling their aids. The solution is trivial - but the NHS can't be ar*ed to fix the problem. Notice on wall to remind nurses to make sure they are in and ON in the morning and whenever a patient leaves the ward. It matters!

  • rate this
    +1

    Comment number 307.

    @305. Further misinformed as there is a pan EU price for all medicines.

    But if you don't like the pharmaceutical industry, simply never ever take one of their medicines no matter how ill you become. Uphold your principles or quieten down...

  • rate this
    0

    Comment number 306.

    @305. So badly informed. I know of a number companies that will not launch cancer drugs in the UK because NICE will not approve them. The knock on effect throughout the EU and maybe the world is too great for them to risk. Other countries embrace these new drugs and their populations benefit. Easy to scape goat an industry. But without their research, where would our medicines come from?

  • rate this
    +2

    Comment number 305.

    Who would trust a pharmaceutical industry ?
    People here are denied treatments provided abroad due to inflated uk prices.
    Surely a bigger issue is Tory Hunt lining up our NHS for his sociophobe contemporaries. Since when was it legal to kill/weaken/starve the population ?

 

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