Low Dose Naltrexone

Nia Griffith Excerpts

Thursday 8th December 2011 Commons Chamber

[Nia Griffith (Llanelli) (Lab)]

First, I want to make it clear that this debate is not about fighting for a very new and expensive drug. Campaigns about drugs are often brought to the attention of Parliament because a patient is fighting to be allowed to have a new and expensive treatment on the NHS. Some of these new drugs are not just expensive because they are new; because of the complex processes required to make them, they will, in fact, often continue to be expensive to produce. Such situations raise dilemmas for decision makers about how access to such drugs can be funded.

This debate is about a very different problem: making an existing drug that is modestly priced available for the treatment of a wider range of conditions. Clinical trials are needed to get full approval for the drug under discussion, but I ask the Minister to consider whether there is any possible way in which it could be made more widely available.

Sometimes patients are faced with unacceptable options for treatment and find themselves researching possible new treatments. That is usually a road that leads to disappointment, but occasionally something useful is stumbled upon, such as low dose naltrexone, or LDN. The problem is that it is what is called an “orphan drug”, which means its patent has expired, so if someone does research on it, a generic manufacturer can subsequently steal the business.

I understand that naltrexone is proved safe in its normal mode of use, and now has a clinical history of 11 years of use in the UK with no problems reported and only minor side effects. LDN is also very low cost, and can be used to treat many conditions that are both chronic and often very expensive to treat with more conventional remedies. Sometimes those more conventional remedies have severe side effects, which then have to be treated with more expensive drugs.

The purpose of this debate is to ask how a drug such as LDN could be made available to patients who ask for it. The most desirable route would be via clinical trials leading to marketing authorisation and then official acceptance from the National Institute for Health and Clinical Excellence and the NHS. A much cheaper and more immediately practical route is to recognise that LDN is a safe choice for patients without many of the risks of drugs currently in use. Doctors could therefore be given official advice not to deny it to patients who want it or wish to acquire it from pharmacists who make it as a “special” at a fair price. There could also be a mechanism for protecting doctors and allowing patients choice. At present, doctors are in a difficult position. If they prescribe anything that is not on an official list, they leave themselves open to criticism, as well as to being sued and possibly losing their right to practise.

The third route is to get it listed as an over-the-counter drug, such as aspirin or paracetamol. I understand that it is considered safer than paracetamol which is sold over the counter, so this might be a reasonable option that would make prescription very easy.

[Annette Brooke (Mid Dorset and North Poole) (LD)]

I congratulate the hon. Member for Llanelli (Nia Griffith) on securing this debate. I am sure that, like me, she has a number of constituents who are benefiting from this drug—I refer particularly to members of the Purbeck and Wareham multiple sclerosis group. It is so frustrating that they cannot get hold of something, through the NHS or in some form that is easily accessible, given that it is definitely making a difference to their lives. I am sure that she would agree that there is a fear that they might not be able to get it one day. What do they do then?

[Nia Griffith]

The hon. Lady is quite right. These people fear that if they change from one GP to another, or if people are less sympathetic towards this drug—obviously this is something that people have to be convinced about—they may well not be able to get hold of it. Unless there is some form of authorisation and some form of making it an official drug that is more widely available, they do run the risk that she describes.

The problem is that many patients feel let down because what they see as perfectly good therapies, which are cheap but out of patent, are being withheld when patients want them. When doctors cannot refuse a patient’s choice, they can still deny it to them, because clearly the issue of their professional conduct is involved. If the NHS recommends another treatment, quite possibly one that costs thousands and has drug company support, it is often difficult to get the research done to prove the validity of a drug such as LDN.

Some 5,000 people in the UK with multiple sclerosis use LDN, and a few thousand more use it for other conditions, such as Crohn’s disease, cancers, fibromyalgia, autism and so on. Their GPs usually prefer not to sign NHS prescriptions for LDN—indeed, they may refuse to do so—but there are instances of GPs then charging patients for private prescriptions, explaining that they are worried that NHS prescribing guidelines would prohibit them from prescribing LDN and so they would end up in serious trouble if they did so. One GP in Glasgow who prescribes a lot of LDN was reported to the General Medical Council for doing so recently. My constituent Andrew Barnett has told me that his GP has said that his lawyers advised against it, so he writes my constituent a private prescription and charges him £8.50 a quarter for doing so. My constituent then also has to pay for the LDN itself, at a cost of £17.50 a month.

Some 100,000 people in the UK have MS, only 12% of whom use drugs offered by the NHS. Some of the drugs available are risky and very expensive, and there are questions about how effective they are. Yet some 5% of MS sufferers choose to use LDN, because they feel that it helps them and does not have the risks of some other treatments. LDN has proved to be safe in trials at very high doses, but it is unpromoted and hard to get. Because people are now making this choice, there must be a way to get this treatment legitimised on the NHS for patients who ask for it. However, we are told that without substantial trials that is not possible. It is very difficult to find a way to fund any such trials because the drug itself is already licensed and therefore drug companies would not be able to recoup the cost of funding the research by marketing the drug; there would be no money in it for them.

We live in an age where information, including real scientific papers and trials reports, is easily available. Patients who are really determined to make the most of their lives, despite terrible illnesses, and who have the wherewithal to look into the research do seek out information and solutions. We have been using LDN in the UK for some 11 years. Naltrexone has been trialled at high doses to treat heroin addiction and is known to be safe, so the only real thing missing is the marketing authorisation from the Medicines and Healthcare products Regulatory Agency—MHRA—for a formulation of low-dose naltrexone, perhaps in liquid or capsule form. A trial would add proof—or not—of its efficacy.

The cost of a trial is probably considerably lower than the cost of a high-tech drug, because the drug itself is so cheap. Estimates suggest that a single trial can be done with some £7 million, but that is just an estimate based on £1,000 per patient a year for the monitoring specialists, plus up to £3,000 a year per patient for LDN. There would be the costs of recruiting 500 to 800 patients and then something to cover the analysis of the results.

There are no systems available, however, for patients to translate their choice of therapy into a legitimate request, even when they vote with their feet in large numbers. Doctors do not see any danger in the choice except that they worry they might be denying people a more effective option, but, as patients have pointed out, if they need what is perceived to be a more effective option, they can take it alongside LDN anyhow.

My constituent Andrew Barnett, who is highly intelligent, scientifically minded and analytical, has made the assessment that LDN seems to have stopped his disease developing further. The constituents of the hon. Member for Mid Dorset and North Poole (Annette Brooke) have confirmed the same belief. My constituent regrets very much that he did not start using it earlier. If he had done so, perhaps some five years ago, he feels that he would still be working and contributing actively to the economy.

Doctors have to be able to say no to treatment choices for the sake of the patient, but when they have no reasons to deny an option, as is the case with LDN, that should not happen. We should try to ensure that we enable them to prescribe LDN. Doctors all tell us that without the trial, they do not have the confidence to prescribe, so patients face the potential loss of supply of prescriptions if they change doctor, quite apart from the cost of private prescriptions, which is hurtful for many patients who have been reduced to living on income support by sickness or disability.

So, the substance of the debate is about patient access to therapies that cannot get the trials they need for reasons such as not being patentable or the lack of profit in the therapy, especially when the therapy is known to be safe. LDN seems to be the most prominent, in that there are no sensible arguments to deny it to patients who want it and those patients report great satisfaction with it in most cases. It is frankly a disgrace that such options are denied to informed patients who ask for them on the NHS. It would save the NHS so much money to allow this. LDN can be supplied for as little as £17.50 a month, compared with some other expensive drugs, such as £60,000 for Copaxone, £15,000 for Avonex or £90,000 a year for another drug that was recently in the news.

The question is how we can get the trials and get people interested. Academics often rely on backing from pharmaceutical companies to put together their plans and proposals for a research project. It is very difficult to find academics who want to spend that time and energy if they do not know that they will get the backing. They could put in a lot of work without getting any funding for any proposal.

We must face up to the problem of what systems we have in place to provide licensing for drugs such as LDN. One doctor I have talked to, Dr Lawrence from Swansea, tells me that LDN not only seems to have acceptable uses for people with multiple sclerosis but, he feels, has enormous potential for the treatment of cancer. That would be a very worthwhile investigation, considering that we spend so much on looking for answers and on treating the various cancer diseases. Sanctioning the use of LDN would also allow doctors to collect clinical data that could be used to monitor and help prove the effectiveness of the drug.

Naltrexone is already an approved drug at higher doses and research and clinical trials have already shown its effectiveness at low doses to treat auto-immune diseases. Patients have difficulty in getting their GPs to write prescriptions and have to get the treatment privately, but it would be preferable for patients to work with their GPs so that their GPs can monitor them. GPs have had to seek legal advice to find out whether they should be prescribing it and lawyers have advised against it, putting them in a very difficult position.

Among the supporters of LDN are GPs, neurologists and oncologists who have seen patients’ diseases not get worse and there are cases where such specialists have supported its use for the patient and stated that in a written letter to the GP. Even then, the GP has felt unable to prescribe the drug for fear of being considered unprofessional.

Campaigners have worked with local health boards and PCTs to try to determine what knowledge there is about LDN and whether GP practices have heard of it or are using it. The responses came back negative. What can we do so that LDN is offered in addition to other drugs currently prescribed on the NHS? This is about patient choice. It would be nice to be able to make LDN available to patients on a much wider basis, but it is important that patients are monitored by their GPs when using any medication, so we do not simply want a free-for-all. We want the proper medical trials, proof and backing that is needed to show whether it is an effective drug, which will enable it to be made more widely available, but there is a real difficulty, despite the fact that the orphan drug could be a cheap option for the NHS, in funding the sort of research needed to trial it. I return to my initial request and ask the Minister whether there is any way he can make LDN more widely and easily available to patients.

--- Later in debate ---

[Mr Burns]

I am grateful to the hon. Lady for raising that point on behalf of her constituents. The short answer is that it is simply because there have been no clinical trials to assess the drug in its low-dosage levels, and so the conditions of the NHS, under the ways in which we operate in the provision of drugs for patients, have not been fulfilled at this stage. If she will wait for a minute or two, I will get to the nub of the point made by the hon. Member for Llanelli about how we could move forward to seek to address that situation. I hope that the hon. Member for Mid Dorset and North Poole (Annette Brooke) will find the way forward helpful and positive.

The hon. Member for Llanelli will no doubt appreciate that it is in everyone’s interest to see a booming medical research industry in the UK, because that leads to real improvements in the lives of patients, their families and carers, and we are determined to support it. We demonstrated our commitment to health research by increasing spending in real terms up until 2015. In August, my right hon. Friends the Prime Minister and the Secretary of State for Health announced a record £800 million, five-year investment in a series of biomedical research centres and units, which will translate fundamental biomedical research into clinical research that benefits patients and the NHS.

The coalition Government are committed to the promotion and conduct of research as a core function of the health service. The Health and Social Care Bill, which is now passing through another place, will turn this into reality by placing appropriate powers and duties on my right hon. Friend the Secretary of State for Health, NHS organisations, Monitor, and local authorities. We will make sure that the systems and processes for commissioning by the NHS Commissioning Board and by clinical commissioning groups promote, support and fund clinical research. The Government will consult on amending the NHS constitution in order to support patients to have access to novel treatments and to be part of the development of wider patient benefits, so that there is a default assumption, with an ability to opt out; that data collected as part of NHS care can be used for approved research, with appropriate protection for patient confidentiality; and that patients are content to be approached about research studies for which they may be eligible to enable them to decide whether they want a discussion about consenting to be involved in a research study.

The clinical practice research datalink will be introduced by the MHRA in partnership with the National Institute for Health Research, building on the NIHR’s research capability programme. This £60 million investment will offer data services, including providing access to data for researchers, data matching and linkage services, and data validation, to support the clinical trial and observational study work of the life sciences research community.

The NIHR will launch an updated UK clinical trials gateway in spring 2012. That website will enable patients and the public to access information about clinical trials and will be a development of the test site launched in March 2011. To increase the number of patients who can benefit from being involved in trials via the gateway, the NIHR has also developed a free smartphone app, which is available for iPhone users and will shortly be available for Android users. It provides a practical and innovative way for patients to access information about clinical trials.

I will now turn to the question of clinical trials that the hon. Member for Llanelli raised and that the hon. Member for Mid Dorset and North Poole raised, by default, in her intervention. I think that this explanation may provide the hon. Member for Llanelli with the basis for making progress in her quest. Clinical trials are a fundamental part of the drug development process, as she accepts. Trials and health research more generally are funded by a range of groups in the UK, in particular by the NIHR, the Medical Research Council, medical research charities and industry. The NIHR welcomes high-quality funding applications for research into any aspect of human health, including the use of LDN. Such applications are subject to peer review and are judged in open competition, with awards being made on the basis of the scientific quality of the proposals. As she has suggested, a new clinical trial will be required to support a licence for the use of LDN.

The MHRA regulates clinical trials on medicines when they are carried out in the UK. That includes granting approval to conduct a clinical trial and ensuring, through inspection, that the highest possible standards are maintained. However, the MHRA does not initiate clinical trials. A clinical trial needs a sponsor. Sponsors have usually come from industry, the NHS or academia. The hon. Lady is seeking Government funding for a clinical trial to prove the efficacy and safety of LDN. I can tell her that funding is available and that university-based researchers can apply for it.

The efficacy and mechanism evaluation programme is funded by the Medical Research Council and managed by the NIHR. It funds evaluation of the clinical efficacy of treatments. If evidence from such evaluations is promising, larger-scale trials can follow. That is one of the purposes for which the NIHR funds the health technology assessment programme. That programme produces evidence on the effectiveness, cost and broader impact of treatments and other types of health care intervention. In the case of LDN, as with all other novel treatments, I cannot prejudge how successful that pathway of research might be, but I can tell the hon. Lady that a pathway does exist, as I have described.

In addition, the hon. Lady expressed concern about whether the systems in place make provision for patients to say what research they would like to happen. I can assure her that patients can make a suggestion for the efficacy and mechanism evaluation programme to consider. Topics prioritised for funding may be advertised, inviting researchers to submit proposals for clinical trials in those topical areas.

I am grateful to the hon. Lady for raising this subject and giving me the opportunity to explain the background to a matter of considerable interest to many people, not least some of her constituents and those of the hon. Member for Mid Dorset and North Poole. I hope the last part of my speech in particular, in which I have explained an existing avenue that they and others interested in LDN may wish to pursue, will be helpful to them.

[Nia Griffith]

May I take this opportunity to thank the Minister for his very full and helpful reply?

[Mr Burns]

And may I, in the spirit of Christmas, thank the hon. Lady very much for the way in which she presented her case? It was quite clear from listening to her speech that she rightly felt very strongly about the issue on behalf of not only her constituents but people up and down the country who need LDN and who, at the moment, are having to go through the procedures that she described.

Question put and agreed to.