National Health Service Commissioning Board and Clinical Commissioning Groups (Responsibilities and Standing Rules) (Amendment) (No. 2) Regulations 2014

Baroness Brinton Excerpts

Tuesday 6th May 2014 Lords Chamber

The way in which medicines for rare diseases are commissioned and provided in this country is important. Although it is fairly early days, there are some discouraging signs, and I am grateful for the opportunity to raise my concerns with the Minister this afternoon. I should have declared my interests as the chair of a foundation trust, a consultant and trainer with Cumberlege Connections, and president of GS1. I beg to move.

[Baroness Brinton (LD)]

My Lords, the regulations, which address an important Cinderella service in the NHS that not many know about, are extremely important. I thank the Specialised Healthcare Alliance for its briefings, which have provided an extremely helpful background. I know that the noble Lord, Lord Hunt, has provided a couple of figures, but it might be worth pointing out that there is a clear distinction between “rare” and “very rare” diseases. It sounds silly to say, but those with very rare diseases would welcome having, for the one in 17 or fewer of the population who will be affected by a “rare” disease—that is the ratio—a reasonable coverage throughout the country, although it may be in specialised areas. However, “very rare” diseases may affect fewer than 500 patients in the UK and, in the case of one or two diseases, may affect perhaps only one or two patients.

With the implementation of the new clinical commissioning groups it has obviously been important to reassess how treatments for rare diseases are commissioned. I do not have to declare an interest because I do not have a rare disease but, as someone who has to access rare biological medicines, I know that my local CCG and many others are struggling with the whole issue of prescribing expensive drugs. However, they cost pocket money when compared with the cost of medicines and treatments that we are looking for as regards rare and very rare diseases. The key is that one-size commissioning will absolutely not fit all, even within the definition of one rare disease, because it would be very unusual to find one clear clinical route. These days, the personalisation of drugs based on genes identification—80% of rare diseases have a genetic component—and the range of co-morbidities with most of these diseases mean that we absolutely must have clear and individualised treatment routes, with a much higher level of understanding of the diseases.

That is why I welcome the prescribed specialised services advisory group, which, for brevity, I shall refer to from now on as the PSSAG—I do not know what the Department of Health is calling it but we may as well add something else into the alphabet soup. It is important that the Secretary of State consults the PSSAG. The regret Motion was laid prior to the publication last week of the recommendation from that special advisory group. Inevitably, the timing is unfortunate, but life is like that. However, I believe that the publication of this report significantly reduces the concerns in the regret Motion about fragmentation and transparency. For example, the sections on membership and process on pages 7 and 8 of the report, some of which the noble Lord, Lord Hunt, referred to, make it very clear that both lay members and representatives from the royal colleges, as well as members with financial and technical expertise who can offer assistance, will come together to look at things. The noble Lord referred to what has to be reviewed but the other elements are equally important. The PSSAG must also consider how activity can be identified to enable separate contracting, monitoring and payment, which I think addresses the noble Lord’s point about what happens when things are devolved to a regional level. It also has to address the running costs associated with separate and direct commissioning, as well as consider defining elements of service to be commissioned. Therefore, I think that I am more reassured than the noble Lord, Lord Hunt.

It will be very important to monitor implementation, partly because, certainly in the early days, it will be difficult to budget for it, in part because of what I said about the personalisation of treatment routes and medication but also because, as we and the commissioners become much more familiar with the changes taking place in the treatments, particularly the gene therapy treatments, we may find that the costs of associated treatments are significantly reduced quite early on if the biologics and other superdrugs, as well as stem cell drugs, begin to work. Therefore, I would be grateful if the Minister could reassure the House about monitoring the situation once the PSSAG gets fully into its stride and commissioning starts to take place.

I have one area of concern, which is perhaps where more than one of the specialist groups that are going to be looking at treatment routes are involved in commissioning a patient’s treatment. Often two, three or possibly even four specialist groups are likely to be involved. In cystic fibrosis there would obviously be a principal neurological one but blood and kidney specialist groups might be involved as well. My fear is that we may end up, as we have done in the NHS in the past, with the position where a patient in a hospital sees lots of different consultants but does not know who has overall responsibility for holding the ring. Is there an equivalent in this sector to make sure that one group has a specific responsibility, partly so that clinicians and therefore their patients are not passed from pillar to post among these different specialist advisory groups?

[Lord Walton of Detchant (CB)]

My Lords, I am grateful to the noble Lord, Lord Hunt, for raising this crucial matter. This issue of rare diseases is of increasing importance with the developments in knowledge and in new forms of treatment that are beginning to emerge. For many years in the NHS, doctors and scientists recognised that there were a good many rare diseases, but those received comparatively little attention save for supportive treatment because no effective drugs were available that were curative or that would at least alleviate significantly the effects of such diseases.

Of course, one has to recognise that many rare diseases still exist in medicine but every single disease, even if it is incurable, can have its effects modified to some extent by pharmacological, psychological and physical means. However, that was not particularly brought to public attention until the past few years, when developments in molecular biology—not least in genetics—highlighted by the rare diseases consortium and by the Genetic Alliance UK meant that in a very large number of rare diseases the causal gene was identified, isolated and localised in the genome. In addition, drugs have begun to emerge which will alleviate or overcome the effects of the genetic defect. For those patients who have a rare disease, affecting up to several thousand individuals, these are known as orphan drugs; for those affecting a few hundred or a few dozen patients, these are known as ultra-orphan drugs, which are very expensive. The number of patients likely to benefit is relatively small so the commercial viability of these remedies is at least a matter of grave concern.

While there are important things to take on board here—we were talking about R&D and AstraZeneca a little while ago—we must recognise the fact that Britain has an outstanding record in medical research and development. As I have often said, today’s discovery in basic medical science and in applied science brings tomorrow’s practical development in patient care. People with rare diseases can be helped by these remedies that are now coming on stream much more rapidly than has been the case in the past. The important thing is this: as the noble Lord, Lord Hunt, said, before the Health and Social Care Act was passed, we had the Advisory Group for National Specialised Services which commissioned services for some people with rare diseases. When the Act was passed, we—those who debated it extensively in this House—were reassured by the knowledge that there was an agreement that highly specialised services would be commissioned by NHS England. In recent debates with the noble Earl, he has been able to reassure us that that organisation has a rare disease advisory group, advising it on the management of these conditions. He was also able to reassure us about the early availability in this field, and in others, of unlicensed drugs in specific circumstances that may be effective in the management of many conditions in medicine—not just for cancer but for rare diseases, too. That is crucial.

However, as the noble Lord, Lord Hunt, said, we are concerned that the services are somewhat fractured in the sense that I have been unable to find out with any great clarity the terms of reference of the prescribed specialised services advisory group. It is not at all transparent at the moment. As the noble Lord said, nothing has been clearly published about its membership, its modus operandi and how it will function, or to what extent it has a relationship, if any, with NICE on the new procedures that NICE is introducing for the examination of orphan and ultra-orphan drugs. We need reassurance and more information on how this group works and, in the interests of public scrutiny, how it makes an effective contribution to health service decision-making. These are matters of great importance because rare diseases, though rare, are a major blight on people in the community. However, we cannot assess human suffering in purely numerical terms. The suffering resulting from many of these rare diseases is in many ways serious and exceptional and deserves very special attention.

For that reason I was very concerned, and I hope that I have misinterpreted the remarks of the director of NHS England, Mr Simon Stevens, in his report to the Commons Health Select Committee. He said that he thought there was a need for the scope of specialised services to be reviewed because he thought that they had been overextended. This is a phase in medicine in the UK when that would cause great concern to patients and their families and to many doctors who are concerned about the treatment of these diseases.