Drugs: Ultra-rare Diseases

Philippa Whitford Excerpts

Tuesday 16th June 2015 Westminster Hall

[Dr Philippa Whitford (Central Ayrshire) (SNP)]

I was a breast cancer surgeon for more than 30 years, and I often experienced the situation that has been described in the debate with my patients and new cancer drugs. We were turned down for Kadcyla earlier this year. With cancer, it is often end-of-life research that later translates to early treatment research. People read things in the paper and say, “Oh, £90,000 for six months of life—that doesn’t make sense.” Inevitably, however, those drugs move forward. We have a different system in Scotland, and while listening to the debate I have been struck by how what is required is a system that is open and can be approached, and which looks from all angles.

In Scotland, the Scottish Medicines Consortium considers drugs as NICE does, and it considers worldwide evidence. It will work up a drug in detail. The balance for us seems to be slightly more on effectiveness than cost, although obviously cost is part of it. Our impression is that, for NICE, cost would sometimes be a bigger component. They are both looking at cost-effectiveness, and we all know there is not an infinite pot of money.

What has changed in our system over the last year is that we have combined our cancer drugs fund with our rare diseases drug fund and simply called it the new drugs fund. The amount in the fund has been quadrupled from £20 million to £80 million, which means that in any year it is a little more flexible in responding to demand, whether that is for drugs for rare diseases or for a new cancer drug. NICE only assesses three drugs a year, so rare drugs are never going to get that work-up. They need a separate system. In Scotland, we have pathways to follow for rare diseases and ultra-diseases.

The biggest change in Scotland in the last year is patient and clinician evaluation. If the evidence for a drug is so strong that it will go through on the nod and there is not an issue, that is fine and PACE is not engendered; but if things look more finely balanced or the drug will not go through, patient groups or drug companies can request a PACE assessment. That will involve expert clinicians, patients and patient groups, and allows people to get slightly outside the numbers and talk about life change, quality of life and money saved in respect of other aspects of the NHS—things that perhaps do not appear in a research paper. That is what is required: a system.

At the end of the day, the system will not produce a favourable result for every single person and every single new drug in the world, but it has to be fair. We cannot have things not being looked at properly, or individual requests being used as the main way of accessing a drug. The system I am talking about is meant to be a transition—supporting young people, for example, who have been on a trial, by giving them access on a compassionate basis, while we get through the paperwork prior to a drug being accepted. It cannot be left as the main method.

[Jonathan Edwards (Carmarthen East and Dinefwr) (PC)]

That system sounds excellent. I commend the Scottish Government on their work. That could be a great help to my constituent, Mr Trystan James, who suffers from tuberous sclerosis complex and is reliant on a clinical trial drug to deal with a life-threatening tumour. Of course, his drug prescription is therefore completely at the discretion of the drugs company and his family are going from one prescription to the next. That relates to what the hon. Member for Strangford (Jim Shannon) said about emotional pressure on families. I commend the Scottish Government on their work.

[Dr Whitford]

The important thing to remember is that if this is all done by individual requests, the NHS does not go to the companies. We need to realise that companies have often made investment over decades and that nine out of 10 drugs they research will go nowhere, but it is important to have a wider debate with companies to get the best price. Hon. Members mentioned that some companies are willing and able to reduce the price to get a drug in.

Drugs are licensed. We must not mix up licensing with funding. Licensing is about asking, “Is this drug safe and proven at a basic level?”, not anyone coming in and saying, “Rare plant juice will cure everything.” These are licensed drugs that we could prescribe—a doctor has the right to prescribe them—but the NHS has to make the decision about whether to fund them; those are funding decisions, not licensing decisions.

It is important that families know what the pathway is and how they move on when their clinician takes a case forward. It is important that they know they can respect decisions and how to lobby at the next step, and that they feel their voice is being listened to. We feel that PACE has, over more than a year, allowed us to do that. Clinicians in Scotland got frustrated about decisions going through without us informing that decision.

There could be a system that sits on the side of NICE, or a sub-group. One of NICE’s three assessments will never be given over to a drug intended for 88 patients when it is also assessing drugs that might be taken by 500,000 people. Rare diseases would always fall behind, and that is why those must have their own system and why the patient voice must be heard in these ways. Obviously, things have changed with the Health and Social Care Act 2012, but I commend such a structure to the Minister.

[Mark Durkan (Foyle) (SDLP)]

The hon. Lady is making a thoughtful contribution. Does she agree that it would be worth all Administrations in these islands, who together form the British-Irish Council, collaborating on these issues, particularly borrowing from the good example being developed in Scotland, and seeing whether there can be common achievement and common advances, and perhaps even creating some common funding stream, as well as the discrete funds that she has talked about?

[Dr Whitford]

Obviously, devolution gave us the power to do things differently, but I do not think that we should re-invent the wheel. Often, we will accept work done by NICE or re-evaluate it quickly, to see whether things should be applied differently, but we do not just go back to the beginning. However, I am sure that ideas can be shared in both directions.

It seems that certain drugs were left as orphans when the system changed. We know that patients with the brain tumour form of tuberous sclerosis, which the hon. Member for Carmarthen East and Dinefwr (Jonathan Edwards) mentioned, can access the drug through the cancer drugs fund, but if they have a kidney tumour and are treated by urologists they are not part of that system and simply will not be aware of it. Such random unfairness exists.

[Jim Shannon]

There is a forum and association, driven by the Health Minister, that discusses matters together with the three regions. A UK-wide strategy is already in place. The process is allowing that to happen already. However, it is delayed and has not happened yet; that is why we are concerned.

[Dr Whitford]

I think it is a matter of what ideas go on the table and what is being discussed in the meetings. Good ideas are going ahead. I commend the idea of including patients and clinicians in evaluations, because the numerical data from trials will often be small due to the nature of the diseases in question, and we will have to look wider. The problem for children is that if these drugs are to prevent deformity, they have to be got in early. People with Morquio already have the changes. We do not know yet how much change could be prevented, or how much saving there could be on a person’s disability in the long term if metastatic breast cancer treatments, which eventually become adjuvant treatments, are given earlier.

I commend the system I have talked about. I know it is difficult and challenging, but it is clearly fair, with an interim period for compassionate reasons, and people know where their voice should be heard.