Stem Cell Transplant Patients

Debate between Liz Twist and Mark Tami

Monday 12th June 2023 Commons Chamber

[Mark Tami]

I thank my hon. Friend for that point. He is entirely right. As part of the transplant process, the immune system is effectively wiped out. That is a necessary part of the treatment, but clearly it leaves the patient fairly defenceless to infections. Once the donor stem cells are given, they will slowly build up a new immune system inside their body, but that takes time—it does not happen overnight—so those patients are often very vulnerable to infections. He made the point that if they put themselves into a dangerous situation because of financial pressure, the worst could happen to them—or at the very least, they could fall back into the medical setting that they were hoping to remove themselves from.

As part of the process, patients strictly isolate themselves in a hospital room for weeks—sometimes months—on end. Even after they leave, they are often weak and, as a result of their inability to work, their household income will obviously suffer. When patients are well enough to be sent home from hospital, there is still a long, gruelling and costly recovery ahead, whether they are the main wage earner or another member of the household, because obviously everyone else has to support them, whether in a caring role or otherwise.

[Liz Twist (Blaydon) (Lab)]

I thank my hon. Friend for giving an excellent explanation of the position and for succeeding in getting an Adjournment debate on the issue. Does he agree that people are hit three times over? They are hit while having the treatment, often hit by reduced income when they have finished their treatment and hit by extra costs from needing heating and food as well as other things, especially now we face such a cost of living crisis.

[Mark Tami]

Absolutely. My hon. Friend makes an important point. Patients must continue to take those extra measures for fear of getting an infection that could be life-threatening. As she said, they are advised to keep warm at all times, to sterilise drinking water and to wash their clothes more often. With the surge in electricity and gas bills, those extra costs are really hitting patients hard.

Another part of the transplant process is having what is known as a clean diet, which basically means that the patient needs to know exactly where their food comes from and how it was cooked, so they tend to cook it themselves, which in the main makes for a more expensive process. Ready meals are not really an option. If someone very kindly prepares something, they cannot take the risk because they cannot be 100% sure where it has come from. Clearly, the record food prices we are now seeing make the situation far worse. We hear on the news that people are going for cheaper options.

NICE Appraisals: Rare Diseases Treatments

Debate between Liz Twist and Mark Tami

Thursday 21st March 2019 Commons Chamber

[Liz Twist]

I certainly agree, and such things happen too often. We need a system that properly evaluates those drugs.

[Mark Tami (Alyn and Deeside) (Lab)]

I have certainly found that NICE sometimes says that the pool of people is not big enough to evaluate, but the clue is in the title: these are rare diseases. NICE cannot carry on doing that, particularly in cases where it is clear that the drug has a really positive effect.

[Liz Twist]

I agree with my right hon. Friend. Indeed, that is the burden of my speech.

As I was saying, I am not going to get into the fine detail of the process. It seemed to us in the all-party group that many conditions, as my hon. Friends have said, face the same problems. The all-party groups for muscular dystrophy and for cystic fibrosis are two that come to mind, but there are many other rare diseases, as we have heard, that do not have all-party groups but face exactly the same difficulties. Since this debate was announced, some other organisations have contacted me to ask me to make sure that we do not forget their concerns.

We found that there are a number of aspects of the NICE appraisal system that are problematic in assessing Kuvan and many other rare drugs. The existence of just two appraisal routes for treatments to be assessed by NICE results in the likelihood of two or more treatments being stuck in the middle by not meeting the restrictive criteria of the highly specialised technology route and therefore being assessed under a single technology appraisal route. Some of them are rare but not rare enough. As we have heard, the majority of treatments for rare diseases are likely to be assessed within the single technology appraisal, which is designed for non-rare treatments. This impacts on both the cost threshold and the approach to evidence, which are all designed for more common diseases.

On lifelong chronic conditions, NICE’s approach values the lifelong cost of treatments. It looks for near future benefits as well. That means it is difficult for chronic diseases such as PKU and treatments that produce lifetime and life-enhancing effects to get access to new treatments. NICE cost appraisals assume that patents do not expire. NICE will assume the existing price of the drug will stay the same. That is illogical as, particularly with older drugs such as Kuvan, the drug will soon go off-patent. This affects the benefits assessment. On non-health costs, NICE performs its calculations based on costs paid and saved by the NHS. That ignores the wider cost to society and individuals caused by diseases like PKU.

I do not seek to criticise NICE staff. They work within a system that we have given them, but it is clear from the many questions to Ministers, debates in this Chamber and in Westminster Hall, the creation of all-party groups, and correspondence with Ministers about individual cases that there is a very real issue here which must be addressed. That is why we are asking, in this motion, for NICE to review its processes to reflect the current issues we face.

Many drug companies have been in touch since this debate was granted to send me briefings. They have been keen to explain their side of the argument and to point out what they see as the problems in the NICE appraisal process for their drugs. There is some overlap with patient concerns, but I am here today to speak on behalf of the community of people with rare diseases, not on behalf of the drug companies. Let me be clear, the fact that this debate is about NICE appraisal processes does not excuse the pharmaceutical companies from their responsibilities. There is a balance to be struck between their need to recover the cost of the development of drugs and make a reasonable profit, and a huge responsibility on them to make their drugs affordable for our NHS.

In this debate, I have focused on PKU and Kuvan. With another drug treatment for PKU on the horizon, Pegvaliase, there is a real worry that even with a drug that may produce really life-changing results for a wider group of patients, those with PKU will again be left without the treatment they need, even when it exists. There are treatments for other rare diseases, too: Spinraza for spinal muscular atrophy and Orkambi for cystic fibrosis, which are not only life-improving but life-extending.

This is quite simple. There are drugs available that can drastically improve the lives of those affected by rare diseases. When I hear that NICE’s appraisal process is an obstacle to improving lives, I feel really angry. We are reducing the lives of children and adults to a cost-effectiveness analysis. We need to find a way forward to amend the appraisal system so that we do not let people fall through the cracks or fall behind. The drug companies must also do their bit to ensure that their drugs are affordable for the NHS, especially when early access via a managed access agreement is being discussed.

That is why today we are calling on NICE to review its appraisal processes and make the necessary changes to stop people falling through the cracks and make available these drugs, which can make such a difference to patients—to people such as my constituent Archie McGovern, whose mum Barbara set me on this path as a new MP.