NICE Appraisals: Rare Diseases Treatments

Part of Backbench Business - Services for People with Autismbackbench Business – in the House of Commons at 4:38 pm on 21st March 2019.

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Photo of Sharon Hodgson Sharon Hodgson Shadow Minister (Public Health) 4:38 pm, 21st March 2019

It is a real pleasure to take part in this very important debate. I start by thanking my hon. Friends the Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon). We are part of the north-east massive, so I am very pleased to be joining them in this debate today. I thank them for their very passionate and heartfelt contributions. I should also mention, as part of the north-east massive—it goes without saying—my hon. Friend Mrs Lewell-Buck. I thank all other hon. Members who spoke for their excellent contributions. They know who they are and I do not need to name them. They were all fantastic.

Throughout the debate we have heard of the heartbreak experienced by patients and their families when they are unable to access life-saving drugs on the NHS. We have heard of their determination to continue fighting to access those drugs, whether by writing to their MP or even by protesting in Parliament square. I was happy to join my hon. Friend Kerry McCarthy there just two weeks ago for the Cystic Fibrosis Trust rally, where people were calling, “Orkambi now!”—they were so loud that we could hear them over the crowds chanting, “No Brexit!” or whatever the shout was at the time.

We must hear patients’ voices in this debate, as it is they and their families who are affected the most by the appraisal process, which is not fit for purpose. The Minister has heard about the real-life experiences of patients throughout this debate, and I am sure that he will continue to listen to them afterwards. I know that he is also in regular communication with patients. In my role as shadow Public Health Minister, I regularly meet patient groups and campaigners, so I know just how important access to these life-saving drugs is to them.

As a constituency MP, I recently met young Riley and his mum Michelle. Riley has phenylketonuria—PKU—and needs Kuvan. He is now 11 and at secondary school. He just wants to blend in with his mates and to be able to go on those first excursions out to the Metrocentre, and perhaps to get something unhealthy to eat from a takeaway, but obviously he cannot do any of that. I asked him about his life and how he felt not having access to Kuvan. He said that it was not fair and that it made him mad. Well, I agree with Riley.

It can take years to get the right diagnosis for a rare disease, so once patients get the diagnosis they are excited and feel that there has been a breakthrough, because they think that they will finally get the treatment they need and deserve. Instead, as we have heard today, they are back at the beginning of the fight, because the life-saving drugs that do exist are not available to them on the NHS. It is one hurdle after another for patients with rare diseases. That is why the Opposition strongly believe that patients should have fast access to the most effective new drugs and treatments. I am therefore pleased to support the motion.

As we have heard, a rare disease is generally considered to be one that affects fewer than five people in 10,000. According to the 2013 UK strategy for rare diseases, it is estimated that in the UK more than 3 million people will suffer from a rare disease at some point in their life. All those patients must have access to the drugs and treatments that they need. However, they are being failed by the NICE appraisal process, which is just not fit for purpose when it comes to assessing the suitability of drugs and treatments for rare diseases.

Patients with rare diseases are squeezed in the middle of two appraisal routes: the highly specialised technology evaluation programme and the single technology appraisal route. The HST evaluation programme is selected for most non-cancer rare disease medicines and is designed for evaluating medicines of that nature, with small patient populations. However, the HST evaluation programme currently lacks the capacity or capability to effectively appraise all new licensed orphan medicines. Since the HST evaluation programme was established in 2013, it has published guidance on eight medicines, which is much fewer than the 45 orphan medicines for non-cancer indications that have been licensed in the same period.

The STA route is designed to appraise treatments for more common conditions and those with existing treatments. This route is poorly suited to considering rare disease medicines, which tend to have small patient populations, a limited evidence base and benefits beyond direct health benefits—something the appraisal process just does not take into account. Some rare diseases are not rare enough for the STA route, and only a handful of medicines are being approved by the HST route. Yes, it is complicated, but it is clear that neither route is working for patients with rare diseases, so patients are missing out on crucial medicines.

Kuvan was licensed in 2008 to treat PKU patients, but it is still not available to patients in England. Orkambi was appraised by NICE in 2016 through STA, but was recommended for use. Three years later, as we have heard, people with cystic fibrosis still have no access to it. That has caused physical and psychological harm to patients and their families. Every day without the drugs that they need makes their condition worse. We must have an appraisal process that captures rare diseases effectively.

Medicines to treat rare diseases are often found to be cost-ineffective, which is why they are not approved for routine commissioning. However, establishing value for money is not straightforward, especially when population groups are small. It does not sit comfortably with me—or, I am sure, with any of us—that cost-effectiveness is prioritised above clinical need, or, as we have heard, the lives of children. Manufacturers want to make a reasonable return on their investment, although some of the figures are huge, but I do not think that that should be a priority. Manufacturers must not hold NICE or NHS England to ransom for their own financial gain.