Immunotherapy works. Why isn’t it routinely available on the NHS?

When Evelyn O’Flynn was diagnosed with stage four non-small cell lung cancer, after seeing the doctor about neck pain, she was told she had between six and nine months to live.

Instead of the standard chemotherapy treatment, she was offered the chance to enrol in a clinical trial for a new immunotherapy treatment called pembrolizumab.

The tumour responded to the therapy and continued to shrink until it was 80 per cent smaller than its original size. ‘I felt so well it was unbelievable,’ she said.

‘What a chance another bit of life gives you, I’m talking about two years and four months. I was given six to nine months to live. I feel so well and I can do everything.’

Earlier this year she had to stop the treatment while her doctors investigated a lump on her neck. During the break the original tumour did not grown and this has been attributed to the residual effects of pembrolizumab.

She is hoping to restart the treatment shortly and said that she is grateful for the extra chance it has given her to spend time with her family.

Lung cancer is the most common cause of cancer death in the UK and only ten in 100 people diagnosed survive their disease for five years or more. Those with metastatic, or stage IV non-small cell lung cancer (NSCLC) have a five year survival rate of about one in 100.

People with NSCLC can suffer a number of distressing symptoms including: difficulty breathing, chest pain and fatigue. There are limited treatment options on the NHS to treat those with NSCLC and these, like the drug docetaxel, can cause severe side effects.

This was an important consideration for Philip Hayter, from Eltham, who was diagnosed with NSCLC after a check for a heart murmur. He also chose to take part in the clinical trial: ‘The only reservations I had was in taking chemo, because you hear all these horror stories of your hair falling out, god knows what else happening and being laid up in bed. This drug they put me on, I don’t feel ill.’

‘It only helps a certain type of cancer; anybody who has that should take it if given the opportunity. I think it’s marvellous, I would never stop taking it unless a doctor told me to.’

Mr Hayter and Ms O’Flynn said they experienced few side effects from the treatment although in clinical trials some patients did report fatigue, decreased appetite, nausea, rash and itching. This is the first time NICE has recommended an immunotherapy treatment for people with this type of lung cancer to be included in the Cancer Drugs Fund, which was set up to allow NHS patients in England access to cancer drugs which are not routinely available on the NHS.

Pembrolizumab works by helping the body’s own immune system to recognise and destroy cancer cells. It does this by blocking a protein called ‘programmed death 1’ protein (PD‑1) on the surface of certain immune cells called T-cells. This triggers the T-cells to find and kill cancer cells. In their final appraisal determination NICE have only recommended that the treatment be used for those cancers that have tested PD-1 positive and do not have certain specific mutations.

NICE has recommended that patients with a PD-1 positive NSCL, whose cancer has spread to other parts of the body, can be given the drug immediately without trying routine chemotherapy first, as previously recommended in January.

The average number of months a person with NSCLC can gain with pembrolizumab, as estimated by the pharmaceutical company’s economic model, was 29 months compared with 14.6 months for standard treatment.

So, why is pembrolizumab still not been recommended as routine NHS treatment for newly diagnosed cases of NSCLC?

In their recommendation NICE have written that there is still considerable uncertainty around the validity of the survival projections. In short, that it is still too early to be sure that estimated survival gain for patients was really better than the standard treatment currently used by the NHS.

Pembrolizumab costs over one thousand pounds per 50 mg and the recommended treatment is 200 mg every 3 weeks by intravenous infusion.

NICE state that their recommendation is made only the basis that the manufacturer will provide discount to the list price of pembrolizumab, although the extent of the discount has not been published.

Further survival data from the ongoing clinical trial and Public Health England is expected to help determine whether this is a cost-effective treatment for the NHS to fund.

Gary Middleton, Professor of Medical Oncology at the University of Birmingham said: ‘This is fantastic news for those people with non-small cell lung cancer that has spread. For the quarter of patients which are suitable for first-line pembrolizumab, it significantly improves survival without significantly impacting the quality of life of patients.’

‘The study that NICE’s decision is based on represents a real milestone in the management of the disease, and NICE’s decision means that people are now able to access pembrolizumab at the point that is right for them, thus leading to better patient outcomes.’