NICE Appraisals: Rare Diseases Treatments

Mary Glindon Excerpts

Thursday 21st March 2019 Commons Chamber

[Mary Glindon (North Tyneside) (Lab)]

It is an honour to follow the hon. Member for Reigate (Crispin Blunt).

I thank the Backbench Business Committee for agreeing to the debate. I also congratulate my hon. Friend the Member for Blaydon (Liz Twist), who is, as she said, my fellow member of the all-party parliamentary group for muscular dystrophy. She made a lucid and compelling case for the review of the NICE appraisal process.

I have had the honour of chairing the APPG for several years. It works closely with our secretariat, Muscular Dystrophy UK, and with patients and carers, on a number of issues that affect the lives of those with muscular dystrophy and other neuromuscular conditions. Perhaps one of the most important issues that we consider is the ability of patients to access treatments for their conditions.

For more than a year, access to the drug Spinraza, manufactured by the company Biogen, has been the focus of the APPG’s concerns about, and frustrations with, the NICE appraisal process. Spinraza is the first and only treatment for patients with spinal muscular atrophy, a rare inherited neuromuscular condition that leads to the gradual loss of the ability to walk, move, breathe and swallow. It currently affects about 2,000 adults and children in the UK. There are several types of SMA, with type 1 being the most severe, usually resulting in the death of infants before they reach their second birthday. However, clinical trials of Spinraza have had amazing results for many of the patients who have tried it. It has been so positive for children with type 1 that over two years ago Biogen opened its global expanded access programme to provide the drug free to type 1 patients.

Spinraza is currently available across 24 European countries and in the US, but for patients in this country access to the drug is being held up by lengthy delays to the NICE appraisal process.

[Mr Jim Cunningham (Coventry South) (Lab)]

Does my hon. Friend agree that the hon. Member for Reigate (Crispin Blunt) is right that there needs to be a new model, and more importantly that something must be done about the cost of drugs? We cannot carry on with the escalation of the cost of these drugs because, as the hon. Gentleman said, that affects many families in different ways.

[Mary Glindon]

That is true, and I think it will be highlighted again and again in this debate.

The delay for patients in this country is made all the more frustrating because the Scottish Medicines Consortium approved Spinraza for children with SMA type 1 last May, and it now has a new ultra-orphan pathway and has speedily reassessed Spinraza, and as a result children and adults with SMA types 2 and 3 will be able to access the drug from next month.

In England the Spinraza appraisal has already been going on for 14 months. In January last year NICE announced that the pathway for the drug would, sadly, be the single technology appraisal, used for common diseases, rather than the highly specialised technology appraisal, which has been spoken about and is used for rare conditions. On 14 August, all hopes were shattered when NICE announced that it did not recommend funding by the NHS as the clinical effectiveness of the drug was not proven and the price was too high.

NICE launched a consultation and held a committee meeting in October to review all responses. There was still no progress for patients. Then, following a previous announcement, on 1 November Biogen closed the expanded access programme for type 1 to all new infants, so although 80 children remain on the programme, any child born after that date with type 1 has no access to this life-saving drug. The process drags on, and NICE had its third committee meeting earlier this month, but as yet no information has been published.

Biogen maintains that the STA process is not appropriate for rare disease medicines, because the smaller patient populations in rare diseases make it inappropriate to expect treatments to achieve the same cost-effectiveness thresholds as medicines in disease areas with much larger patient populations. It has also pointed out that it is very difficult to measure the quality of life in a young paediatric population. However, that is a major determining factor in the STA process, so it is a stumbling block in approving Spinraza. The company still hopes that a managed access agreement can be reached with NICE and NHS England.

The truth is that NICE’s emphasis on cost-effectiveness stands in contrast to the focus on more flexibility and data gathering for future review, which has allowed Spinraza to be approved in Scotland and across Europe. A recent report by MAP BioPharma, “Access to orphan medicines”, highlighted that 75% of rare disease medicines recommended by NICE through an STA between 2013 and 2017 were due largely to rare cancer drugs that are covered by the cancer drugs fund, and none of the only six non-cancer orphan drugs reviewed by NICE through an STA has received a recommendation in line with full marketing authorisation.

The report makes five recommendations for the NICE STA methods review: making changes to the evidence requirements for orphan medicines; drawing from the HST methodology to consider introducing a sliding incremental cost-effectiveness ratio up to £100,000; considering adapting the evidence review group for orphan medicines; embedding formal opportunities for negotiation between companies and NHS England; and considering interim recommendations in line with the cancer drugs fund and the new Scottish ultra-orphan pathway. MAP BioPharma points out that those adaptations would help to level the playing field so that patients, clinicians and companies could be sure that all treatments for rare diseases would be considered under a fair appraisal and that access would not be held back as a result of treatments being referred for an inappropriate appraisal. I hope that those recommendations will be given due consideration by NICE, NHS England and the Department.

Meanwhile, for those awaiting a decision on Spinraza, the anxiety continues. They include families such as that of my seven-year-old constituent, Sam McKie, who has type 2 SMA. Sam loves playing wheelchair football and has played since he was three. He now plays for the Newcastle Magpies wheelchair championship team and is as good as many of the adult players. In fact, he is so good that, in November, the Newcastle United Foundation named him as its disability player of the year. Sam’s dad, Gary, wrote to me, and his words reflect the views of everyone affected by SMA. He said that

“children are facing an agonising and uncertain wait for approval whilst their condition deteriorates. Gaining early access to this drug could see Sam get stronger and gain new abilities. The SMA community would love to be able to access this drug to give our babies and children a chance, a chance they surely deserve. This drug is available now, and timely procedures are stopping our children from accessing it, this is wrong. Please help us.”

Will the Minister hear Gary’s words? Will he take action to ensure that delays do not happen in future? And will he work with Muscular Dystrophy UK and other charities towards making NICE take on board MAP’s recommendations, to help to create a new and fairer system, like that in Scotland, that will deliver for patients like Sam and, as Gary McKie says, give them the “chance they surely deserve”?