NICE Appraisals: Rare Diseases Treatments

Stephen Twigg Excerpts

Thursday 21st March 2019 Commons Chamber

[Stephen Twigg (Liverpool, West Derby) (Lab/Co-op)]

It is a pleasure to follow my hon. Friend the Member for West Ham (Lyn Brown), who made her case with great power and passion. I congratulate my hon. Friends the Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon) on securing this important and timely debate.

Until recently, I had the pleasure of chairing the all-party group on rare, genetic and undiagnosed conditions, which receives secretariat support from Genetic Alliance UK. The all-party group aims to raise awareness in Parliament about such conditions and to ensure that patients and families can access appropriate care and support. My hon. Friend the Member for Hornsey and Wood Green (Catherine West) has taken over as chair of the all-party group. Unfortunately, she had prior commitments today, but otherwise would have been here to take part in the debate.

My constituency in Liverpool is home to the fantastic Alder Hey Children’s Hospital, which works in close partnership with the University of Liverpool and the charitable sector to provide more research opportunities for children with rare diseases and their families. For example, in the rare neuromuscular diseases, Alder Hey has worked with patient-advocate groups and national charities to increase capacity and resources to offer access to clinical trials of new disease-modifying agents. Since 2016, Alder Hey has opened 19 new clinical trials for Duchenne muscular dystrophy alone, enrolling nearly 100 boys in these important trials. By providing excellent, world-class clinical and research expertise and working in partnership with the charitable sector, Alder Hey has been able to provide more opportunities for children with rare diseases to take part in research studies.

In my role as chair of the all-party group, it was a privilege to get to know the rare diseases community. During that time, the issue that was consistently raised with me by patients and their families, clinicians and pharmaceutical companies was the challenge they face attempting to navigate NHS England and NICE’s appraisal processes for rare disease treatment. As other Members have said, patient access to new orphan medicines in the UK lags behind many other European countries, including Germany, France and Spain. The Office of Health Economics found that the UK is slower at making access decisions and approves far fewer medicines for reimbursement than other European countries. According to the recent MAP BioPharma report, which used data from a four-year period, almost a quarter of licensed orphan medicines have not been appraised at all by NICE or NHS England. This can prevent rare disease patients from having any opportunity to access treatment.

The lack of capacity to appraise orphan medicines is just one of the systemic flaws—we have heard about others—with the current NICE appraisal system. The multiple pathways through which medicines can be appraised create further complications and delays. It is often unclear why one route is chosen over the other, so very similar orphan medicines can be subject to vastly different assessment criteria. We also know that patients are often stuck in limbo waiting for the results of private negotiations about price between companies and the NHS years after the market authorisation of their potential treatment.

It is against that backdrop that the all-party group asked Genetic Alliance UK to propose a method of making decisions about rare disease medicines that would be more effective, transparent and fair. The project is called “resetting the model” and it aims to develop a flexible new vision for getting access to rare disease medicines for the UK, and it will be delivered in the coming months. It is absolutely clear that the current NICE appraisal process is simply not fit for purpose and is acting to restrict many rare disease patients from having the opportunity to get access to potentially life-changing treatment.

Let me address a related issue. NICE has been consulting on guidelines for cannabis-based products for medicinal use. On Tuesday, I had the pleasure of meeting my constituent, Lauren Abernethy, who is the mother of Nathaniel. Nathaniel is 10 months old and has a type of epilepsy that is so rare that when he was diagnosed his doctors told Lauren and James, his dad, that Nathaniel is the only known case with this type of genetic mutation recorded anywhere in the world. For the next three months of his life, Nathaniel was in Alder Hey Hospital undergoing tests, and for most of that time he was in critical care.

Lauren was here on Tuesday as part of the “End Our Pain” lobby of Parliament. Owing to the extremely rare type of epilepsy that Nathaniel has, antiepileptic drugs do not work, so Nathaniel was granted the use of Epidolex, a medical cannabis product. He has reacted positively to the treatment, going from being in a state of continuous seizures to now having, on a good day, as few as three to five seizures. However, despite that progress, he continues to live in great pain. He jerks and twitches continuously, which is known as myoclonic jerks, and has up to 100 of these every day. Access to full extract cannabis oil might offer Nathaniel a better quality of life, which he surely deserves, but like so many other children who are suffering from intractable epilepsy, Nathaniel has so far been denied access to that treatment.

I welcomed the decision last November by the Home Secretary to reschedule certain cannabis-based products for medical use, but the reality for patients such as Nathaniel is very different. Only a tiny handful have succeeded in getting a prescription. Patients, some of whom are as young as Nathaniel, are being blocked from access to medicine, which, as the evidence shows, has at least a possibility of relieving their symptoms. I urge the Minister in his response today to talk about how a policy can be put in place, based on evidence, that enables patients with rare diseases who could benefit from medical cannabis getting access to it.

More broadly, may I echo the comments that have been made on both sides of the Chamber and ask the Minister today to set out what he will do to take this crucial set of questions forward? In particular, will he work with Genetic Alliance UK so that we can reset the model, working with NICE and NHS England, so that patients with rare diseases get the fair access to medicines and treatment that, surely, they all deserve.