Phenylketonuria and Kuvan — House of Commons, 2018-03-22
Topic: Health
Victoria Grace Ford (Conservative, Centre-right): I thank the hon. Gentleman very much for his comments. I completely agree that we need to look at the wider issues. In fact, we should also look at what happens if the condition is not successfully managed, because that can be even more expensive. For example, if a woman with PKU gets pregnant and the condition is not well managed, the child she bears will be at very high risk of having substantial long-term disabilities. If a child with PKU does not manage to stick to their diet, they are at risk of permanent brain damage. All those wider costs should be taken into consideration when making this assessment The patient organisation is concerned that NICE may decide to restrict the drug and offer it only to those it considers to be high-need patients. The drug might be given only to patients who have refused to follow the strict diet, so it would actually discriminate against those who have done the right thing and worked so hard to maintain that very difficult dietary control. I say to the Minister: let us to try to break this deadlock. BioMarin and NICE need to engage with each other in a transparent way that has the full support of the patient organisation. The whole patient population should be considered, and those who do the right thing with their diet should not be put at a disadvantage There is a bigger issue. This is not just a debate about one patient or one drug, but a wider one about how we in Britain approach new medicines and treatments, especially for rare diseases. Britain is a world leader in science, especially the life sciences, and we are home to the human genome campus. The amazing, visionary 100,000 genomes project has set us at the forefront of the global revolution in medical research. Our unique NHS gives researchers the ability to access large quantities of reliable and detailed patient data, which helps them to identify very, very specific genetic divergences. That means that medics can increasingly pinpoint the exact cause of a rare condition, and discover specifically which one of a new generation of personalised medicines will give the most effective treatment for an individual’s condition. Life sciences lie at the heart of the Government’s industrial policy. However, if we are to stay at the forefront of world medical research, it is vital that discoveries are not only made here, but trialled, tested, and prescribed here NICE is a world leader in assessing medical health technology, and many other countries have chosen to follow its approach. Nevertheless, the world of medical research is changing exponentially, and if NICE is to continue to hold the confidence of researchers, physicians and patients, it must prove that it can evolve and evaluate even the most innovative treatments, and especially advanced medicines such as cell and gene therapies. When NICE approves a new treatment, we must ensure that the NHS can commission it effectively. Today the NHS cannot commission a drug unless it has been to NICE. Before a drug is granted marketing authorisation, there is the option for the company to make it available through the early access to medicines scheme, but after that marketing authorisation, and before NICE approval, there is no route to funding except through individual funding requests As the Minister knows, most rare diseases are very, very rare—at least some of them are—and the overall cost of treating them is a small part of the NHS budget. We must find a better way for all parties to work together to facilitate the passage of orphan drugs for rare conditions through NICE and the commissioning process I thank colleagues and the Minister for listening to my remarks, and I thank the Cotter family for being here today. Britain is a world leader in medical research, so let us ensure that British patients, such as my constituent, Cait, can be among the first in the world, not the last, to benefit from medical discoveries.