Drugs: Ultra-rare Diseases

Jonathan Edwards Excerpts

Tuesday 16th June 2015 Westminster Hall

[Jonathan Edwards (Carmarthen East and Dinefwr) (PC)]

That system sounds excellent. I commend the Scottish Government on their work. That could be a great help to my constituent, Mr Trystan James, who suffers from tuberous sclerosis complex and is reliant on a clinical trial drug to deal with a life-threatening tumour. Of course, his drug prescription is therefore completely at the discretion of the drugs company and his family are going from one prescription to the next. That relates to what the hon. Member for Strangford (Jim Shannon) said about emotional pressure on families. I commend the Scottish Government on their work.

[Dr Whitford]

The important thing to remember is that if this is all done by individual requests, the NHS does not go to the companies. We need to realise that companies have often made investment over decades and that nine out of 10 drugs they research will go nowhere, but it is important to have a wider debate with companies to get the best price. Hon. Members mentioned that some companies are willing and able to reduce the price to get a drug in.

Drugs are licensed. We must not mix up licensing with funding. Licensing is about asking, “Is this drug safe and proven at a basic level?”, not anyone coming in and saying, “Rare plant juice will cure everything.” These are licensed drugs that we could prescribe—a doctor has the right to prescribe them—but the NHS has to make the decision about whether to fund them; those are funding decisions, not licensing decisions.

It is important that families know what the pathway is and how they move on when their clinician takes a case forward. It is important that they know they can respect decisions and how to lobby at the next step, and that they feel their voice is being listened to. We feel that PACE has, over more than a year, allowed us to do that. Clinicians in Scotland got frustrated about decisions going through without us informing that decision.

There could be a system that sits on the side of NICE, or a sub-group. One of NICE’s three assessments will never be given over to a drug intended for 88 patients when it is also assessing drugs that might be taken by 500,000 people. Rare diseases would always fall behind, and that is why those must have their own system and why the patient voice must be heard in these ways. Obviously, things have changed with the Health and Social Care Act 2012, but I commend such a structure to the Minister.