Accelerated Access Review

Philippa Whitford Excerpts

Tuesday 13th December 2016 Westminster Hall

[Dr Philippa Whitford (Central Ayrshire) (SNP)]

Access to new drugs seems to be almost the commonest theme of debates in Westminster Hall. Having spent years as a breast cancer specialist involved in trials, I can say that it is really frustrating to have access to drugs within a trial and then lose that access when the drug is passed. The United Kingdom can be up to five years behind Europe or America in accessing such drugs. We talk all the time about having more research on brain tumours and other diseases, but that does not help us if, at the end of the research, our patients cannot get the drugs.

Cystic fibrosis is one of the commonest of what we call the rare diseases. It involves a problem with transmission of salts through membranes, which results in incredibly thick mucus that clogs various organs, most commonly the lungs. As the right hon. Member for Leigh (Andy Burnham) mentioned, if it is diagnosed late, damage will already have been done. The earlier patients with cystic fibrosis are treated, the less damage is done and the healthier they are. The life expectancy has changed from childhood to middle age, due to a combination of approaches.

I am shocked to hear that people in England with cystic fibrosis have to pay for their prescriptions, because that would amount to quite a lot of money; they are on a lot of medication. We do not have prescription charges at all in Scotland, because you get an increased rate of people not collecting their prescription, or going to the chemist and saying, “Excuse me, dear, which are the two most important drugs for me to take?” and that ends up not being cost-effective. I would have thought that people with a chronic condition should at least have their names on a list as being exempt. I would have thought that that was the least the Government could do.

The right hon. Member for Leigh said that we have been through this with Kalydeco and many other drugs. Orkambi is a synergistic combination of Kalydeco, or ivacaftor, with lumacaftor, which makes it work much better. They are the first drugs that are not just antibiotics or mucolytics; they are trying to attack the disease itself. In that sense, they are transformative. The problem in the access review is that the definition of “transformative” going forward will not necessarily help those drugs. We do not suddenly find a drug that is a cure for any of these conditions; we move step by step, often adding drugs together or making new discoveries.

There is a real concern among those who develop drugs that in the consultation between NICE and NHS England, the levels considered acceptable for such highly specialised treatments are being changed. The problem is that if we send out the message to people with rare conditions, “I’m sorry; you’re just outside the pale,” we will be letting them down. We need a different approach. I think we need a different conversation for all drugs. The NHS in the United Kingdom brings a cohesive system that allows for follow-up data and allows a lot more information to be sent back to companies over time, which is not easily available in other countries. That should be on the table as part of the negotiations.

I have a real concern, going forward after Brexit, that in this country we will be further down the list for people to even apply for licences here. It may well be that the application to the Medicines and Healthcare Products Regulatory Agency for 60-odd million people in the United Kingdom may well cost an amount very similar to an application to the European Medicines Agency for a market of 450 million. That means we could end up in the same position as Canada, where it often takes about a year before a company decides to apply for a licence. The problem is that if, going forward, companies see that they must pay to apply to NICE, which will turn them down so they will have to pay to apply again, they may just decide that it is not worth the candle. That must be taken into consideration.

Obviously, England has the Cancer Drugs Fund, the idea of which is to allow a little bit of flexibility on access to new drugs, which are often expensive, but it does not help you if you do not have cancer. In Scotland, ours is a new medicines and rare diseases fund, which as a proportion of the population is three times the size of CDF, so it is more flexible. It cannot be a long-term solution for such drugs, or the funds would get sucked up, but it is important that when we are going through a phase of considering the real-life use of expensive drugs we have some flexibility for patients, and not just cancer patients.

We had a debate in the main Chamber last week about the Health Service Medical Supplies (Costs) Bill, and one discussion involved funding. In Scotland, the pharmaceutical rebate goes to fund the new medicines and rare diseases fund. In England, it goes back into core funding, which means that along the line, the beneficiary is the Treasury. If the NHS is managing attribution and access to new drugs in such a way that it gets a rebate, it should be able to use that to access more innovative medicines. That is why the pharmaceutical industry agreed to it. It also creates a better relationship with the pharmaceutical industry. We cannot have a situation where the industry just pulls a price out of the air and we must rise to it—of course we must get value for money—but it is really important that we do not leave people with certain conditions knowing that there is utterly no chance that they or their children will access treatment.

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[Mrs Hodgson]

My hon. Friend is right to raise those concerns. We do not want to move into a new system that will create new unintended consequences. Perhaps the Minister will touch on that in his speech.

Although some are calling for interim solutions to help people who are stuck waiting for the accelerated access review’s recommendations to be implemented, it is also important that the Government get on with implementing those changes. The review was announced more than a year ago and was published two months ago now. It is important to remember that the transformation that we all want to see will not happen straight away, but it is still right that we keep up the pressure for the recommendations to be implemented. There are many such recommendations, and I hope that the Minister will be able to update us today on the progress on each of them. There are two in particular that illustrate what can be done to resolve the deadlock around Orkambi—the immediate establishment of an accelerated access partnership and the setting up of a new flexible strategic commercial unit.

The accelerated access partnership is one way in which, through co-ordination and collaboration across the system, we could see drugs brought on to the market more quickly to benefit patients who need access to them. I would be interested to hear from the Minister what progress has been made on its creation, especially in conjunction with the issues surrounding the deadlock on Orkambi.

It is clear that the strategic commercial unit could help to benefit those who wish to see Orkambi offered on the NHS. The unit could work with those involved in this dispute to end the current deadlock through facilitation of the flexibility and transformational change promised by the accelerated access review. That would go some way towards helping to access data on drugs such as Orkambi and getting them out to patients. There is a willingness out there for that flexibility to be brought into the system; for example, the Cystic Fibrosis Trust has offered to use the UK cystic fibrosis registry to help to provide essential data that can help to prove how effective drugs can be and what more needs to be done. We have already heard how substantial that registry is; it includes 99% of sufferers. I understand that the trust’s offer has been welcomed by all sides in the negotiations but is blocked due to the lack of progress in implementing the changes set out in the review. I hope that the Minister will give us some clarity on when the unit will be created and when we can see a culture shift within the system that will allow for flexibility to accept data and information that show how much effect these drugs have on people’s lives.

[Dr Philippa Whitford]

Does the hon. Lady share my concern about drugs for other conditions, such as sofosbuvir for hepatitis C? Even after they get NICE approval, those more expensive drugs are now being rationed at the NHS England stage. At the moment we are fighting to get through NICE, but it needs to be a smooth path all the way through.

[Mrs Hodgson]

The cost of drugs sometimes leads the NHS into the terrible and unfortunate situation in which rationing seems to become the norm. There can also be a postcode lottery, which is another element that we need to look at. The price of drugs really is the crux of the issue.

In conclusion, I hope that the Minister will offer some insight into the progress being made on the recommendations of the accelerated access review. The case of Orkambi can help to drive through these changes and to end this deadlock, which, as we have heard, is causing unnecessary suffering for those living with cystic fibrosis. The review has established a space for change and for patients to access new and innovative drugs and treatments. It is important that there is no stalling or delay in transforming the system, because people’s lives depend on the changes called for by the review. I am sure that the Minister will keep that in mind when he goes back to his officials.