Cystic Fibrosis Drugs: Orkambi

Rachael Maskell Excerpts

Monday 10th June 2019 Westminster Hall

[Rachael Maskell (York Central) (Lab/Co-op)]

It is a pleasure to serve under your chairmanship, Mr Bailey.

Leading up to this debate, I spoke to constituents, one of whom described the mental strain of knowing that a drug was available but could not be prescribed to their child. Another parent talked about the administration of 22 drugs, and the physio and hospital appointments that were needed, as well as the stress of their child being admitted to hospital. Their child is now 21 years old and there have been no changes, even though there is a miracle drug that could transform their life. Another constituent, Jack, who is seven years old and at the start of his journey, desperately wants hope for his future.

We are at the cutting edge of a generation of pharmaceutical breakthroughs that have the power to transform the lives of people who experience a range of medical diagnoses. It is an exciting point in our journey with medicine, and it could be transformative to patients and carers. To deny therapy is ethically abhorrent. Many Members today have said that this is about negotiation, but I say that negotiation is not the answer. We should not be wrestling over drugs time and time again; we need a completely different framework for addressing the cost of pharmaceuticals.

NICE needs an overhaul to ensure it is fulfilling its role and looking at patients’ life course. I know that it does that work, but it needs to look at its effectiveness over a patient’s life course and at the economic value it can bring not just to medicine, but to the wider economy. It also needs to ensure that the levers are in place for it to look at a portfolio of drugs, as opposed to looking at drugs one by one.

We need to understand how much the NHS can afford to pay for the benefits of new drugs, taking into account the health benefits that could be achieved with the money the NHS must find to pay for them. This assessment, based on a NICE appraisal, could inform a debate about national rebate agreements in the current pharmaceutical price regulation scheme. There are several costs involved: the manufacturing cost, the price the NHS can afford, the NICE assessment and the market price, which is driven primarily by the US market. That is a cause of frustration.

The argument among academics is that a mechanism of a national value-based rebate should reflect the difference between the amount the NHS is willing to pay for the benefits and the manufacturer’s asking price. That would provide better incentives for manufacturers to make long-term investment choices, recoup costs and deliver for the NHS. It would also provide fairer rewards for innovation; manufacturers that produced more effective drugs at affordable prices would not need to pay a rebate, but those that charged more would pay a higher rebate. That would not impact the list price for the global market, and the rebate could be spread across the portfolio to provide better opportunities for manufacturers to bring new products to market.

That would take the politics out of NICE and NHS England. Canada is currently developing something similar, to ensure that drugs reach the places they need to reach. It can be further incentivised: should a manufacturer not supply a product when its production costs mean it could do so without making a loss, it could lose its patent. That would provide the required leverage. We need to hold discussions about this not just across the sector, but with the European Medicines Agency; the impact could be greater with more countries on board.

[David Hanson in the Chair]

People will have seen reports about the generic drug from Argentina, and we have heard about it in the debate today. We cannot allow inequality in access to pharmaceuticals in our country to grow, because it creates health inequalities. People who can pay will be able to afford that drug, and they will have better health outcomes. In the light of the ethics of the debate, the Department of Health and Social Care needs to move fast, now.

We need to be alert when it comes to trade deals with the United States, which is significant to the debate. The US will clearly want to drop the carrot of big pharma in front of Ministers, but that is a serious trap. The President talked about the NHS being “on the table” in his speech about trade negotiations, and we must be alert to the real agenda. It is through big pharma that he will get access to the NHS. As we have already heard, with respect to the size of the NHS drugs budget, that is a way of controlling it. We need to be on top of that.

Instead of drug-by-drug negotiation, we need a framework leading automatically to the leverage required to procure medicines. That will make such a difference. It will remove the uncertainty about different pharmaceuticals, as we move forward through the exciting developments that are taking place. Let us take the politics out of the process and enable NHS England and NICE to do their jobs, even under a new contract. I ask the Minister to do her job and to put a new framework in place.