ALISON O'CONNOR: It’s sick that the cost of drugs dictates who lives and who dies

The NCPE recommended that Orkambi should not be funded ‘at the submitted price’ of €160,000 per patient per year, writes Alison O’Connor

CYSTIC fibrosis sufferers must be dismayed that supply of a drug which they believe could transform their lives has been dismissed on the grounds of cost-effectiveness.

How horrible to be the ones to make that decision. That task fell to our National Centre for Pharmacoeconomics (NCPE), which decided against Orkambi, though it has been hailed as a wonder drug.

However, this might not be the toughest decision the NCPE has made. Supplying the drug over a five-year period to around 500 people who have CF would have cost €392m, roughly the equivalent of two thirds of the price of our new national children’s hospital. To put it another, equally emotive way, it is roughly the amount of money needed to run Temple St Children’s Hospital each year.

Cystic Fibrosis Ireland has described Orkambi, which has been approved by the European Medicines Agency, as a game-changer that would extend the lives of sufferers.

However, the NCPE recommended that Orkambi should not be funded “at the submitted price” of €160,000 per patient per year. That price is outrageously expensive, and a massive try-on by its manufacturers, Vertex. While the NCPE said it did recognise the health benefits provided by the drug, it is not convinced by important aspects of the clinical trial evidence presented by Vertex.

CF is such a horrible disease that it is hard not to feel anything but sympathy for those who suffer from it.

But these Orkambi-like dilemmas are going to become a regular feature of our lives. Orkambi is an indication of far bigger challenges facing us, in terms of new breakthrough drugs that are about to hit the market.

In the three years from 2010, 20 super-expensive new drugs were assessed by the NCPE, but that number shot up to 60 from 2014 to 2015. This year, so far, the figure is 38.

The pharmaceutical industry is hitting a particularly rich vein with new, eye-wateringly expensive drugs for treating conditions such as cancer and cholesterol. We face major ethical and budgetary dilemmas about just how much we will spend on new drugs for individual patients, particularly given that we have a health service in a constant budgetary crisis.

There are lots of competing interests here, not just the powerful drug companies. Individual doctors, who specialise in particular areas of medicine, will understandably advocate on behalf of their patients, and nowhere do we see this more than with cancer doctors.

Professor Michael Barry, head of the NCPE, believes this approach is understandable, but not sustainable. Many of the new cancer, ‘life-saving’ drugs are nowhere near as efficacious as claimed, he said. “The new cancer drugs, in many instances, offer marginal benefits….Oncologists and clinicians, in general, are slow to recognise the modest benefits presented by many drugs.”

This does not fit with the modern narrative around medicine, and particularly around cancer treatment, of almost daily, miraculous new developments and how we need to be availing of all of them. But the cancer doctors are not going to worry, for instance, about the cardiac patients, and how much money is left over for their treatment and drugs.

It also comes down to what sort of a public relations machine surrounds a particular disease and the fear factor associated with it. For instance, as an adult female you are conditioned to be far more concerned about the successes in breast-cancer research than on the cardiac front.

In truth, Prof Barry says — and he has been with the NCPE for 18 years — very few drugs are actual game-changers, despite what we are told. One area of success is the treatment of Hepatitis C: a drug that costs between €50,000 and €100,000 per course cures about 70% of patients. “Last year, we said ‘yes’ to every one of those,” Prof Barry said.

They also said ‘yes’, this week, to Pembrolizumab, known as Pembro, recommending to the Minister for Health that it be covered under the State drug scheme for treatment of skin cancer. It is pricey, with a budget impact of €60m, over five years, but that is six times less expensive than Orkambi.

Prof Barry said that while medical students today are taught about the cost-effectiveness of prescribing certain drugs, previous generations of doctors are used to looking at whether the drug is safe and effective, and not at the cost benefits.

Up to now, we have had a fairly good track record in making new drugs available to patients who need them. However, we are already in the space of not being able to afford the standard of care we have come to expect; this will even be the case for a proven ‘game-changer’ drug, if it is priced way too high.

The new Health Minster, Simon Harris, appealed to the manufacturer of Pembro, MSD, and to Bristol Myers Squibb, which makes a second drug, Nivolumab (also for cancer treatment), to “show some compassion” by making the drugs available free, while the Government decides whether or not to approve them.

But appealing to the better nature of the pharmaceutical industry is hardly sustainable, or realistic. Equally, Minister Harris is right in saying that it is utterly daft that politicians, such as him and the Cabinet, decide who, if anyone, gets these drugs.

The pharmaceutical industry provides drugs that help millions of people worldwide everyday. It also puts pots of money into developing drugs that never make it past the research stage.

But it must realise that it is in danger of pricing itself out of the market, with these new, uber-expensive drugs, which even better-off countries will be unable to afford.

Ultimately, if it continues as is, the industry won’t be able to afford to develop new drugs and we won’t benefit from them when we get sick. If they are extra-greedy now, they are bound to pay later.

This debate, on which drugs to approve, is being had in countries all over Europe, at present. Maybe we need to work out a deal, whereby the companies are paid by actual patient results, but that would require a proper system of monitoring.

There is a backdrop to all of this. It is the ongoing failure of the HSE and the Irish Pharmaceutical Healthcare Association to reach agreement on general drug-pricing and supply. The HSE is looking to significantly reduce how much it has to pay for medicines. Relations between the two sides are not good, and this plays into the circumstances of the controversy surrounding the so-called game-changer drugs.

There have been suggestions of a special fund being set up in the future.

The problem is that to satisfy everyone it would have to be a fund that never runs out, and that is simply not going to happen. Setting realistic boundaries is going to be very tough.


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