Cutting-edge drugs facing funding battle
500 cystic fibrosis patients want Orkambi
Cutting-edge, revolutionary drugs are becoming increasingly unaffordable for publicly-funded health systems like Ireland's.
While the current spotlight is on Orkambi, described as a game-changer treatment for many people with cystic fibrosis, there are more so-called new miracle medicines for cancer and heart failure coming down the line.
For the patients who are desperate for these drugs - that may have the power to increase their lifespan but have exorbitant prices - the demand that governments fund them is perfectly understandable. How can greater life expectancy be reduced to a finite sum of money?
But in a world of health rationing, paying too high a price for these drugs means other services may suffer.
In the case of Orkambi, the annual cost per patient may be up to €150,000 and around 500 people with cystic fibrosis believe they should be allowed to try it.
However, Ireland is not alone in reaching a cost-benefit analysis that, based on that price, it is unsustainable. Patients in the UK, Canada and other countries have been delivered the same bad news.
The decision to turn down funding for the drug looks set to be the conclusion of the HSE's drug committee, which assesses drugs.
The hope is that some more price negotiation or method of making the drug more economically viable is found soon.
The HSE executive makes the final decision, but it is unlikely to overrule the decision of its committee of experts.
Under a protocol agreed earlier this year, the Health Minister can ask Cabinet to give the go-ahead to fund a new drug.
But that only applies where the HSE recommends a medicine and cannot pay for it out of its current funds.
Dr Michael Barry, who heads the pharmacoeconomics section of the HSE, yesterday urged the drugs company Vertex, makers of Orkambi, to put the patient first.
But Vertex, like a lot of drugs companies that produce these drugs, wants its payback for the years of scientific legwork that went into its formulation.
It has cited several reasons, including the small patient population the drug will be suitable for.
It needs to generate enough return to invest in researching new medicines. It also argues that on the patients it works on, the drug has the ability to treat the underlying cause of the disease.
The company was set up 26 years ago and has only turned a profit once.
It recently emerged that the chief executive of Vertex, Jeffrey Leiden, earned a total package of around $36m (€34m).
It's eye-watering remuneration and a clear message from shareholders that they expect performance. The investment gamble by even global giants developing new medicines does not always pay off. In recent days, this was borne out when Pfizer cancelled a planned €400m expansion of its Dublin plant due to the failure of a late-stage pipeline drug to lower cholesterol.
"We are currently awaiting a response from the HSE on our latest proposals," a spokeswoman for Vertex said yesterday.
She said in the meantime, all people currently receiving Orkambi via clinical trials or compassionate use will continue to receive it until agreement is reached.
Philip Watt, of Cystic Fibrosis Ireland, said yesterday this includes around 40 people, 25 of whom have it on compassionate grounds.
Dr Barry suggested that just 125 of the 500 patients will benefit from the drug.
He called for an arrangement where Vertex is paid full price only for those patients on whom it works.
It may be one way of securing these and other blockbuster drugs for patients.