Opinion: Cystic Fibrosis Ireland outline why Orkambi and other 'expensive' drugs are worth it in the long run.
In her recent article, A future of expensive medicines and tough decisions, Aileen Murphy raises some important issues in relation to the difficult decisions faced by Government, especially in relation to the reimbursement of expensive new medications such as the cystic fibrosis (CF) drug, Orkambi.
But there are also some important gaps in the information that she has provided and there are also some statements that Cystic Fibrosis Ireland would contend are both unfair and inequitable to people with CF and indeed unfair to all those with rare diseases in Ireland.
In relation to Orkambi one year after reimbursement, she contends the reviews ‘are mixed’ and that it is not a ‘miracle drug’. We would contend that from the information coming back to Cystic Fibrosis Ireland from both patients, and informally from clinicians and nurses, that the most common response to Orkambi is ‘positive’ and ‘very positive’. Yes, of course, a small minority will not gain from Orkambi, nor will stay on Orkambi for a variety of clinical reasons, but this is true of all drugs.
No one in Cystic Fibrosis Ireland ever stated that Orkambi was a ‘miracle’ drug. However, we do maintain that Orkambi is a game-changing drug, as it is the first drug to treat the underlying cause of CF in over half the CF population in Ireland. Previous drugs, important as they are, treated the symptoms.
Further, Aileen Murphy’s article does not make clear that the deal in Ireland with pharmaceutical company Vertex not only includes Orkambi, but also includes the extension of another drug, Kalydeco, and forthcoming Vertex drugs that are currently showing excellent results in advanced clinical trials and, in some cases, will likely prove even betterthan Orkambi.
She also ignores the fact than many countries in Europe, including the Netherlands, Germany, Italy, Austria and France, have all approved Orkambi and other countries such as the UK are looking at Ireland's innovative CF ‘pipeline’ deal as a possible better way to secure access to these crucial drugs, even though Orkambi was turned down the first time around in England.
The main disagreement we have with Ms Murphy’s analysis is the statement that ‘it is little consolation to those who need treatment now and to those whose access to other healthcare has been delayed owing to the opportunity costs of funding Orkambi’.
Apart from the fact that we have the highest incidence of CF worldwide – and we in Cystic Fibrosis Ireland make no apology for seeking the best services and medications for our patients – this is at best a very simplistic statement. For example, it completely ignores the current agreement between the Government and the pharmaceutical industry in Ireland (IPHA) 2016-2020 which projects savings of over €600 million which would pay for Orkambi and many other orphan drugs.
Further, the Irish government has been rightly criticised in a series of reports, including by the ESRI, highlighting overspending or continued spending on low or no impact drugs over many years rather than focussing on new and cutting-edge drugs that are now becoming available. To be fair, this is fully recognised by the Minister for Health, Simon Harris.
Murphy is right to say that there are tough decisions ahead but patient groups such as Cystic Fibrosis Ireland, the Medical Research Charities Group and the Irish Platform for Patient Organisations, Science & Industry, have been contributing to this discussion with a number of positive suggestions for many months, some of which have been adopted by Government.
Firstly, it was patient groups that proposed that Ireland join with a European grouping of countries (BENELUXA) to ensure that we got the best value for drugs when negotiating with large pharmaceutical companies. Ireland has recently joined with this grouping after flirting briefly with the idea of joining up with English-speaking countries which again patient groups advised from the start was a non-runner.
Secondly, most key stakeholders involved in drug reimbursement in respect of drugs for rare diseases contend that the present system is not ‘fit for purpose’ because, in part, it does not recognise the simple economic equation that if you are dealing with a small number of patients, and you have invested a lot of money into research and development for a new drug, it is inevitable that drugs for rare diseases are going to be much more expensive than drugs for more common conditions.
Thirdly, after much lobbying from patient groups the Government has agreed to set up a specialised rare disease drug therapy reimbursement committee within the National Centre for Pharmaco-Economics (NCPE), with the view to advising on drugs such as Orkambi in future. This is very positive.
We in Cystic Fibrosis Ireland make no apology for saying we have to look after the few as well as the many in our healthcare system, but this is in no way a zero-sum situation.
The views expressed here are those of the author and do not represent or reflect the views of RTÉ.
