Jess Ennis is a UCD Master of Public Policy student. She considers the conflicts over funding and availability of critical medications – specifically Orkambi for cystic fibrosis sufferers.
In July 2015, the FDA approved the combination drug Lumacaftor/ Ivacaftor (Orkambi) which treats the underlying cause of Cystic Fibrosis (CF) for patients with two copies of the F508del mutation. Ireland has the highest rate of CF patients in the world and approximately 550 patients in Ireland are eligible for Orkambi, including myself. However, since the FDA approval, only patients living in the United States (US) Austria and most recently, Germany, are taking the drug. The main issue is that the drug currently costs an astonishing €159,000 per patient per year. If all patients in Ireland were granted this over a 5 year period it would be the equivalent of half the budget for the new national children’s hospital, €400 million. There are also estimates that the drug is only effective for 25% of those patients who take it. For the people who trialled the drug or who are on it on compassionate grounds, the improvements in lung function, weight gain, increased energy levels and a new found quality of life are indisputable results; for which debates of financial statistics are futile.
The HSE is still in negotiations with Vertex (the pharmaceutical company which own Orkambi) trying to bring the price down to approximately €30,000 per patient per year. This price is based on the June 2016, National Centre for Pharmacoeconomics’ (NCPE) cost assessment of Orkambi. This is based on the clinical trial outcomes, including hospital admission avoidance and increased quality of life, safety, cost effectiveness and the budget impact for the HSE. Their recommendations are that Vertex has failed to prove Orkambi is cost effective and the HSE should not reimburse Orkambi at the submitted price. The HSE must follow these recommendation based on the 2013 Health Act (pricing and supply and medical goods). According to Michael Barry from NCPE who advises the HSE, Vertex is nowhere near an acceptable negotiable level. An unfortunate case of institutions putting monetary value on people’s lives and saying public health care investment is not value for money.
On Wednesday 7 December 2016, the CF community protested outside the Dáil in a bid to push the government to fund Orkambi. Roughly 2000 people marched, including people with CF, friends, relatives, those who have lost loved ones to CF and even TD’s. But this is not the first time the CF community have found themselves campaigning for treatment. In fact, for the past twenty plus years, people with CF have been fighting for their rights for sufficient treatment and services across Ireland. These include basic CF needs to avoid the high risk of cross-infection from waiting in emergency departments or in six-bedded wards with other patients with CF. For example, in 2008, various media outlets, in particular Joe Duffy’s radio show, continuously had patients with CF on air to push the government to build the proper isolation units that people with CF require. Only three years ago, people with CF also had to fight for the drug Ivacaftor (Kalydeco) which treats 120 people in Ireland, and which treats the underlying cause of CF and not just the symptoms. In these cases, the CF community proved a strong and powerful interest group who successfully influenced the policy process.
Joe Brolly, an advocate for Cystic Fibrosis Ireland, has described the conflict over pricing and availability of Vertex and Orkambi as a typical feature of the way modern capitalism works. However, I would argue this is more than ‘normal’ modern capitalism: it is a manifestation of corporate greed and a system failure centred on regulatory decision-making. In standard free-market economic theory, if the product (in this case Orkambi) is too high – even allowing for the sunk costs of research and development – other companies would make their own version and allow the market to set the price. However, the demand for Orkambi is relatively low and there is a serious problem in its supply. The FDA granted Vertex a patent on Orkambi and other competition is blocked, so Orkambi is essentially a monopoly drug. Other drug companies will have to wait years to get FDA approval – this is how Vertex is able to demand Orkambi’s extortionate price. Although drug companies claim to dedicate themselves to developing treatments, they are no more than success-driven and profit-making companies. In 2014, the CEO of Vertex took home an inconceivable salary of $45.8 million. The FDA continues to act as lever between the pharmaceutical industry and government. What is evident is a structural influence of the producer interests on policy. A government-enforced monopoly on these drugs has being created and is not set to change anytime soon. The average life expectancy for a person with CF in Ireland is late twenties; time is not an option for many.
Members of Cystic Fibrosis Ireland and various people with CF have been brought into the Dáil by different parties and have participated in consultations on policy on this subject. There is a pluralistic view at work here, which would see the CF interest group moving in and out of the policy process, and our power shifting over time. However, Orkambi goes beyond party politics. On the day of the protest, the Minister for Health, Simon Harris, was in Lisbon, meeting with other European health ministers in an attempt to negotiate the unacceptable drug prices offered by Big Pharma. In a statement released that day, Minister Harris stated: “I intend to continue to intensify my engagement with colleagues in Europe and indeed other countries on this issue. I want to see CF patients receive access to the best treatments possible. That remains my priority.” This strategy by the Minister has the potential to tackle Vertex productively. Using an international governing institution such as the European Union might be the only way of enforcing the ethical obligations of pharmaceutical companies to put patients first and benefit shareholders second.
As a person with CF who could benefit from this drug, and as a public policy student, I can see both sides of this debate. In my opinion, Orkambi is not necessarily the ‘game-changing’ drug the media portrays it to be. However, knowing a drug of hope is being denied to so many of us, based on cost alone, is alarming. Austria has adopted a balanced policy: it will only fund patients who have been shown to benefit from Orkambi after a trial. This is certainly a policy the NCPE and HSE should consider. No doubt, Orkambi has been put on the agenda by the strong interest group which is the CF community. Unfortunately, the bigger issue of the power of drug institutions remains a major concern. What I believe is needed is an international collaborative approach for a clear policy on drugs such as Orkambi. People with CF fight every day to breathe – we should not have to fight these institutions too.
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