Yes 2 Orkambi - Agreement Reached
Statement from Cystic Fibrosis Ireland - April 11 2017
Cystic Fibrosis Ireland (CFI) warmly welcomes the joint statement from the Minister for Health and Vertex that an agreement has been reached between the Health Service Executive (HSE) and Vertex Pharmaceuticals for the reimbursement of the ground-breaking drugs Orkambi (circa 550 cystic fibrosis patients) and Kalydeco (circa 40 cystic fibrosis patients, including 18 patients aged 2-5 years old). The development is especially welcome, coming as it does during Cystic Fibrosis National Awareness Week, with 65 Roses Day taking place countrywide this Thursday.
While we have yet to see the full details of the agreement that has been reached, Cystic Fibrosis Ireland understands that it will be inclusive of all patients who have the potential to benefit from Orkambi and Kalydeco because of their particular CF genotype (almost 600 patients in total).
Cystic Fibrosis Ireland pays tribute to all in the CF community who have spoken out so bravely in favour of Orkambi and Kalydeco in recent months, in particular Jillian McNulty, the Whitmarsh family, Bernie Martin and many others also.
CFI understands that there is a very innovative element to the agreement which is that it is inclusion of ‘pipeline drug therapies’ from the same company that are currently showing promise in the advance stages of clinical trials.
Why are pipeline drugs so important?
Cystic Fibrosis Ireland points out that even with Orkambi and Kalydeco, there will be around 30% of the CF population that still has no drug that treats the underlying cause of their condition in Ireland. This is why a pipeline deal is so important. There also may be better drugs for those on existing Vertex drugs coming down the line.
For example, the Vertex company recently issued very positive news on Phase 3 clinical trials for terzacaftor. The second of these Phase 3 clinical trials tested the safety and effectiveness of tezacaftor in combination with ivacaftor for people with one F508del mutation and a second CFTR mutation that results in residual function. This group does not benefit from an existing drug.
The eight-week study showed that, compared with those on placebo, participants with one F508del and one residual function mutation who took the combination treatment improved their lung function by a very dramatic 6.8 percentage points.
Tezacaftor is likely to be approved by the Food and Drug Administration (FDA) in the United States in late 2017 and by the European Medicines Agency in early 2018.
We understand that these pipeline drugs are part of the agreement.
The high costs will be offset in significant part by other savings
Cystic Fibrosis Ireland recognises the very high cost of these drugs. However, the results of clinical trials demonstrate that Orkambi:
· Slows the progression of cystic fibrosis
· Significantly reduces exacerbations (sudden worsening of the condition) that leads to hospitalisation and can result in a drop of lung function that is never retrieved again
· Reduces the dependency on other drugs used in the treatment of cystic fibrosis, including expensive antibiotics
· Enables a quality of life that may include part-time or full-time work or education for the individual involved, which may not have been possible before, or possible to continue to maintain.
There should never have been a dispute over extending Kalydeco to 2-5 year olds. Kalydeco can potentially be prescribed for about 12% of the CF population in Ireland whereas Orkambi potentially applies to 57% of the CF population, if under 12s are included.
In short, in addition to health gain, Cystic Fibrosis Ireland contends that the approval of Orkambi and Kalydeco will result in significant other savings for the HSE. Cystic Fibrosis Ireland calls for independent monitoring to quantify these additional savings.
Cystic Fibrosis Ireland further calls on the HSE to come to a similar positive conclusion for patients with other conditions desperately seeking a drug therapy including patients with Alpha-1; Duchenne muscular dystrophy and cystinosis.
The role of Cystic Fibrosis Ireland in the drug approval process
Cystic Fibrosis Ireland has sought to play a constructive role throughout this process:
· Cystic Fibrosis Ireland proposed to Minister Leo Varadkar that Ireland should seek to negotiate with other countries in relation to new drugs to ensure the best price is reached. This idea was later adopted by Minister Simon Harris.
· Cystic Fibrosis Ireland gave the HSE five months to negotiate an agreement with Vertex (June-November 2016) as we sought to ‘put on our green jerseys’. It was only when talks broke down did we support intensive public lobbying and protests.
· Cystic Fibrosis Ireland has at all times sought to present the evidence necessary to back up our assertions of the value of these drugs.
CFI warmly thanks the Minister for Health Simon Harris TD, The Taoiseach Enda Kenny TD and the HSE for providing these ground-breaking drugs
For more information: Philip Watt, Cystic Fibrosis Ireland, tel.: 01 4962433 / 087 6370557