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# Cystic Fibrosis
'Undue fear and confusion': Read the emails Vertex sent to Department of Health about Orkambi
A deal on the cystic fibrosis drug was finally reached last month.

jill-6-2 Jillian McNulty CF campaigner Jillian McNulty Jillian McNulty

THE HSE AND Vertex Pharmaceuticals last month struck a deal to make Orkambi and Kalydeco available to cystic fibrosis (CF) patients from 1 May.

Campaigners welcomed the development, which came after long-running negotiations.

CF, an inherited chronic disease that primarily affects the lungs and digestive system, can greatly impact the lifespan of those with the debilitating condition.

About 1,200 children and adults have CF in Ireland, which has more cases of it per head of population than any other country.

Cystic Fibrosis Ireland has said around 500 people in this country have the potential to benefit from Orkambi (patients aged 12 and older) and Kalydeco (children aged two to five).

The cost of Orkambi, about €159,000 per patient per year, was the primary sticking point. In 2016, the National Centre for Pharmacoeconomics (NCPE) assessed the drug and didn’t deem it cost-effective. The deal for both drugs is expected to cost the State in the region of €300 million over the next five years.

Under the Freedom of Information Act, has received copies of the emails exchanged between the Department of Health and Vertex prior to the agreement being reached.

Media reports 

On 27 November, the Sunday Business Post reported that the HSE’s drugs committee was set to recommend against funding the medication.

Three days later, Simon Lem, Vertex’s Vice President for North Europe, wrote to Health Minister Simon Harris about the article.

undue fear Department of Health A section of one of Simon Lem's emails to Minister Harris Department of Health

Lem stated: “Vertex was disappointed to learn via the media over the weekend that the Health Service Executive (HSE) is considering not recommending Orkambi for the treatment of CF in the Republic of Ireland.

“Having continually engaged in good faith with the HSE since Orkambi was licensed over one year ago, Vertex had hoped that an agreement was within reach and is surprised by the latest developments…

“Vertex subsequently met with HSE representatives six times in five months, presenting a range of specific workable proposals that could expedite access for all eligible patients. At no time during the process did we receive a counter-offer or guidance from the HSE on decision making criteria.

“Proposals from Vertex included, for example, guarantees of budget certainty for the HSE, novel access schemes and ways to reduce the timelines on future reimbursement decisions.

“Vertex therefore refutes allegations by the HSE of failing to meaningfully engage and, the publically quoted budget impact figure of €392m is incorrect, unrealistic and does not reflect discussions to date with the HSE.”

Lem said Vertex has “spent over 15 years and hundreds of millions of dollars to date developing medicines for CF”, adding: ”Our mission is to cure the disease and we have a pipeline of medicines we are developing in pursuit of this mission.

Orkambi remains an important step forward for the treatment of CF, helping patients stay out of hospital, breathe more easily, gain weight and live longer lives. Recent clinical data has shown that Orkambi reduces annual lung deterioration by 42%.

“The present situation and manner in which information or misinformation is being communicated is causing undue fear and confusion for the patient community. We remain committed to finding a solution.

“We therefore call on you, as the responsible Minister, to intervene directly ahead of a decision by the HSE in the mutual interests of patients, clinicians and industry.”

‘Meaningful engagement’

In an email sent to Lem on 7 December 2016, Shaun Flanagan, Chief Pharmacist of the HSE’s Corporate Pharmaceutical Unit, asked if representatives for Vertex were “available for additional discussions”.

He added that the HSE was informed two Vertex staff members, Rebecca Hunt and Dr David Gillen, “told a small group of patients at a meeting on 5 December that Vertex were prepared to discuss further options”.

“Can you confirm if this is correct and whether Vertex have moved from the position of 1 December 2016 when you confirmed to us that the “final offer” remained the final offer.”

11/10/2016. Cabinet Meetings Sam Boal / Minister for Health Simon Harris Sam Boal / /

In response, Lem wrote: “We would like to continue our meaningful engagement, and would be available to meet at your earliest convenience.

“We are eager to reach a resolution as soon as possible, so with that in mind, would request the HSE and Vertex ensure the appropriate people are in the room who will be able to conclude an agreement, should one be reached. We are also open to having a representative from the Department of Health in attendance and suggest this could be beneficial to the process.”

In a later email sent to the HSE and Health Minister, Vertex’s Senior Vice President Simon Bedson wrote: “We are very much looking forward to meeting with the HSE and to putting an end to the uncertainty and anxiety that this process has caused to people living with cystic fibrosis (CF) and their caregivers.

Over the past year as we have been working toward an agreement with you, we have gotten to know many of these patients. With each passing day, they are progressively getting sicker and losing lung function. We share their sense of urgency to join the thousands of patients around the world who already have access to Orkambi.

“We believe we have a shared goal – to help these patients obtain Orkambi as soon as possible. As such, we feel strongly that the outcome of our next meeting should be a definitive resolution of this matter, which has already taken too long.

“To achieve this resolution, it’s critical that this meeting happen with the right people around the table, including the senior decision makers from both Vertex and the HSE.”

Bedson also asked the HSE to commit to postponing its final decision on the deal beyond 12 December, “pending resolution of our discussions”.

‘Deeply concerned’

In emails exchanged on 1 February, Lem wrote to Minister Harris, stating: “We are deeply concerned about media reports this morning that the HSE is considering rejecting our proposal that was developed after careful consideration and with input from the agency. We are acutely aware of the distress caused by this sort of speculation and have contacted the HSE to request urgent rassurance that these reports are unsubstantiated.

“More than two weeks have passed since we submitted our proposal to provide access to Kalydeco and Orkambi to the HSE. In this time, We have received daily calls from patients, and their families desperate for a solution. We understand that the proposal is going through the process but we share the frustrations with the apparent lack of urgency to make these medicines available to those who need them.”

concerned Department of Health Department of Health

Lem added that thousand of patients with CF have access to these drugs in other countries and “Ireland should not be an exception”.

In response, Harris’ private secretary David O’Connor told Lem: “The Minister has asked me to reply to thank you for your email and to say that the HSE has indicated that no decision has been reached regarding Orkambi and the matter is currently under consideration by the HSE Directorate. You may be interested to note that the Taoiseach also clarified this matter in the Dail this morning.”

The Dáil exchange he was referring to saw Enda Kenny tell Labour leader Brendan Howlin: “The suggestions in some media reports that the discussions and negotiations have broken down are not true. The HSE re-entered into negotiations with Vertex, the manufacturer of Orkambi, in December following a decision by HSE leadership that it was unable to reimburse Orkambi at the price offered at that time by the company…

The HSE subsequently met with Vertex in December and in January with a view to significantly reducing the cost of Orkambi for cystic fibrosis sufferers so that the State is in a position to fund the drug and provide it for Irish patients.

Kenny went on to say the HSE was considering the issue, adding that a decision on reimbursement would “be made on objective, scientific and economic grounds”.

“Decisions are now based on clinical and medical accuracy in light of the quality of life afforded to the patient and the extent of the improvement,” he added.

‘An extraordinarily difficult time’

Negotiations continued in the subsequent weeks, before a deal was reached on 11 April.

At the time, Harris said he recognised this had been “an extraordinarily difficult time for cystic fibrosis patients, for their families and for their friends as they have been waiting for this process to conclude”.

“With regard to Orkambi for the treatment of cystic fibrosis patients aged 12 and older, following an application by the company, the treatment was assessed in line with the statutory process, following which the HSE called on Vertex to re-enter negotiations.

“Further meetings were held with the manufacturer in December and early January. The discussions with the company broadened to include Kalydeco and further treatments for CF patients…

“I am pleased that we are now in a position to reassure people we are now in the concluding stages of an agreement as I outlined earlier and expect the further work in the coming days will bring this matter to finality,” Harris said.

Read: ‘We are so, so happy that it’s finally here’: Cystic fibrosis patients celebrate deal on Orkambi

Read: After the transplant, I looked in the mirror and my eyes weren’t yellow anymore, they were white’

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