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Alpha-1 Patient Action Group vigil in August. Sam Boal via Rolling News

'Inappropriate and unethical': Varadkar backs life-saving lung disease drug for some users after HSE fails to fund it

21 Alpha-1 patients in Ireland have been receiving Respreeza up until now through a compassionate use programme.

TAOISEACH LEO VARADKAR has said it would be “inappropriate and unethical” if the company providing a life-saving drug to patients with a chronic lung condition was to cut off its supply to some Irish patients.

21 Alpha-1 patients in Ireland have been receiving the Respreeza drug up until now through a compassionate use programme.

However, the HSE failed to reach a price agreement with its manufacturer, CSL Behring, and it has decided not to fund the roll-out of Respreeza to all Irish patients.

In a statement in August, the HSE said that it did not find the clinical benefit of Respreeza to be “sufficiently strong in the context of the proposed cost and budget impact”.

It remains unclear if the manufacturer will cut-off the Respreeza supply to those under the compassionate use programme, as the HSE has received no formal notification of the intention to discontinue access to the compassionate scheme, according to Taoiseach Leo Varadkar.

Patients with the condition, also known as genetic emphysema, have a deficiency of the Alpha-1 antitrypsin protein, which can lead to severe lung, liver and skin problems. Most people with the condition develop emphysema or chronic obstructive pulmonary disease (COPD) at some stage in their lives.

The Alpha One Foundation said the drug “slows the progression of lung disease associated with Alpha-1″ and can lead to “fewer chest infections and less time spent in hospital”.

Speaking in the Dáil today, Varadkar condemned the possibility of the removal of the drug from those on the compassionate use programme:

It is completely inappropriate and unethical for any company to link reimbursement decisions to a compassionate access scheme and I would consider it unscrupulous if the company was not to honour any commitments which it has made to patients.

He said that the Minister for Health Simon Harris has directed the HSE to “seek assurances from the relevant hospital, Beaumont Hospital, that appropriate care arrangements are in place in the event of the access programme being discontinued by the company”.

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‘Insufficient evidence of benefits’

Despite Varadkar’s strong words in relation to those under the compassionate scheme, he backed the HSE’s decision not to reimburse the drug.

“The HSE has carefully considered the pricing and reimbursement of the medicine through its decision-making process and there is insufficient evidence to suggest patients would drive a clinically meaningful benefit from this treatment,” he said.

He said that at the heart of the assessment process is “the rationale that decisions about the reimbursement of medicines in Ireland are made on an objective and scientific basis, taking into account expert opinion, as appropriate, and recognising the health needs of the population and other factors”.

Speaking in August when the decision was made not to reimburse the drug, Professor Michael Barry, Head of the National Centre for Pharmacoeconomics said:

“We know this is a difficult time for patients who have been affected by this decision. However, the cost-effectiveness of this drug has not been demonstrated following submissions from the manufacturer.

“The HSE has to consider the wider population and needs to manage the full range of health services to all of the other patient groups within the finite resources at its disposal.”

Calls for action

The efficacy of Alpha-1 has been recognised by the European Medicines Agency and it has already been approved in 12 European countries. It’s estimated that the drug could benefit up to 60 patients in Ireland.

The Alpha-1 Action Group and the Alpha One Foundation took part in two hour-long vigils in August – the first at the head office of the HSE, and the second at the Department of Health’s Hawkin’s Health offices.

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They called on the HSE and Minister for Health Simon Harris to “put patients first and to hammer out a deal with the pharmaceutical company on making Respreeza available in Ireland”.

“Alpha-1 patients are becoming increasingly concerned and upset as they await a positive decision,” Geraldine Kelly, CEO of the Alpha One Foundation said.

‘We wouldn’t be here without it’

Johnny Hannan (67), from Mallow in Cork, is one of the patients who took part in the clinical trial.

“Respreeza has been clearly deemed to be of benefit for patients, so while policy-makers here might query its effectiveness, you have to wonder if they just think that we patients are not worth the investment,” Hannan said.

“For those of us lucky enough to have had access, we know we wouldn’t still be here without it. Respreeza has the potential to add years back onto people’s lives. For us, it really is a lifeline.”

About 350 people in Ireland have been diagnosed with the most severe form of Alpha-1. It’s thought about 3,000 people have this form of the condition but are yet to be diagnosed, while thousands more have a moderate deficiency.

Read: ‘We wouldn’t be here without it’: HSE will not fund life-saving lung disease drug

More: ‘I’m living proof it works’: Patients with severe lung condition call for access to ‘life-changing’ drug

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