Bittersweet moment for families as drug reimbursed
Almost a year to the day since Anna Cassidy died “gasping for breath”, the drug which had helped sustain her has finally been secured for life for 19 patients who, like Anna, suffer from genetic emphysema.
It is a bittersweet victory for her family, and for the family of Marion Kelly, who also died during a period last year when none of the then 21 patients who took part in a clinical trial could access the therapy.
Marion’s family, who are also approaching the first anniversary of her death, welcome confirmation that the battle to access Respreeza is over for the remaining patients, but say her death was “needless”.
“However, notwithstanding the needless loss of her life and all it has done to our family, today we are hugely consoled by the fact that the others, Marion’s friends and companions in this fight, can be at ease after the HSE finally listened and agreed to put this drug back on,” her family said in a statement.
Anna’s family said that while they welcome the “long-awaited” HSE decision to supply and administer Respreeza, the news “comes with sadness for us as a family, as it will be a year on the 18th November, since Anna tragically passed away, followed by Marion Kelly, both during the period of time without Respreeza”.
“We know that Anna would be delighted with this news for the other patients and their families,” the Cassidy family statement said.
“We as a family have campaigned together with Marion Kelly’s family, the Alpha-1 Foundation and alpha patients themselves to achieve this goal. We would like to thank the HSE for finally doing the right thing, and we will continue to plead for other Alpha-1 patient’s that aren’t included in this group.”
A further 40 patients stand to benefit from the treatment should the State approve full reimbursement.
Respreeza, the only drug proven in a clinical study to slow the progression of genetic emphysema — known as Alpha-1 — was approved by the European Medicines Agency in 2015.
It is available in 12 European countries, but the Irish State had refused to reimburse it after the National Centre for Pharmacoeconomics (NCPE) said its cost-effectiveness “has not been demonstrated”.
The State’s refusal meant that 21 patients, who had availed of Respreeza for more than a decade via clinical trial, had to go without when a row erupted between the HSE and the drug’s manufacturer, CSL Behring, last year.
CSL Behring had continued to make the drug available to trial participants after the trial ended under a compassionate-use programme intended, it said, “to bridge the gap” until the HSE made a decision on whether to reimburse the drug.
However, when agreement could not be reached on what represented a fair price — circa €66,000 per annum per patient — the patients lost out.
The final decision not to reimburse was made in December 2016 after which patients had compassionate access until the end of September 2017, when CSL said it was no longer prepared to cover the cost of administering the weekly infusions.
The HSE also refused to pick up the tab.
During this standoff, when patients had no access to Respreeza over a six-week period, and while the drug sat in a warehouse in Dublin, Anna Cassidy passed away, aged 68, on November 18, 2017.
Two weeks later, on November 1, Marion Kelly, aged 53, lost her fight for life.
Within 24 hours of Marion’s death, the HSE agreed to pay for the administration of Respreeza.
Since then, campaigners such as the Kelly family and the Cassidy family and the Alpha-1 Foundation and Johnny Hannan from Mallow, Co Cork, who has Alpha-1 and is a member of the Alpha-1 Patient Action group, have lobbied tirelessly to have the drug reimbursed.
Yesterday, Johnny said news that the drug will be reimbursed indefinitely is “fantastic”.
“A weight has been lifted off my shoulders,” he said.
However, victory was tinged by the loss of “two wonderful women”, Anna, from Mountcharles, Co Donegal, and Marion, from Nenagh, Co Tipperary.
“They would probably be here today, only for the intransigence of the HSE,” said Johnny.
Geraldine Kelly, chief executive of the Alpha-1 Foundation, said that while they welcomed the decision by the HSE/Department of Health and CSL Behring “to approve the continued supply and administration of the life-changing therapy” for 19 patients, there were “a further 40 or so patients” who would benefit from this treatment.
For these patients, the wait continues until a re-submission by CSL is made to the NCPE for full reimbursement approval, Ms Kelly said.
Marion’s family said the agreement to provide the drug on a long-term basis to 19 patients meant her death “was not entirely in vain”.
“This is a campaign that Marion started and was very passionate about while she was alive and would have wanted us to fight until a positive resolution was found for her fellow Alpha 1 patients” they said.
“Thankfully, today that fight ends and Marion can rest in peace.”
Yesterday. the HSE said it was “finalising an agreement to continue with the compassionate access arrangement in relation to this group of patients at the request of their clinician”, Prof Gerry McElvaney at Beaumont Hospital.
