The challenges posed by pharmaceutical companies seeking massive reimbursement for life-changing drugs has led to a second situation this week where patients face losing access to vital treatment.

The threat by drug firm CSL Behring to withdraw Respreeza, described by respiratory consultant Gerry McElvaney as “the only treatment for genetic emphysema in my lifetime”, follows a decision by the National Centre for Pharmacoeconomic Evaluation (NCPE) not to recommend State reimbursement of the drug.

The decision has led to calls for Health Minister Simon Harris to intervene on behalf of those with genetic emphysema, known as Alpha-1.

Earlier this week, Mr Harris told the Seanad he is collaborating with other EU health ministers to try and force drug companies such as Vertex to introduce viable pricing. Vertex has developed a breakthrough CF drug called Orkambi but is reported to be seeking reimbursement of up to €160,000 per patient per annum, which the HSE is not prepared to approve.

Both the HSE and Vertex indicated last night that they would meet on Wednesday for further discussions.

“The HSE and Vertex are committed to finding a definitive solution that provides rapid access to Orkambi for people in Ireland,” said a spokesperson for Vertex.

The Alpha-1 Foundation is now calling on the minister to review the process for assessing new therapies and to consider the implementation of outcomes-based models of access.

It is also calling on CSL Behring to reduce the price of Respreeza, reportedly around €84,000 per patient, excluding Vat. Currently, 21 patients are in receipt of the drug on a compassionate basis, 19 of whom took part in a clinical trial between 2006 and 2014, for which Prof McElvaney was principal investigator in Ireland.

Prof McElvaney has been treating genetic emphysema since 1986 and Respreeza was “the first time we’ve ever shown we can slow it down”.

Studies have shown the drug reduces the severity of chest infections in patients, and associated hospital admission. Another 40 patients here could benefit. Alpha-1 Foundation CEO Kitty O’Connor has called on CSL Behring to lift its January 31 deadline for withdrawing the therapy — it has been available while the NCPE deliberations were under way.

“These patients have been told by the pharmaceutical company a number of times over the last six months that supply was being stopped — the latest information is that at the end of January, patients will be told whether the treatment will continue,” Ms O’Connor said.

Prof McElvaney said patients are “terrified” of losing access.

“They have been on this drug for almost a decade and it’s been very beneficial. It will be very traumatic for them if it’s withdrawn.”

The HSE received the NCPE report on Respreeza yesterday. A spokesperson said it will “consider the NCPE report carefully in advance of deciding on the next steps and in advance of progressing through the HSE decision-making processes”.


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