- 2 years ago
18 June 2021
Long-term data, patient numbers and a Managed Access Program for Vertex’s latest cystic fibrosis combination therapy TRIKAFTA (elexacaftor/tezacaftor/ivacaftor) are proving to be the stumbling blocks delaying PBAC recommendation, a stakeholder meeting has concluded.
The one-hour meeting held in a Canberra hotel on Thursday was orchestrated by patient organisation Cystic Fibrosis Australia (CFA) “to consider the hurdles and challenges blocking a positive recommendation by the PBAC” as the outcome from the PBAC’s May intracycle meeting at which the therapy was deferred for a second time will be released.
The stakeholder meeting was also attended by Department of Health and PBAC representatives along with senior Vertex executives, clinician Professor Peter Wark and two CF patients. Both CFA and Vertex concluded the meeting was constructive in moving the therapy closing to a PBAC positive recommendation.
“Everyone was committed to open dialogue and to exploring new options – after all, we share a deep urgency about the fate of Trikafta in Australia,” CFA CEO Nettie Burke said.
“The complexity of the issue meant that no firm and unanimous resolutions could be arrived at, but the key holdups can now be clearly outlined.”
Burke has urged Vertex to move quickly on the Department’s requirements and resubmit the drug to the July PBAC meeting.
“Our consumer representatives, together with CFA, implored Vertex to resubmit to the July PBAC meeting and to be creative and innovative when considering MAP criteria,” Burke said. “Vertex needs to make this work. We will wait and see if our pleas are heard and acknowledged.
“Trikafta is still not in our hands, but this week we have moved even closer to broad access. All parties at the Stakeholder Meeting are campaigning strategically and wholeheartedly for a swift resolution.”
Burke said the meeting identified the three issues “still hampering a positive recommendation” to be the long term data for Trikafta, the number of patients expected to use the drug, and the details of a Managed Access Program (MAP). A MAP requires real-world data to be collected to enable future assessment of the effectiveness of the therapy.
Burke committed CFA to use of Australian Cystic Fibrosis Data Registry (ACFDR) data to provide regular information on patient numbers, including Trikafta onboarding and discontinuations.
A spokesperson for Vertex said the company was “pleased to have participated in yesterday’s stakeholder meeting” and thanked Burke for organising.
“The discussions helped raise some of the issues we face in securing access to Trikafta for Australian CF patients. We are pleased that some progress was made in the meeting, however, there is still more that we all need to do to ensure those that could benefit get access to Trikafta,” the spokesperson said.
“The process of funding innovative medicines in Australia is complex and can take time but is a shared goal for all stakeholders in the health system. Vertex is committed to working with the PBAC and the Department of Health to find flexible and creative solutions to ensure all eligible patients who can benefit from treatment have government-funded access to Trikafta as quickly as possible.”
CFA has previously asked that the company provide compassionate access to Trikafta if it does receive a PBAC positive recommendation, with patients echoing the request at the meeting, saying delays negatively impacted peoples’ health and life expectancy.
Professor Wark outlined how Trikafta compared with other modulators and information on international health outcomes, while patients gave their real-world experiences with the therapy.
“It was clear to all that Trikafta will change lives for the better and extend lives so that people with CF can have a fulfilling future,” Burke said.
It is encouraging that our HTA system allows for Stakeholder Meetings with key protagonists. Building relationships aids negotiations and hopefully, this will speed up access for the CF community.