Vertex strikes agreement with Australia for cystic fibrosis pill, ending high-profile standoff
Vertex reached an agreement on Sunday to sell its newest cystic fibrosis drug in Australia, ending a high-profile standoff over access to a life-altering but expensive rare disease drug.
As with most agreements between drugmakers and national payment systems, Vertex and the Australian Pharmaceutical Benefits Scheme didn’t say how much. But they said it will give 2,200 CF patients in the country access to Trikafta, the pill shown to dramatically improve lung function in 90% of CF patients, including 700 who weren’t eligible for previous Vertex drugs because of their particular genetic mutations.
Vertex has clashed repeatedly with national governments over access to its CF drugs, including a newspaper ink-spilling clash with the UK in which former CEO Jeffrey Leiden accused the NHS of “lack[ing] of commitment to children and young people with this devastating disease.”
By the advent of Trikafta, in 2019, the standoff with the UK and other governments’ payers — including in the US — had been resolved. Trikafta was also significantly more effective for most CF patients than previous Vertex drugs, helping mollify objections to the roughly $300,000 price tag the company has put on each of their drugs.
In Australia and other countries, though, the fight dragged on. Last year, Australian regulators approved Trikafta, but an independent pricing committee recommended the government hold off on covering it, saying “cost-effectiveness … had not satisfactorily been established.”
Cystic Fibrosis Australia, a leading local patient group, appeared to slam both Vertex and the Australian government for the standoff, asking Australians to contact their representatives while accusing Vertex of “craven commercialism.”
“Having seen the outstanding clinical trial data we can only surmise that once again ‘money’ is the stumbling block,” they wrote, urging Vertex to give wise compassionate use access while negotiations played out.
The group welcomed the new agreement, writing on Twitter that they were “on Cloud 9.” Coverage will begin on April 1.
We are still on Cloud 9 after yesterday's announcement that Trikafta has been funded on the PBS. Friday the 1st of April cannot come soon enough!https://t.co/ynPS3xjxew pic.twitter.com/pdVMXkJKbO
— Cystic Fibrosis Aust (@CFAustralia) March 28, 2022
Trikafta, however, remains inaccessible in many other countries around the globe, including large swaths of Latin America, Africa and Asia, where cystic fibrosis is less common but still present.
Researchers and advocates have pushed the company to voluntarily allow generic manufacture and sell the drug to low and middle-income countries, as Pfizer and Merck have done for their Covid-19 pills and Gilead did for its hepatitis C cure.
