Australia's Pharmaceutical Benefits Advisory Committee (PBAC) has told the government that, because of new information about Boehringer Ingelheim's oral anticoagulant Pradaxa (dabigatran), "it is of a mind to rescind its March 2011 recommendation" that the drug should be made available on the Pharmaceutical Benefits Scheme (PBS).

The experts' warning comes in a report on the use of anticoagulant therapy conducted for the government by former PBAC chairman Emeritus Professor Lloyd Samson. In its original March 2011 recommendation, the Committee had said that Pradaxa should be available on the PBS for the prevention of stroke or embolism in certain groups of patients with atrial fibrillation.

In its recommendation, the PBAC had noted that Pradaxa: - represented a cost-effective therapy and its use could lead to reductions in morbidity; - the opportunity cost to the government of its listing would be significant; and - a number of patients who are reluctant to take warfarin because of stringent monitoring requirements and interactions with other drugs and foods, but who should be taking oral anticoagulation, would now be treated with Pradaxa and this would likely lead to additional benefits and costs not measured in the trial data presented to the panel.

However, Prof Sansom's new report says that more work is needed before new oral anticoagulants (NOACs) such as Pradaxa can be listed on the PBS. His review finds that some use of Pradaxa in real-world settings has been different to the clinical trial evidence presented to the PBAC in March 2011, including the average age of patients who are likely to use it, the dose they are likely to use and treatment that patients would otherwise be receiving.

In light of the latest clinical information, the review has concluded that the net benefit of NOACs in clinical practice and the subsequent impact on cost-effectiveness "is uncertain," and that the PBAC should review its advice to list Pradaxa on the PBS.

Health Minister Tanya Plibersek said that the PBAC has also told her that, based on the new information, "it is now concerned about whether [Pradaxa] represents value for money at the price offered by the company."

"Because of this, the PBAC has now advised me that it is of a mind to rescind its March 2011 recommendation" for Pradaxa, she added.

The Committee feels there is an appropriate place in clinical treatment for the newer anticoagulants, but would like to see further economic modelling to show their value, in light of the new information, she said. 

"Mindful of the need to finalise its recommendation as soon as possible," the Committee will allow Boehringer Ingelheim and the sponsors of other NOACs to make a late submission to its meeting in March, the Minister added.

"It's important the companies have the opportunity to respond to the Committee's comments, and provide the data and economic modelling it requested," she said.

However, an industry leader said the continued review was "a two-year bureaucratic merry-go-round that has got to stop."

The PBAC recommended Pradaxa for listing on the PBS nearly two years ago, and the government then ordered a review of that decision, said Dr Brendan Shaw, chief executive of research-based industry group Medicines Australia. 

Now the government is "demanding a review of that review - essentially we're having a review of a review of a review," he said.  "If this is a prototype for listing new medicines on the PBS, it's a debacle. It undermines confidence in the PBS listing process. People should not have to wait this long to have access to a medicine that has already been deemed cost-effective by an independent expert committee."