Medicines

Fast-tracked haematology drugs ‘of little additional benefit’: academic


Many high cost haematology and oncology drugs brought into Australia with fast-track TGA approval have offered little additional therapeutic benefit over already available medicines, a study suggests.

Based on publicly-available drug evaluations, the findings have prompted questions over the use of programs like the provisional approval pathway, which allows medicines to receive temporary registration in Australia without full phase III trial efficacy and safety data.

Since July 2017, drug companies have also had the option of registering prescription medicines via a scheme called the priority registration pathway, with a target approval timeframe of 150 working days compared to the standard 255-day turnaround.

Theoretically reserved for urgent applications, this followed a Federal Government review which raised concerns that Australian patients were being forced to wait years for new drugs due to red tape.

The study found 33 medicines had received fast-track registration since the two schemes came online five years ago, representing a quarter of the 132 drugs approved by the TGA in that time.

However, just four drugs offered major additional therapeutic value over options already on offer, said the study author, Associate Professor Joel Lexchin, a health policy analyst at York University in Canada.

These were the antiandrogen medication apalutamide, the enzyme replacement cerliponase alfa, emicizumab for haemophilia A and remdesivir for COVID-19.

One lung cancer drug, lorlatinib, was classified as having a moderate additional therapeutic value, while 11 drugs, mostly for haematology and oncology indications , were found to add little-to-no benefit.

These included acalabrutinib for Mantle cell lymphoma, gilteritinib fumarate for acute myeloid leukaemia and polatuzumab vedotin for large B-cell lymphoma.

A further 17 drugs including enasidenib for acute myeloid leukaemia and zanubrutinib for Mantle cell lymphoma were yet to be evaluated across the three authorities consulted, namely Canada’s Patented Medicine Prices Review Board, the German Institute for Quality and Efficiency in Health Care and Prescrire International, an independent French drug bulletin.

Nevertheless, the evidence suggested many of the ‘priority’ drugs did not warrant expedited approval, Associate Professor Joel Lexchin wrote in Australian Health Review.

“Since their inception, these two pathways have been used to approve the majority of antineoplastic agents,” he wrote.

“They have succeeded in accelerating the review process, but the question remains as to whether these medicines have a larger additional therapeutic value compared to medicines approved through either the standard or orphan pathways.”

It was the same story in Europe and Canada, while 88% of new medicines in the US were approved under the FDA’s priority or accelerated approval pathways, he said.

Notably, a recent analysis had found only 20% of  oncology drugs approved under the FDA’s accelerated pathway showed a survival improvement in confirmatory trials, he added.

But the expedited pathways had succeeded in speeding up the registration process.

In Australia, approvals via the standard pathway took 200 working days on average, compared to 130 working days for the priority pathway and 144 working days for the provisional pathway, Professor Lexchin said.

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