London, UK – Before a drug can be sold in Australia, it must receive approval from the regulator: the Therapeutic Goods Administration (TGA), typically requiring data from a large and lengthy Phase 3 randomized clinical trial. Public funding additionally requires approval from the Pharmaceutical Benefits Advisory Committee (PBAC), for whom value for money must be demonstrated.

The full study, “How Data Packages Lacking Phase III Pivotal Trial Data can Support Regulatory Approval and Reimbursement for Oncologics in Australia,” was published in Value in Health Regional Issues focusing on Asia, Volume 6.

In Australia, if Phase 2 trials indicate potentially substantial clinical benefits over the relevant comparator, regulatory approval and public reimbursement can be achieved for oncology drugs without requiring supportive Phase 3 trial data.

The study objective is to determine whether and under what circumstances oncology drugs lacking comparative Phase 3 data can achieve regulatory approval and public reimbursement in Australia.

Publically available documentation for 6 oncology drugs TGA-appraised without Phase 3 data were extracted, 5 of which obtained regulatory approval. The EMA and FDA (corresponding regulators in Europe and USA) issued recommendations on these indications an average of 1 and 2 years earlier, respectively. PBAC appraised 6 oncology drugs on such a data package, four recommended for public reimbursement and two rejected.

“As we have previously shown for Europe and America, in Australia, early regulatory and reimbursement approval can be achieved for oncology drugs without Phase 3 data. However, both the EMA and FDA appear to make more drugs available, earlier in their developmental pathway, on this basis” says Richard Macaulay, PhD, Senior Consultant of PAREXEL International.

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