AUSTRALIA - CANCER DRUG REIMBURSEMENT ON PHASE 2 DATA

Published Jul 21, 2015
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.

Related Stories

ISPOR Launches New Content on Whole Health

Sep 23, 2025

ISPOR announced that it has launched new website content on whole health, a topic of increasing importance as health systems across the globe grapple with providing the best possible healthcare to patients within constrained budgets.

Landmark Analysis in Value in Health Uncovers Potential Research Efficiency Gains

Sep 15, 2025

Value in Health, the official journal of ISPOR announced the publication of a landmark scoping review that provides a comprehensive mapping of patient preference studies across key medical domains, revealing the first robust evidence base for advancing meta-analyses and benefit transfer methods in healthcare decision-making.

ISPOR Real-World Evidence Summit 28-30 September 2025 | Tokyo, Japan

Sep 9, 2025

ISPOR announced details for its ISPOR Real-World Evidence Summit 2025: Through The Lens of Asia Pacific. The Summit brings together top experts, decision makers, and industry leaders to explore the latest breakthroughs, share cutting-edge research, and discuss innovative solutions to the region’s most pressing healthcare challenges.
Your browser is out-of-date

ISPOR recommends that you update your browser for more security, speed and the best experience on ispor.org. Update my browser now

×