Around the Globe

Australia’s New Clinical Trials Activity Initiative
Focus on Rare Diseases and Cancers
Richard Day
University of New South Wales

n March 2022, the Australian Government announced the Clinical Trials Activity initiative. This initiative will support national and international clinical trials of new treatments, initially focused on rare cancers and diseases as well as unmet medical needs, and aims to attract investigator-led, international clinical trials in any disease facing unmet need. It has been funded through the Medical Research Future Fund (MRFF), a $20 billion (AUD) long-term investment supporting Australian health and medical research that has transformed health and medical research in Australia since its inception in 2015.

In essence, this new funding of $750 million (AUD) commencing in 2022-23 provides “add-on” support for important clinical trials in the wide range of diseases and unmet health needs targeted by MRFF for grant support over the next 10 years. As the MRFF issues “calls” for research in individual disease areas, opportunities to support international investigator-led clinical trials in the disease will be promoted. For example, the call on Multiple Sclerosis Research ($18 million AUD available) announced in April 2022 presents an opportunity for an innovative clinical trial to received substantial assistance. Two of the four “streams” in this call seek applications for clinical trials focused on preventing or treating Epstein-Barr virus infection via vaccines or repurposed pharmaceuticals.

Upon the recent annual celebration of International Clinical Trials Day (May 20), it is timely to note that the Australian Clinical Trials Alliance (ACTA) has announced a new chairman, Professor Steve Webb, and a new chief executive officer, Dr. Stewart Hay. Webb follows founding chair Professor John Zalcberg, who has been an outstanding leader of this organization since its establishment. ACTA “is the national peak body for clinical trial networks, coordinating centres, and quality registries conducting investigator-initiated clinical trials,” and brings together clinical researchers, governments, policymakers, and consumers regarding all aspects of investigator-led clinical trials. For those interested in improving the quality and relevance of clinical trials in Australia, ACTA is always a good place to start.

2021 Trials of the Year

Awarding the ACTA prize for best clinical trial published in the previous calendar year is always an eagerly awaited and hotly contested annual event. Unusually, two clinical trials shared the 2021 Trial of the Year prize: proPSMA and the pneumothorax clinical trial.

The proPSMA trial aimed to achieve better outcomes for men with prostate cancer. To direct more precise treatment of advanced prostate cancer, the study randomized men to conventional CT and bone scan imaging or PET-CT scanning using a gallium marker for a prostate-specific surface antigen (PSMA) tumor marker. The proPSMA results were definitive: PET-CT was significantly more accurate, sensitive (85 percent versus 38 percent), and specific (98 percent versus 91 percent), and led to more changes in treatment (28 percent versus 15 percent). Half as much radiation (8.4 v 19.2 mSv) was another benefit. “This method is already the new standard of care for assessment of men with prostate cancer,” explained Professor Michael Hofman, director of the Prostate Cancer Theranostics and Imaging Centre of Excellence (ProsTIC) at the Peter MacCallum Cancer Centre. The new imaging method is already incorporated into national and international clinical guidelines, and the new label, Ga-68 PSMA, has been approved by FDA.

Pneumothorax (collapsed lung) affects thousands of Australians each year. It means visits to the emergency room to get relief from the severe pain and breathing difficulty. A tube is inserted through the chest wall to remove the air that has leaked from a tear in the surface of the lung into the space between the chest wall and the collapsed lung. This procedure, although the standard of care, is usually painful, can damage organs and nerves, and is a risk for infection and possible additional surgery. Counterintuitively, and contrary to long-standing “usual practice,” “conservative treatment,” even in the face of large collapse, has been shown to result in better outcomes in a landmark study by Professor Simon Brown of the Centre for Clinical Research in Emergency Medicine at Royal Perth Hospital in Western Australia and colleagues. Brown said: “We’ve been putting tubes into people with collapsed lungs since the beginning of the 20th century, thinking we were doing our best to treat the condition; … the study makes it clear that conservative treatment is the best approach even when the lung collapse is large.” In this controlled study across 39 hospitals, 85 percent of those people in the conservative “hands-off” group (162; observation and analgesics) had a “non-inferior” outcome (resolution of pneumothorax within 8 weeks) compared to standard interventional lung drainage (154). Rates of complications were significantly lower. These results have already been picked up by Australia’s national Therapeutics Guidelines, which now recommend initial conservative management independent of the size of the primary spontaneous pneumothorax.

A Less Positive Announcement

Federal funding for NPS MedicineWise, the standout national and internationally recognized organization established to deliver “quality use of medicines (QUM)” outcomes nationally, has been withdrawn. This announcement came as a major shock to the medical and pharmaceutical communities across Australia—and especially the NPS, who had no warning about this budgetary decision. This secretive decision seems mysterious after the recent Sansom review of the NPS that recommended NPS should continue operations as a critical contributor to achieving QUM nationally. National organizations and individuals are rallying to try to reverse this decision, a position for which there is much public and private support.