Clinical trials are tomorrow’s medicine
Endocrinologist Professor Rory Clifton-Bligh has been involved in clinical trials at Royal North Shore Hospital for over 20 years, focusing on conditions such as osteoporosis, thyroid cancer, and diabetes. Patients in the endocrinology clinic have sometimes accessed medication 10 years ahead of other Australians. Many of these drugs have gone on to be listed on the Pharmaceutical Benefits Scheme. “The trials have all been quite transformational in their areas,” he says.
Addressing rare diseases Unfortunately, patients with rare diseases have often faced limited treatment options. However, over the past 10 to 15 years, Rory has seen a significant increase in the number of clinical trials for rare diseases, including some involving his department. The shift has flowed on from the Orphan Drug Act , a US law passed in 1983 that incentivised drug development for rare diseases, making it easier for the US Food and Drug Administration (FDA) to grant approvals, as well as encouraging greater investment. participants for several trials spanning rare bone, thyroid cancer, cholesterol, and lipid diseases. “It’s a fairly diverse portfolio of clinical trials in the rare disease space,” he says. Recruiting subjects can be challenging but multicentre study centres around the world help. Rare diseases are defined differently in parts of the world. In Australia, it is considered rare if five in 10,000 people have the condition while in the US, it’s rare if it affects fewer than 200,000 people. Paradoxically, rare diseases are not rare. Rory points out that rare diseases are more common than people think. Importance of trials He emphasises the broader importance of clinical trials for providing access to new therapeutics and enhancing clinical practice. “What we call best practice is yesterday’s medicine. Clinical trials are tomorrow’s medicine.” Current trials and recruitment challenges Rory and his team are currently enrolling
Professor Rory Clifton-Bligh
12 Tomorrow’s medicine today
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