Kolling Institute researcher and Royal North Shore Hospital endocrinologist Professor Rory Clifton-Bligh has been involved in clinical trials for more than 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 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 to grant approvals.
Current trials and recruitment challenges
Rory and his team are currently enrolling 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 patients can be challenging but multi-centre 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.
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.”