Budget 2018: Boost for clinical trials as patients speak about life as human guinea pigs
For people with chronic or incurable conditions, there’s only one thing scarier than the experimental treatments involved in a clinical trial — getting the placebo.
When faced with the prospect of a life plagued by burdensome pain or an early death, the promise of a clinical trial is often their only hope.
For some it’s a fast track to the latest scientific developments at no cost, but for others it can be too easily viewed as the proverbial panacea, the last great hope for a cure.
Experts say the reality is far different, sometimes clinical trials:
- Test human safety rather than effectiveness
- Are open to only a narrow set of people
- Involve lots of forms and tests
Ultimately the results can be mixed: Sometimes too small to be considered statistically significant or the drug regime is too expensive to get funding subsidies for sometime.
In Australia more than 5 million people took part in clinical trials in the decade leading up to 2015, but internationally our clinical trial rates are lacklustre compared to countries like Switzerland, Denmark and New Zealand.
Now, the number of such trials in the country is getting a boost with the Federal Government announcing $248 million in this year’s budget to boost the number of clinical trials in Australia.
It includes developing a feasibility study to create a one-stop-shop for clinical trials.
What kind of trials are already happening in Australia?
There are already websites that show some of the diverse range of clinical trials taking place here.
There are lots for common conditions like diabetes, arthritis, and cancer.
Mental health conditions also dominate, including ADHD, obsessive compulsive disorder, and social anxiety disorder.
Other common conditions like cluster headaches, plaque psoriasis, hair loss, peanut allergy and food cravings are also being studied.
One study is looking at the effect of video game playing on cognition, physical ability and sleep in older adults.
There is also a randomised control trial of early administration of anti-venom for red-bellied black snake bites.
But for many patients, being part of a clinical trial is really just about doing their part for science.
‘In my case they said it’s all gone’
Sandra Moore with her husband Rob and children Fred, Ellen and Ruby after she finished a melanoma clinical trial. (Supplied)
When school teacher Sandra Moore was diagnosed with stage-three melanoma last year her prospects were bleak.
A bit about trials in Australia
- There were more than 10,000 clinical trials between 2006 and 2015
- Most common diseases studied were cancer, heart disease and mental health
- Obesity and dementia need more study
- $1.1 billion is spent on clinical trials each year
- About one-third are of Australian trials are multi-national
- About 47 per cent are drug trials, others involve behaviour, surgeries or devices
Unusually, her cancer had presented as a lump in her upper arm rather than a suspicious mole.
“It was a surprise, I didn’t actually have a lesion,” Ms Moore said.
“The basic risk with melanoma is that if it’s in our lymph nodes you don’t know if it’s going to come back.”
Ms Moore had already lost a cousin to melanoma so she only told her family she had cancer, not skin cancer, because she didn’t want to alarm them.
Within a week of diagnosis, Ms Moore was approached by experts at the Melanoma Institute to be part of a clinical trial.
The trial, led by Professor Georgina Long, involved undergoing six weeks of treatment to suppress her immune system before she had surgery to remove the cancer from her lymph nodes.
The infusion treatment was designed to shrink tumour cells before surgery and catch any cancer cells circulating through the body.
Ms Moore said the choice to take part was easy because she knew without it, she was already facing up to a 70 per cent chance of her cancer returning.
As a maths teacher, she knew her chances were limited given a previous trial saw three people respond well, four patients have a partial response and three not respond at all.
“When your diagnosed [with] stage-three [cancer] you just assume that your life is really shortened,” Me Moore said.
“I thought ‘well, what have I got to lose’.
“Even if I was one of those with no improvement, you’re being so closely monitored they’re going to know that quickly and see if there’s anything else to do.”
Ms Moore said the team explained the risk of side effects and possible outcomes.
“You know that things can stop working,” she said. “The thing with clinical trials is the staff have all the current information.”
Even with the logistics of the trial, which involved lots of scans, blood tests and biopsies, for Ms Moore, it was very much worth it.
“In my case they said it’s dead, it’s all gone,” she said.
“Everyone is very reluctant to say cure but now everybody is starting to use that on this trial.”
For her the only side effect is a rash that comes and goes. And of course, being alive.
Trial on unique treatment for migraines
For Sydney woman Aurelie Giles, taking part in a clinical trial for migraine treatments was actually about giving back.
The 47-year-old had been getting migraines up to three times a month since her early 20s.
Aurelie Giles took part in a clinical trial after suffering from crippling migraines (Supplied)
Each time she was bed-ridden with nausea, sensitivity to light and crippling pain.
“Since I had my kids, it’s been getting worse,” she said.
“It can be pretty harrowing. I still feel sick sometimes three days later.
“Even working casually I’m in fear about when the next one is going to happen and I’m probably going to miss work.”
So when she saw an advertisement in her local paper seeking participants for a pharmaceutical clinical trial, she decided to take part.
The trial run by The George Institute for Global Health looked at whether migraines could be prevented using blood pressure or cholesterol-lowering medications.
It involved an initial consultation at Royal Prince Alfred Hospital with blood pressure and pathology tests.
Once admitted, Ms Giles had to go to take daily medication, visit the hospital once a month and keep a journal documenting details about her migraines.
Throughout the trial the fear she was actually taking the placebo loomed large.
“I was a bit nervous. I was also thinking if I do take the medication what’s going to happen. I don’t know what’s worse — having the placebo or having the effects of the medication.”
The trial has now concluded and Ms Giles won’t find out for months, maybe even years, if she was given the real medication.
She, at least, thinks she did.
“When I was doing the study the migraines went down a bit,” Ms Giles said. “Maybe I was getting the placebo, I don’t know.”
The mother of two said she was not sure whether she would encourage others to take part in a clinical trial.
Experts also caution that it was important to make sure any trial was being run by a reputable organisation and had ethics approval, because some trials overseas have gone horribly awry.
For Ms Giles, there was the inconvenience of getting to the hospital for tests but she said she unexpectedly received a $150 stipend for her travel costs.
She said she ultimately did it as a way to give back to the health system after getting excellent treatment during a gallbladder removal.
“I felt like I wanted to give back because it’s a pretty good system in Australia.
“I would definitely encourage people to take part, but it’s also very personal too.”
Clinical trial participants ‘are much better off’
Professor John Zalcberg, chairman of the Australian Clinical Trials Alliance, said the new budget funds were part of a concerted push to make Australia a preferred destination for multinational clinical trials.
He said having a one-stop-shop would make it easier for patients and doctors in regional areas.
“Even in the city, it’s hard to find out what trials are going on,” Professor Zalcberg said.
While there were criticisms of some volunteer studies overseas, he said Australia had much better regulation and “constant processes of peer review”.
Here, more often it was hospitals and universities running trials than other interests and their aim was to improve patient outcomes.
“Often it’s been said people in trials get better care and consequently have better outcomes,” Professor Zalcberg said.
“There’s no question in my mind people are much better off.”
Professor Zalcberg said it was important trials had rigorous informed consent processes and patients had reasonable expectations.
“It’s critical people understand what’s going to happen and what’s being done to them,” he said.
Professor Zalcberg said they would like to see more studies in under-represented fields like dementia, but it was a challenge.
“There’s been an awful lot of studies that have been done and they’ve all failed.”