People with Crohn's disease and ulcerative colitis are missing out on more effective therapies as pharmaceutical companies increasingly bypass Australia, leaving some patients facing avoidable surgery and lifelong complications.
Australia’s widening medicines access gap is leaving people with Crohn’s disease and ulcerative colitis without some of the world’s most effective treatments.
And a leading gastroenterologist warns that pharmaceutical companies are increasingly deciding not to launch innovative therapies in Australia because the market is no longer commercially attractive.
This follows the release of Medicines Australia’s Bitter Pill report, which found only 25% of innovative medicines launched globally over the past decade have been reimbursed through the Pharmaceutical Benefits Scheme (PBS), compared with 46% in the UK and 88% in the US.
The report argues that declining investment in innovative medicines and structural problems within Australia’s reimbursement system are discouraging companies from bringing new therapies to Australian patients.
It identifies 18 medicines that companies have either delayed or decided not to pursue in Australia because of the current environment.
For the estimated 180,000 Australians living with inflammatory bowel disease (IBD), clinicians say the consequences are particularly acute because no single medicine works for every patient and many eventually lose response to treatment.
Associate Professor Réme Mountifield, a gastroenterologist, Crohn’s & Colitis Australia board director, and clinical and research lead of the inflammatory bowel diseases service at Flinders Medical Centre, says having access to multiple treatment options was fundamental to modern IBD care.
“We can’t see a patient at diagnosis and say this drug is perfect for you,” she told Gut Republic.
“It’s often an educated guess amongst the advanced therapies. We can’t predict who will respond to each advanced therapy.
“And over time a medication that worked for one patient’s inflammatory pathway may not continue to work.”
That uncertainty means patients often needed to move between therapies over the course of their disease.
“We do get a high rate of loss of response,” Professor Mountifield says.
“For that reason, we need a range of available agents that are effective and that affect different inflammatory pathways in order to achieve remission for patients, so that is important.
“And certainly on the PBS we are fortunate to have a range of advanced therapies that can achieve this.
“But now there’s medications available overseas in similar OECD countries that are, in fact, potentially more effective and have a very favourable safety profile.
“So these are medications that we’re aware of and we want for our patients, and also that patients are aware of and want themselves.”
Most people with Crohn’s disease or ulcerative colitis were diagnosed in adolescence or early adulthood, during their peak study, working, and reproductive years.
Professor Mountifield says uncontrolled inflammation had consequences extending well beyond gastrointestinal symptoms.
“Having active inflammation impairs quality of life. Patients have more hospitalisations and increased healthcare utilisation,” she says.
“It’s bad for patients, but it’s also bad for the economy because you have reduced productivity, reduced workforce participation, and increased healthcare costs.”
Professor Mountifield said there was no doubt patients would benefit from having subsidised access to IL-23 inhibitors, including guselkumab and risankizumab.
“Their safety profile is very favourable and they’re highly effective,” she says.
“In some head-to-head trials they appear to be more effective than our current available agents on the PBS, and they also give a durable response for many patients.”
She stresses the newer medicines would not replace existing therapies but expand treatment options for patients who have exhausted current PBS-funded medicines.
“Some of our patients have cycled through all of the advanced therapies and either lost response or never responded in the first place,” she says.
“We do need these agents, which we know are really good as second-line as well as first-line therapies.”
The Bitter Pill report argues Australia’s challenge is no longer simply lengthy reimbursement timelines but an emerging reluctance among pharmaceutical companies to launch medicines locally at all because Australia’s pricing framework no longer provides a viable commercial return.
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Professor Mountifield says the economics are becoming increasingly difficult for manufacturers.
“One of the problems on the PBS is that these new first-in-class molecules are placed financially on an equal footing with agents that are much cheaper to produce,” she says.
“That is a less favourable environment for pharmaceutical companies to bring in their molecule because their remuneration won’t be anywhere near what it is in other countries.”
Australia also represents a relatively small commercial opportunity despite having one of the world’s highest rates of inflammatory bowel disease, she says.
“We have some of the highest rates of inflammatory bowel disease in the world, but numbers-wise it’s still a relatively small market, so it’s not that favourable for pharmaceutical companies to invest in Australia,” she tells GR.
The consequences are increasingly being felt in clinics.
Without additional treatment options, some patients progress to repeated hospital admissions, prolonged steroid treatment and bowel surgery.
“There’s no question that if there are more drugs available, we’ll see fewer patients having surgery,” Professor Mountifield says.
Surgery often involves removal of part or all of the bowel and may require formation of a permanent or temporary stoma.
“Patients are fairly desperate for more medications to be available that might work because that can potentially avoid or delay surgery,” she says.
She says avoiding pelvic surgery is particularly important for younger patients because of its potential impact on fertility, sexual function, body image, and relationships.
“If we can control inflammation really well with any agent, we know we can reduce the long-term risk of cancer as well,” she says.
“There are so many important outcomes, not just for individual people living with IBD, but for the health system that can be improved by getting in front of inflammation early.”
Emerging evidence suggests the opportunity to alter the course of disease is much earlier than previously thought.
“We used to say we needed to treat aggressively within the first two years,” Professor Mountifield says.
“Now we know it’s really within the first three months. If we can achieve remission with highly effective agents early on, we can really reduce those bad outcomes for patients.”
The Bitter Pill report warns that unless Australia modernises its reimbursement framework, patients will continue missing out on medical breakthroughs routinely available overseas, while companies increasingly direct investment to countries offering more predictable and commercially sustainable access pathways.
Professor Mountifield says that trend is already evident in inflammatory bowel disease.
“They are deterred,” she says of pharmaceutical companies.
“If we’re not a competitive market where they can make money and succeed commercially, our patients will be disadvantaged and our health system will be disadvantaged because these patients flow into inpatient care, steroid use, and lack of workforce participation.”
She says the problem cannot be solved simply through private access because many medicines never reach Australia.
“The drugs don’t get here,” she says.
“We’ve had patients go overseas. There’s some medical tourism going on trying to get access to these agents.
“Outside of clinical trials or another indication, it’s not possible to get access to these new agents readily.
“Most people just can’t afford to either pay for the agents or travel overseas to access them.”
Professor Mountifield says governments, the Pharmaceutical Benefits Advisory Committee, clinicians, and patient organisations need to work together to ensure Australia’s reimbursement system remained attractive enough for companies to launch innovative medicines.
“In the long term, we’re better off controlling active disease and keeping people in the workforce, caring for their children and participating in life,” she says.
