The Medical Technology Association of Australia and device companies joined a range of stakeholders in appearing before a public hearing of the parliamentary inquiry into approval processes for new medicines and medical technologies.
Rare Voices Australia told the committee Australian patients wait longer than those in comparable countries for access to new treatments and the industry views it as a difficult and challenging market.
CEO Nicole Millis said the "perception" is that approval processes in Australia are challenging and uncertain. "Whether or not that is true, that is the perception," she said.
Ms Millis also identified the lack of clinical trials as an issue. "This requires investment in clinical trial infrastructure," she said, highlighting workforce gaps, the lack of registries and administrative burdens.
On health technology assessment (HTA), Ms Millis said Australia's current approach lacks flexibility around the acceptance of evidence.
CEO of the Medical Technology Association of Australia Ian Burgess told the hearing about the dramatic beneficial impact of devices on patients. However, he said current approval processes limit funding for some technologies, including minimally invasive technologies.
Mr Burgess said funding and decision-making processes can be improved in a way that "recognises the unique nature" of devices.
He argued for reform of MSAC processes and expressed concern over the proposed abolition of the Prostheses List (PL) and its replacement with what he described as a "blunt" instrument based on average payments (Diagnosis Related Groups - DRG). The PL determines what devices are funded by private health insurers.
Mr Burgess said the PL currently "provides enormous" value to the private health system and ensures the primacy of surgeon choice.
"Having said that, there are opportunities to improve the Prostheses List," he said, adding this would include patient access to a wider range of new technologies.
"What is being proposed is negative," he said, arguing the proposed DRG-based system would be "very blunt". He said it would move costs away from insurers to private hospitals and even to patients. "Patients could even be forced back into the public system," said Mr Burgess.
"This is a contentious and controversial area," he said.
The MTAA said it supported the reinstatement of processes to allow greater horizon scanning of new medical technologies and called for the adoption of more incentives to promote the development and retention in Australia of local discoveries.
Bowel Cancer Australia, JDRF, Allergy and Anaphylaxis Australia and Fabry Australia appeared in a single session.
Sheridan Campbell of Fabry Australia said over 50 per cent of adults living with the disease (Fabry disease) are not in employment and one quarter have lost employment because of their condition.
Ms Campbell said Australia's treatment guidelines for Fabry need to be urgently reviewed to bring them in line with comparable countries. She said around one-third of patients are currently denied treatment because of the guidelines and that the government should consider greater incentives for companies to bring clinical trials to Australia.
"Australia should not be viewed as a challenging market," she said, referring to the negative perceptions of global biopharmaceutical companies.
"Not everything should be whittled down to a cost-benefit analysis," said Ms Campbell, saying these decisions need to be based on "humanity".
Joni Thomes of Bowel Cancer Australia said the extensive focus and discussion on screening for the disease is not matched by a commensurate focus on access to treatment. She said around one-half of patients are diagnosed with advanced or metastatic disease.
"There is a clear clinical need for new treatment options," she said, adding they currently remain limited.
"We support the model of immediate access to a new drug after its approval," said Ms Thomes. "The PBAC process could run as consumers benefit from access," she said. Ms Thomes said international comparisons show Australian patients wait much longer for reimbursed access to new treatments than those in many comparable countries.
Allergy and Anaphylaxis Australia CEO, Maria Said, there is insufficient research into allergic diseases and that reimbursed access to treatments should be accelerated.
Ms Said a critical area of research is to move away from auto-injectors for the administration of adrenalin to include other and more usable methods.
Mike Wilson of the JDRF (Juvenile Diabetes Research Foundation) said there are innovations on the horizon but that Australia's current assessment and decision-making processes are no long fit-for-purpose.
Some of the groups also questioned the limited approach to consumer input on decision-making bodies, specifically whether it was able to properly represent the experiences of all patients.
The next set of patient group representatives to appear was Migraine and Headache Australia, Australian Pompe Association, Save Our Sons Duchenne Foundation, Cystic Fibrosis Australia and Alpha-1 Organisation Australia.
Lance Dale of Save Our Sons Duchenne Foundation told the hearing "time is critical" for the patients they represent and that means processes need to be reformed. Mr Dale quoted the mother of a patient lamenting the fact treatments are approved and funded overseas but not in Australia.
President of Alpha-1 Organisation Australia, Dr Gaynor Heading, said "structural issues" in Australia mean patients have been denied access to treatment through clinical trials. She said the lack of screening, health prevalence data and MSAC guidelines that "do not consider the needs of the rare disease community" need to be addressed.
"Australians have never had access to funded treatment," she said, adding, "Non-rare disease guidelines should never be used for rare disease" and that patients with Alpha-1 are dying prematurely as a result.
Nettie Burke of Cystic Fibrosis Australia said Australia's approach to HTA is "robust" but that it could be made "better".
Ms Burke said international collaboration is an opportunity and that consumers should be able to review company submissions to MSAC and PBAC. She said this does not extend to access to confidential information but that consumers should be able to review and directly comment on submissions.
Ms Burke also said recent innovations means people living with cystic fibrosis can now live close to a normal life expectancy. She said one consequence is that more of these people are having children, meaning the number of Australians with cystic fibrosis could increase in the years ahead.
Carl Cincinnato of Migraine and Headache Australia highlighted the delay in reimbursed access to the biologics for chronic migraine. He said the treatments have positively changed the lives of many people but there has been no progress to reimbursement through the PBS despite positive recommendations.
Raymond Saich of the Australian Pompe Association said timely treatment is essential given the impact after onset, which can be very early in life. "For us, newborn screening is absolutely vital," he said.
However, while screening in other countries enables diagnosis in around five days, he said it generally takes three-to-six months to diagnose Australian babies with Pompe. Mr Saich said the issue is particularly acute for families in regional and rural areas with babies sometimes dying before diagnosis.