An estimated 19.3 million new cancer cases and almost 10.0 million cancer deaths occurred Worldwide in 2020 (GLOBOCAN 2020). In Australia, cancer is the leading cause of health burden. More than 1 million people in Australia, live today with a previous diagnosis of cancer (AIHW). 20 percent of Australian cancer diagnoses are rare, and they account for 30 per cent of annual cancer deaths (WEHI).
A rare cancer is defined as a cancer that affects 6 people per 100,000 of the population. A less common cancer is one which has fewer than 12 diagnoses per 100,000 of the population. 52,000 Australians are diagnosed with a rare or less common cancer (RLC) every year. Despite there being a 30% incidence, it has a 50% death rate (RCA). While incidence rates have grown over the last 30 years, we have seen there is a much lower mortality rate for Common cancers. In contrast, RLC have continued to increase.
The difference in outcome is largely driven by a large evidence base and greater knowledge of the more common diseases. Greater R&D investment for innovative medicines, earlier diagnosis and available treatments, are increasing survival rates.
Rare Cancer Statistics
There are over 200 different types of rare or less common cancers. Despite many advancements in treatment and medical research these diseases still have major impacts on patients, carers, physicians and communities.
The more prevalent, Common cancers such as melanoma, lung cancer, breast cancer, prostate cancer and bowel cancer. Have seen major progress in treatment options for patients. There is greater awareness and scientific knowledge, with patients living longer. We are seeing a higher volume of drugs in development, and as a result a higher success rate for drugs registered, reimbursed and launched to patients.
What is clear is the mortality rate across RLC has not changed for a long time. There is a clear need among various patient groups unable to access novel therapies fast enough. In the statistics provided by Rare Cancers Australia it is evident that these diseases have a heavy effect on all age groups young and old.
Patient data can help drive change
A key challenge to improve mortality rates and other outcomes is the low patient numbers and irregular diagnoses of the disease. It is difficult to find study participants to meet the stringent criteria of a clinical trial or research study. Limiting the ability to run drug trials or other research projects specific to the cancer population. Without an evidence base, it is then challenging to educate clinicians, improve diagnosis and make new treatments available.
“Data and real world evidence can assist by improving our understanding of the progression of disease, treatment efficacy and selection and importantly understanding the pre-diagnosis patient journey and biomarkers to enable the use of data to assist in the earlier diagnosis of the disease for future patients. For rare disease having a diagnosis is life-changing.”
Greg Hughes, Director Customer Success
When research is conducted and evidence is available it may not meet the robust standards required by regulators and payers. Either sample sizes are too small, the protocol design does not meet evidentiary requirements, there is no comparator, or the population is unclear.
One alternative, is to utilise longitudinal health data. By accessing and integrating the wealth of data captured on a patients longitudinal health journey, to view and understand the course of disease. Understand the signs, symptoms, diagnoses, treatments and outcomes of different diseases. Compare different patient cohorts and understand different populations. Pooling these insights can form a clearer picture of a disease.
We can now use this real world evidence to inform how to improve diagnosis, collect treatment data as evidence and ultimately save lives.
We have demonstrated the value of using longitudinal patient datasets such as the Australian PBS 10% with many key cancers such as melanoma, lung cancer, breast cancer and bowel cancer. Helping pharma companies understand the treatment paradigm to manage their supply chain, help with reimbursement and post market surveillance studies.
However, with rare cancers often the number of patients suffering can be less than 100 nationally. Therefore, the current availability of PBS data being a 10 percent sample cannot provide the insights necessary to add value. This is because the sample size is too small.
“The rare nature of each of these cancers makes analysis difficult without larger datasets, with diagnosis per annum less than 1500 per type of cancer the PBS10% means we are analysing 15 patients and in most cases less. We simply need a larger data sample (eg PBS 50%) and preferably linked to MBS data to enable quality analysis to support TGA and PBAC submissions and real world evidence generation.” Said Greg Hughes.
Prospection have been advocating for some time about the importance of being able to access larger samples in some of these key areas so that companies can have real world evidence (RWE) to put into their PBAC submissions. Discussions are ongoing and we hope that access becomes available sometime in the near future. This is to ensure availably of treatments to help patients suffering from a rare disease
Prospection joins the RCA Mount Kosciusko Challenge
Along with many other Prospection team members, Greg Hughes has chosen to take part in the Kosi Challenge.
“My reason (for doing the Kosi Challenge) is more to do with a shared vision. I have focused 10 years of my career connecting technology, capability, data and good governance to enable health data to be used to assist in the earlier diagnosis and treatment optimisation for patients, in no area is this needed more than in rarer conditions. I absolutely share the RCA 20-30 vision that artificial intelligence is integrated across the cancer continuum to optimise outcomes and I live and breath this every day with the work that Prospection is advancing.”
If you would like to donate to help us reach our goal click the button below. Let’s continue to raise awareness about this much needed cause.
