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How we can overcome the lack of treatment options for rare cancer


Colman Taylor, University of Sydney and John Zalcberg, Monash University

Rare cancers are just that: rare. This means research into each of these particular types of rare cancers is limited, and so are the treatment options. As a consequence, patients diagnosed with rare cancers face significant challenges.

In November 2016, the Australian Senate established a select committee to examine funding for research into cancers with low survival rates. More recently, the health minister announced A$13 million from the Medical Research Future Fund will be used for clinical trials to help achieve better health outcomes for people with rare or uncommon cancers.

The minister also commissioned new work on evaluating cancer medicines that treat multiple tumours and have a specific genetic feature (biological marker). This could improve access to therapies that might benefit some patients with rare cancers.

These recent steps are in recognition of the significant challenges associated with undertaking research into rare cancers. By their nature, rare cancers include small and variable patient populations making gold-standard randomised trials challenging or even impossible.

The lack of evidence resulting from few or no randomised trials creates challenges for registering and reimbursing new medicines. This ultimately leads to a lack of subsidised medicines for these patients. As a result, the improvements seen in patient outcomes related to new therapies for more common cancers like lung cancer, melanoma and bowel cancer over the last two decades do not extend to rare or less common cancers.

What is a rare cancer?

The definition of a rare cancer is debatable. The RARECARE collaboration in Europe uses an operational definition of fewer than six cases per year per 100,000 population. In Australia, the medicines regulator, the Therapeutic Goods Administration (TGA), has recently updated the eligibility criteria for medicines treating rare diseases to fewer than five cases of the disease in a population of 10,000 people.

Historically cancers were categorised by the anatomical location, such as the breast or kidney. But with the discovery of new biological markers, common cancers can be grouped into smaller, more homogeneous and genetically similar subsets. So the number of rare cancers will continue to grow as medical technology advances.

Why don’t they have many available medications?

The lack of government-approved and subsidised medicines to treat rare cancers primarily stems from the lack of evidence supporting their use. Submissions to the current inquiry also cited problems such as a lack of research funding; the need for international collaboration; lack of investment by industry; attracting sufficient interest of researchers and recruiting sufficient patients.

It’s hard to recruit enough patients for research studies.
from www.shutterstock.com

Even if patients can be identified and recruited to a trial, it’s difficult to generate meaningful data from so few patients.

The lack of evidence presents challenges for new medicines trying to meet registration and reimbursement criteria in Australia. To be registered through the TGA, a new medicine must have demonstrated efficacy and safety.

In order for new medicines to be listed on the Pharmaceutical Benefits Scheme (PBS), it must have a demonstrated benefit over standard treatment, as well as being considered an efficient use of tax payer dollars.

New medicines for rare cancers are often expensive, especially when randomised trials are not possible.

What can we do to improve this situation?

With the changing nature of medicine and research, new opportunities are emerging to address the current inequity. The shift to treating patients based on the genetic profile of their tumour rather than the location of the cancer has increased treatment options for rare cancer patients.

To harness the benefits, changes are required with input from multiple stakeholders, including government, industry, clinicians, researchers and patients.

Better access to new medicines ultimately starts with better research. To achieve this, experts have called for additional targeted funding, innovative trial designs, and better partnerships between industry and researchers.

There is also the opportunity to collect better “real world” data via platforms such as the My Health Record, which could supplement existing research and allow performance monitoring of recently approved new medicines.

Organisations such as Rare Cancers Australia and the Cancer Drugs Alliance are liaising with government regarding changes that could improve access to novel medicines for patients with rare cancers. This includes greater input from patients and more flexibility in the way we evaluate medicines for public reimbursement.

The ConversationIt should also be recognised the problems faced in providing innovative treatments to patients with rare cancer extends to rare diseases in general. With modern medicine providing the potential to improve outcomes for patients with rare cancers as well as other serious chronic diseases, we need to have a broader conversation about what we can afford and what we are willing to pay for new medicines.

Colman Taylor, Post-doctoral Research Fellow, The George Institute; Conjoint Senior Lecturer, UNSW; Owner and Director, Health Technology Analysts, University of Sydney and John Zalcberg, Head, Cancer Research Program, Monash University

This article was originally published on The Conversation. Read the original article.

Why we desperately need more focus on gynaecological cancers


A woman’s reproductive system has been called many names, yet still remains a taboo topic.

For many women the words vagina, vulva, uterus, cervix and ovary are not spoken. Rather, euphemisms like “down there”, or “down below” are used. It’s time to start talking about ‘The box’.

The taboo

Talking about our genitalia, and particularly women’s genitalia, is culturally taboo. There are a lot of people who wrongly believe that being diagnosed with a gynaecological cancer means a woman is ‘loose’ or ‘dirty’.

It’s hard to start a conversation about your gynaecological health in the face of such taboos and critical judgment. This discomfort is costing the lives of our mothers, daughters, sisters and friends.

This year 5500 women will be diagnosed with a gynaecological cancer. In the past 25 years overall cancer survival in Australia improved by 19%, but gynaecological cancer survival improved by just 7%.

The Australia New Zealand Gynaecological Oncology Group (ANZGOG) is conducting and promoting cooperative clinical trials and multidisciplinary research to improve the lives of women who have gynaecological cancer.

September is International Gynaecological Cancer Awareness Month, and an important opportunity to raise awareness of gynaecological cancers. ANZGOG’s Save the Box campaign aims to raise awareness and funds for gynaecological cancer research.

Gynaecological cancer symptoms

There are seven types of gynaecological cancer: endometrial, ovarian, cervical, vaginal, vulvar, and two rare pregnancy-related cancers.

The symptoms of gynaecological cancer are vague but include:

  • Abdominal bloating
  • Feeling full
  • Increased frequency in urination or bowel movements
  • Menstrual irregularities
  • Indigestion
  • Fatigue
  • Itching, burning or soreness
  • Lumps, sores or wart-like growths
  • Pain during sexual intercourse
  • Bleeding after menopause

If a woman is experiencing any of these symptoms and they are unusual for her or persistent, she should see her GP. If she is diagnosed with a gynaecological cancer, she should be referred to a Gynaecological Oncologist – see the Australian Society for Gynaecologic Oncologists for a full directory. Statistics indicate that women diagnosed with a gynaecological cancer have the best survival outcomes if they are referred to a gynaecological oncologist and managed as part of a multidisciplinary team.

Perhaps because of the taboos, and also because of the vagueness of some of the symptoms, it can take several visits to the GP before a diagnosis of a gynaecological cancer is suspected and appropriate referral made. Many women who have vulval or vaginal cancers have had symptoms that they’ve been embarrassed about and this has delayed them going to the GP.

Current trends in gynaecological cancer


We are seeing an increased incidence of endometrial cancer as a result of the growing obesity epidemic.

Endometrial cancer is a preventable disease in 4 out of 10 patients through exercise or diet. Women can be referred by their GP’s via an Enhanced Primary Care (EPC) referral to an ESSA accredited exercise physiologist for 5 Medicare reimbursed sessions.


A woman diagnosed with ovarian cancer has only a 40% chance of surviving five years from her diagnosis. Survival has not improved in decades and lags well behind breast (89% survival five years from diagnosis) and other cancers.

New genomic knowledge has radically changed the way we view ovarian cancer. The disease is now classified into seven molecular subtypes, some of which are very rare. We now understand why a one size-fits-all approach to treatment must and can change.

The ANZGOG Ovarian Cancer Alliance for Signal-Seeking Research (OASIS) initiative has been created to catalyse and fund research into improved treatments for ovarian cancer that target the seven molecular subtypes.


On a positive note, with the introduction of the cervical cancer vaccine, the incidence of cervical cancer is predicted to fall dramatically, although this may not be evident for 10 to 20 years. The new cervical screening program, with the emphasis on HPV testing, is also predicted to reduce the incidence of cervical cancer..

The future

Government funding of research is increasingly competitive, meaning that early career researchers, pilot studies, and research into rarer diseases like gynaecological cancers are missing out.

As a non-profit organisation, ANZGOG continues to struggle with funding. Clinical trials are costly and this is the major barrier we face in opening new trials and expanding our clinical trial portfolio.

Why does research matter? Because research leads to improved outcomes through better treatments, better survival and better quality of life.

We’ve seen tremendous advances in the survival rates of women who have breast cancer and many of those improvements are the result of research. Virtually every advance in cancer survival has been made on the back of clinical trials.

The current growth in the development and use of targeted therapies will have a substantial impact. The upcoming challenge will be how to incorporate these drugs effectively into the treatment for ovarian cancer (and perhaps other cancers in the future) in a way that is of greatest benefit to patients.

ANZGOG’s Survivors Teaching Students® – Saving Women’s Lives program brings the faces and voices of ovarian cancer survivors and their caregivers into the classrooms of medical and nursing students. Survivors, through their own personal experiences, are in a unique position to help students become more sensitive to the risk factors associated with ovarian cancer and symptoms of ovarian cancer. Our goal is to increase the number of health care providers who recognise the risk factors and symptoms of ovarian cancer, in the hope that women can be diagnosed earlier, when cure rates are higher.

While many endometrial cancers are cured with surgery alone, there are plenty of unanswered questions about the extent of surgery needed for early endometrial cancer patients, and what additional treatment may be best for those with more advanced disease. ANZGOG is conducting a surgical trial, STATEC, to try to determine the extent of surgery that is best for patients with apparent early stage, but high risk, endometrial cancer.

The improvements in breast cancer survival rates are the shining example of what can happen when we openly talk about the problems, and provide the funds needed to save lives.

‘Save the Box’ is fundraising for gynaecological cancer research conducted by the Australia New Zealand Gynaecological Oncology Group (ANZGOG). Find out more or make a donation today at https://www.savethebox.org.au/

Associate Professor Alison Brand

Alison is Chair of the Australia New Zealand Gynaecological Oncology Group and Director of Gynaecologic Oncology at Westmead Hospital