Coordinated national action is needed to develop an evidence base and standards of care for young Australians with rare and low survival cancers
In November 2016, the Australian Senate established a select committee to explore the impact of funding models on rare and low survival cancer research. CanTeen Australia presented a submission to this inquiry which highlighted the impact of these cancers on adolescents and young adults (AYAs) and the systemic barriers to improving outcomes for patients with rare and low survival cancers. Drawing from that submission, we present the argument for a strategic national approach, including a national trial network, to facilitate cross-sectoral coordination and investment to improve outcomes for AYA patients with cancer and the broader Australian population affected by rare and low survival cancers.
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- 1. Australian Institute of Health and Welfare. Cancer in adolescents and young adults in Australia (AIHW Cat. No. CAN 59; Cancer Series No. 62). Canberra: AIHW; 2011. https://www.aihw.gov.au/reports/cancer/cancer-in-adolescents-and-young-adults-in-australi/contents/table-of-contents (viewed Sept 2017).
- 2. Bleyer A, Budd T, Montello M. Adolescents and young adults with cancer: the scope of the problem and criticality of clinical trials. Cancer 2006; 107: 1645-1655.
- 3. Thomas DM, Seymour JF, O’Brien T, et al. Adolescent and young adult cancer: a revolution in evolution? Intern Med J 2006; 36: 302-307.
- 4. Rare Cancers Australia. Just a little more time: rare cancers update report. Bowral, Australia: Rare Cancers Australia; 2016. https://www.rarecancers.org.au/page/1081/reports-submissions (viewed Oct 2017).
- 5. Bleyer A. Adolescent and young adult (AYA) cancers: distinct biology, different therapy? Cancer Forum 2009; 33: 4-10.
- 6. Tricoli JV, Blair DG, Anders CK, et al. Biologic and clinical characteristics of adolescent and young adult cancers: Acute lymphoblastic leukemia, colorectal cancer, breast cancer, melanom, and sarcoma. Cancer 2016; 122: 1017-1028.
- 7. Bond MC, Pritchard S. Understanding clinical trials in childhood cancer. Paediatr Child Health 2006; 11: 148-150.
- 8. Mitchell AE, Scarcella DL, Rigutto GL, et al. Cancer in adolescents and young adults: treatment and outcome in Victoria. Med J Aust 2004; 180: 59-62. <MJA full text>
- 9. Teenage Cancer Trust. Giving more young people with cancer the opportunity to take part in clinical trials. London: Teenage Cancer Trust; 2014. https://teenagecancertrust.org/sites/default/files/Clinical%20Trials%20Paper_JR_FINAL_April15.pdf (viewed Oct 2017).
- 10. Abrahamyan L, Feldman BM, Tomlinson G, et al. Alternative designs for clinical trials in rare diseases. Am J Med Genet C Semin Med Genet 2016; 172: 313-331.
- 11. Tuffaha HW, Andronis L, Scuffham PA. Setting Medical Research Future Fund priorities: assessing the value of research. Med J Aust 2017; 206: 63-65. <MJA full text>
- 12. Casali PG, Bruzzi P, Bogaerts J, Blay JY. Rare Cancers Europe (RCE) methodological recommendations for clinical studies in rare cancers: a European consensus position paper. Ann Oncol 2015; 26: 300-306.
- 13. Augustine EF, Adams HR, Mink JW. Clinical trials in rare disease: challenges and opportunities. J Child Neurol 2013; 28: 1142-1150.
- 14. Pfister DG. Off-label use of oncology drugs: the need for more data and then some. J Clin Oncol 2012; 30: 584-586.
- 15. Australian Clinical Trials Alliance. Report on the activities and achievements of clinical trials networks in Australia: 2004–2014. Melbourne: National Health and Medical Research Council; 2015. https://www.australianclinicaltrials.gov.au/report-activities-achievements-clinical-trials-networks (viewed Oct 2017).
- 16. Panageas KS. Clinical trial design for rare cancers — why a less conventional route may be required. Expert Rev Clin Pharamcol 2015; 8: 661-663.
- 17. United States Congress. H.R.733 — Recalcitrant Cancer Research Act of 2012. 112th Congress (2011–2012). United States Congress; 2012. https://www.congress.gov/bill/112th-congress/house-bill/733 (viewed Oct 2017).
We thank Peter Orchard and Kimberley Allison for their contributions to the development of this manuscript.
CanTeen Australia received Commonwealth Government funding to support the Australian Young Cancer Patient Clinical Trials Initiative.