Rare tumours have been neglected for decades, with lack of funding, lack of research and lack of visibility. It is encouraging to see how this is changing and how research in rare tumours is not only taking place, but also being highlighted in Presidential Symposium 3 at this year’s ESMO Congress.
Results were presented from the FIRSTMAPPP trial – the first randomised study to explore the role of sunitinib for malignant pheochromocytoma and paraganglioma (MPP). MPPs are very rare malignancies, with an annual incidence of <1 per million population and prior studies have been lacking.
Being fully aware of recruitment challenges in rare tumours, the investigators of the FIRSTMAPPP trial set up an independent data monitoring committee to monitor, among other things, study accrual. The study required participation of multiple centres across France, Germany, the Netherlands and Italy, and a long recruitment period of 8 years to enrol a total of 78 patients. Results are now available and are likely to impact patient management moving forward, which highlights the importance of clinical trials in rare tumours being designed so that patients can be offered evidence-based therapy options.
Additional challenges are sources of funding to deliver these studies. Rare tumours are known to be under-funded; this is especially true where there is competition for resources, which may be most likely allocated to more common cancers. In addition, rare tumours appear to remain of limited interest to pharmaceutical companies.
Clinical trials in rare cancers are challenging, but not impossible to deliver. We most likely rely on international collaborations and team work to make this a reality for all patients diagnosed with rare tumours around the globe and it is exciting to see steps forward being taken.