The burden of rare cancers extends to both society as a whole as well as to patients and their families. Patients with rare cancers often face delays and erroneous diagnoses, ineffective treatment and at times an inability to access clinical experts given their scarcity. The causes and aetiologies of some rare cancers likely differ from more common cancers or their origins remain poorly understood. The rarity of specific cancers may limit the number of clinical trials aimed at improving both treatment and outcomes alike. Furthermore, due to the limited market for drugs targeting rare cancers, developing novel therapies may not top the priority lists of pharmaceutical companies.
To address these shortcomings, precision medicine focuses on identifying the right drug for the right patient at the right dose at the right time. Our response within the RaCaRe Group to this seemingly simple demand combines epidemiological, bioinformatics, molecular and cell culture studies as well as animal models when necessary.