Clinical research on rare diseases of children: neuroblastoma

Early access to new treatment options should not preclude accurate research planning, especially for rare diseases and fragile populations. Taking neuroblastoma as a model case, we analyzed the rationale supporting the search for future therapeutic strategies in the light of preclinical and clinical evidence. We reviewed ongoing randomized trials of pharmacological interventions for the treatment of neuroblastoma retrieved by searching ClinicalTrials.gov and the European Union Clinical Trials Registry (last update March 2016). Our search identified four randomized clinical trial reports. We found poor evidence from preclinical and early clinical research supporting their rationale. Their methodology was questionable too. The urgency to cover unmet needs in difficult clinical settings like rare diseases, particularly those involving fragile populations, cannot justify disorderly research approaches. Under these circumstances, clinical questions should be properly identified and addressed to protect patients and avoid wasteful research.