Related Experiment Video
Updated: Mar 11, 2026
Establishment of Orthotopic Patient-derived Xenograft Models for Brain Tumors using a Stereotaxic Device
Published on: May 2, 2025
Clinical research on rare diseases of children: neuroblastoma
Chiara Gerardi1, Rita Banzi2, Vittorio Bertele'2
1IRCCS - Istituto di Ricerche Farmacologiche Mario Negri, Via La Masa 19, 20156, Milan, Italy. chiara.gerardi@marionegri.it.
Purpose:
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.
Methods:
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).
Results:
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.
Conclusions:
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.
Insights
Accurate research planning is crucial for rare diseases, even with new treatments. This study found limited evidence and questionable methods in neuroblastoma trials, highlighting the need for rigorous approaches to protect patients and resources.
Area of Science:
- Oncology
- Clinical Trial Design
- Rare Diseases
Background:
- Effective treatment strategies for rare diseases, such as neuroblastoma, are urgently needed.
- Fragile populations require careful consideration in clinical research.
- Early access to novel therapies must be balanced with robust research planning.
Purpose of the Study:
- To analyze the rationale and evidence supporting therapeutic strategies for neuroblastoma.
- To evaluate the quality of research planning in the context of rare disease treatment.
- To assess the preclinical and clinical evidence base for ongoing neuroblastoma trials.
Main Methods:
- Systematic review of ongoing randomized clinical trials for neuroblastoma.
- Searched ClinicalTrials.gov and the European Union Clinical Trials Registry.
- Analysis focused on pharmacological interventions and trial methodology.
Main Results:
- Identified four randomized clinical trial reports for neuroblastoma.
- Found limited preclinical and early clinical evidence supporting the rationale for these trials.
- Observed questionable methodological quality in the reviewed trials.
Conclusions:
- The urgency to address unmet needs in rare diseases should not compromise research rigor.
- Disorderly research approaches can waste resources and potentially harm patients.
- Properly defined clinical questions and sound methodology are essential for ethical and effective rare disease research.
