A collaborative global research community challenges rarity and improves evidence.
Across the world, families and adult survivors experience different approaches to care due to lack of solid scientific evidence. Doctors cannot agree on the best way to manage children or provide supportive or lifelong care. We are growing a global research community to increase communication and collaborative effort to advance evidence based care.
Reliable scientific information about retinoblastoma and its treatment is limited. Specialist treatment facilities often work in isolation, and researchers struggle to answer fundamental questions such as:
- How many children in the world develop retinoblastoma?
- How many of those children can access treatment?
- What factors affect a child’s ability to access and complete therapy?
- What treatments are available in different places?
- How effective are these treatments?
- Is the treatment under investigation more or less effective than existing therapies with more or fewer side effects?
Randomized, controlled clinical trials are the gold standard of cancer care, and the only certain way to measure success of a treatment. However, reliable clinical trials require thousands of participants to find small, but important, differences between the new and standard approaches being compared.
For example, to show a 10% (1:10) improvement in effectiveness of a treatment, the trial must contain 1,000 patients. To show a 5% (1:20) improvement, 4,000 patients are needed.
The rarity of retinoblastoma makes it difficult to evaluate therapies and approaches to care. Opinions vary on how to improve early diagnosis and specialist referral. Screening, treatment and follow-up care differs between hospitals and even within the same facility, especially in resource-rich countries. We also know little about the long term psychological effects of treatment.
Communication between treating hospitals is poor, even within one country. As a result, recommended therapy frequently depends on local expertise, personal preference and research interests of the treating doctors, available resources, and beliefs and understanding of the community.
