A patient-turned researcher advocates for transparent science in rare disease research
Richard Rui Yang is a Hong Kong-based researcher affected by Bietti crystalline dystrophy (BCD), a rare retinal degeneration disease characterised by small sparkling crystalline deposits in the posterior retina and cornea. It manifests as nightblindness, decreased vision and, in the end stages of the disease, legal blindness. The disease usually appears in the third decade of life, so Yang became recently a patient.
Yang recently published an article in Orphanet Journal of Rare Diseases to discuss nontransparent science in the rare disease ecosystem. According to the author, the objective of this paper is not to accuse anyone of misconduct, but to emphasize the need for transparency in rare disease research. The researcher states that nontransparent science in rare disease research can lead to significant delays in drug development, ultimately affecting patients.
Through a series of real examples, he illustrates how research is not always reviewed with proper scrutiny. The article highlights the animal model flaws obscured by nontransparent science and its profound implications for orphan drug development. He also points out that lack of transparency can misguide medicine regulators and drug developers, delay or frustrate orphan drug development, or waste limited resources for rare disease research
The article does not have a clear conclusion, but it highlights the need for transparency in rare disease research to ensure that patients receive effective treatments as soon as possible. It is important that patients and caregivers raise their voices against flaws and misconducts in scientific research. Thank you, Richard!
If you are interested in patient advocacy, have a look at the Share4Rare Toolkit for Patient Advocacy.