Why patient data is key to developing treatments for rare diseases

New Delhi: Patient data, including medical records and patient experiences, are essential for scientists to develop new treatments, especially for rare diseases.

The data help identify diagnostic biomarkers, predict disease progression, and help investigators design better clinical trials.

This is important because clinical trials are required to show that a new treatment is safe and effective before a country’s regulators approve it.

Conventional clinical trials involve a large number of participants and include both treatment and control groups. However, for rare diseases, too few people are eligible to participate, and it is not possible to have a control group — making real-world patient data even more crucial for research.