Researchers from 16 countries, including patient advocates and two young people with rare disease experience, examined how teenagers and young adults are involved in rare disease research. They found that while patient engagement is talked about more often, young people affected by rare diseases are largely excluded from shaping research decisions.
This matters because the majority of people living with rare diseases are actually children and adolescents—yet most research is designed without asking them what they need or value.
Teens often care about different things than researchers measure
The study found that researchers typically design studies based on assumptions rather than asking young patients directly. A teenager might care deeply about whether a treatment lets them attend school regularly or maintain friendships, yet traditional research might only measure physical symptoms. This gap means treatments developed without teen input may miss what actually improves their daily lives.
In Singapore and Asia, young people face extra barriers to speaking up
The study acknowledges that cultural contexts matter. In many Asian settings, where respect for authority and family hierarchy are valued, teenagers may feel hesitant to share their views openly in research settings—even when they have crucial insights. The guidance developed by this international team encourages researchers to create safe, age-appropriate ways for young people to contribute, such as one-on-one conversations or peer discussion groups, rather than formal meetings where they might feel uncomfortable speaking.
Ask researchers these questions
- Request to know whether young people helped design the study, not just participated in it.
- Ask how the research measures outcomes that matter to your teen's daily life and goals.
- Clarify what support will be offered so your teen can share their views comfortably.