Children with
rare disease belong to a vulnerable group. When
China’s current medical security system cannot provide
comprehensive health care, they not only
face physical and mental
torture, but also have a higher
risk of
children participating in clinical trials than
adults. So, adequate
protection of
children’s
safety and rights is the key to
ethical review. This
paper analyzed the current status of
drugs clinical trials for
rare disease in
children, including trial difficulties and guarantee system; explained the ethical principles that should be followed in clinical trials, such as the principle of
informed consent and the principle of no harm; and discussed the path of protecting
children’s
safety and rights, so as to raise
awareness and
attention of the importance of
ethical review of clinical trials.