Rare disease and drug treatment
Children with rare disease often need drug treatment as part of their care. Drugs to treat rare disease can be rare ‘orphan drugs’ and many are still in their trial phase when they are used to try and treat people with rare diseases. Sometimes this means that not all of the harms and benefits are known while the drugs are being used in the trial phase. As many rare diseases are progressive, it is especially important that children have timely access to these drugs as they can lead to improvements for people living with rare disease. It is critical that the use of orphan drugs is governed by strong laws and oversight to make sure that the benefits can be harnessed and any potential harm is limited.
https://www.unicef.org.au/Upload/UNICEF/Media/Our%20work/childfriendlycrc.pdf – Article 33