Paediatric rare diseases: Children are not small adults

Around 75% of rare diseases are diagnosed in childhood, with most before the age of two. With 30% of rare disease patients dying before the age of five, the need for different treatment methods for children is clear.

In a new pharmaphorum podcast, web editor Nicole Raleigh speaks with Dr Helen Thackray, chief R&D officer at biotech company BioCryst, as well as an alumnus of the Children’s National Medical Center.

The conversation touches upon incentivisation for manufacturers to develop drugs to treat rare diseases, addressing age-appropriate formulations and recognising the metabolic differences between children and adults, and looks also at clinical trial design.

While Helen and BioCryst have made huge strides in hereditary angioedema (HAE) - where the treatment landscape has completely changed from being androgens heavy to oral prophylactics, on-demand injectables and other options – she believes that the revolution in rare disease is just beginning.

It’s clear that the time for action is now, and serving the paediatric patient population through every stage of the drug delivery process will lead to better adherence and more effective disease management.

You can listen to episode 166a of the pharmaphorum podcast in the player below, download the episode to your computer, or find it - and subscribe to the rest of the series - in iTunes, Spotify, Amazon Music, Podbean, and pretty much wherever you get your other podcasts!