When something is imposed by the medicines regulators, it's not always obvious how the new reality should be navigated. Legislative efforts to mandate clinical testing of drugs intended for children drove the need for different players to act in concert, to figure out how to align to make sure efforts were not siloed and therefore wasted. It took a while, but it became clear that IMI offered the best pre-competitive safe space to do exactly that; in 2018 the project c4c was launched, seeking to establish standards for the best way to carry out clinical trials that involve children.
ITCC-P4, the only other IMI project that directly concerns paediatric medicine, has recruited some of the best minds in the paediatric cancer field to create a testing platform with 400 new preclinical models of high-risk paediatric solid tumours that can be used to carry out early drug testing.
IMI is also moving into research that concerns genetic newborn screening for rare diseases. Diagnosing a newborn with one of thousands of diseases we can’t treat raises an ethical dilemma, and given the kind of infrastructure and that would be needed to build such a screening platform and the challenges in terms of acceptance and safety, ethics, policy, rights and the law it would raise, a public-private partnership seems to be the best option. In June 2020, IMI launched the final Calls for proposals under the IMI2 programme, seeking proposals to speed up the path to rare disease diagnosis, including the genetic screening of new-born babies.
It's time to close the paediatric gap
Paediatric trial network gets put to the test
Lab-grown tumours will help end drought of childhood cancer drugs