Cancer in children remains a major source of suffering: in Europe ~35,000 children are diagnosed with, and ~6,000 children die from cancer each year. Despite major advances in cancer research, children with cancer benefit much less from this progress than adults. The development of new therapeutics for children with cancer is hampered by the rarity of each childhood cancer individually. This makes drug development economically unattractive, and results in a low interest in pediatric cancer from the classical pharmaceutical industry.

Historically, much of the progress in pediatric oncology has been a (often substantially delayed) spill-over from adult developments. Cancer in children is however biologically distinct from adult cancers, and some tumors (for example neuroblastoma) are almost exclusively observed in children, necessitating a development model centered around pediatrics.

To improve this situation, it is not merely a matter of mobilizing more resources, but also of deploying the resources in the most effective way to ensure that research leads to access to novel treatments in the children’s hospital. This purpose fuels the research conducted by Sam Daems, Director at private equity firm Waterland, who tragically lost his daughter to an incurable brain tumor. Sam Daems collaborates on this crucial work with Professor Hilde Stevens at the I3h Institute of ULB, co-directed by Michel Goldman and Mathias Dewatripont.

Building on the input of the pediatric ecosystem, this team has developed a novel economic model that has just been published in the prestigious journal Nature Reviews Drug Discovery. It is based on combining resources from public and private parties, both for-profit and not-for-profit, in a pooled fund which optimally balances risk and returns. This pooled fund will invest in a portfolio of development programmes, each focused on a specific pediatric therapeutic target, reducing the investor risks as much as possible.

Management of the fund will be entrusted to a multidisciplinary team of highly skilled specialists, coordinating the intellectual property aspects and regulatory dimensions, that are critical to the development of a new medicine. Early in the development, and long before the new medicines are approved by health authorities, pre-agreements will be negotiated with the organizations responsible for their reimbursement, to ensure both their economic viability and their availability to all children in need.

The model has been developed in collaboration with international experts in the field of pediatric oncology, and was co-constructed with the team of KickCancer Foundation in Belgium and the Imagine for Margo Foundation in France.

Link to the article in Nature Reviews Drug Discovery: