Each year, an estimated 400 000 children and adolescents develop cancer globally. While great progress has been made in improving survival rates in childhood cancer, there are stark inequities still experienced around the world. Challenges are faced in developing accessibile medicines due to insufficent funding, regulatory hurdles, small patient populations, weak market incentives, and the lack of age-tailored treatments.
On 18 January 2024, the World Health Organization (WHO) developed its first-ever list of priority paediatric cancer formulations, aiming to focus research and development on the specific needs of children. Following this, leveraging expertise of partners in the Global Accelerator for Paediatric Formulations Network (GAP-f) a Target Product Profile (TPP) development group was convened to define optimal and minimum attributes for the 6 priority formulations to be developed in the short term and 2 medicines flagged for the long term. The TPP development group consisted of scientific, clinical, and public health experts from diverse geographic regions and was established to develop a target product profile for the 6 selected priority formulations and to provide clear guidance for manufacturers and product development partnerships.
WHO is now seeking feedback on the draft TPPs developed for cyclophosphamide, etoposide, mercaptopurine, methotrexate, procarbazine and temozolomide. Industry experts, product developers, the scientific community, implementers, clinicians, and health programme personnel currently involved in the management of childhood cancer are all invited to submit their feedback via the table provided below and can review the accompanying draft TPP document for each medicine for reference that includes the meeting findings. Together we can work on making an impact in developing better formulations for children with cancer.
TPP on cyclophosphamide (PO):
TPP on etoposide (PO):
TPP on mercaptopurine:
TPP on methotrexate (PO/IT):
TPP on procarbazine:
TPP on temozolomide:
Deadline for the call is 31 March 2025