The UK government has announced its support for the Rare Therapies Launch Pad, a new programme that will develop a pathway for children with rare conditions to access individualised therapies. The programme’s first project will...
Government bringing together experts in the field to drive forward progress in tackling cancer in children and young people. The taskforce presents an opportunity to act and will consider genomic treatments, new diagnosis tools, research...
The FDA has announced the availability of the Pediatric Drug Development: Regulatory Considerations — Complying With the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act Guidance for...
The FDA has approved Omisirge (omidubicel-onlv) from Gamida Cell Ltd. Omisirge is a substantially modified allogeneic cord blood-based stem cell therapy, administered as a single intravenous dose and indicated for the treatment of hematological malignancies. It is...
Report to the European Commission on companies and products that have benefited from any of the rewards and incentives in the Paediatric Regulation and on the companies that have failed to comply with any of...
A recent commentary published in the American Journal of Medical Genetics discusses the need to transition away from a “one disease at a time” model in newborn screening and drug development, and potential strategies for addressing this...
A study has been published in Singapore Medical Journal providing an overview of the existing treatment strategies for paediatric genetic diseases (PGDs), including recent advancements.
A new study published in the European Journal of Pediatrics explores the role paediatric biobanks can play in overcoming some of the challenges to improving biomedical research for children. Particularly in the case of rare diseases, which...
A recent study published in Cancer Medicine proposes strategies which could help incentivise the development of drugs specifically intended for paediatric cancers in Europe and the United States. While such incentives already exist for both paediatrics and...
Today, the U.S. Food and Drug Administration issued draft guidance that, when finalized, will provide the agency’s perspective on the ethical considerations for including and protecting children in clinical trials. The draft guidance is intended to assist...
