Gabriel Revon-Riviere, Leah C Young, Elizabeth A Stephenson, Kathy Brodeur-Robb, Sarah Cohen-Gogo, Rebecca Deyell, Thierry Lacaze-Masmonteil, Antonia Palmer, Rulan S Parekh, James A Whitlock, Daniel A Morgenstern
Innovative therapeutic approaches are needed to alleviate the burden of life-limiting, rare, and chronic conditions affecting children, adolescents, and young adults (CAYA). This includes a need for improved access to both clinical research and to non-approved or off-label therapies, together with, ultimately, more therapies achieving regulatory approval in Canada. The single patient study (SPS), also known as an open label individual patient (OLIP) study, was introduced by Health Canada to open access to non-marketed drugs where a clinical trial is not readily available, but the drug is considered too investigational to be managed on a standard Special Access Program...
November 2023: Paediatrics & Child Health