Phase I study to assess safety and tolerability of investigative medication in pediatric patients with Sanfilippo syndrome (MPS IIIA)
A specialty healthcare company developing treatments for rare diseases sought our support for an early-stage pediatric trial program testing an investigational medicine in Sanfilippo syndrome. We were commissioned to run a nine-month online campaign to raise the profile of the program and to find and refer up to four of the nine patients needed for […]
Patient preferences for injectable drug-delivery options
A pharmaceutical company commissioned us to survey sickle cell patients to understand how people living with the condition handle their pain crises and hospital visits, and how they would feel about different ways to take or receive a new injectable medication. Specifically, the client wanted to know if any factors influence preference for auto-injector devices […]
Longitudinal, real-world data to support US FDA submission
A specialist pharmaceutical company commissioned us to recruit a group of people living with pouchitis, a rare gastrointestinal condition, and to track how it affects their lives over a six-month period. The objective was to generate real-world data to support a future regulatory submission to the US FDA, for approval of the company’s investigational drug. […]