We were delighted to present a paper at the EphMRA UK chapter one day meeting on Tuesday 21st March. Head of Analytics, Richard Goosey, and Director, Emilie Braund, co-presented ‘Small populations, big challenges: How to conduct quantitative research in rare diseases’.
Like all other types of drugs, therapies that can help people with rare diseases require accurate forecasts and demand assessments to be able to bring them to a successful launch. And post launch, performance still needs to be tracked effectively. With such small populations, finding sufficient sample to make quantitative evaluations can be extremely difficult but is nevertheless highly necessary.
In their presentation, Richard and Emilie explored some of the key challenges of conducting rare disease market research, demonstrated how to overcome them using a range of small sample analytical techniques, and outlined what is possible with small data. Through a series of recent project case studies, they outlined what is possible with small data and explained how advanced analytical techniques can be used to forecast demand, assess optimal clinical trial end points, inform pricing, communications, brand tracking and targeting.