Rare trials you can trust - Biostatistics
The Unique Contribution of Biostatisticians Specializing in Rare Conditions.
Although difficult, it is possible to run high quality trials involving small numbers of people scattered around the globe. For the challenges experienced when running rare disease studies you need an expert team who will work seamlessly together to design and deliver something that fits. There is a group of people who quietly form part of this expert team. These people sit behind trials and provide a pivotal foundation. The term ‘biostatistics’ may not be at the top of news articles, but biostatistics is at the core of every single piece of research.
It is the critical element that is needed so that we know how much we can trust the results. Biostatisticians working in the field of rare disease are, themselves, uniquely qualified for the challenges the area brings – people with years of experience who are driven to use their skills every day to help others.
What’s Inside?
This white paper aims to shed light on the difference that biostatisticians specialising in rare conditions bring to this united trials-team.
- The role of biostatistics.
- The 3 key areas for designing a successful trial to test the benefit-risk of a drug in rare disease:
- Natural history of the condition in question – understanding what you’re seeing and knowing what to measure.
- Constraints of study delivery e.g. small population and study design optimisation, simulations of the trial to optimise trial performance.
- Power to make a difference – address the aspect of disease that matter – be it in terms of quality of life, treatment of a disease, or symptom control.