Webcast: Small populations, big challenges: How to conduct quantitative research in rare diseases

Misti Paul, Steven Helm & Sarah Reynolds - February 2021

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Although there are around 7,000 classified rare diseases that affect millions of people worldwide, comparatively few treatments are currently available to treat them. In recent years, many countries including the US have begun incentivising investment into research and development and have implemented fast-track processes that expedite their approval, increasing patient access to therapy.

As a result, orphan drugs are predicted to represent over 20% of global prescription sales by 2022 (excluding generics). With forecasted sales worth $209bn per year, they present a major opportunity.

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.

Watch the webcast in which Director of Advanced Analytics Misti Paul, Associate Director Steven Helm and Senior Analytics Executive Sarah Reynolds explore some of the key challenges of conducting rare disease market research and demonstrate how to overcome them using a range of small sample analytical techniques. Together, they outline what is possible with small data and how Research Partnership can help to provide you with valuable rare disease insights.

What are the key takeaways?

  • Get a brief overview of the rare disease marketplace
  • Learn about the range of quantitative research objectives that can be achieved when using small samples
  • Discover how our advanced analytical techniques can be used to forecast demand, assess optimal clinical trial end points, inform pricing, communications, brand tracking and targeting.

Watch the webcast

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