Accurate forecasting in rare and ultra-rare diseases is one of the biggest challenges in biopharma today. With tiny patient populations, fragmented data, and fast-evolving treatment landscapes, traditional forecasting models often fall short. This white paper unpacks why forecasting in rare diseases demands an entirely different mindset, and how companies can evolve their methodologies, tools, and culture to keep pace.
Why does this matter?
As precision medicine and gene therapies push the boundaries of what’s possible, the commercial and analytical demands on rare disease forecasters are higher than ever. Yet, misjudging patient populations, uptake rates, or access barriers can have million-dollar consequences, both financially and for patients waiting for treatment.
This white paper helps pharma and biopharma leaders understand:
- Why conventional models break down when applied to rare and ultra-rare diseases
- How to build data-driven, flexible forecasting frameworks despite limited patient data
- What leading companies are doing differently to achieve credible, defensible forecasts
What’s inside this white paper
Drawing on expert interviews and real-world analysis, including data from Evaluate Pharma, this paper explores:
- Forecasting pitfalls and how to avoid them for rare and ultra-rare diseases: The top four mistakes companies make, plus how to overcome them with smarter data and cross-functional collaboration.
- The data challenge: Why fragmented registries and underdiagnosis distort forecasts, and what to do about it.
- Advanced forecasting approaches: How to blend epidemiological insights, analog selection, and scenario planning for more robust results.
- Role of patient advocacy and real-world data: How partnerships beyond the company can refine forecasts and strengthen market readiness.
- Technology and platform innovation: How cloud-based forecasting solutions are helping organizations overcome “Excel fatigue” and achieve a single source of truth.
What you’ll gain from this paper
Whether you’re a forecaster, commercial strategist, or market access leader, this white paper will help you:
- Recognize early warning signs of overconfidence in forecasting models
- Understand how to model uncertainty transparently and set credible expectations
- Learn from real-world examples of over-forecasting and under-forecasting in rare disease launches
- Identify data and infrastructure gaps that could derail your next rare disease forecast
Forecasting rare diseases isn’t just about numbers; it’s about delivering clarity where uncertainty rules. Download the white paper now to discover how leading companies are reimagining forecasting for the next generation of rare and ultra-rare therapies.
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