Advancing the Battle Between Rare and Orphan Diseases
RareValue Accelerates and Improves the Performance of Investments
Treatments in development for rare and orphan diseases represent a powerful opportunity for patients to see a far different and better future; and a promising opportunity for companies that can cut through the fog and overcome the data desert that complicates any investment thesis.
But the biopharmaceutical industry faces profound challenges in overcoming unclear epidemiology and planning with uncertain commercialization potential. In short, the amount of usable data and insights, small patient populations, and imperfect analogs conspire to defer or deter investment and progress. The question is how to unlock the treatment potential for rare diseases while creating a sound investment thesis for individual investments and the portfolio at large.
BluePrint Orphan set out to address this challenge - to create a decision model for preclinical and clinical stage investments to help unleash the full treatment and commercialization potential in rare and orphan diseases. The objective was to create a robust and repeatable analytic for the 7,000 rare and orphan diseases that represent different risk/reward dynamics, assess the potential of molecules - and do this while enabling filtering by therapy type (e.g. gene therapy, cell therapy) and considering regional differences.
We developed RareValue to create a robust, repeatable model to synthesize and quantify “ground-up” signals – and place this in an interactive tool that can become a living decision support system for investment and portfolio planning decisions.
The model itself is based on numerous individual market opportunity assessments. In executing these efforts, we found a common algorithm that best reflected the opportunity from 5 factors that we think matter most in determining the investment potential:
The market is rapidly turning its attention to rare and orphan diseases as the ability to improve lives is matched with extraordinary commercial opportunities. It is an attractive but data- and insights-poor market which may defer or deter investments. BluePrint Orphan’s RareValue model breaks through the fog and uncertainty, giving biopharmaceutical companies the ability to make informed decisions at the individual and portfolio level. RareValue becomes an important accelerant to advancing the market with the hope of improving the lives of those affected by rare and orphan diseases.
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