What’s Hot in 2020: Rare Diseases
Discovery
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Joe Cornicelli

What’s Hot in 2020: Rare Diseases

To meet the needs of rare disease drug developers CROs needs better models and innovative partnering   

The pharmaceutical and biotech industries are investing heavily in discovering and developing treatments for rare and orphan diseases, defined in the US, as having patient populations of less than 200,000 individuals. Yet with the existence of ~7,000 rare diseases, it’s estimated that there are between 25 – 30 million Americans afflicted. Over the last 2 years, 40% of FDA approvals were for rare and orphan drugs, and Deloitte estimates that 20% of all prescriptions will be written for these and other drugs of the same ilk by 2024. 

The emphasis in this area has its challenges for the CRO industry. Many animal models for these conditions do not exist and must be created. This takes time and may result in models that don’t fully recapitulate key features of human disorders. Biologic, cellular and genetic therapies are designed to modulate human targets and could fail to recognize the animal orthologues or create an immunologic response. The industry is also re-examining its outsourcing model, shifting away from transactional interactions to a more strategic, thought-partner relationship with CROs. This change in approach and strategic direction will advance the development of relevant models and accelerate the path to approval. Most important, it provides hope to those families dealing with devastating diagnoses.