Breakthrough therapies for rare diseases command premium pricing

The global orphan drugs market presents plenty of opportunities for new drug development – while there are only 172 approved orphan therapies, over 6,800 orphan diseases exist according to the US National Institutes of Health (NIH).

Advancements in drug discovery capabilities coupled with regulatory and financial incentives are helping generate rich, competitive pipelines of breakthrough treatments with true disease modifying properties. Pharmaceutical and biotechnology companies are now rolling out therapies for serious, rare diseases – going beyond palliative care and targeting the underlying pathology to slow down or stop disease progression – as they value the financial and philanthropic rewards this brings.

A new analysis from Frost & Sullivan's Product and Pipeline Assessment of the Global Orphan Drugs Market identifies rare cancers as the orphan therapeutic area with the highest level of drug development activity. Other disease areas witnessing considerable drug development activity include blood/lymphatic system diseases, infectious/parasitic diseases, neurological diseases, metabolic diseases, and immunological/inflammatory diseases.