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The Orphan Drug Boom: Gold Rush Or Flash In The Pan?

Executive Summary

Rare disease drug development has ballooned in just three years, powered by the success of independent biotechs like Alexion, Big Pharma’s entrée into the field, and pressure from regulators and payors that is dis-incentivizing development of traditional primary care drugs. The positive momentum has intensified dealmaking in the orphan drug space and is giving investors confidence to back rare disease-focused start-ups. Some have coined the resulting movement the “orphan drug bubble,” but interest isn’t likely to deflate soon – at least not as long as Big Pharma continues to invest in the area and industry is able to sustain a favorable reimbursement climate for ultra-premium-priced drugs.


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