Orphan Drugs a Growing Source of New Business Activity


In 2013, fully one third of approved New Molecular Entities (NMEs) were orphan drugs. Back in 1983, when Congress first established the orphan-drug designation, there was only one new such treatment per year across the industry; in the ensuing decades, that number has jumped to almost 200 per year. Today, nearly 33% of FDA-approved drugs are for rare diseases.

Many orphan drugs are approved for diseases that were previously untreated, and therefore likely to command high prices. Having a quicker approval process, specific tax benefits and extended protection from competition post-approval, orphan drugs represent an attractive niche opportunity for the outsourcing sector. Indeed, the consensus forecast suggests that by 2018 orphan drug sales will constitute around 16% of the entire prescription market.

The PharmSource Lead Sheet enables you to quickly identity potential new business opportunities with bio/pharmaceutical companies doing orphan drug development. These opportunities are tagged in the weekly reports, and searchable in the database in three easy steps using the Search for Leads feature:

1. Select the time period of interest
2. Select the Orphan/Fast Track tab, then click the box for Orphan
3. Export leads to Browser view or directly to an Excel download

Users can further refine their selections by Geography, Therapeutic Category, Dosage Form, etc.

So far in 2014, 122 molecules from Phase I to Phase III have been registered as Orphan Drugs, a significant increase over the same period last year.

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Saul is PharmSource’s Director of Market Intelligence. He brings over 15 years of experience in market analysis, having worked in business intelligence roles at Evaluate Pharma, Cardinal Health (now Catalent) and Abbott Laboratories. Prior to that, he was a Post-doctoral fellow in Neuropharmacology at the Universities of Birmingham and Bristol.  Saul holds an MSc (Neuroscience) from the Institute of Psychiatry (King’s College London) and a D.Phil in Neuropharmacology from the University of Oxford.

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