Key highlights in EvaluatePharma’s Orphan Drug Report 2014:
- A record 260 orphan drug designations were granted in the US in 2013
- Orphan drug sales to grow 11% per year through 2020, compared to 5% for drugs (excluding generics) treating larger patient populations
- Vertex’ VX-809 in combination with Kalydeco was 2013’s most valuable R&D orphan drug based on NPV, while Pharmacyclics’ Imbruvica ranked as the most promising new orphan drug approved by the FDA in 2013
- Bristol-Myers Squibb is set to leapfrog Novartis as the leader in orphan drug sales in 2020 thanks to cancer immunotherapeutic Opdivo
- Phase III orphan drug development cost is half that of non-orphans but development time for orphans is not shorter than for non-orphan drugs
- Anticipated return on investment for a Phase III/filed orphan drug is 1.89 times greater than for a non-orphan drug
Download Available Here. (Source: Evaluate, 2014).