Leader Board: 2020

R&D Spend vs. Return on Investment

By 2020:Worldwide orphan drug sales forecast

to be $176 billion.

Compound Annual Growth Rate

for orphan drugs will be 2x overall drug market,excluding generics.

BMYSales

Drug

R&D

Revlimid (lenalidomide)

VX-809 + Ivacaftor

BMY set to climb to #1 position in orphan drug sales in 2020

#1 orphan drug in 2020

Most valuable R&D orphan drug

Phase III orphan drug development

costs ½ that of non-orphan drugs,

potentially ¼ after US tax breaks.

Phase III orphan drugs expected ROI

to be 1.89x non-orphan drugs.

Median Cost per Patient Differential

19x higher for orphan drugs

Compared to non-orphan

$98,534 vs $5,153

$755 million | Orphan (Top 10)

Avg. USA Sales 5 Years After Launch(2013 FDA Approvals)

Avg. Phase III Trial Size(2013 FDA Approvals)

$1,755 million | Non-Orphan (Top 10)

1,310 patients | Orphan (Top 10)

Orphan Drugs

Sales 2020

Non-Orphan Drugs

Orphan drugs will be 19.1% of worldwide prescription sales, excluding generics.

Trends in Sales

Download the full report at www.evaluategroup.com/orphandrug2014Copyright @ 2014 Evaluate Ltd. All rights reserved.

6,811 patients | Non-Orphan (Top 10)

Orphan Drug Report 2014