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The Bright Future of Orphan Drugs

Posted by | 2:04pm on Thursday, June 6, 2013
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orphan drugs will be 15.9% of worldwide prescription sales by 2018 (excluding generics.)  

I believe that the pharmaceutical industry is moving towards the rare disease marketing model. I’ve built my entire company around this idea and so was interested to read the industry overview, Orphan Drug Report 2013 from EvaluatePharma. The data reinforces the idea that the orphan drug niche is a growing sector within the pharmaceutical industry. This report predicts worldwide orphan drug sales to total $127 billion by 2018 with a compound annual growth rate of +7.4% per year between 2012 and 2018. The authors forecast that orphan drugs will be 15.9% of worldwide prescription sales by 2018 (excluding generics.)

In 1998 orphan drugs represented only 5.1% of worldwide prescription sales. The success of the US Orphan Drug Act in 1993 has led to similar measures being adopted in other markets, including Japan in 1993 and the European Union in 2000. In 2012 European orphan drug designations were up 44% and Japan designations up 33%. In the US the number of new orphan drug designations declined 7% in 2012 to 188, from a peak of 203 in 2011.

Interestingly, on average it takes 9 months for an orphan drug to receive FDA approval which is only 1 month less than the 10 months for non-orphan therapies. The report also highlights that for some rare diseases there is a lot of competition. For example, Acute Myeloid Leukemia is the indication with most filed orphan drug designations in EU.

Top companies
The report forecasts that Novartis will maintain the top position in orphan drug sales to 2018 with BMS, GSK and Sanofi moving up. See the chart below for their predictions.

Worldwide Orphan Drug Sales (2012/18): Top 20 Companies & Total Market

WW Orphan Sales ($bn) WW Market Share Rank
Company 2012 2018 % CAGR 12-18 2012 2018 Chg. (+/-) Chg. (+/-)
1 Novartis 10.9 11.8 +1% 13.1% 9.9% -3.8% -
2 Roche 9.0 9.4 +1% 10.8% 7.4% -3.4% -
3 Celgene 4.9 8.6 +10% 6.0% 6.8% +0.8% +2
4 Pfizer 5.4 7.2 +5% 6.6% 5.6% -0.9% -1
5 Sanofi 2.9 5.5 +11% 3.5% 4.3% +0.8% +4
6 Bayer 4.2 5.2 +3% 5.1% 4.1% -1% -
7 Bristol-Myers Squibb 1.7 4.7 +18% 2.1% 3.7% +1.6% +9
8 GlaxoSmithKline 1.4 4.1 +20% 1.6% 3.3% +1.6% +12
9 Alexion Pharmaceuticals 1.1 3.7 +22% 1.4% 2.9% +1.5% +12
10 Baxter International 2.5 3.2 +4% 3.0% 2.5% -0.6% +2
11 Eli Lilly 2.6 3.0 +2% 3.1% 2.3% -0.8% -1
12 Biogen Idec 2.9 2.9 +0% 3.5% 2.3% -1.2% -4
13 Shire 1.7 2.8 +9% 2.0% 2.2% +0.2% +4
14 Merck KGaA 3.0 2.6 -2% 3.6% 2.0% -1.6% -7
15 Johnson & Johnson 1.8 2.6 +7% 2.1% 2.0% -0.1% -
16 Novo Nordisk 1.5 2.4 +8% 1.9% 1.9% +0% +2
17 Vertex Pharmaceuticals 0.2 2.2 +53% 0.2% 1.7% +1.5% +28
18 Amgen 2.6 2.0 -4% 3.1% 1.6% -1.5% -7
19 Actelion 1.8 1.8 +0% 2.2% 1.4% -0.8% -5
20 Merck & Co 1.8 1.6 -2% 2.2% 1.3% -0.9% -7
Total Top 20 64.0 87.3 +5.3% 77.1% 68.5% -8.6%
Other 19.0 40.1 +13.2% 22.9% 31.5%
Total 83.0 127.4 +7.4% 100.0% 100.0%
Source: EvaluatePharma® (23 APR 2013)
Note: All sales analysis based on EvaluatePharma®’s clean ‘Orphan’ sub-set of products, which excludes products where less than 25% of lifetime sales come from orphan indicators.

The future for rare disease therapies looks bright, which is excellent news for the pharmaceutical companies who produce them as well as the patients who are waiting for them. What do you think about this data?

Image courtesy of D Sharon Pruitt on Flickr (CC BY).

About Wendy White

Since founding Siren Interactive in 1999, Wendy has been recognized as a thought leader at the intersection of niche pharma brands, patient empowerment and online marketing. Her vision for how the internet can facilitate interactions and provide crucial information that patients, caregivers and their healthcare providers previously struggled to find has propelled Siren to the forefront of relationship marketing for rare disorder therapies.

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