The orphan drug area is relatively new but fast growing. Over the next weeks, Black Swan Consulting will summarise information on this class of drug products. Please also see http://black-swan-consulting.com/what-is-cooking/Orphan-drugs.

1. What is an orphan drug?

2. Orphan drug categories
Products normally fall into three categories*:
1. Products intended to treat a specific rare disease
2. Products withdrawn from the market for economic or therapeutic reasons. For example
thalidomide
3. Products that have not been developed either because they are derived from a research
process that cannot be patented or because they concern important markets which are,
however, not creditworthy
2*www.orpha.net

3. Rare means rare!
§ US (FDA) definition: drugs and biologics which are defined as those intended for
the safe and effective treatment, diagnosis or prevention of rare diseases/disorders
that affect fewer than 200,000 people in the U.S.
§ EMA (EU) definition: drugs intended to treat fatal or severely debilitating diseases
affecting less than 5 per 10,000 inhabitants -­> less than 225,000 EU patients
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*www.orpha.net

4. Ultra-­orphan drug
§ Not an official regulatory category
§ Prevalence of ultra rare orphan disease is less than
1:50,000 people
– Fabry disease
– Gaucher disease
– Cystinosis
– Paroxysmal nocturnal hemoglobinuria
§ Examples of drugs:
– Soliris, Alexion
• paroxysmal nocturnal hemoglobinuria
• approx US$ 400,000 p.a.
• 1-­2 cases per million
– Naglazyme, BioMarin
• Maroteaux-­Lamy syndrome
• approx US$ 400,000 p.a.
• may be 1 case per million (newborn)
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5. What is special about orphan drug?
§ To encourage companies to develop drug and medical devices for rare and
potentially non-­profitable disease areas regulators offer certain incentives:
– Market exclusivity
– Enhanced patent protection
– Research support
– Regulatory support (fast track and fee waivers)
– Tax relief
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Theoretically, or under normal circumstances, rare or orphan
diseases are unattractive to companies due to a combination of high
R&D costs and low number of patients available for treatment.
Moreover, for most products a strong health economical argument
cannot justify needed price point.

6. US orphan drug act of 1983
§ Additional benefits include priority FDA review and waiver of regulatory fees
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Funding and support to perform clinical trials
A 50% tax credit of clinical development costs
Market exclusivity for 7 years upon registration

7. Impact of US orphan drug act of 1983
§ Fewer than 10 orphan drugs supported by industry came to market between 1973 and 1983.
§ More than 400 drugs and biologic products for rare diseases since 1983
§ The Orphan Grants Program has been used to bring more than 45 products to marketing approval.
§ The Humanitarian Use Device Program has been the first step in approval of more than 50 Humanitarian Device
Exemption approvals.
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8. EU orphan medical products
(legislation adopted in 1999 and later amended several times)
§ Additional EU research grants are available for development
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Direct access to the centralised procedure
Free scientific advice and 50% reduction in fees
Market exclusivity for up to 10 years

9. Impact of EU orphan medical product legislation
§ Approximately 50 products were approved over the first 10 years of
the programme
§ Many companies have failed when trying to bring an orphan drug to
the EU market. Biggest predictor of success is previous success
(increase chance of registration success 17 fold)
§ Programme picking up speed
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10. BSC delivers competent and practical solutions to its life-­sciences clients´
strategic and commercial problems helping them to make and successfully
implement optimal business decisions.
Black Swan Consulting
Questions
www.black-­swan-­consulting.com peder.andersen@blackswanconsulting.ch
+41 79 500 1 500