Download DIA 2011 Annual Meeting

Obtaining Orphan Product
Designation
Challenges and pitfalls
Marlene E. Haffner, MD, MPH
President and CEO
Haffner Associates, LLC
[email protected]
Requirements for Designation
• Population less than 200,000 in the US
• Reasonable consideration that the product
will have utility for the stated indication
• Need some data – preferably clinical or
animal
• IND helpful but not a requirement
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Obtaining your designation
• Orphan Products “tip sheet”
• http://www.fda.gov/ForIndustry/DevelopingPr
oductsforRareDiseasesConditions/Howtoapp
lyforOrphanProductDesignation/ucm135122.
htm
• Aka – www.fda.gov/orphan - how to apply for
orphan products designation
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What is the Disease – what is a subset
• Define the disease
• Is that what you are treating or are you
treating a symptom of the disease?
• What is a subset of a disease?
A part of a disease that can be treated by the
drug. However, the disease as a whole cannot
be treated by the drug. WHY??
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When is a subset valid
• NOT because that is what you wish to study
• Because the drug is not useful for the “larger” disease
– It is too toxic
– Genetic markers make it ineffective – HER2 +,
KRAS mutant – except in the subset
• More effective treatments for other segments of the
disease – surgery for stage 1
• Pediatric manifestations of a disease are valid
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Not always straightforward
• Erythropoietin for anemia of:
– ESRD
– Anemia of prematurity
– Blackfan Diamond syndrome
• Product for obesity in
– Obese adults
– Prader Willi syndrome
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How is subsetting determined
• Review the disease
• What is the mechanism of action in the
disease
• Are you treating the disease or a symptom of
the disease – if a symptom, then is the
symptom prevalence attributed only to “that”
disease – post-op pain vs pain
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Defining Prevalence
• If an oncology drug – NIH SEER Data –
http://Seer.cancer.gov
• If a metabolic disease – published literature; Mendelian
inheritance (McKusick) – www.OMIM.org
• If infectious disease – start with CDC
• Sometimes hospital discharge summaries
• All other – published literature – texts and current
articles in peer reviewed literature
• Not always easy
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Establishing Prevalence
• Use of experts – only in VERY rare diseases
• Discuss issues of defining prevalence in your
designation request
• Be straightforward. The OOPD (Office of
Orphan Products Development) reads the
literature
• If a range – OOPD will assume upper end
• Importance in higher numbers - >100,000
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Non Profitability in 7 years
• Has occurred only 3 times since 1983
• Twice for products to treat narcotic addiction
• Once for a product already marketed for
another indication and about to go off patent
• Difficult hurdle; must open books for review
• Developing an unprofitable product is “not
the usual.”
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THANK YOU
?
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