obtaining orphan product designation challenges and pitfalls marlene e. haffner, md, mph president...
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Obtaining Orphan Product Designation
Challenges and pitfalls
Marlene E. Haffner, MD, MPHPresident and CEOHaffner Associates, [email protected]
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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/HowtoapplyforOrphanProductDesignation/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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