Obtaining Orphan Product Designation

Challenges and pitfalls

Marlene E. Haffner, MD, MPHPresident and CEOHaffner Associates, LLCmhaffner3@verizon.net

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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