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Orphan Products

Orphan Products Surrogate endpoints Biomarkers Accelerated approval Fast Track Approval Priority Review and more…. Marlene E. Haffner, MD, MPH Haffner Associates, LLC Arizona Innovation Conference September 20, 2011. Orphan Products. Treats a population of < 200,000 in the US

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Orphan Products

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  1. Orphan ProductsSurrogate endpointsBiomarkersAccelerated approvalFast Track ApprovalPriority Review and more….Marlene E. Haffner, MD, MPHHaffner Associates, LLCArizona Innovation ConferenceSeptember 20, 2011

  2. Orphan Products • Treats a population of < 200,000 in the US • Same review and development standards as for a non-orphan product • Numbers of patients in clinical trials will be smaller • FDA has two special units to deal with orphan products Office of Orphan Products Development/Office of the Commissioner Associate Director for Orphan Products in the Office of New Drugs (OND)/CDER

  3. Orphan Products – incentives for development • 7 years exclusive marketing for that drug for that disease • Tax credits for clinical development – 20 yr carry forward; 1 year fall back • Waiver of NDA filing fees ($1.5 million FY 2011) • Grant support - $450,000/annum for 4 years

  4. Orphan Products • More than 7,000 rare diseases • More than 350 drugs for orphan diseases have been developed since 1983 • Burgeoning field – large and small companies involved • Money to be made • Review times may be faster as majority of orphan diseases serious or life threatening

  5. Surrogate Endpoints • Measure of effect of effect of treatment that may correlate with a real endpoint • No guaranteed relationship • Needs validation • E.g. cholesterol is related to heart disease, but many with high cholesterol do not develop heart disease. • So cholesterol lowering drugs lower cholesterol but that alone does not prevent death. Proof of efficacy in preventing heart disease took 5 years to show.

  6. Surrogate endpoints • May be able to shorten a clinical trial with the us of VALIDATED endpoints • Great interest currently; may not correlate

  7. Biomarkers • Indicates a disease state or some physiological state of the organism • May be used to measure the progression of disease or the effects of treatment • Rheumatoid Factors as a marker for Rheumatoid Arthritis; elevated PSA for prostate cancer • May relate to how a drug will/will not act in the body – KRAS gene expression in Colorectal Cancer

  8. Biomarkers • May assist in identifying high-risk individuals so they can either be treated prior to onset of disease of shortly thereafter • Test must be rapidly available and easy to carry out • Must be validated and related to a positive outcome

  9. Accelerated Approval • Treatment of Serious or life-threatening illness • Must have meaningful benefit over existing therapies • May utilize a surrogate endpoint that is “reasonably likely…to predict clinical benefit” • May have restricted prescribing • Most likely will have post marketing requirements

  10. Fast-Track Mechanism • Must address a serious or life threatening illness • Potential to treat unmet need • Multiple meetings with FDA to discuss development plans • Options to submit sections of the NDA/BLA as they are ready

  11. Priority Review • Begins on submission of NDA/BLA • Shortens review time from 10 months to 6 months • May also have “Fast Track”

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