AzCI presents: Specialty Programs in FDA Review and Approval
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Transcript of AzCI presents: Specialty Programs in FDA Review and Approval
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ARIZONA CENTER
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Innovation Center
The Arizona Center for Innovation (AzCI) assists technology companies turning their innovative ideas into successful businesses through:
• Focused programs • World-class expertise • High-quality facilities
Access:• Other technology
entrepreneurs• Collaborative,
creative environment
• Advantage of hands-on support
• Successful business leaders
Arizona Center for Innovation
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AzCI
Startup and emerging technology companies• Community and UA spin-outs
Technology• Aligned with UA Tech Parks and UA
International• Softlandings
Who
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AzCI
Technology Areas: • Security and Defense• Mining• Agriculture and Water• Biotechnology• Intelligent Transportation and Vehicles• Renewable EnergyInformatics/Big Data/Advanced Manufacturing/Imaging/Optics/Photonics
Focus
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And more…
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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
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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
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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
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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.
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May be able to shorten a clinical trial with the us of VALIDATED endpoints
Great interest currently; may not correlate
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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
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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
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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
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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
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Begins on submission of NDA/BLA Shortens review time from 10 months to 6
months May also have “Fast Track”
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https://techparks.arizona.edu/azci
©Arizona Center for Innovation 2015