azci presents: specialty programs in fda review and approval

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Page 1: AzCI presents: Specialty Programs in FDA Review and Approval
Page 2: AzCI presents: Specialty Programs in FDA Review and Approval

ARIZONA CENTER

Page 3: AzCI presents: Specialty Programs in FDA Review and Approval

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

Page 4: AzCI presents: Specialty Programs in FDA Review and Approval

AzCI

Startup and emerging technology companies• Community and UA spin-outs

Technology• Aligned with UA Tech Parks and UA

International• Softlandings

Who

Page 5: AzCI presents: Specialty Programs in FDA Review and Approval

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

Page 6: AzCI presents: Specialty Programs in FDA Review and Approval

And more…

Page 7: AzCI presents: Specialty Programs in FDA Review and Approval

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

Page 8: AzCI presents: Specialty Programs in FDA Review and Approval

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

Page 9: AzCI presents: Specialty Programs in FDA Review and Approval

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

Page 10: AzCI presents: Specialty Programs in FDA Review and Approval

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.

Page 11: AzCI presents: Specialty Programs in FDA Review and Approval

May be able to shorten a clinical trial with the us of VALIDATED endpoints

Great interest currently; may not correlate

Page 12: AzCI presents: Specialty Programs in FDA Review and Approval

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

Page 13: AzCI presents: Specialty Programs in FDA Review and Approval

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

Page 14: AzCI presents: Specialty Programs in FDA Review and 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

Page 15: AzCI presents: Specialty Programs in FDA Review and Approval

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

Page 16: AzCI presents: Specialty Programs in FDA Review and Approval

Begins on submission of NDA/BLA Shortens review time from 10 months to 6

months May also have “Fast Track”

Page 17: AzCI presents: Specialty Programs in FDA Review and Approval

https://techparks.arizona.edu/azci

©Arizona Center for Innovation 2015