From Bench to Bedside to Practice: a Practical Course Genetic Alliance Annual Conference July 28, 20

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From Bench to Bedside to Practice a Practical
Course Genetic Alliance Annual ConferenceJuly
28, 2006

• How do We Get to Treatments?
• The Orphan Drug Act Incentives to Drug
  Development
• Marlene E. Haffner, M.D., M.P.H.
• Director, FDA Office of Orphan Products
  Development

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The Mission of The Office of Orphan Products
Development (OOPD)

• To assist and encourage the identification,
  development, and availability of safe and
  effective products for people with rare
  diseases/disorders.

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Why is our mission important?

• Millions of Americans suffer from one of the
  approximately 6,000 identified rare diseases.
• The same is true in Europe.
• No effective treatment is available.
• 85 are Serious/Life Threatening.
• 50 are Pediatric.

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How do we Achieve our Mission?

• Orphan Drug Designation Program
• Grants Program
• Humanitarian Use Device Designation Program
• Interacting with government agencies, medical and
  research communities, pharmaceutical and device
  industries, professional organizations, rare
  disease groups, and concerned citizens

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What Are Orphan-Drug Designation Incentives?

• Seven-year marketing exclusivity upon FDA
  approval
• Other incentives
• Tax credit equal to 50 of clinical investigation
  expenses
• Exemption of PDUFA application fees
• Assistance in drug development process
• Orphan products grant funding
• OOPD assistance

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Marketing Exclusivity - Most Sought Incentive
Received upon FDA approval of specific
drug for specific indication

• 7 years following FDA market approval
• FDA cannot approve same drug for same indication
  during exclusivity period
• Except with consent of sponsor
• If sponsor cannot provide sufficient quantities

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Eligibility for incentives of the Orphan Drug
Act Designation

• Two of the criteria applied
• - Prevalence lt 200,000
• - Rationale for use of the drug for the
  proposed indication.

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The Orphan Designation Process

• Sponsor submits application to OOPD for specific
  product/specific disease indication.
• Designation must be received prior to New Drug
  Application (NDA)
• Does not affect timing of investigational new
  drug (IND).

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Since 1983

• 1619 active designated orphan products
• 294 approved orphan products
• 227 drugs
• 67 biologics
• Potentially treat 15 million patients

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Unanticipated Positive Outcomes of the Orphan
Drug Act

• Profitability of the product chronicitiy of
  disease.
• Building Biotech science.
• Building large and small pharmaceutical firms.
• Support to the U.S. economy.
• Development of cutting-edge technology.

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Development of Orphan Industry

• Many Small-to-Medium Sized Pharmaceutical
  Companies have been established to develop rare
  disease therapies.
• A large number of biotechnology firms have
  prospered as a result of the U.S. Orphan Drug
  Act.
• Major pharmaceutical organizations have
  subsidiaries devoted to the development of orphan
  drugs.

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Trends in Orphan Product Development

• Targeted therapies
• Recombinant therapies
• Monoclonal antibody therapies
• Addressing the Human Genome

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Grant Support for Investigation of Rare Disease
Treatment

• Supports mainly early trials
• Primarily awarded to academic researchers
• FDA funds approximately 20 new grants per year
• Provides 200,000 to 350,000 in total costs per
  year -- up to 3 years

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40 Products Approved Through Research Funded By
Orphan Grants Program
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Challenges in Clinical Trial Design for Rare
Diseases

• Getting usable clinical data early
• Population Size
• Geographic Distribution
• Disease Severity
• Informed Consent
• Data
• Anecdotal
• Case Reports

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Orphan Products Must Be Just as Safe and Effective

• Orphan products have same review standards as
  non-orphans.

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OPD Web Site
http//www.fda.gov/orphan Your Link to

• Overview of the FDA Office of Orphan Products
  Development
• Guidelines for designation application
• List of designated and approved orphan products
• Grant application information
• List of ongoing orphan grant studies

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Meet the OOPD Staff
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Questions