Orphans at FDA: The Fundamentals

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Orphans at FDA The Fundamentals

• Timothy Coté MD MPH, Director
• Office of Orphan Products Development, FDA
• Presented in Copenhagen, Denmark
• November 5, 2008

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By the conclusion of this talk, the student
audience will be able to.

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Learning Objectives

• Explain the rationale of the Orphan Drug Act of
  1983.
• Name the four principle functions of the FDA
  Office of Orphan Product Development.
• Give the two criteria for FDA orphan drug
  designation status and contrast this with the
  EMEA criteria.
• Cite the three financial incentives of orphan
  drug status.

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Learning Objectives, Continued

• Explain the OOPDs role in device regulation.
• Describe the FDAAA 2007 incentive for tropical
  disease
• Know the eligibility criteria for an orphan
  product grant.
• Compare/contrast the regulatory roles of OOPD and
  the review divisions.

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Explain the rationale of the Orphan Drug Act of
1983.

• They are like children who have no parents, and
  they require a special effort.---Congressman
  Henry Waxman

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Name the four principle functions of the FDA
Office of Orphan Product Development.

• Designate drugs as having orphan status.
• Award grants for the clinical development of
  these drugs.
• Regulate orphan devices through the Humanitarian
  Use Device (HUD) program.
• Serve as FDAs rare disease focal point with
  outreach to patient groups and industry.

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Give the two criteria for FDA orphan drug
designation status and contrast this with the
EMEA criteria.

• Rare disease or condition prevalence of lt200,000
  Americans.
• Medical rationale Basis of evidence that the
  drug holds promise for being effective in
  treating the disease.
• EMEA, 1999 Add to these significant benefit
  and seriousness criteria.

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Orphan Designation and Approvals
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Diseases/Conditions Targeted by Designated Orphan
Drugs
2000-2006 Data
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Cite the three financial incentives of orphan
drug status.

• Orphan exclusivity
• 7 years
• Better than a patent
• Exemption from fees
• Tax credits (50) for clinical development costs.

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Explain the OOPD's role in device regulation.

• Separate legislation established two interlocked
  programs
• OOPD confers Humanitarian Use Device (HUD)
  status.
• Criteria Device for disease with 4,000 persons
  per year (Incidence).
• CDRH review division awards Humanitarian Device
  Exemption (HDE) to HUDs.

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Describe the FDAAA 2007 incentive for tropical
disease drugs

• Drugs for diseases that are common elsewhere but
  rare here qualify for orphan status.
• Little to no development in this area for past
  50 years.
• FDAAA 2007 established Priority Review Vouchers
  (PRVs) to create incentives for their development.

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• Priority Review Voucher Mechanics

Marketing Approval for Tropical Disease Drug
(Orphan) Tropical Disease Drug
Successful NDA/BLA
Voucher generation
Priority Review Voucher
Voucher Transfer
(Possible Blockbuster) Other Drug NDA/BLA
Big PhRMA Company
?
FASTER
Voucher Redemption
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Know the eligibility criteria for an orphan
product grant.

• 400K/yr for 4 yrs 10-20 new grants per year.
• Clinical Trials for an Orphan product.
• For-profit or not-for profit
• Domestic or International
• Drug or device

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History of OOPD Grants Program
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Compare/Contrast FDA/OOPD v. Review Divisions
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QUIZ!!

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Recent Danish Designations.
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Summary

• Orphan products development are the future.
• FDA/OOPD stands ready to help.
• Thank you for this opportunity to make new
  friends.