The Office of Orphan Products Development Grant ProgramJanet Whitley, Ph.D.Office of Orphan Products

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The Office of Orphan Products Development Grant
ProgramJanet Whitley, Ph.D.Office of Orphan
Products DevelopmentFood and Drug Administration
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Office of Orphan Products Development

• Orphan Drug Designation
• Drugs/biologics
• Orphan Products Grants
• Drugs, Biologics, Devices, Foods
• Humanitarian Use Device Designation
• Humanitarian Device Exemption

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Grant Program Objective

• To fund clinical research that will accelerate or
  assist in the approval of products which
  demonstrate promise for rare diseases/disorders.
  

Basic Research Funding
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Grant Program Objective

• To fund studies leading to publications in
  peer-reviewed journals.
  
• Supports mainly Phase 1 and 2 trials
• Funding may be used to cover portion of larger
  trial
  
• Importance of funding

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Grant Support for Investigation of Rare Disease
Treatment

• Awarded to both academic and industry-sponsored
  research
  
• FDA funds approximately 20-30 new grants per
  year
  
• Provides 150,000 to 300,000 in direct costs per
  year -- up to 3 years

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OPD Grant

• 50/400 grants awarded to industry sponsors
• Grantees are US and abroad
• Orphan Designation not required
• New drug/product or new use of approved product

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Funding

• FY 2003
• 101 responsive applications
• 15 received funding
• Currently 65 Active grants

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Orphan Grant Application Process

• Issue Federal Register notice (RFA).
• Meets program review criteria.
• Review panels
• Ad hoc panels of outside experts review
  scientific merit
  
• Regulatory review
• Prepare summary statements.
• Present to Advisory Council.
• Advise - Fund - Monitor.

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Grant review Responsive to Program Review
Criteria?

• Study is a clinical trial.
• Prevalence is fewer than 200,000 U.S. patients.
• Study must be performed under an active IND/IDE
  (except medical foods).
  
• Budget is within limits.
• Availability of sufficient product.

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Grant review Scientific and Technical Merit?

• Soundness of the rationale
• Appropriateness of study design
• Statistical justification for proposed enrollment
  - POWER
  
• Potential for patient accrual
• Qualifications of investigator support staff

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Grant review Scientific and Technical Merit?

• Adequacy of resources and environment
• Budget Justification
• Informed consent documents and IRB approval
• Potential for completion of study within stated
  time and budget

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Not funded because

• Non-responsive application
• Overall quality of application clarity and
  detail
  
• Scientific merit
• Preliminary data, poor study design, weak
  rationale, sketchy analysis plan
  
• Compelling nature of study
• Resubmissions that dont follow/address panels
  suggestions/concerns
  
• Feasibility
• Number of patients, drug supply, too much to do
  in too little time
  

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Management of Funded Grants

• Assignment to Project Officer
• Liaison between grantee and FDA review division.
• Enrollment goals/achievement.
• Quarterly progress updates.
• Site visits to assure grant compliance.
• Sponsor acquisition.
• Continuation funding dependent on progress.

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Grant Program Accomplishments

• 36 product approvals supported by OPD grants
• Hundreds of publications, abstracts, and
  presentations have been produced as a result of
  orphan product grant studies.
  

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FY2005 Request for Applications (RFA)

• Federal Register Announcement
• August
• OPD website
• Application Due Dates
• October 2004
• April 2005

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Responsibilities of the Office of Orphan Products
Development in Evaluating Humanitarian Use
Devices (HUD)
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Background

• Makers of new medical devices ordinarily must
  show that products are safe and effective.
  
• For humanitarian purposes, FDA will approve
  devices limited to use only in rare
  diseases/conditions if manufacturers demonstrate
  the safety and probable benefit to patients.

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HUDs vs HDEs

• Two-step process
• 1 HUD FDA will consider the use and size of
  the proposed patient population
  
• 2- HDE Device is evaluated for safety and
  probable benefit in the CDRH Office of Device
  Evaluation.

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What is a HUD?

• A Humanitarian Use Device (HUD) is a medical
  device intended to benefit patients in the
  diagnosis and/or treatment of a disease or
  condition that affects or is manifested in fewer
  than 4,000 patients per year in the United States.

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What is an HDE?

• A Humanitarian Device Exemption (HDE) is a
  provision that exempts sponsors of devices for
  orphan diseases from the effectiveness
  requirements of the medical device law, provided
  the device meets safety conditions and will not
  expose patients to significant or unreasonable
  risk.

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Diagnostic/prevention devices

• Sponsor must demonstrate that the less than 4,000
  patients/year would be subjected to diagnosis by
  the device in the U.S.
  
• PKU screening in newborns
• Rare disease
• 4,000,000 newborns screened yearly

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How is a HUD Review Performed?

• Evaluate rationale and population.
• Approve, disapprove, or request additional
  information.
  
• Review clock 45 days from receipt of
  request/supplemtent.

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Refusal to approve HUD request

• Population 4,000
• OPD consults with review division re use
• Sponsor points to approved HUD population
• Medically plausible basis to limit the use of a
  product to a particular population
  
• Usually risk-to-benefit
• Feasibility of assay/rationale
• Cost not a factor (not in regs)

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Statistics

• Since October 1996
• 138 requests received.
• 96 devices designated as HUDs by OPD.
• 36 devices approved as HDEs by CDRH.

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Contact Information

• Website www.fda.gov/orphan
• Information on HUDs, Orphan Designation, and
  grants (RFA)
  
• Phone [phone]
• Snail 5600 Fishers Lane
• HF-35, Room 6A55
• Rockville, MD 20857