Regulatory Boot Camp Bringing a by fjwuxn


									Regulatory Boot Camp: Bringing a Product to Market and
Avoiding Regulatory Pitfalls

                                 December 13, 2007
                                 Mass Medical Society
                                 Waltham, MA
Insert Picture Here
                                 Robert Yocher, M.H.Sc., RAC
                                 VP RA/CQC
                                 Genzyme Corp.
Wide Variety of Devices
   Medical devices vary in terms of the risks that they present:
   Permanent implant or short term patient contact
   Circulating blood contacting or patient fluid contact
   Patient contacting or non-patient contacting
   Energy emitting or passive
   Diagnostic or Therapeutic
   Tool or treatment
Risk Based Classification Provides Flexibility
 Class III devices: Most complex, highest risk
 Breakthrough technology or high risk (life sustaining,life
 Premarket review and approval required through a
  Premarket Approval Application (PMA) or
  Humanitarian Device Exemption (HDE)
 Submitter required to demonstrate reasonable assurance
  of safety and effectiveness
 Clinical trials necessary ($$$$$$!)
 Examples: High intensity focused ultrasound devices,
  drug eluting coronary stents, photodynamic therapy
  devices, deep brain stimulators
Expect Two Submissions
 Investigational Device Exemption and
  PreMarket Approval Application
 Most stringent most lengthy of Device pathways
  (typically 1-3 yrs. or more)
 Contains sufficient “valid scientific evidence” that
  provides “reasonable assurance” that the device is
  safe and effective for its intended use or uses.
Premarket Approval Options
 Expedited PMA
 Streamlined PMA
 Standard PMA
 Modular PMA
 An applicant can request Expedited Review for
  Standard or Modular PMAs
Reality –doesn’t make much difference!
Expedited PMA Review
 Geared toward devices that have a major public
  health impact
 Device that is intended to treat or diagnose life-
  threatening or irreversibly debilitating diseases or
  conditions; and
 Meets one of the following criteria:
Expedited PMA Review Criteria
 Represents a breakthrough technology with a clear
  clinically meaningful advantage over existing
 Has no approved alternative;or…
 Offers significant advantages over approved
  alternatives; or…
 Is in the best interest of patients.
Expedited PMA Review
 Expedited review entitles the marketing application to
  review before other pending applications
    Streamlined PMA Process
 Designed for devices using well-known technologies with well
  known disease processes
 PMAs may qualify as Streamlined if:
    the device has a review guidance; or
    two or more previous PMAs have been approved for the same type
     of device; or
    the device has a study protocol jointly developed by the
     manufacturer and FDA
 Contact FDA to determine eligibility. Submit study protocol to
  FDA for review
 Use abbreviated Summary of Safety and Effectiveness by
  attaching Executive Summary to labeling
 Manufacturing inspection may be deferred if FDA completed a
  GMP inspection within past 2 yrs
 Final Review within 180 day cycle
Standard and Modular PMAs
 Standard
     IDE submitted and approved
     Complete PMA submitted
 Modular
     PMA shell developed during IDE process
     IDE submitted and approved
     Modules are submitted to FDA as they are completed
      FDA reviews modules over time
   Both have the same content requirements
Content of a PMA
 A PMA application must contain:
   General information (name and address of applicant, Table of
   Summary of Safety and Effectiveness
   Description of the device and its manufacturing process
   Reference to performance standards
   Environmental assessment
Content of a PMA
    Non-Clinical studies
    Clinical Investigations
    Financial certification and/ or disclosure statements
    Labeling
    Literature review/bibliography
    other information requested by FDA
    Sample device, if requested
   “FDA regulations provide 180 days to review the PMA and make a
    determination. In reality, the review time is normally longer. Before
    approving or denying a PMA, the appropriate FDA advisory committee
    may review the PMA at a public meeting and provide FDA with the
    committee's recommendation on whether FDA should approve the
    submission. After FDA notifies the applicant that the PMA has been
    approved or denied, a notice is published on the Internet (1)
    announcing the data on which the decision is based, and (2) providing
    interested persons an opportunity to petition FDA within 30 days for
    reconsideration of the decision.”
 accessed 12/10/07
Timeline & Milestones
 Filing review 45 days
 BIMO inspections 70-90 days
 Manufacturing Inspection 90 -120 days
 Day 100 meeting
 Prep for panel 2-4 weeks
 Panel hearing
 Labeling negotiations
 Final determination
 Post market responsibilities
User Fees
   FY 2008 Device Review User Fees (U.S. Dollars) Type of ApplicationStandard Fee/Small Business
    Fee (<$100 million sales) Premarket Application (PMA, PDP, BLA, PMR)

*NOTE: The fee is waived for the first premarket application from firms with gross receipts or sales <$30
   $185,000/$46,250 PMA/BLA/PDP
   $185,000/$46,250 Premarket Report (PMR) - premarket approval application for a reprocessed device
   $185,000/$46,250 Efficacy Supplement (for BLA)
   $138,750/$34,688 Panel-track Supplement
   $ 27,750/$6,938 180-day Supplement
   $ 12,950/$3,237 Real-time Supplement
   $ 2,960/$1,480 30-Day Notice/135-Day PMA Supplement**
   $ $6,475/$1,619 Annual Fee for Periodic Reporting on a Class III Device**

         * PMA=Premarket Approval; PDP=Product Development Protocol; BLA=Biologics License Application; PMR=Premarket
          Report (for a reprocessed device)
          **New for FY08
Fees and Exemptions
   Exemptions and Waivers
   The following types of applications require no fee.
   Special PMA Supplements-Changes Being Affected
   Express PMA Supplements
   Humanitarian Device Exemption (HDE)
   BLA for a product licensed for further manufacturing use only.
   The following exemptions or waivers apply.
   Fee Exemptions and Waivers (No Fee for These) Category
    Exemption or Waiver First premarket application (PMA, PDP,
    BLA, or premarket report) from a small business with gross
    receipts or sales <$30 million . One-time waiver of the fee that
    would otherwise apply. Any application for a device intended
    solely for pediatric use. Exempt from any fee. If an applicant
    obtains an exemption under this provision, and later submits a
    supplement for adult use, that supplement is subject to the fee
    then in effect for an original premarket application. Any
    application from a State or Federal Government entity. Exempt
    from any fee unless the device is to be distributed commercially.
 PROs                           CONs 
  High regulatory bar means         Expensive
   fewer competitors                 Time consuming
  Competitors cannot use your       Restrictive Manufacturing
   data                              Restricted device
  Competitors must spend the        Labeling, advertising &
   time and money                     promotion restrictions
  Liability protection?
 Try to de novo classify to Class II (510k)
 Limit your claims
 Hire an experienced RA professional
 Hire experienced consultants
 Look for partners
 Resist changes by assessing risks
 Expect to take longer and be more expensive than
  you planned.

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