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PDUFA Legislative Update

PDUFA Legislative Update . NORD Corporate Council Annual Meeting. Paul Kim, Foley Hoag LLP May 15 2012. Current Situation Overview of Senate, House Bills Outlook. Current Situation Overview of Senate, House Bills Outlook. Rapid, Bipartisan Action. S. 2516 and H.R. 5651

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PDUFA Legislative Update

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  1. PDUFA Legislative Update NORD Corporate Council Annual Meeting Paul Kim, Foley Hoag LLP May 15 2012

  2. Current Situation • Overview of Senate, House Bills • Outlook • Current Situation • Overview of Senate, House Bills • Outlook

  3. Rapid, Bipartisan Action • S. 2516 and H.R. 5651 • Dingell: passage is usually mired in controversy… the fact that there is no controversy indicates the extraordinary success and strength of the Committee • H.R. 4156, the EXPERT Act (Expanding and Promoting Expertise in Review of Rare Treatments Act); H.R. 3211, Humanitarian Device Reform Act; H.R. 4132, FAST Act; H.R. 2245, the Preserving Access to Life-Saving Medications Act; GAIN Act

  4. Outstanding Issues • Drug importation (McCain-Sh. Brown) • National Drug Supply Chain Monitoring (Waxman-Matheson-Bilbray-McKinley ) • Online Pharmacy and Telehealth (Cassidy, Feinstein) • LPAD (FDA, IDSA, obesity) • Cosmetic Regulatory Reform (Pitts-Pallone-Dingell) • OTC/BTC Concepts

  5. Historic Reauthorizations Clinical Trials.Gov Fast Track REMS Off Label Mandatory Labeling “Better” BPCA Mandatory PMS 1 Plus S/E DTC User Fees Least Burdensome MDUFA 3 Party Review “Best” BPCA/PREA Cosmetic Safety Reagan-Udall Citizen Petition MDUFMA Adcomm COI Biodefense R&D BPCA/PREA bsUFA, GDUFA Antibiotics Select Agents Shortages PDUFA Zoonotic Supply/Pedigree Supplements PREA GAIN Act 1992 PDUFA 1997 FDAMA 2002 Bioterrorism Act 2007 FDAAA 2012 PDUFA V TREAT Act

  6. Human Drug Reviews 2012 $1840 2006 $767 2003 $553 1993 $100

  7. FDA Funded By Fees

  8. Current Situation • Overview of Senate, House Bills • Outlook • Current Situation • Overview of Senate, House Bills • Outlook

  9. Bill Comparison • Title I-IV – Drug, Device, Generic, Biosimilar User Fees • Title V – Pediatric Drugs and Devices • Title VI – Medical Device Regulatory Improvements • Title I-IV – Drug, Device, Generic, Biosimilar User Fees • Title V – BPCA/PREA Reauthorization • Title VI – FDA Administrative Reforms • Title VII – Medical Device Regulatory Improvements

  10. Bill Comparison • Title VII – Drug Supply Chain • Title VIII – Generating Antibiotic Incentives Now (GAIN) • Title IX – Drug Approval and Patient Access • Title X – Drug Shortages • Title XI – Other Provisions • Title VIII – Drug Regulatory Improvements • Subtitle A – Drug Supply Chain • Subtitle B – Medical Gas • Subtitle C – GAIN • Subtitle D – Accelerated Approval • Subtitle E – Critical Path Reauthorization • Subtitle F – Misc • Title IX – Drug Shortages

  11. User Fees Summarized • PDUFA V: FY2013 = $693M • MDUFA III: FY2013 = $97.7M and $595M/5 years • GDUFA: $299M annually incl. one-time $50M ‘backlog fees’ in first year • BsUFA: may not exceed total costs “for the process for the review of biosimilar biological product applications”

  12. Biosimilar User Fees • Early Product Development • NO base funding - $20M • “Four Legged Stool” • Initial and Annual Product Development Fees (10%); as well as Application and Supplement; Product Establishment; and Product Fees • Encompasses pre-licensure inspections, post-market safety activities

  13. PDUFA V Enhancements • Enhanced NME Reviews – ‘validation period’ to improve first cycle performance • Investments for Regulatory Science – including focus on rare diseases • Structured Risk-Benefit Assessment – account for disease severity, unmet need • Post Market Safety – standardize REMS, validate Sentinel

  14. MDUFA Enhancements • Average Total Time Goals – not just FDA clock – and Improved FDA Days • Improving Presubmission and Midpoint Dialogue • Greater Agency Accountability – new key metrics and independent review

  15. Pediatric Reauthorizations • Permanently authorizes BPCA, PREA, Pediatric Advisory Committee, ODAC Pediatric Subcommittee • Clarify exclusivity awards for Written Request studies; labeling changes due to inconclusive or non-S/E studies do not qualify • Pediatric study plan:phase 2 meeting; Pediatric Review Committee (PeRC) review of significant modifications to Written Requests or PREA study plans • Tracking of deferrals, ‘follow up; letters, assessments of past Written Requests resulting in labeling changes

  16. Approval and Access • Codifies or amends current fast track and accelerated approval authorities, clarifying types of evidence and endpoints on which FDA can rely • Senate: “Breakthrough therapy” intended to treat a serious or life-threatening disease or condition, and preliminary clinical evidence may demonstrate substantial improvement over existing therapies

  17. Approval and Access • Senate: require FDA to ensure that opportunities exist for consultation with stakeholders from the rare disease community and to maintain a list of external scientific and medical experts to consult on products for rare diseases • House: requires guidance development to address “how to incorporate novel approaches to the review of surrogate endpoints based on patho-physiologic and pharmacologic evidence … especially in instances where the low prevalence of a disease renders the existence or collection of other types of data unlikely or impractical”

  18. GAIN Act • New qualified infectious disease products (QIDPs) gain 5+years of NCE exclusivity, new use exclusivity, orphan exclusivity • QIDPs are antibacterial or antifungal drugs intended to treat serious or life-threatening infections, including those caused by qualifying pathogens, listed and revised by FDA • Sponsors may obtain written SPA-style recommendations

  19. Device Reforms • Expand Sentinel system; Assess recalls for risk mitigation strategies; IDEs/Clinical holds, UDI regulations; Clarify ‘least burdensome’; HDE Reform and Custom devices; FDA documentation/review of denial/disapproval; De Novo w/o NSE; 3rd Party Review/Inspections Reauthorized; Device Modifications with withdrawn guidance • Senate only: Reclassification by order, PMS as condition of approval; Section 522 PMS, Changes in Interpretation=GGP • House only: Scheduled down-classification, Int’l Harmonization, Fora; Transparency in Clearance

  20. Humanitarian Device (HDE) Reform • Lifts profit prohibition for new category of HDES: “intended for the treatment or diagnosis of a disease or condition that does not occur in pediatric patients, or that occurs in such numbers that the device’s development is ‘impossible, highly impracticable, or unsafe’ • Annual distribution number (ADN) now for “number of devices needed to treat, diagnose or cure 4,000 individuals in the United States during any calendar year” •  Existing HDEs may seek determination

  21. Custom Devices • Clarifies device customization for small (<five/year), unique (“treating a sufficiently rare condition, such that conducting clinical investigations would be impractical”) populations • If device unavailable in U.S. and no other devices are domestically available to treat, it does not have to comply with the premarket approval and performance standard requirements if intended to meet special needs of a physician and manufactured on a case-by-case basis to accommodate unique physiology • Requires final guidance on replication of multiple devices not later than two years after the date of enactment

  22. Supply Chain • Required Domestic and Foreign Registrations and Records Inspection; Risk-Based Inspections; Exchange of Information Protected from Disclosure • Extraterratorial Jurisdiction; Amending Adulteration Standard (GMPs, “knowingly”); Admission of Imported Drugs; Enhanced Penalties for Counterfeits • House only: Unique Facility Identifier, Adulteration: Delay or Limit to Inspections, Destruction of Counterfeit Drugs, Registration of Commercial Importers, “Valid Prescription” • Senate only: Drug Excipients, Electronic Registration, 3rd Party Drug Auditors, DHS: Failure to Allow Foreign Inspection

  23. Drug Shortages • Amends section 506C: “(permanent) discontinuance” or interruption of life-supporting; life-sustaining; intended for use in prevention or treatment of debilitation disease • (Senate) Used in emergency medical care or surgery, not a radiopharmaceutical or (House) plasma protein derived • Senate only: Applications or inspections may be expedited; task force and strategic plan, Internal consultation prior to enforcement; 3rd party mechanism to report; FDA records of shortages, notifications, actions to mitigate; FDA may apply to biologicals and vaccines • House only: Drug shortage list, Manufacturing changes may be expedited, Hospital repackaging exempt from registration; Annual FDA report; DOJ report

  24. Current Situation • Overview of Senate, House Bills • Outlook • Current Situation • Overview of Senate, House Bills • Outlook

  25. FDA Priorities in 2013 • In FY2013, asking for $4.5B budget, 17% hike • Secure user fee reauthorizations, implement reforms • Implement Food Safety Modernization Act • Boost global inspections • Improve Agency science base • Improve CDRH review performance to avoid congressional micromanagement 

  26. Balancing Equities Performance • 35 NMEs in 2011 - most in a decade • 16 Priority Review approvals - 10 orphans, and 2/3 in single cycle • More cancer therapies faster in US v EU Pressures • Continued scrutiny of regulatory burden, role in facilitating innovation, job creation • Expectation of “safety” contrasts to gaps in global inspections, pharmacovigilancecapabilites

  27. 2012 Legislative Priorities

  28. Looking Ahead • Elections.Changes dynamics, truncates legislative year • Chopping block. President proposes $320B in health savings, House Republican Ryan budget proposed cutting $6.2 trillion over 10 years and fundamentally restructuring Medicare • Bush tax cuts expire in December. President conceded to extending all income levels at end of 2010 – plus Debt Ceiling • Budget Control Act sequestration cuts set to begin with $108B in 2013, $1.2 trillion over 10 years, half in defense but exempting half of federal spend, e.g. Medicaid • SCOTUS rules on ACA. Closely divided Court to rule on ACA, individual mandate, related coverage provisions, Medicaid expansion; unlikely to take Anti-Injunction Act “pass”

  29. Budget Simplified U.S. tax revenue $2,170,000,000,000 Federal budget $3,820,000,000,000 New debt $1,650,000,000,000 National debt $14,271,000,000,000 BCA annual cut $108,000,000,000 Annual family income $21,700 Family budget $38,200 New debt $16,500 National debt $142,710 BCA annual cut $1,080

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