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

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Presentation on theme: "PDUFA Legislative Update NORD Corporate Council Annual Meeting Paul Kim, Foley Hoag LLP May 15 2012."— Presentation transcript:

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

2 NORD Corporate Council | 2 Current Situation Overview of Senate, House Bills Outlook

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

6 NORD Corporate Council | 6 Human Drug Reviews

7 NORD Corporate Council | 7 FDA Funded By Fees 7

8 NORD Corporate Council | 8 Current Situation Overview of Senate, House Bills Outlook

9 NORD Corporate Council | 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 NORD Corporate Council | 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 NORD Corporate Council | 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 NORD Corporate Council | 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 NORD Corporate Council | 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 NORD Corporate Council | 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 NORD Corporate Council | 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 NORD Corporate Council | 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 NORD Corporate Council | 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 NORD Corporate Council | 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 NORD Corporate Council | 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; 3 rd 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, Intl Harmonization, Fora; Transparency in Clearance

20 NORD Corporate Council | 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 devices 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 NORD Corporate Council | 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 NORD Corporate Council | 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, 3 rd Party Drug Auditors, DHS: Failure to Allow Foreign Inspection

23 NORD Corporate Council | 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; 3 rd 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 NORD Corporate Council | 24 Current Situation Overview of Senate, House Bills Outlook

25 NORD Corporate Council | 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 NORD Corporate Council | 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, pharmacovigilance capabilites

27 NORD Corporate Council | 27 2012 Legislative Priorities PDUFA Reauthorization Pediatric Exclusivity Drug shortages Supply chain security Progressive & accelerated approvals Advisory comm. conflicts of interest REMS Orphan drug issues Deficit Reduction Part D rebate, noninterference Part B payment Medicaid rebate Increased copays on Part D dual eligibles Restricting patent settlements Prepare for broader Medicare reform Sequestration ACA Implementation IPAB Innovation Center/duals coordination Comparative effectiveness research Rx coverage (essential health benefits) Sunshine Act Prepare for possibility of large changes to ACA Other Importation 340B

28 NORD Corporate Council | 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 NORD Corporate Council | 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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