pdufa legislative update nord corporate council annual meeting paul kim, foley hoag llp may 15 2012

PDUFA Legislative Update

NORD Corporate CouncilAnnual Meeting

Paul Kim, Foley Hoag LLPMay 15 2012

NORD Corporate Council | 2

Current Situation Overview of Senate, House Bills Outlook



Rapid, Bipartisan ActionS. 2516 and H.R. 5651Dingell: 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


Outstanding IssuesDrug 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


Historic Reauthorizations

1992PDUFA

1997FDAMA

2002Bioterrorism

Act

2007FDAAA

2012PDUFA 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


Human Drug Reviews

1993$100

2003$553

2006$767

2012$1840


FDA Funded By Fees

2008 2010 2011 20120

500

1000

1500

2000

2500

3000

3500

4000

4500

User FeesBA

7


Bill ComparisonTitle 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


Bill Comparison

Title VII – Drug Supply Chain

Title VIII – Generating Antibiotic Incentives Now (GAIN)

Title IX – Drug Approval and Patient Access

Title X – Drug ShortagesTitle 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


User Fees SummarizedPDUFA V: FY2013 = $693MMDUFA III: FY2013 = $97.7M and $595M/5

yearsGDUFA: $299M annually incl. one-time

$50M ‘backlog fees’ in first yearBsUFA: may not exceed total costs “for the

process for the review of biosimilar biological product applications”


Biosimilar User FeesEarly Product DevelopmentNO 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


PDUFA V EnhancementsEnhanced NME Reviews – ‘validation period’

to improve first cycle performanceInvestments for Regulatory Science –

including focus on rare diseasesStructured Risk-Benefit Assessment –

account for disease severity, unmet needPost Market Safety – standardize REMS,

validate Sentinel


MDUFA EnhancementsAverage Total Time Goals – not just FDA

clock – and Improved FDA Days Improving Presubmission and Midpoint

DialogueGreater Agency Accountability – new key

metrics and independent review


Pediatric ReauthorizationsPermanently authorizes BPCA, PREA, Pediatric

Advisory Committee, ODAC Pediatric SubcommitteeClarify 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


Approval and AccessCodifies 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


Approval and AccessSenate: 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”


GAIN ActNew 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


Device ReformsExpand 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


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


Custom DevicesClarifies 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


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


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


FDA Priorities in 2013

In FY2013, asking for $4.5B budget, 17% hikeSecure user fee reauthorizations, implement

reformsImplement Food Safety Modernization ActBoost global inspectionsImprove Agency science baseImprove CDRH review performance to avoid

congressional micromanagement


Balancing Equities

Performance35 NMEs in 2011 - most in a decade16 Priority Review approvals - 10 orphans, and 2/3 in

single cycle More cancer therapies faster in US v EUPressuresContinued scrutiny of regulatory burden, role in

facilitating innovation, job creationExpectation of “safety” contrasts to gaps in global

inspections, pharmacovigilance capabilites


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


Looking AheadElections. Changes dynamics, truncates legislative yearChopping 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”


Budget SimplifiedU.S. tax revenue $2,170,000,000,000Federal budget $3,820,000,000,000New debt $1,650,000,000,000 National debt$14,271,000,000,000BCA annual cut $108,000,000,000

Annual family income $21,700Family budget $38,200New debt $16,500National debt$142,710BCA annual cut $1,080

pdufa legislative update nord corporate council annual meeting paul kim, foley hoag llp may 15 2012

Documents

sentinel slide

independent review slide

drug approval

medications act gain

drug supply chain title

devices title

provisions title

labeling changes slide