FDA: From Appropriations to

ApprovalsSteven GrossmanHPS Group, LLC

PRESENTATION TO Digestive Disease National

CouncilMarch 8, 2009

Contact: sgrossman@hpsgroup.com (301) 879-5600© HPS Group, LLC 2008-2009

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The Food and Drug Administration

Promotes and protects the public health by ensuring consumers have access to:

Safe foods, and Safe and effective medical products, including

drugs, biologics and medical devices. FDA is one of the world‘s most admired

consumer protection agencies…is widely respected for its leadership in science-based regulation.

FDA-regulated products account for almost 25 cents of every consumer dollar spent in the US.

FDA Budget Basics FDA’s budget is relatively small:

$ 2.04B appropriated; $600M in user fees (assumes passage of FY 09 omnibus legislation)

83% of FDA costs are staff-related: salary, benefits, rent, supplies, telecom, travel, etc.

FDA needs about $100 million per year to “stay even” with increased costs

FDA’s base has eroded even while it was given new responsibilities…and it operates in a world of increased globalization and scientific complexity

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Congress Keeps Adding Responsibilities, 1996-2006

1996 - Freedom of Information Act (FOIA)

1996 - Safe Drinking Water Act Amendments

1996 - Animal Drug Availability Act1996 - Food Quality Protection Act1996 - Economic Espionage Act of

1996 1996 - Electronic Freedom of

Information Improvement Act1996 - Comprehensive

MethamphetamineControl Act

1996 - Health Insurance Portability and Accountability Act (HIPAA)

1996 - Drug-Induced Rape Prevention Punishment Act

1997 - Food & Drug Administration Modernization Act (FDAMA)

1997 - Better Pharmaceuticals for Children Act

1997 - PDUFA II(Family Impact Assessments)

1998 - Antimicrobial Regulation Technical Corrections Act

1998 - Sec. 615 Ag. Research, Extension and Education Reform Act

1998 - MQSA Reauthorization1998 - Sec. 654, Omnibus Approps1999 - Government Employees Training Act1999 - Fed. Financial Assistance Management

Improvement Act2000 - Responsible for Clinical Laboratory

Improvement Amendments (CLIA)2000 - Approps Act (FDA) - FY 20012000 - Medicine Equity and Drug Safety Act2000 - Prescription Drug Import Fairness Act2000 - Approps. Act (HHS) Sec. 516, HPV-Condom Labeling

Review2000 - Ryan White AIDS Care Act2000 - Date Rape Drug Prohibition Act2000 - Children’s Health Act 2000 - Technology Transfer Commercialization

Act2001 - Animal Disease Risk 2002 - Medical

Device User Fee and Modernization Act (MDUFMA)2002 - Hatch-Waxman-Amendments2002 - Drug Importation Report 2002 - Farm Security & Rural Investment Act2002 - Bioterrorism Act

2002 -2002 - PDUFA III Best Pharmaceuticals for Children Act2002 - Rare Diseases/ Orphan Product Development 2002 - E-Government Act2003 - Mosquito Abatement for Safety and Health Act2003 - Animal Drug User Fee Act2003 - Pediatric Research Equity Act (PREA)2003 - Medicare Prescription Drug and Modernization Act2004 - Minor Use and Minor Species Animal Health Act2004 - Food Allergen Labeling and Consumer Protection Act2004 - Medical Devices Technical Corrections Act2004 - National Defense Authorization Act2004 - AIDS (PEPFAR)2004 - Project BioShield2004 - Anabolic Steroid Control Act2004 - MQSA Reauthorization 2004 - Homeland Security Presidential Directive (HSPD) #12, Identification Standard2005 - Protecting America in the War on Terror Act2005 - Patient Safety & Quality Improvement Act 2005 - Medical Device User Fee Stabilization Ac2005 - Stem Cell Therapeutic and Research Act 2006 - Combat Meth Act

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FDA Receives Less Funding Than Its Local School

District…

http://montgomeryschoolsmd.org/aboutAlliance for a Stronger FDA—March 2009

* = Assumes passage of FY 2009 omnibus appropriations bill

FY07CR FY08 FY09

Montgomery County (MD) Public Schools $1.85B $1.98B $2.07B

FDA (appropriated funds) $1.57B $1.72B $ 2.04B*

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...Yet It’s Reach is Global

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Implementation of FDAAA Strong shift toward safety concerns over efficacy

Statutory initiatives blended with administrative changes Transparency imbedded in the new approaches Woodcock: approval standards have not changed

Implementation of REMS/mandatory Phase 4’s REMS replaces Risk-Maps, as of March 25, 2008 First uses judicious; REMS unblocked drugs stalled for

years Implementation initially slowing drug approval decisions Newly approved drugs to be re-reviewed at intervals Hot phase 4 priorities: immunogenicity, patient registries,

risk communication, validation of subset treatment options

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Working Without a Commissioner

FDA is extraordinarily complex, every move has worldwide consequences, many unintended

FDA had acting commissioner for half of Bush Administration...a worrisome trend

Challenge to identify new Commissioner, get him/her confirmed quickly, and help them get a team in place

Not about competency of FDA civil service leadership

Many decisions can't or won't be made without a permanent, confirmed Commissioner

RESCUE THE PRESIDENT FROM THE PEANUT CRISIS

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Integrating New Science into Traditional Clinical

Paths Accepted means of proving safety and efficacy vs.

new scientific knowledge (e.g. oncology, Alzheimer’s) End-points/methodologies (biological/surrogate/statistical) Products that don't fit traditional paradigms Challenges in the construction of real-world clinical trials  Disease symptomology vs. fundamental biological

processes Late stage treatment vs. earlier interventions

Critical Path for the future; what about today? Study designs now = products FDA sees 2010-2015 Where pathways are unclear: clinical trial costs

escalate, cutting edge science slows, R&D may stop

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Balancing Safety with Patient Risk and Need

FDA heading toward near-absolute safety standard; costly for industry, not necessarily good for patients

Severely-ill patients require more balanced, more individualistic and less paternalistic approach

FDA standards varied/inconsistent on patient risk vs. access to potentially-useful therapy. Obvious: stage 4 cancer vs. gastric reflux disease Difficult: 12% of patients get a clear and durable

response; 88% suffer beyond the parameters of their disease

More difficult: patients with non-life threatening debilitating conditions (e.g. blindness) may be willing to take more risks than patients with a life-threatening but manageable disease

Subtle: Alzheimer's patient/family willing to take great risks in early stage/intact; fewer risks in late stage/mentally absent

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Staying Focused on Priorities • FDA’s responsibilities exceed its resources• Gaps in inspections, safety, information technology• Constant pressure from globalization• Challenge from innovation—human, microbial, viral• Some mission creep inevitable• Might new missions risk FDA focus and effectiveness?

• Tobacco• Re-importation• Comparative effectiveness/reimbursement wars• Follow-on biologics

• Time to re-assess foods and medical products in the same agency?

A strong FDA benefits all Americans:

Patients, consumers, health professionals, industry ....and the whole world benefits, too.