P HILLIPS C ONSULTING G ROUP, LLC 513(g)s and Marketing Applications for 513(g)s on The "FDA...

PHILLIPS CONSULTING GROUP, LLC

513(g)s and Marketing Applications for 513(g)s on

The "FDA Effect": Tough Going for Medical Innovation?

AAAS 2015 Annual MeetingSan Jose Convention CenterSan Jose, California February 12-16, 2015


The Short Answer …

Achieving FDA authorization to market innovative technology in the US is extremely difficult and challenging!

This said, my objective is to provide insight into the factors that influence FDA decision-making and behavior and to foster an understanding and appreciation for why FDA does what it does.

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Outline

FDA’s Mission and Proclaimed Performance The “FDA Effect”

• Today’s regulatory landscape• The US Regulatory Pendulum

Determinants of Agency Performance• FDA culture – factors that impact agency risk

tolerance• Law and administrative procedures• Regulatory Science

Closing Remarks

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FDA’s Mission1

The Administration shall—

(1) promote the public health by promptly and efficiently reviewing clinical research and taking appropriate action on the marketing of regulated products in a timely manner;

(2) with respect to such products, protect the public health by ensuring that—

(B) human and veterinary drugs are safe and effective;(C) there is reasonable assurance of the safety and effectiveness of devices intended for human use;

(3) participate through appropriate processes with representatives of other countries to reduce the burden of regulation, harmonize regulatory requirements, and achieve appropriate reciprocal arrangements; and(4) … carry out paragraphs (1) through (3) in consultation with experts in science, medicine, and public health, and in cooperation with consumers, users, manufacturers, importers, packers, distributors, and retailers of regulated products.

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1 21 U.S. Code § 393(b)


The Administrations FY 2015 Budget2

FDA’s Scope is Vast, Complex, and Evolving Rapidly• The products FDA regulates represent >20 cents of every

consumer dollar spent on products in the US. FDA is a Bargain

• Every American pays about $8/year for the vast array of protections and services FDA provides.

FDA Delivers Results• FDA’s drug approval system continues to lead the world in

both quality and speed. - Three quarters of all significant pharmaceutical advances that were

approved anywhere in the world in 2013 were approved first by FDA.

• FDA also achieved significant reductions in medical device application review times and application back logs.

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2 http://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Reports/BudgetReports/UCM388309.pdf

http://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Reports/BudgetReports/UCM388309.pdf




Today’s Regulatory Landscape

Entering a period of political uncertainty • 2014 is the start of year 3 in a 2nd term of the

administration• The 114th Congress has the largest Republican majority

since 1931• Will the current FDA leadership remain steadfast and

engaged? Agency tolerance for “risk” is low3

• FDA management reluctant to make difficult decisions• Scientific reviewers consistently require additional

information Inadequate FDA resources

• Aspects of the infrastructure have improved, but remain subpar

• Recruitment is good, but retaining talent is problematic Substantial industry unrest exists

• Complaints/appeals related to FDA decisions are substantial

• Attracting investment in new technology remains a challenge

3In this context, “risk” refers to being held responsible for an untoward outcome related to exposure to an FDA regulated product..


The Regulatory Pendulum

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The Regulatory Pendulum

The culture of the organization drives FDA to the left whereas budget constraints and economic reality drive FDA to the right

Leadership dictates the direction/length of swing • Consider the David A. Kessler, MD3 era:

- Strong leadership skills - Avid supporter of a “strong FDA” with emphasis on

enforcement - Drove pendulum to the far left

• Contrast with Bruce Burlington, MD4 - Strong leadership skills- Demanded scientific rigor and timely decisions- Drove pendulum toward the center

3FDA Commissioner 1990-19974CDRH Director 1993-1999


The Regulatory Pendulum (continued)

FDA turns sharper and faster to the left than to the right• Deputy Commissioner Jane E. Henney, MD5 - “I

will take the heat” FDA is an easy target

• Sherwin Gardner6 - “FDA is a slow moving target that bleeds profusely when hit”

• The actions of Senator Charles E. Grassley (R) and Representative Henry A. Waxman (D) have pushed the pendulum to the left

5Deputy Commissioner of Operations from 1992-1994; FDA Commissioner 1999-20016Deputy Commissioner 1972-1979


FDA Culture

Traditionally FDA is very conservative (i.e., slow and deliberate)

Order achieved through “control” is the objective, unless external forces dictate otherwise

Risk averse, particularly during periods of oversight and criticism

Under-funding is always the “excuse” for poor performance


FDA Culture (continued)

Science emphasized over law The FDA realities

• deregulation stifles agency growth and limits employee opportunities

• citing deficiencies and documenting observations demonstrate scientific rigor and commitment to public health

• procrastination rewarded over decisiveness • FDA personnel leverage industry’s desire for

FDA market authorization to achieve objectives

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Law and Administrative Procedures All FDA actions are subject to the law and

implementing regulations• The agency regulates specific products … data

that should be relevant may not be for decision-making

• There are limits on how FDA can use information … in general, data in regulatory submissions cannot be used for purposes not intended by submitters

• The processes by which regulatory decisions are reached are governed by strict procedures

The promulgation, implementation, compliance, and enforcement of regulations consume time and resources12


Regulatory Science

Science is most often limited by the human mind, current technological capabilities and funding

The science that fuels regulatory processes (i.e., “regulatory science”) is further encumbered by law and regulations• Regulatory science refers broadly to the

scientific and technical foundations upon which regulations are based

Scientific investigators have freedom, but not when the output is the basis for FDA decision-making

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Closing Remarks

FDA is here to stay and achieving FDA approval is a challenge and it will remain a challenge … the agency’s value is linked to the difficulty associated with navigating the regulatory processes

The best that we can hope for is reasonable regulation, coupled with clarity and predictability

No drug, device or biologic is absolutely safe and effective … judgment is required to determine if the regulatory threshold for approval is met.

Understanding the factors that impact FDA can steady the pendulum

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