US Life Sciences Market:

Key Legal Features

March 26, 2013

US FDA Update

Karl M. Nobert

karl.nobert@squiresanders.com

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Navigating the U.S.

Food and Drug Administration

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2012 FDA Organization

CDRH

Center for Devices

and Radiological Health

FDA

Center

For Tobacco

CFSAN

Center for Food Safety

and Applied Nutrition

CBER

Center for Biologics and

Evaluation Research

CVM

Center for

Veterinary Medicine

CDER

Center for Drugs and

Evaluation Research

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2013 FDA Organization

Office of

Global Regulatory

Operations and

Policy

Office of

Food and

Vet Medicine

Office of

Medical Products

and Tobacco

Office of

Operations

FDA

Office of

Commissioner

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Regulatory Framework

• Federal Food, Drug, and Cosmetic Act (“FDC Act”)

• Code of Federal Regulations (“CFR”)

Implements the statutory provisions of the FDC Act

Title 21 – Covers FDA regulations on food and drugs in “Parts”

• Guidance Documents

• Compliance Manuals

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Regulatory Pathways

for Market Entry

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Food Regulation Key Agencies and Statutes

Food and Drug Administration

• Federal Food, Drug, and Cosmetic Act

• Fair Packaging and Labeling Act

• Bioterrorism Act of 2002

United States Department of Agriculture

• Food Safety and Inspection Service

Federal Meat Inspection Act

Federal Poultry Products Inspection Act

Federal Egg Products Inspection Act

• Animal Plant Health Inspection Service

Animal Health Protection Act

Plant Variety Protection Act

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Food Regulation - Key Points

• Safe for Human Consumption

• Not Adulterated

• Not Misbranded (Properly

Labeled)

• Facility Registration

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Drug Regulation - The Basics

• The Center for Drug Evaluation and Research (“CDER”)

• Types of Drug Regulated:

- Rx (Branded and Generic)

- Over-the-Counter

- Homeopathic

• Drug Definition [21 U.S.C. § 321(g)(1)]:

- Official Compendia

- Intended for use in diagnosis, cure, mitigation, treatment, or

prevention of disease in man or other animals

- Intended to affect structure or function of body of man or animal

- Any component of items 1-3 above.

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Investigational New Drug (“IND”) Application

New Drug Application (“NDA”)

Pre-market review and approval

Safety & Efficacy

Current Good Manufacturing Practices

(“cGMPs”)

User Fees

Drug Regulation - Approval Basics

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Medical Device Regulation - Classification

3 Classes of Medical Devices Based on Perceived Risk and

Control Levels

• Class I (low risk)

General Controls

• Class II (medium risk)

Special Controls

510(k) Notification

• Class III (high risk and novel devices)

Special Controls

PMA

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Dietary Supplement Regulation - Definition

A product (other than tobacco) intended to supplement the diet that bears or contains one or more of the following dietary ingredients:

• Vitamins

• Minerals

• Herbs or other botanicals

• Amino acids

• Concentrate, metabolite, constituent, extract or combination of above listed ingredients

Under the Food, Drug and Cosmetic Act, dietary supplements are

regulated as a category of foods, and have been since at least

1938

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Cosmetic Regulation – Characteristics

• Articles intended for Cleansing (except some soap)

• Beautifying

• Promoting Attractiveness

• Altering appearance

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Cosmetic Regulation - Products

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Introduction to Labeling,

Marketing and Promotion

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• Immediate Container

• Package Insert

• Outer Packaging

• Shipping Label

• Client Information Sheet

The Product Label – What is Included?

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Product Labeling – What is it?

All marketing and promotional materials including:

• websites (product sites, meta tags)

• detailers / brochures

• trade show materials (handouts, posters)

• publications (journals, magazines)

• advertisements (TV and radio ads, internet pop-ups)

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The Standard for Adequate Substantiation

• Competent and Reliable Scientific Evidence

FTC standard relied on by FDA

"tests, analyses, research, studies, or other evidence based on the

expertise of professionals in the relevant area, that has been conducted

and evaluated in an objective manner by persons qualified to do so, using

procedures generally accepted in the profession to yield accurate and

reliable results." See Vital Basics, Inc., C-4107 (Consent April 26, 2004);

see also In Re Schering Corp., 118 F.T.C. 1030, 1123 (1994).

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Types of Claims & Risks

1) Disease Claims

2) Unsubstantiated Claims

of Effectiveness

3) Fair Balance / Omission of

Risk Information

4) Unsubstantiated Superiority

Claims

5) Consumer

Testimonials

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Enforcement Action

and Mitigating Risk

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Avoiding FDA Enforcement Action

• Untitled Letter

• Warning Letter

• Seizure

• Injunction

• License Revocation

• Criminal Penalty

• Civil Money Penalty

• Disgorgement of Profits

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How Does FDA Find Out About Violations?

• Inspections

• Random Sampling

• Adverse Event Reports

• International Cooperation

• Trade Complaints by Competitors

IP Portfolio Protection

Rahul Pathak, Ph.D. J.D.

rahul.pathak@squiresanders.com

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Patentability - Outline

• Patents are awarded to subject matter that is:

Patentable subject matter

Useful

Novel

Non-obvious

Described and enabled

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Patentable Subject Matter

• Categories

Processes, machines, manufactures, compositions of matter

– Excludes

» Transitory signals

» Naturally occurring organisms

» Human beings

• Exceptions

Laws of nature, physical phenomena, abstract ideas

Unless

– Practical application of an exception

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Useful Subject Matter

• Criteria

Credible

– Accepted by one of ordinary skill in the art

Specific

– Specific to subject matter claimed, not to class of the invention

Substantial

– Real world use

• Examples

Treating or preventing microbial infection

DNA fragment

Uncharacterized protein

Isolated protein for treating Alzheimer’s disease

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Novel Subject Matter – Effective March 16

• All elements of subject matter are not in the prior art

• Prior art

All patents, printed publications, public uses, on sale, or available to

the public

– Before filing date of patent application

All patents and published patent applications by another worldwide

– With earlier effective filing dates

– “Worldwide” differs from EP practice

• Exceptions

Disclosure by or from inventor within one year of filing

Commonly owned patents and patent applications

Both differ from EP practice

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Non-Obvious Subject Matter – March 16

• Differences between the claimed invention and the prior art

• Claimed invention as a whole would have been non-obvious to a

person of ordinary skill in the art

Substantial differences from prior art

Insubstantial differences, with evidence of unexpected results

• Prior art includes patent applications earlier filed

Differs from EP practice

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Patent Application

• Claims, claims, claims

• Detailed description

How to make and use the invention

Best mode

– Differs from EP practice

• Drawings

Optional

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Patent Examination

• Subject matter

• Utility

• Novelty

• Non-obviousness

Often contentious

• Description and enablement

Also often contentious

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Post-Grant Proceedings – March 16

• Ex parte reexamination

• Post grant review

Novelty, obviousness, written description, enablement, indefiniteness,

subject matter

Trial at the Patent Trial and Appeal Board

12-18 months

• Inter partes review

Prior patents or printed publications

Trial at the Patent Trial and Appeal Board

12-18 months

• Transitional program for covered business methods

Method or apparatus for data processing

– Except for technological inventions

May be filed by party charged with infringement

Any grounds of unpatentability

Dynamics of the Research

and Development / Clinical

Trials Market in the US

Maureen Bennett

maureen.bennett@squiresanders.com

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Dynamics of US R&D/Clinical Trials

• Global Dynamics

Continued Impact of Patent Cliffs

Continued Impact of Health Care Reform/Pricing Pressures

Consolidation of In-house R&D resources

Increased in Industry Joint Development Programs

Increased outsourcing to CROs/AROs/Specialty Providers

Variable availability of capital from financial markets

Variable Availability of Government Funding/NIH in the US

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Dynamics of US R&D/Clinical Trials

• New Models of Innovation

Transcelerate

Industry/AMC Broad Collaboration Platforms

Target Validation Consortium

State Programs

Personalized Medicine/Unmet Needs

Social Media Recruitment

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Dynamics of US R&D/Clinical Trials

• Recent US Regulatory Developments Affecting Clinical Trials

New HITECH Regulation-Flexibility in Authorizing Future Research

FDA Approval of Remote Clinical Trial Monitoring

HHS Consideration of Centralized IRBs

FDA Final Guidance re: Financial Disclosures

Physician Payment in the Sunshine Act

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Dynamics of US R&D/Clinical Trials

• Negotiating Clinical Trial Agreements with US

AMCs/hospitals/physician practices

• Contrast with UK System

No model agreements like NHS Trust

Variation in budget formats

IRBs

No regulatory requirement for insurance, but…

Indemnity negotiations

PI Oversight Issues

For questions regarding

CLE credit, please contact:

Robin Hallagan

robin.hallagan@squiresanders.com

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Worldwide Locations

• Cincinnati

• Cleveland

• Columbus

• Houston

• Los Angeles

• Miami

• New York

• Northern Virginia

• Palo Alto

• Phoenix

• San Francisco

• Tampa

• Washington DC

• West Palm Beach

• Bogotá+

• Buenos Aires+

• Caracas+

• La Paz+

• Lima+

• Panamá+

• Santiago+

• Santo Domingo

• Beirut+

• Berlin

• Birmingham

• Bratislava

• Brussels

• Bucharest+

• Budapest

• Frankfurt

• Kyiv

• Leeds

• London

• Madrid

• Manchester

• Moscow

• Paris

• Prague

• Riyadh

• Warsaw

• Beijing

• Hong Kong

• Perth

• Seoul

• Shanghai

• Singapore

• Sydney

• Tokyo

North America Latin America Europe & Middle East Asia Pacific

+ Independent Network Firm