FDA’s Role in Encouraging Innovation in Combination Products

Danelle R. Miller, Esq.

Regulatory Counsel, Roche Diagnostics

For

The Combination Products Coalition

FDA/DIA Cross-Labeling Workshop

May 10, 2005

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Creativity is thinking up new things. Innovation is doing new things.

Theodore Levitt

FDA’s Role in Encouraging Innovation in Combination Products Barriers to Innovation

The Scope of FDA’s Authority

Tools for Encouraging Innovation

Policy options

Barriers to Innovation

As a rule, men worry more about what they can’t see than about what they can.

Julius Caesar

Barriers to Innovation: Why Companies Don’t Want to Collaborate Potential impact on their

product’s safety-effectiveness profile

Intellectual property and confidentiality concerns

Conflicting business strategies

Resource priorities

Costs

Product liability issues

Interest in competing products (drugs or delivery systems, for example)

Existing partnerships

Barriers to Innovation: Why Companies Don’t Want to Collaborate Control

Risk of having someone else’s “issues” control your resources

Potential limits on flexibility Changes have greater impact

Manufacturing and quality concerns

Desire to develop and retain internal expertise

Familiarity or lack of familiarity with a potential “partner” or product

Differing perceptions on market size and what constitutes “success” Ex: A sizeable market to a small device company may not

appear as large to a major pharmaceutical company

Additional regulatory exposure

The Scope of FDA’s Authority

Great minds have purposes, others have wishes. 

Washington Irving

FDA’s Authority

FDA derives its authority from the Food, Drug and Cosmetic Act (“Act”).

The Act gives FDA broad authority. For example, FDA can:

Promulgate regulations for enforcement of the Act

Some limitations Approve drugs and devices,

license biological products Regulate use of drugs, biologics

and devices, including those used for investigational purposes

Require manufacturers to include “adequate directions for use” in labeling

FDA’s Authority

AND Take enforcement action against

“misbranded” devices or drugs, including:

Devices that are “dangerous to health when used in the dosage or manner, or with the frequency or duration prescribed, recommended, or suggested in the labeling thereof”

Devices for which labeling is “misleading.” In determining whether labeling is misleading, FDA should look to whether “labeling fails to reveal [material] facts . . . Under such conditions of use as are customary or usual.”

FDA’s Authority

FDA’s authority is not all-encompassing. For instance, FDA does not have the authority to:

Require that companies work together Mandate that a manufacturer seek approval for new uses

of its product Association of American Physicians and Surgeons,

Inc. v. FDA Force manufacturers to “mutually conform” their

labeling to include new uses As a practical matter, 3.2(e)(3) does not work

Open or reference proprietary information in a drug sponsor’s file for another sponsor seeking approval of a combination product

Except: 505(b)(2)

FDA’s Authority

In fact, industry would object to the agency playing such an active “matchmaker” role

Tools for Encouraging Innovation

Teams work better when they work together.

Alice Vernon

Tools for Encouraging Innovation

FDA cannot require companies to work together, but FDA can encourage companies to collaborate in a way that leads to innovation.

Existing tools include:User fee waiversExpedited review and

approval timesPower of persuasion –

“just ask”

Tools for Encouraging Innovation

FDA can seek additional tools from Congress, including:Additional period of

(drug) market exclusivity for voluntary collaboration in developing innovative combination products

Tax incentives for voluntary collaboration

Policy Options

Action is eloquence. 

William Shakespeare

Policy Options

When companies refuse to collaborate, FDA has options for proactively supporting innovation while protecting the public health:

Section 505(b)(2) of the Act

Require labeling changes related to safety issues

Policy Options Facilitate independent operation of a combination product

sponsor.

Require sponsor to assure that: The combination product is effective under the

circumstances described in combination product labeling.

The combination product will continue to be effective under the circumstances described in the labeling.

Apply post-approval requirements to combination products Controls (ex: change controls). For example, allow for a

post-approval testing sequence that would catch changes and ensure continued safety and effectiveness.

Studies Registries

Consider a mechanism for notifying combination product sponsor of changes to component not under the sponsor’s control