FDA/CDRH Public Meeting

Oversight of
Laboratory Developed Tests -
Patient Considerations

Genentech
A Member of the Roche Group
July 19, 2010
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 Overview

• Genentech’s position on personalized

healthcare (PHC) and patient health & safety

• Genentech’s Citizen Petition: Regulation of In

Vitro Diagnostic (IVD) Tests

• IVD tests and patient safety

• Conclusions
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 Genentech’s Position on Personalized
Healthcare and Patient Health and Safety
• Personalized healthcare (PHC)
– RIGHT drug to the RIGHT patient
– An integral part of our strategy is to provide patients with safe,
efficacious, clinically differentiated medicines.
– IVD assays that provide information at a molecular level are key
to PHC.

• In the context of PHC, patient health and safety depend on:

– A thorough evaluation of the safety and efficacy of the medicines
used to treat patients
– Combined with appropriate assessment of the accuracy and
clinical utility of all IVD tests that significantly inform prescribing
of drugs.

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 Genentech’s Citizen Petition
• In Dec. 2008, Genentech submitted a Citizen Petition to FDA which set
forth:
– Patient-focused reasons why FDA should exercise its regulatory
authority over all IVD tests.
• Some LDTs are entering the market without review of the
evidence for claims made to support their use in patient care.

– Framework for using the FDA’s current risk-based classification

system for necessary and appropriate review of LDTs
• Should be calibrated to the risk posed by the test, so that it
does not stifle innovation in PHC.
• All claims should be scientifically validated and reviewed by FDA
to ensure that health care professionals and patients have
access to validated diagnostics to help guide their therapeutic
decision-making.

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 Diagnostic Tests and Patient Safety

• Without appropriate regulation of all IVD tests, patients

are at risk.

• Use of diagnostic tests that make unsubstantiated claims

intended to guide specific therapeutic decision-making
threatens patient health and safety.

• Potential risks to patient health and safety include:

– Patients receiving inappropriate treatment (thus
exposing them to unnecessary side effects or possible
treatment failure)
– Patients not receiving appropriate treatment.
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 Diagnostic Tests and Patient Safety
(cont’d)
• Regulation of LDTs should be comprehensive including :
– Analytical and clinical performance
– Monitoring of test performance in order to protect
patients (e.g., through post marketing surveillance
and medical device reporting).
• Example: Patient test recall in Jan. 2009 for
possibly inaccurate LDT Vitamin D testing
affecting thousands of patients who were tested
over two years

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 Genentech’s Continued Concern:
Our Patients
• Today, LDTs continue to enter the market without sufficient review
of scientific or clinical evidence for claims made to support their
use in patient care.
– LDT manufacturers promote their tests without FDA regulatory oversight.
– LDT manufacturers promote responsiveness to therapies without FDA review
of data.

• In contrast, a Genentech-identified biomarker which predicts

responsiveness to therapy would require full regulatory review prior
to approval of the test, inclusion in labeling and any promotion or
use.

• Genentech is concerned that the current environment is unsafe for

patients and possibly creates situations that could result in
inappropriate treatment.

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 Conclusions
• With a focus on PHC, diagnostic tests play an
increasingly important role in clinical decision making
and disease management.

• LDTs that have not been properly validated for their

intended use put patients at risk.

• Patient risks include failure to receive appropriate

treatment, and/or receive inappropriate treatment.

• A risk-based application of FDA oversight to LDTs is the

appropriate approach to achieve the desired public
health benefit.
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