Companion In Vitro Diagnostics and Oncology Drug Approvals Gregory - - PDF document

As Presented at the NCCS Cancer Policy Roundtable October 18-19, 2011

1

1

Companion In Vitro Diagnostics and Oncology Drug Approvals

Gregory H. Reaman, M.D.

Associate Director Office of Hematology Oncology Products Center for Drug Evaluation and Research FDA

2

Disclosures and Disclaimer

• No financial relationships to disclose.
• No discussion of off label or investigational

use of specific products/devices.

• The views expressed are those of the

speaker and do not necessarily represent the opinion of the Food and Drug Administration.

As Presented at the NCCS Cancer Policy Roundtable October 18-19, 2011

2

3

Outline

• Regulation of Investigational Use Devices
• In Vitro Companion Diagnostic Devices:

– Definition – Review and approval of IVD Companion Diagnostic Devices – Coordinated development with new therapeutic products – Recent examples

4

In Vitro Diagnostic Products: Regulation

• Research use
• Investigational use
• Diagnostic use
• Review program for human subject

studies: Investigational Devices Exemption (IDE)

As Presented at the NCCS Cancer Policy Roundtable October 18-19, 2011

3

5

IDE Review

• Patient safety; risk-benefit
• Knowledge to be gained from

investigation: clinical impact

• Alignment with plans for later development

and FDA review

– Eligibility – Population enrichment – Therapeutic intervention decision-making

6

Investigational Device Requirements for Clinical Trial

• Fully specified device
• Adequate assessment of analytical

performance

– Analytes, reagents, equipment, software

• Instructions
• Clinical/preclinical data to justify exposure
• Testable hypothesis

As Presented at the NCCS Cancer Policy Roundtable October 18-19, 2011

4

7

In-vitro Diagnostic Device

• Provides information that is essential for

the safe and effective use of a therapeutic

• product. Its use with a particular product

is stipulated in the label of both the device and product as well as any generic equivalents.

– Identify patients most likely to benefit – Identify patients at increased risk for adverse reactions – Monitor treatment response

8

Review and Approval

• Novel therapeutic product –

contemporaneous development

• Delayed IVD Companion Diagnostic

Device review and approval – subsequent labeling changes

– New products for life-threatening conditions – Already approved products – safety issue, not supplemental indication

As Presented at the NCCS Cancer Policy Roundtable October 18-19, 2011

5

9

Concept for Co-development

• Scientifically robust and efficient

development plans and regulatory review and approval for in-vitro diagnostics and drug and biologic products

• Inter-center coordination: CDER, CBER,

CDRH

• Co-development does not mean that drug

and diagnostic products cannot be marketed separately

10

Cont’d

• Prevent scientific/technological issues

associated with one product from negatively impacting development of the

• ther
• Early discussion between sponsor,

manufacturer, and review centers (pre- IND, IDE process) encouraged.

As Presented at the NCCS Cancer Policy Roundtable October 18-19, 2011

6

11

2 Recent Proofs of Principle

• Crizotinib (Xalkori) in ALK+ lung cancer
• Vemurafenib (Zelboraf) in V600E BRAF

mutant melanoma

• Early co-development for population

enrichment

• Accelerated time-lines
• Early expectation that these examples will

be the rule rather than the exception.