On June 21, 2023, the FDA issued a final guidance, Oncology Drug Products Used with Certain In Vitro Diagnostic Tests: Pilot Program, to announce and describe the FDA’s voluntary pilot program for certain CDER-regulated oncology drug products1 used with certain corresponding in vitro diagnostic (IVD) tests.
Under current policy, in certain scenarios the FDA may decide to approve a treatment that requires use of a companion diagnostic even if a corresponding companion diagnostic does not receive marketing authorization when the treatment is approved. In these cases, tests offered as laboratory developed tests (LDTs) are being used for patient treatment decisions. FDA has generally exercised enforcement discretion for LDTs, meaning that, at this time, FDA generally has not enforced applicable requirements for these devices. FDA intends to pilot a new approach to provide greater transparency regarding performance characteristics that certain tests for oncology biomarkers used for selection of oncology drug products should meet. Through this transparency FDA also seeks to support better and more consistent performance of LDTs, resulting in better drug selection and improved care for patients with cancer.
The voluntary pilot program announced in this guidance is intended for drug product sponsors with regard to:
(1) CDER-regulated oncology drug products for which FDA determines that use of an IVD test is needed to identify the patient population, but that are candidates for approval without marketing authorization of such a test at the same time as the drug product authorization, as described in existing policy where:
- use of an IVD test is needed to identify the intended patient population;
- no satisfactory alternative treatment exists; and
- the anticipated benefits from the use of the drug product are so pronounced as to outweigh the anticipated risks from approval of the drug product without an FDA-authorized companion diagnostic.
(2) corresponding clinical trial assay(s)
- that use the same technology as a previously FDA-authorized companion diagnostic for any indication (for example, next generation sequencing (NGS), polymerase chain reaction (PCR), Sanger sequencing, immunohistochemistry (IHC), and fluorescent in situ hybridization (FISH)); and
- for which there is a well-validated reference method, well-validated comparator method, and/or well-characterized materials (for example, appropriate clinical samples, cell lines) that can be used to support test accuracy.
This pilot does not alter the standards for approval of the oncology drug products or for marketing authorization of the corresponding companion in vitro diagnostics.
Statement of Interest for the Pilot
Interested drug product sponsors should submit a statement of interest to their Investigational New Drug (IND) applications, New Drug Applications (NDA), or Biologic License Applications (BLA), as appropriate, to be considered for the voluntary pilot program.
Please see the details discussed in the guidance on the information to include in the statement of interest.
Upon receipt of the statement of interest, FDA will follow up with no more than 9 sponsors to request specific information to enable FDA to make a decision concerning acceptance into the pilot.
Timeframe for Pilot
As discussed in the guidance, the initial phase of the pilot program, starting on June 21, 2023, is anticipated to last up to one year.
Templates for Collecting and Providing Performance Characteristics and Validation Information for Clinical Trial Assays
- Next generation sequencing test template
- Polymerase chain reaction test template
- Sanger sequencing test template
- Immunohistochemistry test template
- Fluorescence in situ hybridization test template
These templates are part of the guidance, Oncology Drug Products Used with Certain In Vitro Diagnostic Tests: Pilot Program. These templates may be used to provide the data and information that should be submitted to the FDA, when requested by FDA, regarding clinical trial assays (CTAs) used in the pivotal clinical trial(s) for oncology drug product(s) under the pilot program. The CTA templates are intended to help oncology drug sponsors collect and provide validation information and performance characteristics for the CTAs used in the pivotal clinical trial(s) for the drug product.
The FDA intends to make available, on CDRH’s website, the minimum recommended performance characteristics, based on the CTAs, for in vitro diagnostics to be used with each approved drug product under the pilot.
For questions about the pilot program regarding clinical trial assays (CTAs) and other IVDs, contact the CDRH Office of In Vitro Diagnostics at OncologyPilotCDRH@fda.hhs.gov.
For questions about the pilot program regarding CDER-regulated oncology drug products, contact Reena Philip (OCE) at 301-796-6179, or by email at Reena.Philip@fda.hhs.gov.
- Companion Diagnostics
- Oncology Drug Products Used with Certain In Vitro Diagnostic Tests: Pilot Program
- Federal Register Notice
1For the purposes of this guidance, references to oncology drug products include both human drug products and biological drugs products regulated by CDER, unless otherwise specified. This pilot is currently only available for certain oncology drug products within CDER.