The basic promise of personalized medicine is predicated in large part on the expanding availability of biomarker-based testing. A majority of companion diagnostics (CDx) are in vitro diagnostic tests providing essential information for the safe and effective use of a corresponding therapeutic product – CDx help predict whether a specific treatment is suitable or not for a given patient. CDx tests will identify responsive patient sub-populations to a specific drug or a range of medicinal products, or those likely to experience adverse drug effects.
Ideally, CDx are co-developed together with therapeutics (Rx). Drug developers and diagnostic manufacturers coordinate CDx-Rx co-development, to bring both drug and diagnostic test to the market.
Today, there is little regulatory guidance in the US related to CDx approval in the context of the associated drug development, and almost none in Europe. With the forthcoming European IVD regulation, it is anticipated that CDx will be classified as high-risk tests, and specific requirements will be added to get these products CE marked (i.e. demonstrate the clinical utility of the test). While these changes bring more robustness to CDx development, they also have a great impact on cost and time to the market. As for all IVDs, it is essential to anticipate these regulatory changes in Europe, when co-developing CDx and the associated medicinal product.
Key challenges encompass:
- Establishing a CDx-Rx co-development plan
- Risk and quality management
- Transitioning from biomarker discovery platform to the one preferred for clinical use
- Clinical utility and performance evaluation
- International regulatory approvals
- Co-Labeling of drug and CDx
- Market access strategy and value demonstration to payers
- Position product and claims
- Use of Commercially Available Diagnostics as CDx
- Advise on Rx-CDx co-development plan
- Co-assess medicinal products and diagnostic regulatory pathway
- Develop roadmaps towards international market approvals
- Advise on/conduct regulatory bodies strategic and product development meetings (EMA, FDA)
- Assist with risks analysis and risk management plan
- Optimize protocols for Rx and CDx clinical evaluation
- Verification and Validation Activities for CDx
- Review performance evaluation and CDx regulatory dossiers (EU, US)
- Optimize quality and vigilance systems
- Advise on co-labeling & advertising
- Identification of the appropriate CDx developer