Article Summary:
The US Food and Drug Administration (FDA) has issued draft guidance on developing cancer drugs in combination with other treatments. Several stakeholders, including drugmakers and research advocacy groups, have provided comments requesting more clarity and expansion of the guidance.
Specifically, stakeholders like Friends of Cancer Research (FoCR) and the American Association for Cancer Research (AACR) have asked the FDA to provide more details on how it will assess the contribution of each treatment in a combination therapy. They requested the FDA to expand on the data sources, including real-world data, that can be used to evaluate the drugs.
The groups also asked the FDA to acknowledge situations where full factorial trial designs may not be appropriate, such as in cases of synthetic lethality, strong biologic co-dependency, investigational drugs with limited monotherapy activity, or in rare biomarker-defined populations. They suggested the FDA should provide more examples and clarify acceptable alternative trial designs in these scenarios.
Additionally, stakeholders like AACR requested the FDA to incorporate toxicity and therapeutic index considerations into its contribution of effect (CoE) evaluation, aligning it with the clinical decision-making process.
Drugmakers such as Novartis, EMD Serono, and Pfizer also provided comments, seeking more clarity on the use of external data, safety information, and the development of drugs belonging to the same class in combination therapies.
Overall, the stakeholders aim to ensure the FDA’s guidance provides clear, flexible, and pragmatic pathways for developing cancer drugs in combination with other treatments.