FDA draft benefit-risk assessment guidance – a cancer treatment perspective

Factoring patient assessments into regulatory decisions

On September 29, 2021, the U.S. Food and Drug Administration (FDA) published a draft guidance for the industry entitled “Benefit-Risk Assessment for New Drug and Biological Products” to clarify how a drug’s benefits, risks, and risk management options factor into certain pre-and post-market regulatory decisions. This document is in alignment with the International Council for Harmonization (ICH) guidance for the industry. This guidance development came about due to industry stakeholders indicating the need for understanding how benefit-risk assessment considerations inform what types of evidence they generate in their drug trials. This activity shows that the FDA’s Center for Drug Evaluation and Research, and the Center for Biologics Evaluation are increasingly factoring patient experience into regulatory decisions. Moreover, industry stakeholders are responding with increased interest in including patient experience data in their drug trials.

Clinigma® perspective: 
The guidance draft outlines that patient experience data can help to identify endpoints that measure or predict clinical outcomes of importance to patients. For diseases that are increasingly being treated as chronic conditions with the continued improvement in drug development, clinical outcomes such as burden and health-related quality of life are critical. Cancer is increasingly considered a chronic condiction with more people relying on daily drug treatment to manage their diagnosis. Cancer treatments are in many cases associated with late-stage complications, which can have a significant impact on the way that patients survive. Being able to document better how patients assess their survival, also when taking into acount the late-stage complications is paramount in cancer survivorship. Involving patient experience in drug development can help to identify what matters most in patients’ lives in relation to their diagnosis, treatments, and survival (incl. late-stage complications).

Importantly for Clinigma®, the guidance draft discusses the use of methods such as Patient Preference Information (PPI) as a way of collecting and operationalising patient experience data. Our rigorous reserach methodology, matched with out team of global experts, is perfectly suited for ensuring that distinct patient voices from various locales are incorporated into the benefit-risk assessment for drug development processes.