FDA draft benefit-risk assessment guidance – deadline for input 29th November

The Food and Drug Administration (FDA) draft benefit-risk assessment of new drugs

A key aspect of Clinigma’s work is capturing patients’ experiences for clinical development – before, during, and after clinical trials. These experiences include the benefits and risks of clinical trial treatments. Types of benefits include clinical benefits, as well as important characteristics of the medical products, such as convenience (e.g. a more convenient dosing regimen or route of administration). This may lead to improved patient compliance or benefits that affect those other than the patient. Risks include drug interactions, risks identified in the non-clinical data, risks to those other than the patient, and risks based on current knowledge of the drug. Factors such as potential misuse, abuse, or diversion of the drug can also be considered.

A recent FDA draft guidance released in September 2021, Benefit-Risk Assessment for New Drug and Biological Products Guidance for Industry, aims to clarify for drug sponsors and other stakeholders how considerations about a drug’s benefits, risks, and risk management options factor into certain premarket and postmarket regulatory decisions. The FDA’s Risk-Benefit Framework includes analysis of the condition, current treatment options, treatment benefits, and risks/risk management.

Reminder: The deadline for submitting comments to the Benefit-Risk Assessment guidance draft is on the 29th of November.

Assessing the benefit-risk perceptions of patients in a clinical trial is often complex. It requires a thorough assessment of the available evidence, recognition of the data gaps, and careful consideration of multiple factors. These include the severity of the condition, the patient population, and the current treatment landscape/trial/therapeutic context.

Clinigma® has recently made a Standardised Interview Manual (SIM) that can operationalise the smooth integration of interviews in clinical trials. The SIM includes a specific ‘Benefit-Risk’ module, aimed at understanding the treatment benefits that are of most importance to the patients, their perceptions of the extent to which the trial treatment will benefit them, how the risks associated with the treatment are acceptable to obtain these benefits, as well as questions concerning the patients’ broader acceptance of uncertainty.

The SIM is a working document that accommodates this latest FDA draft guidance, alongside findings from other projects such as the European Medicines Agency (EMA) Preference/Benefit-Risk Methodology Project. This ensures that the interview questions posed to patients are both appropriate and relevant.

Clinigma’s new manual is user-friendly and tailorable to any clinical trial. It brings clarity and value to pharmaceutical companies through Clinigma’s focus on what matters to the patients, which decision criteria/endpoints are important, the relative importance of these decision criteria/endpoints, and the acceptability of trade-offs when weighing benefits versus risks of treatments.

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