Regulation in a New Era of Cancer Drug Development

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Regulation in a New Era of Cancer Drug Development: Capturing Patient Voice

Ari Gnanasakthy, MBA, MSc, Principal Researcher Patient-Reported Outcomes and Assessments
RTI-Health Solutions

As a Health Outcomes researcher, it is critical to remain abreast of trends in the regulatory environment. I attended a workshop about the assessment of PROs in cancer clinical trials jointly organized by the FDA and ASCO. I'd like to share with you some of what I heard.

It became clear during the workshop that the PRO measures that are currently used in cancer studies are outdated. Questionnaires are lengthy and contain questions that may be irrelevant to current-day cancer studies. 

The main takeaway from the workshop was the recommendation to assess four core PRO domains in cancer studies. These four domains are: disease-specific symptoms, symptomatic adverse events, physical functioning and overall impact of side effects.

The FDA suggested that items for these four domains may already exist in current PRO measures available within PROMIS, FACIT, EORTC and the PRO-CTCAE item bank. While this suggestion makes total sense, there are some aspects of this measurement strategy that is yet to be fleshed out. For example, how to combine items with different number of response options?

Although the recommendation by the FDA was generally welcomed, not everyone agreed. There were some that questioned the value of assessing symptomatic adverse events from the patients’ point of view.

But – as one panel member asked -  don’t you first want to understand your own product? 

It’s no longer business as usual. We are entering a new era of capturing patients’ voice in cancer drug development. These FDA recommendations will not only to help sponsors and regulators understand the products but will also help patients and providers make choices that matter the most to patients.  

Please contact me if you’d like to discuss this further. 

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