FDA response to Citizen Petition to include Quality of Life comparisons in RCTs

Copying a snippet of the FDA response to advocate’s petition (that would be me) along with links to the full text and the original petition. The issue remains. Quality of life is integral to the assessment of risk/benefit. The sponsor can include QoL comparisons selectively – when they anticipate a win.

"Dear Mr. Schwartz:

This letter responds to your citizen petition, which was received by the Food and Drug
Administration (FDA or Agency) on March 11, 2022 (Petition).

The Petition requests that FDA amend the 2018 guidance for industry titled Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics (2018 Guidance) to require or strongly urge supplementary comparison of Qualify of Life-related patient-reported outcome (QoL-PRO) measures for certain surrogate endpoints in cancer clinical trials.

Specifically, the Petition requests that in amending the 2018 Guidance, FDA take the following actions:

• Provide guidance to clinical trialists, drug sponsors, and Institutional Review Boards regarding the need to capture and compare QoL-PROs as secondary (supplemental) endpoints – particularly when the primary endpoint is a surrogate for clinical benefit based on tumor imaging.

• Help to set standards for secondary QoL-PRO reporting, beginning with
ClinicalTrials.gov. This so that what is reported can be readily utilized to interpret the study results in order to guide clinical practice and better-informed patient choice.

FDA has carefully considered your Petition and acknowledges the importance of the issues it
raises. …

Full text of FDA response:

Full text of our petition:


Karl, you and I should found an activist group called ‘Antifda’.

This sounds familiar. Have only done a cursory read so far.

FDA closes out series of patient-focused drug development guidance

The FDA on Wednesday released its fourth (of four) patient-focused drug development guidance documents, with this latest draft featuring long-awaited advice on how to collect and submit data on the individual experiences of patients to inform regulatory decisions.

So-called clinical outcome assessment (COA)-based endpoints feature prominently in the draft, with the FDA saying COAs are “intended to reflect, directly or indirectly, how patients feel, function, or survive.” But there are lots of dos and don’ts when it comes to COAs, and sponsors may frequently encounter challenges, many of which are outlined in the 45-page draft.