FDA Finalizes Guidance on PRO Assessment Tool for COPD Trials

Regulatory NewsRegulatory News | 26 March 2018 |  By 

The US Food and Drug Administration (FDA) on Monday finalized guidance to help sponsors developing drugs to treat chronic obstructive pulmonary disease (COPD) to use the St. George’s Respiratory Questionnaire (SGRQ), a patient-reported outcome measure (PRO) assessment tool, in clinical trials.

The 8-page guidance, which was revised in draft form in May 2016, notes that sponsors can can use the SGRQ as a PRO assessment of efficacy in submissions to investigational new drug applications, new drug applications and biologics license applications.

Sponsors can obtain scores from the SGRQ either using a longer 50-item questionnaire or a 40-item version, though the shorter one “does not have a defined recall period” while the longer one has different recall periods of one year, three months or four weeks, FDA says.

“A sponsor should discuss with the review division the use of the SGRQ for stratification or enrichment purposes early in the protocol development phase,” the guidance adds.

FDA also says sponsors should “only use the total score,” which is made up of three components: “(1) symptoms — frequency and severity of symptoms; (2) activity — effect of disease on common daily physical activities; and (3) impacts — psychosocial effects of the disease.”

“The minimum clinically important difference for the total score between patients and within-patient has been determined to be at least 4 units on the SGRQ scale (Jones 2002; Jones 2005). For the FDA, no evidence exists to support the use of other values,” FDA notes.

Responder analysis is FDA’s preferred primary method for reporting results from questionnaire data.

“This analysis compares patients who improve with patients who deteriorate or do not change,” FDA explains.

“A sponsor can present responder analyses as the responder rate for each arm and the difference in the responder rates, or the odds ratio. Other analyses may be appropriate, and the sponsor should discuss these with the review division.”

Chronic Obstructive Pulmonary Disease: Use of the St. George’s Respiratory Questionnaire as a PRO Assessment Tool: Guidance for Industry


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