The FDA has issued a new draft guidance titled “Chronic Obstructive Pulmonary Disease: Developing Drugs for Treatment,” a revision of a 2007 draft guidance with the same title. Among other changes, the 2016 revision includes an appendix regarding use of the St. George’s Respiratory Questionnaire (SGRQ) in COPD studies.
In the Federal Register announcement, the agency says, “FDA acknowledges that the landscape of clinical trials has evolved since 2007 and therefore is encouraging public comment on the body of the guidance in addition to public comment on the SGRQ information added in Appendix A.” According to the announcement, comments on the new draft guidance should be submitted by July 19, 2016.
The draft guidance recommends that patient and/or evaluator-reported outcome measures, including symptom scores, activity scales, and health-related quality-of-life instruments such as the SGRQ should be considered.
With regard to biomarkers, the guidance says that “With the exception of lung function tests, there are no well-validated biomarkers or surrogate endpoints that can be used to establish efficacy of a drug for COPD” but notes that other biomarkers can be considered. However, the guidance says, “With the possible exception of the high-resolution CT, none of these biomarkers are sufficiently validated to date for use as the primary evidence of efficacy or for supporting specific labeling claims.” While novel endpoints can be considered, it says, “A single study should not be used to establish both the validity of a novel primary endpoint and the efficacy of the drug in question.”
In the section on inhaled drugs, the guidance specifies that the device should be designed with COPD patients in mind, specifically that “For breath-actuated devices, the inspiratory flow-rate that will be necessary to activate the device should be such that a COPD patient can easily generate that level of flow. The device should have a dose indicator or counter that informs patients of the number of doses remaining. The device should be durable and the dexterity required to use the device should be within the capability of COPD patients who may often be elderly and may have co-existent arthritides. Phase 3 studies should assess device durability in patients’ hands and assess whether patients can follow the instructions to use the device effectively.”
Read the Federal Register announcement.
Read the 2016 draft guidance.
