CHEST Guidelines-The-St.-George’s-Respiratory-Questionnaire- (1)

The-St.-George’s-Respiratory-Questionnaire- (1)

Pdf Summary

The development of new medicines is hampered despite advancements in understanding human biology and disease. Conventional tools for clinical assessments are outdated, making it difficult to evaluate new biologically-based treatments. Regulatory bodies like the FDA have instituted programs to develop and qualify new drug development tools (DDTs). These programs require collaboration among various stakeholders, including consortia led by the FDA and other organizations.<br /><br />The COPD Biomarker Qualification Consortium (CBQC) is one such collaborative effort aimed at qualifying new DDTs for chronic obstructive pulmonary disease (COPD). This consortium engages pharmaceutical companies and academics to select appropriate biomarkers for clinical trials, pooling data from various sources.<br /><br />One vital development has been the inclusion of the St. George’s Respiratory Questionnaire (SGRQ) in the FDA's draft guidance as a clinical outcome assessment (COA) measure for COPD. This recognition allows drug sponsors to use the SGRQ for new drug applications, offering some certainty that positive results using this measure will be considered pivotal, albeit in conjunction with another measure.<br /><br />Despite these advances, challenges remain. Preparing a dossier for new DDTs involves significant costs and effort, comparable to a new drug application, and the process often faces significant delays due to limited resources at regulatory agencies. The FDA maintains a compendium listing COAs used in clinical trials, but only a fraction are qualified, highlighting the need for more resources and streamlined processes.<br /><br />Future efforts will necessitate accelerated paths for COA and biomarker qualifications, given substantial unmet needs in COPD and other conditions. The integration of measures like the SGRQ marks progress, but ongoing support from initiatives like the 21st Century Cures Act will be essential in fostering innovation, reducing morbidity and mortality, and bringing new therapeutics to market.

Keywords

drug development

biomarkers

COPD

FDA

St. George’s Respiratory Questionnaire

clinical trials

regulatory agencies

21st Century Cures Act

biologically-based treatments

clinical outcome assessment