Article type
Abstract
"Background:
Clinical practice guidelines (CPG) for rare diseases, such as immune thrombocytopenia (ITP), pose a unique challenge for guideline developers and methodologists as there is a high demand for recommendations on their management, while high-quality evidence is scarce. To address pivotal questions effectively, guideline developers must carefully balance the relevance of key questions (KQ) to patients and clinicians while efficiently utilising resources in selecting an appropriate methodologic approach.
Objectives:
This pilot project aims to provide CPG developers with a transparent stepwise approach of KQ refinement and prioritisation for systematic evidence synthesis to increase the relevance of the resulting panel recommendations and thus improve patients’ care.
Methods:
Following the initial presentation of KQs by thematic authoring groups of the ITP guideline project, we created a standardised template posing signalling questions that address major concepts of systematic evidence synthesis and the foundation of good practice statements (i.e. consensus recommendations). Figure 1 details the workflow structure. We organised workshop meetings with the involved thematic groups to guide them through the refinement and prioritisation of questions.
Results:
Initially, we were presented with a list of 208 KQs posed by nine thematic groups. Following the stepwise approach, we created a map of interlinked KQs. By consolidating KQs across groups and excluding those that did not lead to actionable recommendations or meet the criteria for good practice statements, the total number was reduced to a preliminary list of 76 KQs, with five pending decisions. Thereof, 38 were selected and prioritised for systematic evidence synthesis, and 38 were chosen to be addressed via consensus (Figure 2). A systematic search yielded 161 records, from which we identified 27 eligible guidelines or evidence summaries, with one CPG qualifying for ADOLOPMENT consideration.
Conclusions:
Through this streamlined approach, the team successfully condensed and refined a large number of questions, leading to a focused set of patient- and clinician-relevant KQs designated for systematic evidence synthesis and consensus work-up. In conclusion, this pilot project offers a transparent workflow of KQ refinement and prioritisation, feasible to address challenges posed by the scarcity of high-quality evidence in creating CPGs for rare diseases. "
Clinical practice guidelines (CPG) for rare diseases, such as immune thrombocytopenia (ITP), pose a unique challenge for guideline developers and methodologists as there is a high demand for recommendations on their management, while high-quality evidence is scarce. To address pivotal questions effectively, guideline developers must carefully balance the relevance of key questions (KQ) to patients and clinicians while efficiently utilising resources in selecting an appropriate methodologic approach.
Objectives:
This pilot project aims to provide CPG developers with a transparent stepwise approach of KQ refinement and prioritisation for systematic evidence synthesis to increase the relevance of the resulting panel recommendations and thus improve patients’ care.
Methods:
Following the initial presentation of KQs by thematic authoring groups of the ITP guideline project, we created a standardised template posing signalling questions that address major concepts of systematic evidence synthesis and the foundation of good practice statements (i.e. consensus recommendations). Figure 1 details the workflow structure. We organised workshop meetings with the involved thematic groups to guide them through the refinement and prioritisation of questions.
Results:
Initially, we were presented with a list of 208 KQs posed by nine thematic groups. Following the stepwise approach, we created a map of interlinked KQs. By consolidating KQs across groups and excluding those that did not lead to actionable recommendations or meet the criteria for good practice statements, the total number was reduced to a preliminary list of 76 KQs, with five pending decisions. Thereof, 38 were selected and prioritised for systematic evidence synthesis, and 38 were chosen to be addressed via consensus (Figure 2). A systematic search yielded 161 records, from which we identified 27 eligible guidelines or evidence summaries, with one CPG qualifying for ADOLOPMENT consideration.
Conclusions:
Through this streamlined approach, the team successfully condensed and refined a large number of questions, leading to a focused set of patient- and clinician-relevant KQs designated for systematic evidence synthesis and consensus work-up. In conclusion, this pilot project offers a transparent workflow of KQ refinement and prioritisation, feasible to address challenges posed by the scarcity of high-quality evidence in creating CPGs for rare diseases. "