An Additional Criterion to Appraisal of Guidelines for Research and Evaluation II Might Increase the Efficiency in Guideline Development for Rare Diseases: Results of a National Delphi Study

Abstract

Introduction

Rare diseases significantly impact patients, and there is a need for clinical practice guidelines (CPGs) or other guidance documents as well as tools to assess their trustworthiness.

Objective

The goal was to define a minimum set of criteria for the appraisal of guidance documents for rare diseases. Guidance documents include CPGs, but also other, less stringently developed documents to guide clinical practice.

Study Design

In 2022, we started a Delphi consensus procedure to develop criteria specifically for rare disease guidance documents. Participants were 23 delegates from 18 medical specialists' associations, 3 patient organizations for rare diseases, and 2 other medical organizations in the Netherlands. The Delphi procedure consisted of three rounds. In the first round, the participants were asked whether six prespecified criteria, selected from AGREE II, were relevant, and to add other important criteria. In the second round, all collected criteria were prioritized, and in the third round, the criteria with the highest priority were ordered by the participants. In an online meeting, the remaining criteria were reformulated.

Results

Six of the seven final criteria correspond to the AGREE II criteria. The participants proposed a new criterion focusing on efficient use of limited resources, namely the inventory of problem areas as a basis for specific clinical questions to which the recommendations would provide answers.

Conclusion

The current set of criteria for rare disease guidance documents helps evaluate the trustworthiness and enhance equitable healthcare quality for rare disease patients. Although the aim was that the minimum set of criteria reduces the time and effort for developing quality criteria for rare diseases, the participants' perceived need for methodological rigour prevailed.