Article type
Abstract
Background
In the Netherlands, many national quality documents exist for rare diseases, which do not have the official status of a clinical practice guideline (CPG) but are used as a guidance for clinical practice. Especially for rare diseases, it is difficult to develop guidelines, as topics are plenty and resources and evidence are (often) limited. Therefore, national quality documents may provide a helping hand in the day-to-day practice of many clinicians when faced with a disease they do not encounter on a daily basis.
Objectives
In this study, we have appraised quality documents for rare diseases that are used in the Dutch clinical setting and do not have the official status of a CPG.
Methods
We randomly selected 20 quality documents on a wide variety of rare diseases from medical specialists’ associations and patient associations. Two researchers independently evaluated the quality of the Dutch national quality documents according to a set of 10 criteria that was specifically developed for the appraisal of quality documents and based on the AGREE II criteria.
Results
The number of criteria met by the assessed documents varied from 0 to 7 (with a median of 3) on a total of 10 criteria. A systematic literature search and selection was only described in one document. Also, in only 1 document, the conflicts of interest and actions to address these conflicts were addressed. We noticed that many documents altogether lacked a description of the methods.
Conclusions
This study shows considerable limitations in the transparency and methodological rigor of Dutch quality documents for rare diseases. Quality documents, also for rare diseases, need to adhere to quality standards, excluding bias and undue influence as much as possible. There is a need for rigorous quality standards but also for an efficient methodology to enhance the feasibility of the development of quality documents for rare diseases. A first step toward improving the quality of rare disease quality documents without adding much workload would be the addition of a methods paragraph. With increased transparency, recommendations can be more accurately interpreted, thereby enhancing the quality of quality documents, and finally of patient care.
In the Netherlands, many national quality documents exist for rare diseases, which do not have the official status of a clinical practice guideline (CPG) but are used as a guidance for clinical practice. Especially for rare diseases, it is difficult to develop guidelines, as topics are plenty and resources and evidence are (often) limited. Therefore, national quality documents may provide a helping hand in the day-to-day practice of many clinicians when faced with a disease they do not encounter on a daily basis.
Objectives
In this study, we have appraised quality documents for rare diseases that are used in the Dutch clinical setting and do not have the official status of a CPG.
Methods
We randomly selected 20 quality documents on a wide variety of rare diseases from medical specialists’ associations and patient associations. Two researchers independently evaluated the quality of the Dutch national quality documents according to a set of 10 criteria that was specifically developed for the appraisal of quality documents and based on the AGREE II criteria.
Results
The number of criteria met by the assessed documents varied from 0 to 7 (with a median of 3) on a total of 10 criteria. A systematic literature search and selection was only described in one document. Also, in only 1 document, the conflicts of interest and actions to address these conflicts were addressed. We noticed that many documents altogether lacked a description of the methods.
Conclusions
This study shows considerable limitations in the transparency and methodological rigor of Dutch quality documents for rare diseases. Quality documents, also for rare diseases, need to adhere to quality standards, excluding bias and undue influence as much as possible. There is a need for rigorous quality standards but also for an efficient methodology to enhance the feasibility of the development of quality documents for rare diseases. A first step toward improving the quality of rare disease quality documents without adding much workload would be the addition of a methods paragraph. With increased transparency, recommendations can be more accurately interpreted, thereby enhancing the quality of quality documents, and finally of patient care.