Article type
Abstract
BACKGROUND. Rare diseases are characterized by their low prevalence, with most of them being chronic or potentially life-threatening conditions. Given their low prevalence, providing specialized treatment to patients with these types of diseases is challenging. Therefore, in 2017, the European Commission approved the first European Reference Networks (ERNs). ERNs bring together health care providers from across Europe to improve access to diagnosis and treatment for complex or rare medical conditions that require a high concentration of knowledge and resources. In this context, the ERN Clinical Practice Guidelines (CPGs) and Clinical Decision Support Tools (CDSTs) program were established and funded by the European Commission. A consortium was established consisting of 5 health technology assessment (HTA) agencies and 2 academic institutions.
OBJECTIVES. The objectives of this program were to provide a common methodology to harmonize the process of developing CPGs and CDSTs in the ERNs and to provide technical support to the ERNs to enhance their capacity to produce high-quality CPGs and CDSTs.
METHODS. Throughout the 48-month project duration (2020–2024), 4 work packages were developed: (A) governance structure, which includes establishing the Advisory Body and Expert Panels; (B) methodologies for the development, evaluation, and implementation of CPGs and CDSTs; (C) training and capacity development; and (D) support in the production of CPGs and CDSTs, including de novo development, evaluation, and adaptation.
RESULTS. An Advisory Body group with experts from rare diseases fields was established, and they have supported the development of a set of 13 Methodological Handbooks for the development of CPGs and CDSTs of rare diseases. Additionally, a training program with 2 different levels (basic/intermediate and advanced) were carried out to train clinicians in the development, appraisal, and implementation of CPGs and CDSTs. Finally, 22 out of the 24 ERNs were supported for the development of 28 CPGs and/or CDSTs. In total, 111 systematic reviews were conducted on 35 rare diseases.
CONCLUSIONS. This project has contributed significantly to the development of CPGs and CDSTs in the context of rare diseases, wherein guidelines are currently scarce, and it has harmonized the guideline development process among the different ERNs.
OBJECTIVES. The objectives of this program were to provide a common methodology to harmonize the process of developing CPGs and CDSTs in the ERNs and to provide technical support to the ERNs to enhance their capacity to produce high-quality CPGs and CDSTs.
METHODS. Throughout the 48-month project duration (2020–2024), 4 work packages were developed: (A) governance structure, which includes establishing the Advisory Body and Expert Panels; (B) methodologies for the development, evaluation, and implementation of CPGs and CDSTs; (C) training and capacity development; and (D) support in the production of CPGs and CDSTs, including de novo development, evaluation, and adaptation.
RESULTS. An Advisory Body group with experts from rare diseases fields was established, and they have supported the development of a set of 13 Methodological Handbooks for the development of CPGs and CDSTs of rare diseases. Additionally, a training program with 2 different levels (basic/intermediate and advanced) were carried out to train clinicians in the development, appraisal, and implementation of CPGs and CDSTs. Finally, 22 out of the 24 ERNs were supported for the development of 28 CPGs and/or CDSTs. In total, 111 systematic reviews were conducted on 35 rare diseases.
CONCLUSIONS. This project has contributed significantly to the development of CPGs and CDSTs in the context of rare diseases, wherein guidelines are currently scarce, and it has harmonized the guideline development process among the different ERNs.