From: Evaluation of Clinical Practice Guidelines for Rare Diseases in Japan
| Keywords | Explanations |
|---|---|
| Intractable Disease Consultation Support Center | The centers offering support to address psychological and social issues, which are in each prefecture to support their daily lives and to improve the quality of life for patients living with rare/intractable disease in the community (43). The centers offer services such as consultation and information provision on formal support programs available to patients living with rare/intractable disease, promoting social engagement, organizing employment support through counseling for adaptation to the workplace and arranging employment opportunities, coordinating of peer supports, and holding workshops for the patients and their family (43), (44). |
| Japan Intractable Diseases Information Center | Information support by a MHLW project, which is the reference source on rare/intractable diseases online. The website is designed to allow access equally to stakeholders on information about incurable diseases. The website contains information such as various Acts or Programs for rare/intractable diseases, designated rare/intractable disease projects, national research projects, clinical trials, centers of experts, patient associations, practice guidelines, and knowledge for the public (45). |
| Patient Association | Peer support. Informational, educational, psychological, and practical support based on extremely similar illness experiences (46). |
| Medical Subsidy System | Financial support as regulated by the Rare/Intractable Disease Act. Patients with rare/intractable disease are required to submit a clinical survey form, which is marked by a designated rare/intractable disease clinician, to the local healthcare center in each prefecture. When the criteria for financial support are met, the patient is eligible for the support (47). |
| Designated Rare/Intractable Diseases | A term indicates the diseases subjected to the medical subsidy system regulated by the Rare/Intractable Disease Act (47). This term can serve as a signpost to make healthcare providers aware of non-medical resources available to patients living with rare/intractable diseases. The term also indicates the organization of national specialized research groups for each disease to overcome it (28), (45), (47). |
From: Evaluation of Clinical Practice Guidelines for Rare Diseases in Japan
| Characteristics | CPGs included |
|---|---|
| Published year | |
| 2015 | 5 |
| 2016 | 6 |
| 2017 | 32 |
| 2018 (until August) | 12 |
| Edition | |
| First edition | 29 |
| Revised edition | 26 |
From: Evaluation of Clinical Practice Guidelines for Rare Diseases in Japan
| Domains/items | Domain score*,† | Item score‡,† |
|---|---|---|
| Scope and Purpose | 69% (47%, 78%) | |
| 1. The overall objective(s) of the guideline is (are) specifically described. | 5 (3, 6) | |
| 2. The health question(s) covered by the guideline is (are) specifically described. | 5 (5, 6) | |
| 3. The population (patients, public, etc.) to whom the guideline is meant to apply is specifically described. | 5 (4, 6) | |
| Stakeholder Involvement | 42% (25%, 61%) | |
| 4. The guideline development group includes individuals from all relevant professional groups. | 4 (3, 5) | |
| 5. The views and preferences of the target population (patients, public, etc.) have been sought. | 1 (1, 3) | |
| 6. The target users of the guideline are clearly defined. | 6 (3, 6) | |
| Rigor of Development | 28% (17%, 53%) | |
| 7. Systematic methods were used to search for evidence. | 5 (1, 6) | |
| 8. The criteria for selecting the evidence are clearly described. | 3 (1, 5) | |
| 9. The strengths and limitations of the body of evidence are clearly described. | 2 (2, 4) | |
| 10. The methods for formulating the recommendations are clearly described. | 2 (1, 3) | |
| 11. The health benefits, side effects, and risks have been considered in formulating the recommendations. | 3 (2, 5) | |
| 12. There is an explicit link between the recommendations and the supporting evidence. | 4 (3, 6) | |
| 13. The guideline has been externally reviewed by experts prior to its publication. | 2 (1, 5) | |
| 14. A procedure for updating the guideline is provided. | 1 (1, 3) | |
| Clarity of Presentation | 69% (42%, 86%) | |
| 15. The recommendations are specific and unambiguous. | 5 (3, 6) | |
| 16. The different options for management of the condition or health issue are clearly presented. | 5 (3, 6) | |
| 17. Key recommendations are easily identifiable. | 5 (5, 7) | |
| Applicability | 31% (13%, 46%) | |
| 18. The guideline describes facilitators and barriers to its application. | 2 (2, 5) | |
| 19. The guideline provides advice and/or tools on how the recommendations can be put into practice. | 3 (2, 5) | |
| 20. The potential resource implications of applying the recommendations have been considered. | 3 (2, 5) | |
| 21. The guideline presents monitoring and/or auditing criteria. | 2 (1, 3) | |
| Editorial Independence | 38% (17%, 50%) | |
| 22. The views of the funding body have not influenced the content of the guideline. | 3 (2, 5) | |
| 23. Competing interests of guideline development group members have been recorded and addressed. | 3 (2, 4) | |
| Overall Guideline Assessment | ||
| 1. Rate the overall quality of this guideline. | 3 (2, 5) |
From: Evaluation of Clinical Practice Guidelines for Rare Diseases in Japan
| Keywords | n = 55 | Percentage |
|---|---|---|
| Intractable Disease Consultation Support Center | 3 | 5% |
| Japan Intractable Diseases Information Center | 12 | 22% |
| Patient Association | 17 | 31% |
| Medical Subsidy System | 21 | 38% |
| Designated Rare/Intractable Diseases | 36 | 65% |
