Abstract
BackgroundRare diseases are defined as life-threatening or chronically debilitating diseases with a prevalence of 50 out of 100,000 individuals or less. Orphan medicinal products (OMPs) are intended for the treatment of rare diseases. The assessment of quality of evidence in small populations is often complex. Many generic tools are unfit. Therefore, the aim of this study was to develop and validate a new tool to assess the quality of OMPs' clinical evidence (COMPASS).MethodsFirstly, a draft version of the COMPASS tool, developed by the authors and consisting of three parts, was amended based on suggestions obtained in four rounds of expert consultation. Secondly, the tool was put through three rounds of validation. The data source was information provided on the Orphanet website and in European Public Assessment Report (EPAR) document of the European Medicines Agency.ResultsThe first pilot round revealed a high (92.2%) inter-rater agreement for part one of the tool. After further improvements, the final inter-rater agreement was 86.4% for part two (on methodological quality) and three (on quality of reporting) of the tool. The COMPASS tool does not attempt to score or rank the quality of clinical evidence, but rather to give an outline of various, key elements with respect to quality of clinical evidence of OMP studies.ConclusionsThe COMPASS tool can be applied to assess the quality of evidence of an OMP based on information in the registration dossier, for example by local reimbursement agencies, pharmacists or clinicians. In that way, the tool can contribute to making reimbursement and/or treatment decisions increasingly more founded on the principles of evidence-based decision making.
Highlights
Rare diseases are defined as life-threatening or chronically debilitating diseases with a prevalence of 50 out of 100,000 individuals or less
Non-binding recommendations for the approval of cancer drugs and biologics were issued by the Food and Drug Administration (FDA) several years ago [21]
In conclusion, we developed and validated a new tool for the assessment of clinical evidence of Orphan medicinal product (OMP)
Summary
Rare diseases are defined as life-threatening or chronically debilitating diseases with a prevalence of 50 out of 100,000 individuals or less. The aim of this study was to develop and validate a new tool to assess the quality of OMPs' clinical evidence (COMPASS). It is estimated that there are currently between 5,000 and 7,000 rare diseases [2]. Studies to evaluate the effect of an OMP in patients with rare diseases are often hampered by the difficulty of enrolling a sufficient number of patients [3,4]. How do we define quality of clinical evidence? According to GRADE (Grading of Recommendations Assessment, Development and Evaluation), quality of evidence reflects the extent of our confidence that the estimates of an effect are correct [8]. The draft version of the tool was adapted in accordance with all relevant issues, as deemed upon consensus by the researchers, raised at all consultations
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
