Building a trusted framework for uncertainty assessment in rare diseases: suggestions for improvement (Response to "TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases").

SE Grimm*, X Pouwels, BLT Ramaekers, B Wijnen, S Knies, Janneke P.C. Grutters, MA Joore

*Corresponding author for this work

Research output: Contribution to journalLetterAcademicpeer-review

1 Citation (Scopus)
9 Downloads (Pure)

Abstract

The aim of this letter to the editor is to provide a comprehensive summary of uncertainty assessment in Health Technology Assessment, with a focus on transferability to the setting of rare diseases. The authors of “TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases” presented recommendations for reducing uncertainty in rare diseases. Their article is of great importance but unfortunately suffers from a lack of references to the wider uncertainty in Health Technology Assessment and research prioritisation literature and consequently fails to provide a trusted framework for decision-making in rare diseases. In this letter to the editor we critique the authors’ tool and provide pointers as to how their proposal can be strengthened. We present references to the literature, including our own tool for uncertainty assessment (TRUST; unrelated to the authors’ research), apply TRUST to two assessments of orphan drugs in rare diseases and provide a broader perspective on uncertainty and risk management in rare diseases, including a detailed research agenda.
Original languageEnglish
Article number62
JournalOrphanet Journal of Rare Diseases
Volume16
Issue number1
DOIs
Publication statusPublished - 1 Feb 2021

Fingerprint

Dive into the research topics of 'Building a trusted framework for uncertainty assessment in rare diseases: suggestions for improvement (Response to "TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases").'. Together they form a unique fingerprint.

Cite this