Using Expert Judgement to Inform Decision Making WHEN the Evidence Base Is Limited: A Case Study in Haemophilia
Author(s)
Martin A1, Asghar S1, Morgan G1, Burke T1, Maruszczyk K2, Pedra G1, O'hara J1, Sun S3
1HCD Economics, Daresbury, UK, 2HCD Economics, Warszawa, MZ, Poland, 3Shire US Inc., a Takeda company, Cambridge, MA, USA
OBJECTIVES: Benefits associated with physical activity (PA) for people with haemophilia (PWH) may include improvements in joint, bone and muscle health. However, the factor VIII activity level required to avoid a bleeding episode with PA is unknown. We therefore sought to determine if clinically acceptable estimates could be generated using a modified version of the Sheffield Elicitation Framework (SHELF), a formal approach for eliciting judgements regarding probability distributions. METHODS: A panel of 9 haemophilia experts which included five consultant haemotologists, two clinical specialist physiotherapists, one haemophilia nurse consultant and one patient advocate participated. Based on the 2017 National Hemophilia Foundation (NHF) PA descriptions, clinical experts estimated factor levels at which a bleed could be avoided. The estimates were elicited using an adapted version of the SHELF Roulette method. Probability distributions for each scenario were combined and presented to the panel of experts. RESULTS: All responses were judged to be correct applications of the SHELF method and estimates were combined. The final set of outcomes and approach were accepted by the panel and may be used to inform future research and evaluation for factor VIII activity level required to avoid a bleeding episode with PA. CONCLUSIONS: Important health care decisions are required often where the evidence base is limited. SHELF is a formal approach for harnessing expert judgements. SHELF can be used to promote evidence-based practice by use of a methodological tool to facilitate the combination of expert judgement to guide health care decisions.
Conference/Value in Health Info
2020-11, ISPOR Europe 2020, Milan, Italy
Value in Health, Volume 23, Issue S2 (December 2020)
Code
PRO111
Topic
Clinical Outcomes, Health Technology Assessment, Methodological & Statistical Research
Topic Subcategory
Clinician Reported Outcomes, Decision & Deliberative Processes, Missing Data, Relating Intermediate to Long-term Outcomes
Disease
Rare and Orphan Diseases