Broadstreet is pleased to be presenting several virtual posters at this year’s ISPOR meeting.
VISUALIZING NETWORK META-ANALYSES IS NOT TRIVIAL – A NOVEL TAKE ON THE NETWORK DIAGRAM
Popoff, E;1 Powell, L;2 Johnston, K1
1Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada
This study sought to create a novel visualization framework that concisely describes several feasibility assessment, network, and network meta-analysis (NMA) output features simultaneously. Separate visualizations were developed for networks incorporating continuous and binomial outcomes as well as for incorporating time-varying survival outcomes. The technique was found to work best for star-shaped networks with adaptations possibly required for other geometries or applications outside NMAs.
CHARACTERIZING KEY CLINICAL OUTCOMES AMONG PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY: RESULTS FROM A 20-YEAR POPULATION-BASED STUDY
Qian, C;1 Klimchak, AC;2 Szabo, SM;1 Gooch, KL;2 Dragan, R;3 Prior, HJ;3 Mah, J4
1Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada; 2 Sarepta Therapeutics, Cambridge, MA, USA; 3University of Manitoba, Winnipeg, MB, Canada; 4University of Calgary, Calgary, AB, Canada
This study provides valuable insight into the occurrence of key clinical events among those with Duchenne and contributes to the evidence regarding the natural history of the disease. Among this cohort, the median age for a diagnosis of scoliosis was 12 years, severe respiratory outcomes were recorded at 14 years, cardiomyopathy at 13 years and median age at death was 19 years old. This work contributes to our understanding of the considerations and potential limitations for estimating key clinical outcomes in Duchenne patients using real-world administrative data.
THE IMPACT OF KEY HEALTH STATE TRANSITIONS ON HEALTH-RELATED QUALITY OF LIFE IN DUCHENNE MUSCULAR DYSTROPHY: A QUALITATIVE STUDY
Szabo, SM;1 Audhya, IF;2 Bever, A;1 Feeny, D;3 Malone, DC;4 Neumann, PI;5 Mickle, AT;1 Iannaccone, ST;6 Gooch, KL2 1Broadstreet HEOR, Vancouver, BC; 2Sarepta Therapeutics, Inc., Cambridge, MA; 3McMaster University, Hamilton, ON; 4The University of Utah, Salt Lake City, UT; 5Tufts School of Medicine, Boston, MA; 6The University of Texas Southwestern, Dallas TX USA.
The objective of this study was to understand patient perspectives on the health-related quality of life (HRQoL) impacts of key changes in health that characterize the progressive loss of physical function experienced by individuals with Duchenne. Of note, while patients reported loss of ambulation and initiating nocturnal ventilation as having substantive impacts on quality of life, patients considered loss of upper limb function to be the most challenging transition due to accompanying loss of independence. The findings from this study are valuable as they highlight clinical transitions and events in DMD that are important from patient and caregiver perspectives.
TIME TRADE-OFF UTILITY VALUES FOR HEALTH STATES CHARACTERIZING PROGRESSIVE MUSCULAR DEGENERATION IN DUCHENNE MUSCULAR DYSTROPHY
Audhya, IF;1 Szabo, SM;2 Bever, A;2 Malone, D;3 Feeny, D;4 Neumann, PI;5 Bolatova, T;2 Iannaccone, ST;6 Gooch, KL1 1Sarepta Therapeutics, Inc., Cambridge, MA, 2Broadstreet HEOR, Vancouver, BC, 3The University of Utah, Salt Lake City, UT, 4McMaster University, Hamilton, ON, 5Tufts School of Medicine, Boston, MA, 6The University of Texas Southwestern, Dallas TX USA
This study aimed to estimate societal utility values for a comprehensive set of health states characterizing the clinical and patient relevant course of Duchenne, that describe the impact of key disease phases on HRQoL and activities of daily living. Of note, utility values for non-ambulatory health states with preserved or moderately impaired upper limb function were found to be substantially higher than previously published estimates for non-ambulatory health states. These findings help to document the effect of Duchenne on HRQoL from the societal perspective. If you have questions about any of these projects, please get in touch at [email protected]
