Published On: 6/2/2023

Event Recap Blog: TARGET-NASH Analysis Presented at ISPOR 2023 and Target RWE Leaders Present at PMSA 2023

In May, Target RWE participated in some conference events across the country to share new real world data (RWD) findings from our nonalcoholic steatohepatitis (NASH) community and to give an insightful presentation on data curation and impact on the field of research.

Researchers from academia, industry, and Target RWE collaborated to estimate NASH progression in a natural history cohort stratified by both baseline fibrosis stage and inflammation using real world data from the longitudinal, observational TARGET-NASH registry. The analysis was presented recently at the International Society for Pharmacoeconomics and Outcomes (ISPOR) annual meeting in Boston.

Disease progression of NASH includes assessed by a progression of fibrosis on a scale of F0 to F4 (the most severe level or cirrhosis) and inflammation using the nonalcoholic fatty liver disease activity score (NAS), which is a composite score of steatosis, lobular inflammation, ballooning, and fibrosis.

“We need more data to tell real-world patients – let alone inform health-economic models – about the rate of fibrosis progression in NASH,” said Elliot Benjamin Tapper, M.D., Associate Professor, Gastroenterology, Internal Medicine, Transplant Hepatology, and lead author on the poster. “We found that NAS scores are a feasible and informative way to predict fibrosis stage progression and enable modeling of earlier disease progression in NASH. The NASH transition probabilities reflected annual change for evaluating NASH progression in the context of fibrosis stage.”

Data from adult patients enrolled in the U.S. in Target RWE’s longitudinal observational cohort study TARGET-NASH with biopsy-confirmed NASH, paired biopsies, and NAS, were used to determine the transition probability of NAS and the conditional probability of fibrosis progression. As NAS ≥4 is an inclusion criterion for NASH clinical trials, researchers examined this categorically.

A total of 152 patients with paired liver biopsies and NAS were included in the analysis. Patients had a median age of 57 and were White (90%), Female (61%), with a mean BMI of 33 kg/m2. Multinomial logistic regression from Target RWE advanced analytics was adapted to estimate transition probabilities. Based on the results of the analysis, in the NAS <4 group (n=27), the annual transition probability to NAS ≥4 was 0.29 (95% confidence interval [CI]: 0.1-0.47); in the NAS ≥4 group (n=44), the probability of transitioning to NAS <4 was 0.37 (95%CI: 0.22-0.53). Regardless of initial NAS, the fibrosis stage was more likely to remain the same or increase versus decrease.

At the Pharmaceutical Management Science Association (PMSA) 2023 conference in San Diego, Target RWE leaders gave an important discussion on the impact of medical data curation approaches on data quality and its profound implications for research insights. Presented by Chief Commercial Officer, Lutz Schlicht, PhD, and SVP of Commercial Analytics & Data Curation, Ewa J. Kleczyk, PhD, the talk provided an overview of medical data curation - emphasizing its crucial role in transforming raw and complex healthcare data into a structured, usable format and the federal guidelines enforced by the FDA for drug development.

“Proper curation of medical data is essential to ensure its quality and reliability, enabling researchers to derive meaningful moments of meaning and fuel evidence-based decision-making,” said Lutz Schlicht, PhD. “We appreciate the opportunity to share the innovative ways that Target RWE is providing the most comprehensive, fit-for-purpose data curated from our well-established disease communities at PMSA this year.”

In addition to ISPOR and PMSA, Target RWE was also a sponsor and exhibitor for Digestive Disease Week (DDW) where the company promoted its TARGET-GASTRO community which captures RWD from a variety of gastroenterology indications including Crohn’s disease, ulcerative colitis, and eosinophilic gastrointestinal disorders.

Target RWE announced the launch of 35 new disease state registries earlier this year, which include the entire spectrum of immune-mediated inflammatory conditions to capture valuable real world patient data to better understand these complex conditions and how treatment approaches may overlap.

Learn more about Target RWE data solutions and the TARGET-NASH community here.