Real-World Persistency for Inflammatory Bowel Disease Biologics Using Patient Registry Data.

Background: Although it is a truism that drugs benefit patients only when taken, surprisingly little is known about real-world drug-use persistence and discontinuation, even for expensive biologic drugs. Methods: We used longitudinal self-reported drug-use data from the inflammatory bowel disease (IBD) Partners registry of people with IBD to construct Kaplan-Meier drug-use persistency graphs for biologic drug-use spans that started between 2017 and 2022. Results: We examined 2034 drug-use spans for 1594 survey participants. Most of the biologic drugs had a 75%+ persistency rate around the one-year mark and 60%+ persistency at the 3-year mark. The overall persistency and the differences in persistency between drugs were aligned with published literature. Conclusions: This analysis demonstrates the feasibility of collecting IBD-specific patient-reported drug persistency data via a voluntary patient registry. Patient-reported persistency provides real-world drug persistency data and the patient's perspectives as to why they discontinued use of the drug-a combination of data and perspective that is not available from any other real-world medical record, claim, and pharmacy data source that are valuable to physician, patients, payers, healthcare policymakers, and health technology assessment organizations.

QALYs: The Math Doesn't Work.

The quality-adjusted life-year (QALY) is a metric widely used when assessing the cost-effectiveness of drugs and other health interventions. The assessments are used in the development of recommendations for pricing, formulary placement decisions, and health policy decisions. A new bill, H.R. 485, the Protecting Health Care for All Patients Act of 2023, was approved by the US House Energy and Commerce Health Subcommittee that will, if passed, end the practice of using QALYs in all federal programs.1,2 Proponents of the ban say that QALYs undervalue the positive effects of therapeutics on people with disabilities.3 We share their concerns. Furthermore, our review of the mathematical properties of QALYs, including an analysis of quality-of-life utility (QOL utility) data recently collected from patients with inflammatory bowel disease (IBD), has led us to conclude that QALYs are an inappropriate metric of drug and treatment cost-effectiveness for all people, both disabled and nondisabled, and should not be the basis for US healthcare policy decisions.

The Development and Initial Findings of A Study of a Prospective Adult Research Cohort with Inflammatory Bowel Disease (SPARC IBD).

BACKGROUND: Clinical and molecular subcategories of inflammatory bowel disease (IBD) are needed to discover mechanisms of disease and predictors of response and disease relapse. We aimed to develop a study of a prospective adult research cohort with IBD (SPARC IBD) including longitudinal clinical and patient-reported data and biosamples. METHODS: We established a cohort of adults with IBD from a geographically diverse sample of patients across the United States with standardized data and biosample collection methods and sample processing techniques. At enrollment and at time of lower endoscopy, patient-reported outcomes (PRO), clinical data, and endoscopy scoring indices are captured. Patient-reported outcomes are collected quarterly. The quality of clinical data entry after the first year of the study was assessed. RESULTS: Through January 2020, 3029 patients were enrolled in SPARC, of whom 66.1% have Crohn's disease (CD), 32.2% have ulcerative colitis (UC), and 1.7% have IBD-unclassified. Among patients enrolled, 990 underwent colonoscopy. Remission rates were 63.9% in the CD group and 80.6% in the UC group. In the quality study of the cohort, there was 96% agreement on year of diagnosis and 97% agreement on IBD subtype. There was 91% overall agreement describing UC extent as left-sided vs extensive or pancolitis. The overall agreement for CD behavior was 83%. CONCLUSION: The SPARC IBD is an ongoing large prospective cohort with longitudinal standardized collection of clinical data, biosamples, and PROs representing a unique resource aimed to drive discovery of clinical and molecular markers that will meet the needs of precision medicine in IBD.

Simulation and visualization of flow pattern in microarrays for liquid phase oligonucleotide and peptide synthesis.

Microfluidic microarrays have been developed for economical and rapid parallel synthesis of oligonucleotide and peptide libraries. For a synthesis system to be reproducible and uniform, it is crucial to have a uniform reagent delivery throughout the system. Computational fluid dynamics (CFD) is used to model and simulate the microfluidic microarrays to study geometrical effects on flow patterns. By proper design geometry, flow uniformity could be obtained in every microreactor in the microarrays.