Verifying the value of existing frameworks for formulary review at a large academic health system: assessing inter-rater reliability.

BACKGROUND: The value assessment framework (VAF) is one approach to assessing the evidence and value of medications. VAFs are a way to measure and communicate the value of medications and other health care technologies for decision-making purposes. Given the increasing number of high-cost medications, challenging formulary inquiries, and critiques of currently available tools, health systems need to explore a standardized way to incorporate value assessment into formulary decision making. OBJECTIVES: To (a) evaluate existing VAFs by measuring inter-rater reliability among typical clinicians completing formulary reviews and (b) explore general implications of applying these tools to formulary decision making for all medications at a large academic health system. METHODS: This was a retrospective, observational study at a single health system. A list of medications added, denied, and removed from the system formulary from September 1, 2013, through August 31, 2018, was collected. Published VAFs, such as the American Society of Clinical Oncology (ASCO) Value Framework, European Society of Medical Oncology (ESMO) Magnitude of Clinical Benefit Scale, National Comprehensive Cancer Network (NCCN) Evidence Blocks, American College of Cardiology/American Heart Association Value Framework, and the incremental cost-effectiveness ratio (ICER) calculation were applied by 3 different reviewer groups. The primary outcome was inter-rater reliability among the 3 different reviewers for a given framework. Cohen's weighted kappa and the intraclass correlation coefficient (ICC) were used to assess inter-rater reliability. RESULTS: The frameworks were applied to 94 medications. The VAFs with the highest ICCs between all 3 raters were NCCN (0.635; 95% CI = 0.387-0.823) and ASCO (0.634; 95% CI = 0.370-0.832), both indicating moderate inter-rater reliability. The VAFs with the lowest ICCs were ESMO (0.368; 95% CI = 0.126-0.611) and ICER (0.159; 95% = CI -0.018-0.365), with ICCs corresponding to poor reliability. CONCLUSIONS: Because high-cost medications are a challenge to health systems, VAFs may be beneficial to target formulary decision making in this setting. Applying VAFs proactively may improve interrater reliability and usability in formulary decision making. DISCLOSURES: No outside funding supported this study. The authors have nothing to disclose.

The Missing Link: Evolving Accessibility To Formulary-Related Information.

BACKGROUND: Formulary management is a key component to ensuring the safe, effective, and fiscally responsible use of medications for health systems. One challenge in the formulary management process is making the most relevant formulary information easily accessible to practitioners involved in medication therapy decisions at the point of care. In September 2014, Froedtert and the Medical College of Wisconsin (F&MCW) implemented a commercial formulary management tool (CFMT) to improve accessibility to the recently aligned health-system formulary. The CFMT replaced an internally developed formulary management tool. OBJECTIVES: The primary objective was to determine pharmacist end-user satisfaction with accessibility to system formulary and formulary-related information through a new CMFT compared with the historical formulary management tool (HFMT). The secondary objective was to measure the use of formulary-related information in the CFMT and HFMT. METHODS: The primary objective was measured through pharmacist end-user satisfaction surveys before and after integration of formulary-related information into the CFMT. The secondary objective was measured by comparing monthly usage reports for the CFMT with monthly usage reports for the HFMT. RESULTS: Survey respondents reported being satisfied (52.5%) or very satisfied (18.8%) more frequently with the CFMT compared with the HFMT (31.7% satisfied and 2.5% very satisfied). Between October 2014 and January 2015 the frequency of access to formulary-related information increased from 92 to 104 requests per day through the CFMT and decreased from 47 to 33 requests per day through the HFMT. CONCLUSIONS: Initial data suggest incorporating system formulary-related information and related resources into a single platform increases pharmacist end-user satisfaction and overall use of formulary-related information.

Enzyme Replacement or Substrate Reduction? A Review of Gaucher Disease Treatment Options.

BACKGROUND: Gaucher disease is a rare lysosomal storage disease resulting from a deficiency or reduced activity in the acid ß-glucocosidase enzyme. Only 1 treatment option was available for 15 years, but several new treatment options have come to market since 2003. OBJECTIVE: The article will detail the pathophysiology and review current therapies in the literature for all 3 major clinical types of Gaucher disease, with a focus on considerations for selecting therapy in type 1 disease. METHODS: Extracted and summarized applicable studies and reviews from Cochrane Review, ClinicalTrials.gov, CINAHL, IPA, and PubMed. RESULTS: Enzyme replacement therapy is preferred for the management of Gaucher disease. Current literature does not favor any enzyme replacement product over another. However, velaglucerase alfa and taliglucerase alfa theoretically have a lower risk of immunogenicity reactions compared with imiglucerase. Alternative treatments for type 1 disease include substrate reduction therapy; however, these treatments require evaluation of patient-specific variables (eg, genotype evaluation, renal function) and consideration of adverse effect and dosing profiles. Evaluation of current literature found no substrate reduction therapy is preferred over another. There are no approved therapies for type 2 and type 3 disease, but enzyme replacement therapy may be used with limited efficacy for symptom management. CONCLUSION: Enzyme replacement therapy is preferred for treating type 1 Gaucher disease and substrate replacement therapy may be considered in patients who do not tolerate or cannot receive enzyme replacement therapy.