Sodium Phenylbutyrate and Taurursodiol: A New Therapeutic Option for the Treatment of Amyotrophic Lateral Sclerosis.

OBJECTIVE: To review the safety and efficacy of sodium phenylbutyrate and taurursodiol (SP + T) in slowing progression of amyotrophic lateral sclerosis (ALS) compared with pre-existing therapies. DATA SOURCES: A PubMed (from January 1, 2009, to April 13, 2023) and ClinicalTrials.gov search conducted using sodium phenylbutyrate, taurursodiol, AMX0035, riluzole, and edaravone. Additional articles were identified by hand from references. DATA SELECTION AND DATA EXTRACTION: This included English-language articles evaluating SP + T efficacy or safety in humans for decreasing neuronal death and slowing the progression of ALS. DATA SYNTHESIS: In one phase II clinical trial that encompassed an open-label extension phase, disease severity, assessed by the rate of decline in overall score on the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised with higher scores indicating more functional ability, was -1.24 points per month with active drug and -1.66 points per month with placebo (difference, 0.42 points per month; 95% CI, 0.03-0.81; P = 0.03). Post hoc analysis found survival benefit of median 4.8 months with active medication compared with placebo. RELEVANCE TO PATIENT CARE AND CLINICAL PRACTICE IN COMPARISON WITH EXISTING DRUGS: SP + T is a new US Food and Drug Administration-approved oral suspension for the treatment of ALS. Patients who received active medication through the phase II trial showed decreased rates of disease progression. Overall, SP + T could be considered a potential agent for the treatment of ALS which has a high unmet need. CONCLUSION: SP + T is an option for the treatment of ALS; however, additional data regarding efficacy in phase III trials with long-term safety profile considerations, as well as trials to compare current therapy with SP + T, are needed.

Inclisiran: A First-in-Class siRNA Therapy for Lowering Low-Density Lipoprotein Cholesterol.

OBJECTIVE: To review the current pharmacology, pharmacokinetics/pharmacodynamics, safety, and efficacy of inclisiran in lowering lipid levels. DATA SOURCES: A PubMed (from December 1, 2014 to April 15, 2022) and ClinicalTrials.gov search was conducted using ALN-PCSsc, ALN-60212, PCSK9si KJX-839, and inclisiran. Additional articles were identified by hand from references. STUDY SELECTION AND DATA EXTRACTION: We included English-language articles evaluating inclisiran pharmacology, efficacy, or safety in humans for lowering low-density lipoprotein cholesterol (LDL-C). DATA SYNTHESIS: Inclisiran is a novel small interfering RNA-based therapy administered as a twice-yearly subcutaneous injection. By binding to the messenger RNA (mRNA) precursor of proprotein convertase subtilisin/kexin type 9 (PCSK9), inclisiran inhibits expression of the PCSK9 gene, resulting in increased recycling and expression of LDL receptors and decreased levels of LDL-C. Like PCSK9 inhibitors, inclisiran was associated with a comparable extent of LDL-C reduction in several phase II/III trials. Compared with placebo, inclisiran was found to have similar adverse events except for injection-site reaction. RELEVANCE TO PATIENT CARE AND CLINICAL PRACTICE: Currently, inclisiran lacks data on clinical outcome improvement or long-term safety. However, it may play a role in patients with atherosclerotic cardiovascular disease (ASCVD) or ASCVD risk equivalent if optimal LDL-C cannot be achieved by statins and PCSK9 inhibitors cannot be tolerated. The drug may be used for heterozygous familial hypercholesterolemia. CONCLUSION: Inclisiran is an effective and safe medication for lowering LDL-C levels. Additional data regarding efficacy on cardiovascular outcomes and long-term safety profile with inclisiran are needed.

Improved learning experience with modified case studies courses in a pharmacy curriculum.

BACKGROUND AND PURPOSE: Many pharmacy programs have dedicated case studies (CS) courses; however, universal standards on how to implement CS courses do not exist. Understanding the process of changing CS courses at an institution may help others to recognize and overcome potential barriers. This study describes the implementation process of changes that occurred in CS courses and evaluates the impact of the changes on students' learning experiences. EDUCATIONAL ACTIVITY AND SETTING: Faculty members involved in CS courses made changes that focused on small group discussions, increasing faculty availability, decreasing student-to-faculty ratio, providing immediate and detailed feedback, and facilitating active learning. After the implementation of the new CS course design, two surveys were administered to evaluate the impact of the new design, one to a single cohort of pharmacy students and one to faculty involved in the CS courses. FINDINGS: Seventy-two students completed the survey (80% response rate). The majority of students preferred the following aspects of the new CS course design: more objective SOAP (subjective, objective, assessment, plan) note rubric with detailed descriptions on point allocation, small group discussion within a classroom, and faculty-facilitated case review in group discussion. Having the same, readily available instructors reviewing a case in each class was also an important factor in their learning experience. The faculty survey resulted in similar findings but with concerns of increased workload and teaching an unfamiliar topic. SUMMARY: Overall, the new CS course design provided a better learning experience for pharmacy students compared to the previous CS course design.

The Pharmacist's Expanding Role in HIV Pre-Exposure Prophylaxis.

The efficacy of pre-exposure prophylaxis (PrEP) to prevent HIV has been firmly established; however, the success of PrEP largely depends on access to care as well as high levels of medication adherence. One of the key areas of focus for the National HIV/AIDS Strategy for 2020 in the United States calls for full access to comprehensive PrEP services where appropriate and desired, with support for medication adherence. Despite advances and advocacy for PrEP since approval for adults in 2012, large rates of prescribing disparity exist among gender and race/ethnicity. In 2016, only 3.7% of all PrEP users were women and only 11.2% were black. As one of the most widely accessible health care resources, pharmacists are well positioned to improve patient understanding, promote medication adherence, provide key risk reduction counseling, and enhance PrEP efficacy. Pharmacists' knowledge and accessibility in nearly every urban and rural community can be leveraged as part of a comprehensive HIV prevention strategy to expand access to care and improve population health. As trusted health care professionals, pharmacists develop a strong rapport with patients and may be the key to address current disparities in PrEP prescribing patterns as well as serve as an essential liaison between patients and other members of the multi-disciplinary care team. The purpose of this review is to summarize available data on pharmacist involvement in various models of care providing PrEP services and to identify opportunities to maximize and expand the role of the pharmacist to improve access to PrEP.