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Purpose This project aimed to characterize the resources necessary for pharmacists to support the required steps for obtaining and handling investigational drugs outside of a study protocol in the individual patient and intermediate-size population Expanded Access Program (EAP) processes. The second aim was to characterize the types of EAP requests received. Summary This retrospective, single-center, observational study was performed by reviewing EAP requests initiated at Duke University Hospital (DUH) between August 1, 2017, and February 11, 2023. The annualized cost of unreimbursed EAP study services was projected to be approximately $196,500 at DUH for 2023. Of the 168 EAP requests submitted after the institutional policy requiring pharmacy and therapeutics (P&T) committee approval was established, 162 (96.4%) were approved by the P&T committee. Conclusion Given the lack of published information on a pharmacist-led workflow related to EAP services, this study sought to share DUH's process for managing EAP requests. As there is no mechanism for reimbursement of EAP services, they can be difficult to manage given the labor resources required. Further work is needed to recoup unreimbursed investigational drug service labor costs to ensure compassionate use programs can be implemented in a manner that is financially sustainable for a health system. [ABSTRACT FROM AUTHOR]

Purpose Health-system pharmacists play a crucial role in monitoring the pharmaceutical pipeline to manage formularies, allocate resources, and optimize clinical programs for new therapies. This article aims to support pharmacists by providing periodic updates on new and anticipated novel drug approvals. Summary Selected drug approvals anticipated in the 12-month period covering the second quarter of 2024 through the first quarter of 2025 are reviewed. The analysis emphasizes drugs expected to have significant clinical and financial impact in hospitals and clinics, as selected from 52 novel drugs awaiting US Food and Drug Administration approval. New cellular and gene therapies for cancers continued to strengthen the pipeline, in addition to new drugs targeting previously untreatable conditions. Several novel drugs are being developed for rare and ultra-rare diseases such as hemophilia, Niemann-Pick disease type C, hereditary angioedema, and aromatic l -amino acid decarboxylase deficiency. Conclusion The current drug pipeline includes new drugs with various indications for cancers and rare diseases as well as diabetes, acute coronary syndrome, chronic skin disorder, and chronic obstructive pulmonary disease. [ABSTRACT FROM AUTHOR]

Purpose This project compared the time to complete investigational prescription drug order verification by investigational drug service (IDS) pharmacists in an onsite vs offsite (hybrid) staffing model, evaluating the impact of remote work on verification time. Methods Parenteral IDS drug orders from August 2019 through June 2022 were analyzed. Electronic medical records were timestamped for each order, first verification, second verification, and medication administration. The project was divided into four 6-month time periods to allow for 2 independent comparisons of onsite vs hybrid staffing models. A survey was given retrospectively to IDS pharmacists for the last hybrid timeframe to assess work satisfaction and burnout. Results A total of 4,398 orders were evaluated. The time from order entry to first verification was 14 minutes (n = 1,248) during the first onsite time period compared to 13 minutes (n = 1,042) during the first hybrid period (P = 0.003). The time for order verification was similar between the second onsite and hybrid periods (12 minutes [n = 1,041] vs 10 minutes [n = 1,067], respectively; P < 0.0001). The staff satisfaction survey for the hybrid model showed high levels of job satisfaction and no self-reported burnout. Conclusion The remote hybrid staffing model did not result in a meaningful change in the time from order placement to medication administration within the IDS pharmacy. Hybrid staffing also resulted in greater job satisfaction among IDS pharmacists. This study highlights the benefits of implementing remote work practices in the pharmacy practice setting. [ABSTRACT FROM AUTHOR]

Purpose There are currently no consensus guidelines on establishing metrics for investigational drug services (IDS). Because of the complexity of research protocols, it remains difficult for sites to track pharmacy productivity and create a baseline for IDS growth within the institution, as well as to perform benchmarking with peer institutions. The goal of this study was to help establish practical guidance for IDS metrics and site utility as applicable. Methods This was a survey-based project conducted by the metrics subgroup of the Hematology/Oncology Pharmacy Association (HOPA) IDS special interest group (SIG), which was formed specifically for this analysis. Three surveys developed by the metrics subgroup were sent to members of the IDS HOPA SIG to gather metrics. The first survey included questions about what metrics IDS sites currently collect. The identified metrics were then condensed into categories. Through a consensus-based approach, standardized definitions were established and applied to future surveys. The 2 subsequent surveys sent to HOPA SIG members helped create a list of top recommended metrics that are recommended for every IDS site to track. Results A total of 3 surveys were sent to 75 recipients, with the response rate ranging from 24% to 38%. From these surveys and consensus with the metrics subgroup, 5 top recommended metrics were identified: (1) active protocols; (2) dispenses; (3) new clinical trials initiated; (4) patients treated; and (5) clinical interventions. Conclusion These recommended metrics should serve as guidance and allow for standardization to help ensure adequate resources are available for IDS pharmacy staff. These recommendations should serve as a basis for standardization and benchmarking with peer institutions. [ABSTRACT FROM AUTHOR]

Purpose Health-system pharmacists play a crucial role in monitoring the pharmaceutical pipeline to manage formularies, allocate resources, and optimize clinical programs for new therapies. This article aims to support pharmacists by providing periodic updates on new and anticipated novel drug approvals. Summary Selected drug approvals anticipated in the 12-month period covering the first quarter of 2024 through the fourth quarter of 2024 are reviewed. The analysis emphasizes drugs expected to have significant clinical and financial impact in hospitals and clinics, as selected from 59 novel drugs awaiting US Food and Drug Administration approval. This year's pipeline includes recently added drugs with various indications including oncology, infectious diseases, genetic disorders, and rare diseases. New cellular and gene therapies are rapidly evolving and being studied for several rare diseases and cancers. Conclusion More oncology agents, including gene therapies, oral agents, and monoclonal antibodies, are in the pipeline this year. Additional diseases targeted by new novel drugs, including cellular and gene therapies, are hemophilia, nonalcoholic steatohepatitis, Alzheimer's disease, and rare diseases such as galactosemia and epidermolysis bullosa. [ABSTRACT FROM AUTHOR]

Purpose Health-system pharmacists play a crucial role in monitoring the pharmaceutical pipeline to manage formularies, allocate resources, and optimize clinical programs for new therapies. This article aims to support pharmacists by sharing new and anticipated novel drug approvals. Summary Selected drug approvals anticipated in the 12-month period covering the second quarter of 2023 through the first quarter of 2024 are reviewed. The analysis emphasizes drugs expected to have significant clinical and financial impact in hospitals and clinics, as selected from 58 novel drugs awaiting Food and Drug Administration (FDA) approval at the time of data extraction. The pipeline includes drugs with various indications, such as oncology, inflammatory conditions, and rare diseases. Key developments in oncology are highlighted along with notable advancements in treating myelofibrosis, metastatic colorectal cancer, and low-grade gliomas. Cellular and gene therapies are anticipated to emerge prominently as treatment options for severe hemophilia A and sickle cell disease. Several monoclonal antibodies targeting autoimmune diseases are awaiting FDA approval. Conclusion Several new novel drugs in the pipeline are intended for use in treating cancers, autoimmune conditions, and rare diseases such as sickle cell disease. [ABSTRACT FROM AUTHOR]

Purpose Research pharmacy effort required to safely and compliantly manage investigational products (IP) varies between studies. No validated tool exists in the United States to evaluate these differences in effort. The Vizient Pharmacy Research Committee Investigational Drug Services (IDS) Subcommittee previously developed a systematic complexity scoring tool (CST) through expert consensus to assign a complexity score for pharmacy effort. This project seeks to develop and validate complexity categories based on CST scores. Methods Vizient member institutions in IDS assigned a CST complexity score and a perceived complexity category (low, medium, or high) for study initiation and maintenance. Receiver operating characteristic (ROC) curve analysis defined the best CST score cutoff points for each complexity category. Comparing the CST-assigned to the user-perceived complexity category determined whether the CST-assigned complexity category aligned with practitioner assignment. Results A total of 322 responses were used to determine complexity score categories. The AUC values for study initiation and maintenance were 0.79 (P < 0.001) for the low/medium boundary and 0.80 (P < 0.001) for the medium/high boundary, suggesting the performance of the CST is good. The agreement between CST-assigned and user-perceived complexity categories was 60% for study initiation and 58% for maintenance. The Kendall rank correlation coefficient between the raters and ROC categories was strong, with a value of 0.48 for study initiation and 0.47 for maintenance. Conclusion Development of the CST allows IDS pharmacies to objectively measure the complexity of clinical trials, which is a significant step towards assessing workload and guiding resource allocation. [ABSTRACT FROM AUTHOR]

Purpose This study is an analysis of the changes to workload and operations of UNC Health's investigational drug service (IDS) brought about by the coronavirus disease 2019 (COVID-19) pandemic. Methods Workload statistics were collected and analyzed for trend changes to illustrate operational changes necessitated by the COVID-19 pandemic within the IDS pharmacy at UNC Health. Results Multiple workload metrics declined at the beginning of the COVID-19 pandemic, followed by an increase in the metrics for many categories as the pandemic continued. Notably, monthly inventory added initially decreased by 37.5%, later leveling off but showing increased variability. Fills dispensed and monitoring visits both decreased by 34.5% from the first quarter (Q1) to Q2 of 2020. Both metrics returned to or slightly exceeded prepandemic levels by the end of the study period in March 2021. Patient enrollment decreased 76% from February to May 2020 before dramatically increasing in Q3 of 2020 and Q1 of 2021 with the initiation of COVID-19 vaccine studies. The average time to study startup increased for trials not related to COVID-19 and decreased for COVID-19–related trials. There has been no major impact on the number of open protocols throughout the course of the pandemic. Conclusion Despite initial decreases in workload following the start of the COVID-19 pandemic, IDS operations returned to and, in some cases, exceeded prepandemic levels. [ABSTRACT FROM AUTHOR]

Purpose Training represents a considerable portion of research activities and is vastly different for each clinical trial. This variation is partially explained by the lack of detailed regulations surrounding training procedures, which hinders the ability of investigational drug service (IDS) staff to plan their workload. The aim of this study was to quantify the workload associated with trial-specific training of IDS staff. The secondary aim was to identify the factors associated with training complexity. Methods A retrospective study was carried out in the IDS of a mother and child university hospital. Trial-specific documents on which the pharmacy staff was trained were analyzed. Workload was calculated by measuring reading time. The readability of each document was determined by the Flesch Reading Ease score. The complexity of the trials was established using the scoring method of Calvin-Lamas et al. The influence of the following factors on training was assessed by analysis of variance: sponsor type, research phase, and research focus by medical specialty. Results A total of 93 clinical trials and 433 documents were included. Investigator's brochures were the longest (a mean [SD] of 107 [46] pages; P < 0.0001) and most difficult documents to read (mean [SD] readability score, 25.5 [4.4]; P < 0.0001). Trials with industry sponsors required a significantly longer overall reading time (mean [SD], 12.26 [6.72] hours; P < 0.0001). On average, a mean (SD) of 9.42 (7.16) hours of reading were necessary to train one employee for a clinical trial. Conclusion This study is the first to document reading time necessary for training of IDS staff. The training workload varied by sponsor type, while the research phase and medical specialty had little impact. IDS units would benefit from a tool that could identify complex trials. [ABSTRACT FROM AUTHOR]

The authors convey their thoughts on recommendations for future pandemic preparedness from the Hematology/Oncology Pharmacy Association Investigational Drug Services Special Interest Group. Topics mentioned include the role of the pharmacist in clinical research, reimagination of clinical trial patient care, and mailing of investigational drugs to patients.

Purpose The process of providing treatment with investigational drugs through expanded access is explained. Roles and informational resources for pharmacists are discussed. Summary Expanded access is a regulatory pathway for the treatment of serious or life-threatening diseases or conditions with investigational agents outside of clinical trials. In the setting of no available therapies or ineligibility for clinical trials, a patient and their treating physician may pursue therapies that are not approved by the Food and Drug Administration (FDA). The drug manufacturer, FDA, and institutional review boards are required stakeholders in the expanded access process. Other pathways for obtaining investigational agents outside of clinical trials, including federal Right to Try and emergency use authorization, exist but differ in their level of involvement of these key stakeholders. Pharmacists are equipped to be involved in therapy identification, risk vs benefit evaluations, therapy preparation and administration, supportive care, transitions of care, and regulatory compliance. Specific websites, publications, and organizations can aid in navigating expanded access. Conclusion Combining elements of traditional clinical care and research, expanded access involves direct treatment with non–FDA-approved agents outside of a clinical trial. Healthcare providers should be aware of the possibility of providing investigational treatments after all approved options have been exhausted. [ABSTRACT FROM AUTHOR]

Purpose To report historical patterns of pharmaceutical expenditures, to identify factors that may influence future spending, and to predict growth in drug spending in 2022 in the United States, with a focus on the nonfederal hospital and clinic sectors. Methods Historical patterns were assessed by examining data on drug purchases from manufacturers using the IQVIA National Sales Perspectives database. Factors that may influence drug spending in hospitals and clinics in 2022 were reviewed—including new drug approvals, patent expirations, and potential new policies or legislation. Focused analyses were conducted for biosimilars, cancer drugs, generics, COVID-19 pandemic influence, and specialty drugs. For nonfederal hospitals, clinics, and overall (all sectors), estimates of growth of pharmaceutical expenditures in 2022 were based on a combination of quantitative analyses and expert opinion. Results In 2021, overall pharmaceutical expenditures in the US grew 7.7% compared to 2020, for a total of $576.9 billion. Utilization (a 4.8% increase), price (a 1.9% increase) and new drugs (a 1.1% increase) drove this increase. Adalimumab was the top drug in terms of overall expenditures in 2021, followed by apixaban and dulaglutide. Drug expenditures were $39.6 billion (a 8.4% increase) and $105.0 billion (a 7.7% increase) in nonfederal hospitals and in clinics, respectively. In clinics and hospitals, new products and increased utilization growth drove growth, with decreasing prices for both sectors acting as an expense restraint. Several new drugs that are likely to influence spending are expected to be approved in 2022. Specialty and cancer drugs will continue to drive expenditures along with the evolution of the COVID-19 pandemic. Conclusion For 2022, we expect overall prescription drug spending to rise by 4.0% to 6.0%, whereas in clinics and hospitals we anticipate increases of 7.0% to 9.0% and 3.0% to 5.0%, respectively, compared to 2021. These national estimates of future pharmaceutical expenditure growth may not be representative of any particular health system because of the myriad of local factors that influence actual spending. [ABSTRACT FROM AUTHOR]

The article highlights the critical role that the investigational drug services (IDS) pharmacist plays in ensuring safe medical practices for patients receiving investigational medications. Topics include current gaps in investigational drug practices, IDS work group recommendations, and IDS participation in use of interactive response technology (IRT).

Purpose Results of the 2020 ASHP national survey of pharmacy practice in hospital settings pertaining to pharmacy operational changes implemented in response to the coronavirus disease 2019 (COVID-19) pandemic are presented. Methods Pharmacy directors at 1,437 general and children's medical/surgical hospitals in the United States were surveyed using a mixed-mode method of contact by email and mail. Survey completion was online. Results The response rate was 18.7%. Seventy-three percent of hospitals implemented changes to hospital units, including 46% that increased intensive care unit bed capacity; 94% made changes to pharmacy supply chain acquisition, changes to products, and/or increased inventory. Staffing changes were implemented by 69% of hospitals, with the most common being staffing reductions (55%) and salary reductions (16%). Medication-use changes were implemented by 86% of hospitals, with treatment guidelines for COVID-19 treatment (79%) and opening compassionate use or investigational drug studies (55%) being the most common. Changes in sterile compounding processes were implemented by 84% of hospitals. Personal protective equipment (PPE) shortages led to 71% of hospitals modifying PPE use standards in sterile compounding. Eighty-seven percent of hospitals changed operational activities, such as changing medication return practices (56%), medication reconciliation processes (46%), intravenous medication recycling (38%), and discharge counseling (37%). Hospitals experienced shortages of many medications, including albuterol inhalers (60%), sedatives and anesthetic agents (58%), neuromuscular blockers (43%), corticosteroids (34%), cardiovascular agents (24%), investigational agents (24%), and dialysis solutions (6%). Conclusion The pharmacy profession responded to myriad threats to operations and patient care during the COVID-19 pandemic in 2020. [ABSTRACT FROM AUTHOR]

The article discusses operational challenges in protecting staff from occupational exposure encountered while handling investigational medicine product (IMP) shipments at two health-system research pharmacies. The IMP shipped by the clinical trial sponsor to the research pharmacy lacked labels indicating that the product shipment is dangerous, causing the staff who opened the shipment to be exposed to possible hazards. Adding hazardous risk to IMP shipping container labels is recommended.

Purpose A method to evaluate the hazardous exposure potential of investigational drugs was developed in order to comply with hazardous drug handling standards. Summary New nationwide standards require health-system pharmacies to recognize potential occupational risks and protect employees from any hazardous exposure. United States Pharmacopeia general chapter 800 (USP<800>) provides recommendations on handling precautions for commercial hazardous drugs. Recommendations for investigational drugs are less clear, with USP<800> suggesting more widespread protections when information is deemed insufficient to assess the risk. The investigational drug services pharmacy at a freestanding pediatric hospital developed a method to evaluate the hazardous potential of investigational drugs in order to determine the likelihood that a drug held an occupational handling risk. The goal of the project was to ensure compliance with hazardous drug handling standards and provide adequate employee protection while minimizing the financial burden on the health-system pharmacy. Conclusion Investigational drugs that meet any of 4 defined criteria should be subject to hazardous drug handling precautions until adequate safety data are available. [ABSTRACT FROM AUTHOR]

The article reports on a general handout that details requirements linked with the use of investigational oral chemotherapy medication. Topics mentioned include a paper released by the Hematology/Oncology Pharmacy Association in 2014 that calls for best practice standards within an investigational drug service, oral chemotherapy education sheets with information to patients with cancer and caregivers, and the increased needs and risks associated with the use of oral investigational medications.

Purpose The development, structure, and implementation of an innovative residency program designed to help meet a growing need for pharmacists with specialized expertise in investigational drug use and clinical research are described. Summary Clinical research has become an increasingly complex field, but prior to 2017 there were no U.S. specialty residency training programs focused on pharmacists' role in drug development and the care of patients enrolled in clinical trials. In 2016 Johns Hopkins Hospital (JHH) launched an initiative to develop residency training standards specific to the areas of investigational drug use and clinical research. The residency development process consisted of creation of a residency development committee; a needs assessment, including formation of a diverse panel of internal and external experts to guide identification of key competency areas and development of residency goals and objectives; design of the program's structure, including a framework for required and elective rotations; submission of an application for pre-candidate status to the ASHP Commission on Credentialing; and recruitment efforts. Conclusion The JHH investigational drugs and research residency, a combined PGY1 and PGY2 program with 5 competency areas, 14 goals, and 49 objectives, was granted pre-candidate status by ASHP in November 2016. The first resident began the program in June 2017. [ABSTRACT FROM AUTHOR]

The article focuses on the highlights of the Vax Fest 24-hour COVID-19 mass vaccination drive-through event held by Ochsner Health in New Orleans, Louisiana in March 2021. Topics discussed include the emergence of the event as the biggest COVID-19 vaccination in the state which led to the vaccination of almost 5,000 people, inclusion of live music, dancing and mascot appearances at the Vax Fest, and the willingness of residents in the area to receive vaccination.

Purpose Leveraging pharmacy technicians to take on advanced roles can enable pharmacists to practice at the highest level of their license. Technicians trained through traditional venues may not have the necessary knowledge and skills to adequately take on these roles. To meet this need, a technician residency program was developed and implemented in a health-system pharmacy. Summary The technician residency program was designed to develop competency in the medication use process as assessed through achievement of 30 objectives based on successful completion of rotations, projects, and didactic examination. The program duration was 50 weeks with rotations which included compounding, technician-check-technician cart fill and floor stock replenishment, investigational drug services, regulatory compliance, and other functions. The resident also completed several projects to improve data analytics, communication, and leadership skills. By the end of the program, the residency program director and preceptors determined that the resident achieved 83% (25/30) of objectives and had developed competency in the medication use process superior to technicians with several years of experience. The resident was ultimately hired in a specialty technician position and was found to perform effectively in that role. Conclusion The program allowed the technician resident to gain a comprehensive understanding of health-system pharmacy practice and continue to develop skills and abilities that would typically take several years of work experience to be achieved. [ABSTRACT FROM AUTHOR]

The authors convey their concerns on some of the ethical issues associated with non-inferiority trials. Topics mentioned include benchmarks by which the ethics of human research can be measured, the importance of placebo-controlled trials in some circumstances, and the null hypothesis of a non-inferiority study.

The article presents the American Society of Health-System Pharmacists (ASHP) guidelines for investigational drug product management as of April 2018. Topics discussed include their purpose, the value of and models for a clinical research pharmacy, and such areas of coverage as facilities, security, and limited staff access, temperature control and monitoring, and site qualification. Also noted is the need for standardization at study sites to improve patient safety and protect data integrity.

The article analyzes the impact of precision medicine on the U.S. health care industry. It discusses the impact of the Precision Medicine Initiative and the 21st Century Cures Act on therapeutic innovation in the future. Some of the changes in the regulation of drug approvals by the Food and Drug Administration (FDA) are discussed, as well as strategic recommendations for pharmacists and pharmacy leaders on practicing precision medicine.

In the article, the author discusses the strategies and consideration in the implementation of the U.S. Pharmacopeia chapter 800 (USP<800>) in the U.S. She focuses on the review of investigational medications for clinical trials at the Altman Clinical and Translational Institute at University of California (UC) San Diego Health using an assessment of risk (AOR). Also cited is the duty of pharmacies to classify hazardous drugs to protect staff from occupational exposure.

Purpose. The importance and key components of clinical medication reviews for participants in oncology clinical trials are described, and drug- drug interactions (DDIs) associated with new oncology drug classes are discussed. Summary. Use of investigational drugs is a mainstay of adult oncology clinical trials and has led to discovery of new oncology drug classes, including immunotherapy agents and oral targeted therapies, as well as novel chemotherapy delivery methods. As sponsor-supplied DDI information on investigational drugs and drug classes is typically limited and often inconsistent, a clinical medication review to assess the potential for DDIs is recommended for all patients enrolling in oncology clinical trials. A simplified approach to performing such reviews includes (1) evaluating the trial protocol for DDI risks, (2) meeting with the patient face-to-face to perform the review, (3) making medication-related recommendations based on the findings of the patient encounter, and (4) documenting review findings in the medical record. Pharmacists can create a personalized "concomitant-medication review guide" listing key medication-use information in table format to assist other clinicians in preventing and assessing DDIs during a patient's clinical trial participation. Conclusion. Each investigational drug and new drug class in oncology has a unique DDI profile. It is essential that patients be screened for DDI risks prior to clinical trial participation and that pharmacists and clinical investigators have clear guidelines for managing DDIs. Performing a clinical medication review is one approach to simplifying this process and ensuring patient safety. [ABSTRACT FROM AUTHOR]

Purpose. The pharmacology, clinical efficacy, and safety profiles of evolving therapies for the management of chronic heart failure (HF) and acute decompensated heart failure (ADHF) are described. Summary. HF confers a significant financial burden despite the widespread use of traditional guideline-directed medical therapies such as angiotensin-converting enzyme inhibitors, angiotensin receptor blockers, β-blockers, and aldosterone receptor antagonists, and the rates of HF-related mortality and hospitalization have remained unacceptably high. In response to a demand for novel pharmacologic agents, several therapeutic compounds have recently gained approval or are currently under review by the Food and Drug Administration. Sacubitrilvalsartan has demonstrated benefit in reducing cardiovascular mortality and HF-related hospitalizations in clinical trials, while ivabradine and ferric carboxymaltose have proven efficacious in reducing HF-related hospitalizations. Lastly, the role of serelaxin in ADHF is currently under investigation in an ongoing Phase III study. While large, outcome-driven clinical trials are fundamental in informing the clinical application of these therapeutic agents, careful patient selection is imperative to ensuring similar outcomes postmarketing. In addition, optimization of current guideline-directed medical therapy remains essential as new therapies emerge and are incorporated into guideline recommendations. Additional therapeutic agents currently undergoing investigation include bucindolol hydrochloride, cimaglermin alfa, nitroxyl, omecamtiv mecarbil, TRV027, and ularitide. Clinical practitioners should remain abreast of emerging literature so that new therapeutic entities are optimally applied and positive patient outcomes are achieved. Conclusion. Recently introduced agents for the treatment of patients with HF include sacubitril-valsartan, ivabradine, and ferric carboxymaltose. Additional agents worthy of attention include serelaxin and other therapies currently under investigation. [ABSTRACT FROM AUTHOR]

Purpose. The pharmacology, pharmacokinetics, clinical efficacy, and safety of Viekira, as well as its place in hepatitis C virus (HCV) therapy, are reviewed. Summary. Ombitasvir 25 mg-paritaprevir 150 mg-ritonavir 100 mg plus dasabuvir 250 mg (Viekira) is approved in the United States as a combination direct-acting antiviral agent for treatment-naive or treatment-experienced patients with HCV genotype 1 infection, including those with compensated cirrhosis. It is the first coformulated direct-acting antiviral that targets different stages of the virus's life cycle. Viekira is administered as an oral, interferon-free regimen. Phase III clinical trials demonstrated that Viekira administered with or without ribavirin can achieve sustained virological response rates of e90%. These results are notable because they show that high virological cure rates can be achieved without peginterferon and ribavirin. Viekira is also effective for special patient populations, such as individuals coinfected with HIV, liver transplant recipients, and those with advanced renal disease. The most frequently reported adverse effects among patients associated with Viekira without ribavirin were nausea, pruritus, and insomnia. During clinical trials, the most common adverse effects among patients receiving Viekira with ribavirin were fatigue, nausea, pruritus, insomnia, and weakness. Conclusion. Viekira, the first coformulated direct-acting antiviral that targets different stages of the HCV life cycle, is an interferon-free treatment for HCV genotype 1 infection. It is associated with a virological cure rate of e90% and treatment durations of 12 and 24 weeks. Viekira is also effective and safe for patients who have undergone liver transplantation, are coinfected with HIV, or have advanced kidney disease. [ABSTRACT FROM AUTHOR]

Purpose. Emerging treatment options for the management of chronic hyperkalemia in the outpatient setting are reviewed. Summary. Current treatment options for the management of hyperkalemia are limited and often accompanied by serious adverse effects. Two investigational drugs for the treatment of hyperkalemia are being evaluated in Phase III trials: sodium zirconium cyclosilicate and patiromer. Both of these drugs are administered orally and act by enhancing potassium's removal, predominantly through the gastrointestinal tract. The safety and efficacy of sodium zirconium cyclosilicate and patiromer were evaluated in Phase II and III trials. Both agents were studied in patients with chronic mild-to-severe hyperkalemia, chronic kidney disease (CKD), or heart failure as well as those taking a renin-angiotensin system (RAS) inhibitor, an aldosterone antagonist, or both therapies. These clinical trials found that sodium zirconium cyclosilicate and patiromer normalized serum potassium levels quickly and maintained normalized serum potassium levels over several weeks. Both medications caused a rapid decrease in serum potassium, with two studies examining efficacy endpoints for 12 weeks or longer. The overall frequency of adverse effects in these clinical trials was low, with gastrointestinal adverse events being the most commonly observed. Conclusion. Options for the management of hyperkalemia, particularly chronic hyperkalemia in the outpatient setting, are limited. Both sodium zirconium cyclosilicate and patiromer are emerging therapies that may provide long-term management of hyperkalemia, particularly in patients with underlying heart failure or CKD as well as those taking an RAS inhibitor, an aldosterone antagonist, or both. [ABSTRACT FROM AUTHOR]

Purpose. The chemistry, pharmacokinetic and pharmacodynamic properties, efficacy, and safety of the recently introduced combination antimicrobial agent ceftolozane-tazobactam are reviewed. Summary. Ceftolozane-tazobactam (Zerbaxa, Cubist Pharmaceuticals) is a cephalosporin β-lactam and β-lactamase inhibitor marketed as a fixed-dose combination agent for the treatment of complicated urinary tract and intraabdominal infections. Its dosing and chemistry provide expansive antimicrobial coverage of gram-negative organisms, including Pseudomonas aeruginosa, and stable activity against many β-lactamases, as well as coverage of most extended-spectrum β-lactamase-producing organisms and some anaerobes. Ceftolozane-tazobactam is susceptible to hydrolysis by carbapenemase enzymes but is not affected by other resistance mechanisms such as efflux pumps and porin loss. Clinical trials demonstrated that combination treatment with ceftolozane-tazobactam plus metronidazole had efficacy comparable to that of levofloxacin in patients with complicated urinary tract infections, including pyelonephritis, and comparable to that of meropenem against complicated intraabdominal infections. A Phase III trial of ceftolozane-tazobactam versus meropenem for treatment of bacterial pneumonia, including ventilator-associated pneumonia, is underway. Adverse effects reported with ceftolozane-tazobactam use are comparable to those seen with other β-lactams (e.g., hypersensitivity, nausea, diarrhea, headache). Initially, ceftolozane-tazobactam may be reserved for targeted therapy against multidrug-resistant pathogens. Conclusion. Ceftolozane-tazobactam is a new cephalosporin with enhanced activity against multidrug-resistant P. aeruginosa and other gram-negative pathogens. [ABSTRACT FROM AUTHOR]

Purpose. Results of a survey assessing trends and innovations in the use of pharmacy technicians and other nonpharmacist staff in the research pharmacy setting are reported. Methods. A Web-based survey was distributed to Internet communities of members of the American Society of Health-System Pharmacists and the University Health- System Consortium involved in investigational drug research and related practice areas. The survey collected data on the characteristics of institutions with pharmacy department staff dedicated to such research activities and the participation of pharmacists, technicians, and other staff in key areas of research pharmacy operations. Results. Survey responses from 51 institutions were included in the data analysis. Overall, the reported distribution of assigned responsibility for most evaluated research pharmacy tasks reflected traditional divisions of pharmacist and technician duties, with technicians performing tasks subject to a pharmacist check or pharmacists completing tasks alone. However, some institutions reported allowing technicians to perform a number of key tasks without direct pharmacist supervision, primarily in the areas of inventory management and sponsor monitoring and auditing; almost half of the surveyed institutions reported technician involvement in teaching activities. In general, the reported use of “tech-check-tech” arrangements in research pharmacies was very limited. Some responding institutions reported the innovative use of nonpharmacist staff (e.g., paid interns, students and residents on rotation). Conclusion. Although the majority of research pharmacy tasks related to direct patient care are performed by or under the direct supervision of pharmacists, a variety of other essential tasks are typically assigned to pharmacy technicians and other nonpharmacist staff. [ABSTRACT FROM AUTHOR]

A correction to the article "Establishing an Investigational Drugs and Research Residency at an Academic Medical Center" by Molly Wascher et al, published in the November 15, 2019 issue.

Purpose. An analysis of trends in U.S. pharmaceutical spending is presented, including projections for drug expenditures in nonfederal hospital and clinic settings in 2014. Methods. Trends in pharmaceutical expenditures and developments likely to influence future spending, including new drug approvals and patent expirations, were analyzed using data from the IMS Health National Sales Perspectives database. Projections were based on a combination of quantitative and qualitative analyses and expert opinion. Results. Total prescription sales for the 12 months ending September 2013 were approximately $326 billion, 0.7% lower than sales during the previous 12 months; pharmaceutical spending by clinics and nonfederal hospitals grew by 4.5% and 1.8%, respectively. Vaccines were among the products driving large sales increases in clinic settings, with alteplase and pegfilgrastim topping the list of fast-growing drugs by hospital expenditures. Few new drug approvals anticipated in 2014 are expected to result in major expenditures by hospitals and clinics. Expansion of access to health care and other changes related to the Patient Protection and Affordable Care Act, as well as continued improvement in the U.S. economy, may drive growth in pharmaceutical spending over the next 12-24 months. Conclusion. Growth in U.S. prescription drug expenditures is expected to rebound in 2014, with a projected 3-5% increase in total drug expenditures across all settings this year, including a 5-7% increase in clinic spending and a 1-3% increase in hospital spending. Health-system pharmacy leaders should carefully examine local drug-utilization patterns to determine their respective organization's anticipated spending in 2014. [ABSTRACT FROM AUTHOR]

The article reviews the Viltepso viltolarsen antisense oligonucleotide for treating Duchenne muscular dystrophy (DMD) from NS Pharma Inc.

Purpose. Factors likely to influence drug expenditures, drug expenditure trends in 2012, and projected drug expenditures for U.S. nonfederal hospitals and clinics in 2013 are discussed. Methods. Prescription drug expenditure data for 2011 through September 2012 were obtained from the IMS Health National Sales Perspectives database. Expenditure projections were based on a combination of quantitative and qualitative analyses, combined with expert opinion. Results. Total prescription sales in the United States for the 12-month period ending September 2012 were $326.0 billion, a 0.8% increase from the previous 12 months. This rate of growth was the lowest in recent history and can be attributed to modest increases in expenditures for new products (3.3%) and the prices of existing products (5.9%), coupled with a marked decline in overall volume. and mix (-8.4%). For the 9 months ending in September 2012, total prescription expenditures grew 2.7% when compared with the same period in 2011. Oncology products remained important expenditures for hospitals and clinics. Antineoplastic agents were the top medication class for expenditures in nonfederal hospitals, and oncology products accounted for 32.2% of drug expenditures in the clinic setting in the first 9 months of 2012. Conclusion. For 2013, we project a 1-3% increase in total drug expenditures across all settings, a 2-4% increase in expenditures for clinic-administered drugs, and a 0.5% decline to 1.5% increase in hospital drug expenditures. Health-system pharmacy leaders should carefully examine their own local drug-utilization patterns to determine their own organization's drug expenditure forecast. [ABSTRACT FROM AUTHOR]

Purpose. The published evidence on the pharmacology, pharmacodynamics, pharmacokinetics, safety, and efficacy of a promising investigational agent for managing type 2 diabetes is evaluated. Summary. Canagliflozin belongs to a class of agents-the sodium-glucose cotransporter 2 (SGLT2) inhibitors-whose novel mechanism of action offers potential advantages over other antihyperglycemic agents, including a relatively low hypoglycemia risk and weight loss-promoting effects. Canagliflozin has dose-dependent pharmacokinetics, and research in laboratory animals demonstrated high oral bioavailability (85%) and rapid effects in lowering glycosylated hemoglobin (HbA1c) values. In four early-stage clinical trials involving a total of over 500 patients, the use of canagliflozin for varying periods was associated with significant mean reductions in HbA1c (absolute reductions of 0.45-0.92%) and fasting plasma glucose (decreases ranged from 16.2% to 42.4%) and weight loss ranging from 0.7 to 3.5 kg. More than a dozen Phase II or III clinical trials of canagliflozin in adults are ongoing or were recently completed, but the final results of most of those studies have not been published. Adverse effects reported in clinical trials of canagliflozin include urinary tract and genital infections, occurring in about 10% of patients. Additional and larger Phase III clinical trials to delineate the potential role of canagliflozin and other SGLT2 inhibitors in the management of diabetes (including studies involving the elderly, children, and patients with renal or hepatic dysfunction) are planned or currently underway. Conclusion. Canagliflozin and other investigational SGLT2 inhibitors have a novel mechanism of action that may offer a future alternative treatment pathway for managing type 2 diabetes. [ABSTRACT FROM AUTHOR]

Purpose. The pharmacology, pharmacokinetics, efficacy, and safety of apixaban are reviewed. Summary. Apixaban is an oral, direct, selective factor Xa inhibitor with a rapid onset of action. It has a plasma elimination half-life of 12 hours and has been administered in a twice-daily dosing regimen in clinical trials without the need for anticoagulation monitoring or dosage adjustment. Apixaban has multiple elimination pathways, and its pharmacokinetics is not substantially altered by patient age, sex, race, or ethnicity. The results of three Phase III trials indicated that apixaban was similar to or more effective than enoxaparin for preventing venous thromboembolism (VTE) in patients undergoing total hip or knee replacement, with similar or lower rates of bleeding. Two Phase III trials found that apixaban was more effective for stroke prevention than either aspirin or warfarin in patients with atrial fibrillation (AF), with a similar (versus aspirin) or improved (versus warfarin) safety profile. A Phase III trial evaluating apixaban plus antiplatelet monotherapy or dual-antiplatelet therapy in patients with acute coronary syndrome ended early due to clear evidence of a clinically important increase in bleeding among patients randomized to apixaban without any meaningful reduction in ischemic events. The adverse-event profiles for apixaban and comparators have been similar in studies conducted to date. Conclusion. Apixaban, a new anticoagulant, appears to offer an efficacy and safety profile comparable with that of enoxaparin for preventing VTE after orthopedic surgery, with the advantage of oral administration. In patients with AF, apixaban is more effective than either warfarin or aspirin for stroke prevention, with an acceptable safety profile. [ABSTRACT FROM AUTHOR]

The article describes a set of guidelines developed by pharmacists of the investigational drug service (IDS) established by the University of Michigan Hospitals and Health Centers. The guidelines are aimed at helping pharmacists prepare instructions for dispensing, inventory management, and accountability of investigational drugs in a systematic manner. Information on the dispensing guideline template, its structure and its use in training pharmacists before the start of a study is included.

The article discusses the implications of the COVID-19 pandemic on the conduct of clinical trials and on the management of investigational products (IP) to be used in clinical settings. Topics mentioned include the termination of in-office visits to ensure patient safety, the responsibility of trial sponsors concerning the use of self-administered IP, and the importance of temperature control during IP shipments.

The article discusses the ongoing investigational drug therapy in the area of pharmacy practice in response to U.S. cases of Ebola virus disease (EVD). Topics discussed include the use of brincidofovir, an investigational oral nucleotide, during emergency for treating EVD patients, the need for coordination among pharmacy and medical staff for the acquisition and management of investigational therapy and the need for meticulous attention to details such as the disposal of medication containers.

The article reports on a federal right-to-try law that aims to broaden access to investigational drugs. Topics mentioned include amendment of the Federal Food, Drug, and Cosmetic Act, thoughts from clinical research pharmacist Marjorie Phillips about an obstacle to the success of the right-to-try law, and U.S. Food and Drug Administration's individual patient expanded-access investigational new drug application process.

This section offers news briefs on the U.S. pharmaceutical and healthcare industry as of February 15, 2018. Topics covered include U.S. Food and Drug Administration's approval of the marketing of 46 drugs and therapeutic biological products in 2017, the 2017 Fred M. Eckel Pharmacy Leadership Award given to Stephen M. Caiola, and the 2017 Clifton J. Latiolais Award given to Karen L. Kier.

The article offers information about the tisagenlecluecel which is a chimeric antigen receptor (CAR) T-cell immunotherapy approved by the U.S. Food and Drug Administration (FDA), and marketed as Kymriah by pharmaceutical company Novartis.

The article discusses the U.S. Food and Drug Administration (FDA) draft guidance entitled "Drug and Device Manufacturer Communications with Payors, Formulary Committees and Similar Entities-Questions and Answers." Topics include healthcare economic information on DFA-approved products that can help payors in planning future coverage budgets and reimbursements, the Pharmacy and therapeutics (P&T) committees, and cost-effectiveness models.

The article presents information on several new drugs and dosage forms including the Calaspargase pegol-mknl injection from Servier Pharmaceuticals, Diclofenac epolamine topical system from IBSA Pharma, and Levitiracetam extended-release tablets from Sun Pharmaceutical Industries.

The article reports on the importance of language and cultural competence of performance standards in providing care for patients within the lesbian, gay, bisexual, and transgender (LGBT) community. Topics include healthcare disparities that affect LGBT patients, the need to include LGBT topics in pharmacy education, and healthcare providers' lack of knowledge on how to be welcoming to their LGBT patients.

The article discusses an integrated investigational drug service (IDS) model for the preparation of inpatient, outpatient, investigational and commercial oncology doses. It mentions the importance of an integrated IDS model within an oncology service line to ensure the dispensation of the correct therapies at the correct time, factors considered for model implementation including content experts, access to protocol information and standard practices, and the operational structure of the model.