Drug Formulary Decisions Research Articles

Handling of New Drug Safety Information in the Dutch Hospital Setting: A Mixed Methods Approach.

IntroductionThe implementation of new drug safety information and Direct Healthcare Professional Communications (DHPCs) in hospitals is important for patient safety.ObjectivesThe aim of this study was to gain insight into which procedures and practices are in place to handle new drug safety information and particularly DHPCs in the Dutch hospital setting.MethodsWe first conducted focus groups including medical specialists and hospital pharmacists, focusing on handling of drug safety information at the individual and organisational level. A survey was then developed and distributed among hospital pharmacists in all Dutch hospitals to quantify the existence of specific procedures and committees to handle drug safety information and DHPCs.ResultsEleven specialists and 14 pharmacists from six hospitals participated in focus groups. Drug safety information was usually considered before drugs were included in formularies or treatment protocols. Furthermore, drug safety information was consulted in response to patients experiencing adverse events. DHPCs were mostly dealt with by individual professionals. DHPCs could lead to actions but this was very uncommon. Completed surveys were received from 40 (53%) of the hospitals. In 32 (80%), the hospital pharmacy had procedures to deal with new drug safety information, whereas in 11 (28%) a hospital-wide procedure was in place. Drug safety was considered in committees concerning drug formulary decisions (69%) and antibiotic policies (63%). DHPCs were assessed by a hospital pharmacist in 50% of the hospitals.ConclusionsDrug safety information was used for evaluation of new treatments and in response to adverse events. Assessment of whether a DHPC requires action was primarily an individual task.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40264-022-01149-4.

PCN82 - Assessment of Criteria for Formulary Decision-Making of Cancer Drugs in the Public Sector in Malaysia – A Pilot

Multicriteria decision analysis (MCDA) approach is being pursued to improve and support formulary decisions for cancer drugs in the public sector in Malaysia. Decision criteria are the key component in the MCDA framework. Prior to a nationwide survey to determine healthcare professionals’ decision criteria preference, this preliminary survey was conducted with the primary aim to identify important criteria for formulary decisions relevant to local context. Secondary objective was to determine if doctors had different criteria preference for selection of cancer drugs versus other drugs. Drug formulary decision criteria identified through literature review were integrated into a survey and administered online using Google Forms. Snowballing technique was used to select respondents who were pharmacists and doctors working in the Ministry of Health. Respondents assessed the relevance of each criterion listed by answering “yes” or “no”. An open-ended question asking for additional criterion was included. The questionnaire for doctors explored if there would be a difference in the criteria selected if the formulary decision was for a cancer drug. Descriptive statistical analysis was performed using Microsoft Excel 2013, v15.0. A total of 21 responses from 10 pharmacist and 11 doctors were received. Approximately 40% of the respondents are members of formulary decision committees. 82% of the doctors are non-oncology specialist. From the list of 14 criteria, effectiveness, safety, cost effectiveness and budget impact received more than 80% agreement from both groups. Respondents recommended three additional criteria: direct cost, ease of adherence and other countries experience. Two non-oncologist reported the use of different criteria for cancer drug selection. This study highlights how views on the type and relevance of formulary decision criteria can vary according to the health system and stakeholder group. Thus its important to localize criteria to ensure a multicriteria approach relevant for local decision making can be implemented.

Assessment of the Level of Satisfaction and Unmet Data Needs for Specialty Drug Formulary Decisions in the United States.

Formulary management within a limited budget is critical, especially for specialty drugs, which are used for serious medical conditions and are very expensive. Despite attempts to summarize the pertinent evidence, it is uncertain whether data needs of formulary decision makers for specialty drugs are satisfied. To assess the level of satisfaction of specialty drug formulary decision makers with regards to the strength of current available data sources and unmet needs regarding clinical, economic, and unpublished evidence. This study targeted pharmacists and physicians involved with formulary decision making at health plans or pharmacy benefit management companies at the national, large regional, and local levels. 95 individuals were invited to participate (without compensation) in a 21-item, web-based survey (Qualtrics), which was open from June 14 to July 31, 2014. The responses were coded for descriptive and statistical analysis. Statistical analyses included the Kruskal-Wallis test, analysis of variance, and the Mann-Whitney-Wilcoxon test. Of 95 pharmacists or physicians, 40 respondents initiated the survey, and 33 respondents completed the survey (response rate = 34.7%). Drug formulary decision makers infrequently rated data evidence strength (17.1% "always"). Clinical data evidence strength was rated highest with published randomized controlled trials (RCTs; mean [SD] = 4.06 [0.87] of 5.0), while participant organizations' internal data were rated highest for economic data evidence strength (mean [SD] = 3.91 [1.07] of 5.0). Decision makers rated the highest unmet need as more data generated from head-to-head RCTs (mean [SD] = 2.94 [0.25] of 3.0) and cost-effectiveness analyses (mean [SD] = 2.53 [0.67] of 3.0). The participants believed manufacturers might be in the best position to satisfy their desire for head-to-head RCTs (mean [SD] = 4.31 [1.09] of 5.0). Despite a variety of data sources, drug formulary decision makers continue to rely on published RCTs or internal economic analyses as having the strongest evidence strength. The study respondents believed that pharmaceutical manufacturers would be best able to satisfy the greatest clinical data unmet need, that is, head-to-head RCTs in specialty drug formulary decisions. This study was not funded by any company or pharmaceutical manufacturer. Navarro has worked as a consultant for Biogen, Purdue Pharma, and Novartis and has offered expert testimony on behalf of AstraZeneca. The authors declare no other potential conflicts of interest. Study design was contributed primarily by Navarro, along with Choi. Choi took the lead in data collection and interpretation, assisted by Navarro. Both authors contributed equally to manuscript writing and revision.

A Prescription for Improving Drug Formulary Decision Making

Gordon Schiff and colleagues present a new tool and checklist to help formularies make decisions about drug inclusion and to guide rational drug use.

Cost Shifting and Timeliness of Drug Formulary Decisions in Atlantic Canada

Our objectives were to investigate the timeliness of formulary decision-making in Atlantic Canada, including the Common Drug Review (CDR) process and the adoption of positive CDR recommendations by Atlantic Canadian provincial public drug plans, and to determine the degree of cost shifting to private payers. Dates of formulary listing decisions from Atlantic Canadian provincial drug plan formularies and utilization analyses from Medavie Blue Cross were used to calculate the timeliness of decisions and cost shifting from public payers to a private payer. The median time period between the issuance of a positive CDR recommendation and the addition of a drug to an Atlantic Canadian provincial drug plan was 26.7 weeks (σ=19.1). Cost shifting to employer-sponsored health plans provided by Medavie Blue Cross was minimal. There is significant variation in the timing of provincial drug formulary listings among the four Atlantic Canadian provinces and the uptake of CDR recommendations. Atlantic Canadian provincial governments should support the mandate of the CDR by aiming for a more timely consideration of recommendations.

The Limits of Cost-Effectiveness Analysis

The competitive worldwide economic environment and ever-increasing costs of health care have created a setting in which understanding costs and making sure that we achieve good value in health care are paramount. One approach to seeking value is through the use of cost-effectiveness analysis. Although this science is now several decades old, it has been refined over the last several years, with increasingly sophisticated statistical and standardized methods.1,2 Is cost-effectiveness analysis useful? Does it help in medical decision making and in allocation of scarce resources? In the accompanying article, “Cost, Effectiveness, and Cost-Effectiveness” Diamond and Kaul3 argue that cost-effectiveness analysis is not a useful approach. Although we agree with many of the points that Diamond and Kaul raise, we do not agree with their conclusion. Cost-effectiveness analysis involves an assessment of both cost and effectiveness. The distribution of each needs to be understood. A cost-effectiveness analysis is only as valid as its underlying measures of effectiveness and cost, a discussion that is beyond the scope of this article. However, the methods to make these assessments vary considerably. There are standards for cost-effectiveness, but at times, perfectly adhering to these standards is not realistic, and compromises are often made that may be entirely scientifically legitimate.4 Cost-effectiveness is, by nature, incremental. Thus, it is necessary to look at the added costs compared with a control group. Selection of the appropriate control group is a challenge itself. At times, the appropriate control is placebo, and at other times, it is active therapy; the appropriate control is dependent on the clinical question being asked. However, when cost-effectiveness analysis is conducted using data from a clinical trial, the selection of the control group will not be a decision that the analyst can affect (at least after the trial has been completed). When …

Emerging Lessons From The Drug Effectiveness Review Project

The Drug Effectiveness Review Project (DERP) is an alliance of fifteen states and two private organizations, which have pooled resources to synthesize and judge clinical evidence for drug-class reviews. The experience shines a bright light on challenges involved in implementing an evidence-based medicine process to inform drug formulary decisions: When should evidence reviewers accept surrogate markers and assume therapeutic class effects? How open and participatory should review procedures be? Should reviewers consider cost-effectiveness information? What is the appropriate role of the public sector in judging evidence? The DERP illustrates that attempts to undertake evidence-based reviews, apart from the methods themselves, which continue to evolve, involve questions of organization, process, and leadership.

The Arrival of Economic Evidence in Managed Care Formulary Decisions

Managed care plans have traditionally resisted using economic evidence explicitly in drug formulary decisions, even as they used ever more aggressive and sophisticated processes for managing care. In recent years, this has changed as health plans have begun to adopt evidence-based and value-based formulary submission guidelines. The guidelines have the potential to serve as a national unifying template for pharmacy and therapeutics committees to consider clinical and economic information in a systematic and rigorous fashion. However, many questions remain about their use and about the nature of communications (called "unsolicited requests") from plans to drug companies for information. This article describes the unsolicited request process and its potential impact on the use of economic evidence in formulary decisions.

Clinical monograph for drug formulary review: systemic agents for psoriasis/psoriatic arthritis.

Significant advances in the pharmacologic treatment of psoriasis, most notably the introduction of the biologic agents efalizumab and alefacept, have occurred recently. In addition, another biologic agent, etanercept, was recently approved for the treatment of psoriasis and psoriatic arthritis, thus adding to the list of biologic agents approved for the treatment of these disease states. A review was conducted by the Drug Information Service of a pharmacy benefits manager (PBM) to determine the relative merits and place in therapy of commonly used systemic agents for the treatment of psoriasis and psoriatic arthritis. To provide readers with a comprehensive clinical monograph on psoriasis and psoriatic arthritis agents, written with a managed care perspective, as used in actual drug formulary decision making by a PBM. The drug formulary of this PBM is designed to provide health plans with an evidence-based review of drugs, therapeutic classes, and disease states with a managed care focus. For each therapeutic class or disease review, an extensive and thorough literature search of MEDLINE is conducted for efficacy, safety, effectiveness, and humanistic and economic data. Drug/disease-state databases (UpToDate online, MICROMEDEX), U.S. Food and Drug Administration clinical reviews, key Internet sites, medical/pharmacy-related news sites, clinical guidelines, and AMCP dossiers are also reviewed. Formulary drug monographs produced by the Drug Information Service of the PBM include a critical analysis and summary of disease-oriented and patient-oriented clinical outcomes, effectiveness, and humanistic data. Additional data considered and included in the formulary review process are clinical attributes, patent expirations/generic competition, off-label or pending indications, and pharmacoeconomic data. The biologic agents do not appear to be as efficacious as traditional systemic therapies but are associated with fewer long-term toxicities that often limit treatment duration with traditional systemic agents. Although no head-to-head comparisons between alefacept and efalizumab exist, efalizumab appears to offer slightly higher efficacy rates, while alefacept has a longer duration of action. Etanercept at the higher approved dose appears more efficacious compared with efalizumab or alefacept for the treatment of psoriasis, and it is the only biologic currently approved for the treatment of psoriatic arthritis. Efalizumab and alefacept are generally well tolerated, but rebound flare of psoriasis is associated with efalizumab, thus requiring continuous treatment to avoid a flare in disease. Efalizumab and etanercept can be self-administered by the patient, while alefacept and infliximab require administration by a health care professional. Systemic therapy is reserved for patients with moderate-to-severe psoriasis or patients with psoriatic arthritis. The biologic agents are not as efficacious as traditional therapies but, due to better tolerability, are gaining acceptance in the treatment of psoriasis and psoriatic arthritis. The biologic agents differ in efficacy rates and are generally well tolerated. Clinical attributes, overall efficacy, and economic costs associated with the biologic agents will be significant factors in selecting agents for the treatment of psoriasis and psoriatic arthritis.

Health plan budget impact analysis for pimecrolimus.

Budget impact models are useful tools for managed care organizations to make drug formulary decisions. The objective of this study was to estimate the incremental budgetary change in per-member-per-month (PMPM) medical and pharmacy costs for atopic dermatitis (AD) or eczema after the introduction of pimecrolimus cream 1%, a topical calcineurin inhibitor. Estimates of the percentage of patients seeking care, treatment patterns, and quantities of medications dispensed for AD were measured using 2001 and 2002 medical and pharmacy records in a proprietary database for health plans distributed throughout the United States. Approximately 2.5 million health plan members had continuous health insurance coverage during the study period. Costs for medications were assigned using the 2003 wholesale acquisition cost, and costs for physician visits were based on average 2003 Medicare reimbursement rates. Efficacy data from clinical trials were used to model the impact of pimecrolimus on subsequent physician visits. Sensitivity analyses were performed to evaluate the impact of varying the percentage of patients seeking care, practice patterns, medication quantities, percentage of pimecrolimus users, and levels of patient cost sharing. The estimated percentage of health plan members seeking care for AD in 2001 was 3.2%. The estimated total cost PMPM for AD treatment prior to introduction of pimecrolimus was 0.362 dollars for all covered lives, assuming no patient cost sharing. In the year after its introduction, 5.2% of the AD population filled a prescription for pimecrolimus. The incremental increase in pharmacy benefit cost was 0.008 dollars PMPM in 2003 dollars, but the total incremental medical and pharmacy cost was 0.002 dollars PMPM after accounting for the projected reduction in physician visit costs, representing a 0.7% increase in all AD-related costs. Based on sensitivity analyses, the incremental total cost PMPM after the introduction of pimecrolimus ranged from -0.004 dollars to 0.026 dollars. Using claims data for the medical treatment of AD in 2001-2002 and the utilization of pimecrolimus, the addition of pimecrolimus as a treatment option for AD had a minimal impact on PMPM costs for AD-related care in 2003 dollars. As with all pharmacoeconomic models, health plans should perform their own budget forecasting using assumptions derived from their own pharmacy and medical claims data.

Funding the new biologics--public policy issues in drug formulary decision making.

One function of drug formularies is to allow health care providers to exert some control over spending. Decisions about whether to include a given medication in a formulary are based on estimates of its costs and effectiveness, relative to other treatment strategies. These decisions are made from a societal perspective, as opposed to that of individual patients, which sometimes results in conflicts. The clinical response to a medication often varies widely among subjects, which means that a small subgroup of patients might benefit dramatically, while others with the same disease do not. The result would be that a drug might appear not to be cost effective in an economic analysis, even though it is of proven value for some patients. New and innovative medications are assessed according to high standards of cost effectiveness, even though established treatments are wasteful of valuable health care resources. Moreover, quality-adjusted life-years (QALYs) discriminate against certain patient groups, including those with diseases that are associated with a high morbidity but a low mortality. Such patients often incur high indirect costs, including loss of employment income and costs incurred by family caregivers that QALYs do not reflect. Therefore, even though QALYs are transparent and widely applicable, they are not necessarily appropriate in the evaluation of a particular therapeutic intervention. A new paradigm should be developed for evaluating emerging therapies. An example would be a risk-sharing approach, whereby the pharmaceutical industry and public insurers share in the costs and rewards of introducing new treatments. This would have implications for the prices charged for new medications.

Reassessing the relevance of pharmacoeconomic analyses in formulary decisions.

In 1992, Lipsey[1] provided a review of institutional drug formularies and the relevance of pharmacoeconomic analyses in drug formulary decisions. The past 5 or 6 years have seen considerable work in the development of guidelines for pharmacoeconomic evaluations, and growth in the use and applications of pharmacoeconomic evaluations. Such growth has given cause for a reassessment of the relevance of pharmacoeconomic analyses in formulary decision-making. We believe that pharmacoeconomic analyses are as necessary now as in 1992 when Lipsey[1] provided his review. However, we also believe that pharmacoeconomic analyses are not the ‘be-all and end-all’ in the formulary decision-making process. Evidence on the safety, efficacy and, ultimately, the effectiveness of drugs is always important; these 3 factors are important prerequisites for formulary inclusion. The relevance of any one particular pharmacoeconomic evaluation will depend on the context of the formulary decision process, and the data available at the time of the decision. In this regard, it is important to consider a number of factors including the goals decision-makers are faced with. Depending on the incentive structure of the pharmacy and therapeutics (P&T) and formulary committees at different levels (e.g. institutional, regional, national), the decision-making goals may differ and therefore, the required information and the perspective of the evaluation may differ. Another important determinant of the relevance of pharmacoeconomic evaluations will be the degree of accountability for claims made within a pharmacoeconomic evaluation. The purpose of this article is to review some of the above issues as they relate to, and impact on, the use of pharmacoeconomic analyses in formulary decisions. We discuss some points initially raised by Lipsey,[1] and add to the discussion important implications of the role of pharmacoeconomic analyses as seen in recent literature and experiences in the formulary decision-making process. Through these points, we will attempt to show that, while economic evaluations of pharmaceuticals will continue to be relevant in this process, there are several important elements that will determine to what extent pharmacoeconomic information may impact the final decision.

Pharmacy benefit management, cost-effectiveness analysis and drug formulary decisions

Pharmacy benefit management companies (PBMs) have evolved over the past decade in response to the increased demand for health care cost containment. Their activities include the implementation of drug formularies and the negotiation of rebates from manufacturers. Our analysis of this industry is based on interviews and materials provided by the top five ranked PBM companies which account for over 80% of beneficiaries covered within formulary plans. The formularies of these companies are relatively inclusive, but they are becoming more restrictive over time. At present the use of cost-effectiveness (C-E) studies in the formulary decisions of PBMs has been limited. In this regard, the surveyed PBMs emphasized that most C-E studies have not compared therapeutic substitutes in populations with characteristics that are similar to those of their clients. Pharmacy benefit management companies also have had strong incentives to focus narrowly on drug costs because they typically manage drug benefits on a “carved-out” basis. However, PBMs anticipate a growing future role in the integrated management of patient care (disease management) for certain high cost chronic diseases and conditions. All of the leading firms we surveyed have disease management programs in development. The importance of C-E studies to PBM decisions is expected to increase significantly as disease management programs are implemented. The data infrastructure inherent to the PBM industry and the increasing number of employees with advanced training in pharmacoeconomics will permit firms to perform their own internal C-E studies. They are also establishing various alliances and joint ventures with drug manufacturers, health maintenance organizations, and academic institutions to perform these analyses. The leading PBMs tend to favor active participation in the development of methodological approaches to C-E studies over government regulations such as those proposed by the FDA in 1995.