Pharmacy Committee Research Articles

National Survey of Pharmacy and Therapeutic Committees in Saudi Arabia: Scope, Structure and Responsibilities

National Survey of Pharmacy and Therapeutic Committees in Saudi Arabia: Scope, Structure and Responsibilities

Medicine-use evaluation of doxazosin prescribing to inform formulary recommendations.

It is estimated that >50% of all medicines are inappropriately prescribed, dispensed or sold. In 2018, the Western Cape Government Health (WCGH) Provincial Pharmacy and Therapeutics Committee (PPTC) endorsed an investigation of doxazosin use in provincial public sector healthcare facilities using a medicine-use evaluation (MUE). This was based on potential inappropriate prescribing, high usage and high costs associated with doxazosin. To determine whether doxazosin was being prescribed in accordance with recommendations from standard treatment guidelines and essential medicines lists for hospital and primary healthcare level, as well as from the WCGH provincial code list. The study design consisted of a retrospective multicentre prescription review. The study population included all adult outpatients who were prescribed doxazosin, which was to be dispensed by the WCGH Chronic Dispensing Unit. Criteria for evaluation included indications for doxazosin administration and total daily dose prescribed. The study sample comprised 171 patients. The main indication for doxazosin administration was as a fourth-line agent for the treatment of hypertension (82%). The remaining indications were not guideline approved (18%). The total daily doses identified were 4 mg (62%), followed by 8 mg (36%) and 2 mg (2%). All of these were approved dosages as per guideline recommendations. MUEs are an untapped resource for medication-use monitoring and practice improvement. MUEs offer a distinct, cost-effective approach to improve medicine use in all settings. This MUE revealed a relatively minor concern with doxazosin prescribing. MUEs can be beneficial to inform formulary changes and re-evaluate medication-use restrictions.

PSY3 DISEASE BURDEN OF RHEUMATOID ARTHRITIS IN CHINA

Rheumatoid arthritis (RA) is a heterogeneous, systemic and autoimmune disease with symmetric polyarthritis. Patients have a higher incidence of disability and functional limitations with a longer course. RA not only causes a decline in patients' physical function, quality of life and social participation, but also imposes a huge economic burden on patients' families and society. This study was to explore the disease burden of RA in China. The disease burden model was built in Microsoft Excel 2016 and the economic burden of disease was categorized into direct medical, direct non-medical, and indirect costs. Treatment pattern, quality of life, epidemiological and cost data were extracted from a systematic review of literature and public databases, real world data from Hospital Pharmacy Committee of Chinese Pharmaceutical Association were also included in analyses. Data synthesized were examined and the data-gaps were filled by in-depth interviews with clinical and health economic experts, and by advisory board meetings (Delphi Panel). One-way sensitivity analyses of key parameters were also conducted. In China, the prevalence of RA is about 0.41%. According to the calculations, the annual total cost of RA in China in 2018 was about ¥12,673,622,280 RMB, among which direct medical cost accounts for 33.3%, direct non-medical cost accounts for 29.8%, and indirect cost accounts for 37.0%. The indirect costs are high in current RA management as the effective treatment for RA is insufficient in China, leads to a heavy societal burden due to productivity loss caused by consequential deformity and disability. In order to improve the outcomes and quality of life of RA patients, it is needed to improve the awareness of the disease in patients and physicians, to facilitate the coverage of biological agents by reimbursement list, and to follow the standardized clinical treatment pathways for management of RA.

Managing conflicts of interest in pharmacy and therapeutics committees: A proposal for multicentre formulary development.

While many countries have central agencies responsible for formulary development, within the United States, each hospital, health care system, or insurance provider has their own pharmacy and therapeutic committee, leading to both inefficiencies and inequalities across formularies. The number and variety of processes within pharmacy and therapeutic committees also increases the likelihood that conflicts of interest will influence the development of formularies. We sought to determine how such influences could be reduced by reviewing international evidence related to the presence and harms of conflicts of interest in formulary development. Several approaches have been taken to reduce the influence of conflicts of interest in pharmacy and therapeutics committee processes, including include disclosure, recusal, exclusion, universal consideration and dual committees. The feasibility of each of these approaches is considered in the context of the United States. A proposal is drawn from the discussion of various approaches to conflicts of interest in pharmacy and therapeutics committees: multicenter formulary development. Multicentre formulary development, where resources are pooled across institutions, may lead to a reduction in the influence of conflicts of interest in pharmacy and therapeutics committee processes in the United States, increasing the chances of including the most safe, efficacious and cost-effective drugs on formularies.

Pharmacist perspectives and considerations for implementation of therapeutic oncology biosimilars in practice.

An overview of therapeutic oncology biosimilars, the U.S. biosimilars regulatory pathway, and the clinical development of selected biosimilar products is provided, including discussion of considerations in adopting biosimilars into oncology practice. Biosimilars are biologic agents that are highly similar to and have no clinically meaningful differences from an approved reference product in terms of safety, purity, and potency. There is a large market for cancer biologics, and approval of biosimilars has the potential to increase access to care and reduce costs. An abbreviated regulatory pathway for the development and approval of biosimilars defines a stepwise approach to demonstrating biosimilarity and conducting clinical comparative trials to confirm equivalent pharmacokinetics, efficacy, safety, and immunogenicity to the reference product. Three therapeutic biologics (bevacizumab, trastuzumab, and rituximab) have been used extensively in the treatment of a variety of cancers and are targets for biosimilar product development. Preclinical and clinical experience with 2 recently approved biosimilars to bevacizumab and trastuzumab is reviewed. Challenges faced by pharmacy and therapeutics committees when considering oncology biosimilars for formulary inclusion are discussed. Increased adoption of biosimilars could potentially lower treatment costs and improve access to biologics for patients with cancer. Key considerations in formulary review of biosimilars include the quality and quantity of data from comparative clinical trials, economic factors, manufacturer reliability, and challenges associated with incorporating biosimilars into practice.

Risankizumab.

Each month, subscribers to The Formulary Monograph Service receive 5 to 6 well-documented monographs on drugs that are newly released or are in late phase 3 trials. The monographs are targeted to Pharmacy and Therapeutics (P&T) Committees. Subscribers also receive monthly 1-page summary monographs on agents that are useful for agendas and pharmacy/nursing in-services. A comprehensive target drug utilization evaluation/medication use evaluation (DUE/MUE) is also provided each month. With a subscription, the monographs are available online to subscribers. Monographs can be customized to meet the needs of a facility. Through the cooperation of The Formulary, Hospital Pharmacy publishes selected reviews in this column. For more information about The Formulary Monograph Service, contact Wolters Kluwer customer service at 866-397-3433.

Formulary Drug Reviews: Brexanolone Injection.

Each month, subscribers to The Formulary Monograph Service receive 5 to 6 well-documented monographs on drugs that are newly released or are in late phase 3 trials. The monographs are targeted to Pharmacy & Therapeutics Committees. Subscribers also receive monthly 1-page summary monographs on agents that are useful for agendas and pharmacy/nursing in-services. A comprehensive target drug utilization evaluation/medication use evaluation (DUE/MUE) is also provided each month. With a subscription, the monographs are available online to subscribers. Monographs can be customized to meet the needs of a facility. Through the cooperation of The Formulary, Hospital Pharmacy publishes selected reviews in this column. For more information about The Formulary Monograph Service, contact Wolters Kluwer customer service at 866-397-3433.

Implementation of hyperlinks to medication management policies and guidelines in the electronic health record.

The implementation and maintenance of a process for adding and removing hyperlinks to medication management policies and guidelines approved by a pharmacy and therapeutics (P&T) committee into the electronic health record (EHR) are described. Medication management policies and guidelines approved by the P&T committee are published on the University of Utah Health intranet, making it possible to add hyperlinks to this information within the EHR. Adding these hyperlinks allows policy and guideline information to be available to clinicians on the medication ordering, verification, and administration screens without requiring a separate search of the intranet. In a quality-improvement project, all medication management policies and guidelines posted on the intranet were reviewed for relevance to the medication ordering, verification, and administration processes. Hyperlinks to relevant policies and guidelines were implemented into the EHR for specific medications. At the beginning of the review, 100 unique drugs associated with 1 or more hyperlinks were identified. The hyperlinks referenced a total of 33 Web documents: 8 policies and 25 guidelines. There are 74 medication management policies and 78 medication management guidelines approved by the P&T committee at University of Utah Health. After investigator review, 12 of 74 policies (16%) and 41 of 78 guidelines (53%) were deemed relevant during the medication ordering, verification, and administration processes. The review and hyperlink implementation process took a total of 101 hours. A continual review process was developed to enable addition and removal of hyperlinks as appropriate. Providing direct access to relevant medication management policies and guidelines approved by the P&T committee during the medication ordering, verification, and administration processes via hyperlinks in the EHR makes formulary information readily accessible by appropriate staff. These hyperlinks may also improve adherence to formulary information, reduce medication expenditure, and improve safety and therapeutic outcomes of medication therapy.

Use of Auditing and Feedback in an Outpatient Hospice Setting: Quality and Pharmacoeconomic Oversight

BackgroundClinicians play an important role in containing pharmaceutical spending at the patient level, as well as ensuring efficacy and quality outcomes, yet little research has examined how to achieve this goal. MeasuresUsing auditing and feedback (A&F) as part of a Pharmacy and Therapeutics (P&T) Committee, we evaluated our community-based hospice program's prescribing habits for opioids, antipsychotics, and antidepressants and calculated oral pharmaceutical prescription costs per-patient-day. Quality of care was reflected by patient pain scores in electronic medical records. InterventionOur P&T Committee adopted an A&F approach to monitor and assess provider prescribing habits and cost. An already-existing pain quality improvement program assessed care quality. OutcomesPain relief either improved or was maintained while medication costs were reduced by over $1.00 per-patient-day from 2010 to 2011. Conclusions/Lessons LearnedAn active, hospice P&T Committee featuring A&F can significantly affect medication costs for a hospice program while maintaining or improving patient outcomes.

Andexanet Alfa (Andexxa) Formulary Review.

Andexanet alfa, a recombinant modified human "decoy" factor Xa (FXa) protein, is the first and only available antidote approved by the Food and Drug Administration to manage life-threatening or uncontrolled bleeding associated with the anti-Xa agents. It binds to direct and indirect anti-Xa oral anticoagulants with high specificity to reverse their inhibitory effects and restore the activity of FXa. Andexanet alfa is administered via two different dosing regimens, standard and high dose, based on the specific FXa inhibitor, dose, and time since the patient's last dose of FXa inhibitor. The approval for andexanet alfa is supported by data from two phase 3 studies (ANNEXA-A, ANNEXA-R) and preliminary data from the phase 3b/4 ANNEXA-4 trial. The first study found that andexanet alfa rapidly reduced anti-Xa activity by 92%-94% in healthy volunteers taking apixaban or rivaroxaban. The ANNEXA-4 study found that the median anti-Xa activity decreased by 89%-93% in patients with major bleeding taking apixaban or rivaroxaban. However, thrombotic events occurred in 12 of 67 patients (18%) during the 30-day follow-up in ANNEXA-4. Additionally, only 40% of patients had restarted anticoagulation and, in this group, the rate of thrombotic events was 12%. Four patients had a thrombotic event within 3 days after andexanet alfa treatment. The wholesale acquisition cost of the standard dose regimen is $24,750, and the high-dose regimen is $49,500. The estimated annual drug budget of treating 10-100 patients ranges from $248K to $495M. Effective October 1, 2018, Medicare will provide an add-on payment for andexanet alfa of up to $14,063 per qualifying case to Inpatient Prospective Payment System-participating acute care hospitals. In this formulary review for a health system's pharmacy and therapeutics committee, andexanet alfa clinical trials and medication package insert were summarized and, after consulting with clinical experts from our institutions, practical recommendations for use were generated to ensure appropriate and safe use of this agent.

Formulary Drug Review: Sufentanil Sublingual.

Each month, subscribers to The Formulary Monograph Service receive 5 to 6 well-documented monographs on drugs that are newly released or are in late phase 3 trials. The monographs are targeted to Pharmacy & Therapeutics Committees. Subscribers also receive monthly 1-page summary monographs on agents that are useful for agendas and pharmacy/nursing in-services. A comprehensive target drug utilization evaluation/medication use evaluation (DUE/MUE) is also provided each month. With a subscription, the monographs are available online to subscribers. Monographs can be customized to meet the needs of a facility. Through the cooperation of The Formulary, Hospital Pharmacy publishes selected reviews in this column. For more information about The Formulary Monograph Service, contact Wolters Kluwer customer service at 866-397-3433.

Medicamentos e hospitais públicos: o impacto da implantação de comissões de farmácia e terapêutica

Este trabalho verificou os impactos econômicos e de acesso, além dos níveis de essencialidade das listas de medicamentos de uma rede hospitalar pública após a implantação de Comissões de Farmácia e Terapêutica (CFT) por meio do método de Planejamento Estratégico Situacional (PES). Foi observado uma redução de 27,8% do número total de itens, um aumento de 13,6% do percentual de itens pertencentes a Rename, de 10,5% pertencentes a lista da OMS e de 14,7% indicados por diretrizes clínicas. Em análise econômica observou-se uma redução de aproximadamente 12% dos custos da lista, demonstrando o grande potencial de contribuição para otimização dos recursos públicos.

A Quality Improvement Initiative to Decrease Inappropriate Intravenous Acetaminophen Use at an Academic Medical Center.

Background: Following availability in the United States in 2011, intravenous acetaminophen (IV APAP) was added to many hospital formularies for multimodal pain control. In 2014, the price of IV APAP increased from $12/g to $33/g and became a top 10 medication expenditure at our institution. Objective: To promote appropriate IV APAP prescribing and reduce costs. Design, Setting, Participants: Quality improvement project at a 562-bed academic medical center involving all inpatient admissions from 2010 to 2017. Interventions: Using Plan-Do-Study-Act (PDSA) methodology, our Pharmacy & Therapeutics (P&T) committee aimed to reduce inappropriate use of IV APAP by refinement of restriction criteria, development of clinical decision support in the electronic medical record, education of clinical staff on appropriate use, and empowerment of hospital pharmacists to enforce restrictions. Measurements: Monthly IV APAP utilization and spending were assessed using statistical process control charts. Balancing measures included monthly usage of IV opioid, IV ketorolac, and oral ibuprofen. Results: Five PDSA cycles were conducted during the study period. Monthly spending on IV APAP decreased from the highest average of $56 038 per month to $5822 per month at study conclusion. Interventions resulted in an 80% annual cost savings, or an approximate savings of $600 000 per year. Usage of IV opioids, IV ketorolac, and oral ibuprofen showed no major changes during the study period. Conclusions: IV APAP can be restricted in a safe and cost effective manner without concomitant increase in IV opioid use.

Abstract 242: Comparison of Pharmacist-Managed Initial Dosing of Direct Oral Anticoagulants with Non-Pharmacist Practitioner Prescribing Patterns

Background: There is currently a lack of data regarding pharmacist-managed DOAC (direct oral anticoagulant) therapy. The objective of this study was to assess the impact of pharmacist-managed initial dosing of apixaban and rivaroxaban utilizing a Pharmacy and Therapeutics Committee approved guideline when compared with general practitioner/cardiologist practitioner prescribing practices for patients with Non-Valvular Atrial Fibrillation (NVAF). Methods: A retrospective, chart review was performed during the period of January 2015 to January 2018 and included adults 18-95 years old, who were prescribed apixaban or rivaroxaban for documented NVAF. Patients eligible for this study were split up into two cohorts: a pharmacist-managed cohort and a non-pharmacist-managed cohort. The primary endpoint of the study was the percentage of patients who received appropriate initial apixaban or rivaroxaban dosing. Any incorrect initial doses of apixaban or rivaroxaban were then analyzed to determine the reason for error. The secondary endpoints included thromboembolic and hemorrhagic adverse events. Exploratory analyses included: provider follow up-time, inappropriate DOAC use in valvular atrial fibrillation (VAF), and correct transition from warfarin to DOAC. Results: For the primary endpoint, the pharmacist practitioner was significantly more accurate when initially dosing apixaban and rivaroxaban compared to non-pharmacist practitioners (97% [63 out of 65] vs. 74% [48 out of 65] correct prescriptions, p=0.001). When evaluating the reason for error in prescribing, non-pharmacist providers were significantly less likely to dose the patient appropriately based on renal function or drug interactions (p=0.003). For the secondary endpoint of hemorrhagic or thromboembolic events, there were no statistically significant differences among the two provider types. The exploratory analyses revealed that 3% (2 out of 65) of the patients in the non-pharmacist managed cohort had VAF and received a DOAC for antithrombotic therapy. The patients in the pharmacist managed cohort had zero patients receive a DOAC for VAF. The pharmacist was also significantly better at transitioning patients from warfarin to DOACs correctly compared to non-pharmacist practitioner (100% [12 out of 12] vs. 50% [3 out of 6], p=0.025) and following up with patients within a four-month time frame compared to non-pharmacist practitioners (100% [65 out of 65] vs. 82% [53 out of 65], p=0.001). Conclusion: Additional research is required to evaluate the benefits of pharmacist-managed anticoagulation; however, based on the results of this study, pharmacists are significantly better equipped to initiate DOAC therapy for patients with NVAF. Based on the findings of the study results, pharmacist-managed DOAC services may be transferrable to a variety of different disease states requiring anticoagulation therapy.

Drug formulary decision-making: Ethnographic study of 3 pharmacy and therapeutics committees.

An ethnographic study of 3 pharmacy and therapeutics (P&T) committees was conducted to characterize the process by which evidence is weighted and decisions are made. We recorded discussions and conducted an analysis of the transcripts from the 3 P&T committees for a 1-year period. We examined the content and ideas expressed during deliberations and synthesized themes to give a broader picture of the issues arising. Committee discussions were transcribed and the segments of each meeting that addressed any new formulary additions were then analyzed. Using constant comparison method, we generated a series of topic codes to characterize and classify that portion of the discussion. At 26 meetings across the 3 sites, 24 new drug formulary additions were discussed. A total of 1,093 discussion segments were identified and mapped to 7 broad categories related to discussion of evidence of need, efficacy/indications, safety, misuse potential, cost issues, committee decision-making issues, and discussion related to operationalizing use and implementation at the local institution. Overall, the leading category of discussion was efficacy/indications followed by evidence of need, operational issues, and cost issues, with some variation by site. The committees devoted substantially greater portions of their discussion to the logistics of using the drugs in their institutions than they did safety issues. We identified wide variations in specific drugs being considered and the relative amount of time devoted to various issues related to these drugs being discussed. We found discussions generally did not follow a systematic, standardized, rigorous, and reproducible approach. Discussions tended to be more idiosyncratic, individualized, varying from drug to drug, and at times devoted to a variety of tangential issues raised by committee members. P&T committee discussions at all 3 sites tended to be idiosyncratic and individualized, varying from drug to drug, and at times devoted to a variety of issues more tangentially raised by committee members. All spent less time talking about drug safety, in each case roughly half the time that they devoted to discussions of efficacy.

National Corporate Pharmacy and Therapeutic Committee at the Ministry of Health, Saudi Arabia

Pharmacology, Toxicology and Biomedical Reports,2018,4,3,24-27.DOI:10.5530/PTB.2018.4.9Published:September 2018Type:Review ArticleAuthors:Yousef Ahmed Alomi, Saeed Jamaan Alghamdi, and Radi Abdullah Alattyh Author(s) affiliations:Yousef Ahmed Alomi1, Saeed Jamaan Alghamdi2, Radi Abdullah Alattyh3 1The Former General Manager of General Administration of Pharmaceutical Care, The Former Head, National Clinical pharmacy and pharmacy practice, The Former Head, Pharmacy R and D Administration, Ministry of Health, Riyadh, SAUDI ARABIA. 2General Administration of Pharmaceutical Care, Ministry of Health, Riyadh, SAUDI ARABIA. 3General Administration of Pharmaceutical Care, Ministry of Health, Riyadh, SAUDI ARABIA. Abstract:Objective: To review the Ministry of Health Corporate MOH Pharmacy and Therapeutic committee system at the Ministry of Health foundations in the Kingdom of Saudi Arabia. Method: It is a retrospective analysis of Ministry of Health Corporate MOH Pharmacy and Therapeutic committee system at Ministry of hospitals institutions. Its analysis illustrated within the Pharmacy strategic plan 2012-2020. The analysis process used. The modified pharmacy business model system and Project Management Procedure. Results: Ministry of Heath drug Corporate MOH Pharmacy and Therapeutic committee established with clear vision, mission and goals. The committee had human or economic and other resources described in the review. To assure the continuity of the system; the risk management was used and described. Besides, the monitoring and controlling of the system were demonstrated. The closing stage with convention to operation project shown in the Analysis. Conclusion: The Ministry of Health Corporate MOH Pharmacy and Therapeutic committee system founded though Pharmacy strategies and regulations. The Ministry of Health Corporate MOH Pharmacy and Therapeutic committee has regular development accordingly at all Ministry of Health hospitals and primary healthcare centers in the Kingdom of Saudi Arabia. Keywords:Ministry of Health, Pharmacy and Therapeutic Committee, Saudi ArabiaView:PDF (242.23 KB) PDF PDF Images National Corporate Pharmacy and Therapeutic Committee at the Ministry of Health, Saudi Arabia

Implications of the Parenteral Opioid Shortage for Prescription Patterns and Pain Control Among Hospitalized Patients With Cancer Referred to Palliative Care

The recent parenteral opioid shortage (POS) has potential implications for cancer-related pain management in hospitalized patients. This study compared changes in opioid prescriptions and clinically improved pain (CIP) among patients treated by an inpatient palliative care (PC) team before and after our institution first reported the POS. A cohort study of 386 eligible patients with cancer treated at a comprehensive cancer center 1 month before and after the announcement of the POS. We reviewed data from electronic health records, including patient demographics, opioid type, route of administration, and dose. Board-certified palliative care specialists assessed CIP at follow-up day 1. The announcement of the POS by the institution's pharmacy and therapeutics committee on February 8, 2018. The primary outcome was to measure the change in opioid prescription patterns of physicians, and the secondary outcome was to measure the proportion of patients who achieved CIP before and after announcement of the POS. Of 386 eligible patients, 196 were men (51%), 270 were white (70%), and the median age was 58 years (interquartile range, 46-67 years). Parenteral opioids were prescribed less frequently by the referring oncology teams after the POS (56 of 314 [18%]) vs before the POS (109 of 311 [35%]) (P < .001). The PC team also prescribed fewer parenteral opioids after the POS (96 of 336 [29%]) vs before the POS (159 of 338 [47%]) (P < .001). After the POS (vs before the POS), significantly fewer patients achieved CIP on follow-up day 1 (119 [62%] vs 144 [75%] of 193; P = .01). Multivariate analysis showed that before the POS, patients had an 89% higher chance of achieving CIP on follow-up day 1 (odds ratio, 1.89; 95% CI, 1.22-2.94; P = .005). There was a significant change in opioid prescription patterns associated with the POS. Furthermore, after the POS, fewer patients achieved CIP. These factors have potential implications for patient satisfaction and hospital length of stay.

Application of pharmacoeconomics to formulary management in a health system setting.

A novel value-based approach to evaluate costly specialty drugs for formulary addition was developed. In February 2016, Stanford Health Care launched the specialty drug subcommittee (SDSC), a subcommittee of the pharmacy and therapeutics committee, responsible for the formulary review of specialty pharmaceuticals. A process was developed for value-based review that includes not only consideration of clinical trial data and institutional acquisition costs but also internal patient outcomes and a cost-effectiveness model using internal financial data. A Markov model was developed to assess the value of trabectedin, which was approved for formulary addition in April 2016, relative to the addition of dacarbazine. The economic model and internal patient outcome analysis were presented to the prescribing oncologist and the SDSC for review. Internal data revealed that fewer patients than had been estimated received trabectedin, with outcomes significantly worse than those observed in the clinical trial leading to Food and Drug Administration approval. In the cost-effectiveness model, trabectedin had higher costs and poorer outcomes compared with dacarbazine. Based on the economic model, low utilization, and real-world outcomes, trabectedin was removed from formulary and a restrictive treatment pathway for nonformulary use, developed by the primary prescriber, was implemented. This process has since been applied to 11 more specialty drugs. Internal cost-effectiveness models in combination with real-world patient outcomes data can be effective formulary management tools. Engagement and collaboration with the requesting provider are key to developing thoughtful treatment pathways.

Enzymatic replacement therapy for lysosomal storage disorders: Drug evaluations review in Spain

IntroductionIn the European Union companies only need to demonstrate that the risk-benefit balance of the new drug is favourable to obtain the authorization to sell new drugs. Hence a comparison with available treatments, a cost-effectiveness analysis and the place in the therapy of the new drug are not required. Therefore, it is necessary to carry out these analyses in an additional study. In Spain there is no national government agency, which conducts a centralized evaluation and makes decisions on funding and are the Pharmacy and Therapeutics committees that take mandatory decisions for the entire region. ObjectivesTo identify all drug assessments and health technology assessment reports of the enzymatic replacement treatments for the four LSD considered in this study, including Mucopolysaccharidosis (MPS), Gaucher, Fabry and Pompe disease at the national, regional and hospital level; and to summarize the efficacy in terms of outcome measures, adverse events, economic impact and final recommendations and indicate potential improvements. Methods19 reviews at the regional and hospital level and one therapeutic positioning report at national level for MPS were considered for this. ResultsIn general, the drugs evaluated in LSD have proved to be effective in the most frequent clinical forms of disease. However, there are clinical phenotypes that still do not have available an effective treatment. ConclusionsIt is expected that new therapies, such as intrathecal therapies or gene therapy could be evaluated in the different types of LSD demonstrating positive effects, especially in the previous phenotypes.

Use of economic predictions to make formulary decisions.

The accuracy of cost savings and reimbursement predictions for medications added to an academic medical center formulary was assessed. Formulary changes over a 5-year period were reviewed by the investigators. Medications were included if the medication was added to formulary and the monograph included cost savings or reimbursement data that indicated a positive net margin. The primary endpoints were percent predicted cost savings and net margin per medication based on medication cost only. Secondary endpoints included the percent of medications with at least 100% predicted cost savings or net margin and evaluation of median percent predicted savings or net margin individually. The pharmacy and therapeutics committee reviewed 558 formulary agenda items, 184 of which were selected for further analysis. In total, 19 medications were identified as having a predicted monetary advantage. The endpoints of percent predicted cost savings and net margin yielded a median of 76.5% (range 72.9-188.71%) (n = 3) and 148.2% (IQR 108.9-543.3%) (n = 16), respectively. For 13 (68%) of 19 medications, the percent predicted cost savings or net margin was at least 100%. Economic predictions utilized for formulary management at an academic medical center generated net positive monetary value for medications where predicted cost savings or reimbursement factored into the decision to add a medication to the formulary.