Academic literature on the topic 'Drug and Therapeutics Committees (DTCs)'

Background: As a key partner of Ministry of Health (MOH) Ethiopia, The Clinton Health Access Initiative (CHAI) had been implementing the Child Survival Project (CSP) since October 2015. Strengthening DTC was one of its focuses to improve overall supply chain management (SCM). The objectives of this study are to review the evolution of DTCs in Ethiopia from their early years to current practice and identify the major hindering factors for their functionality. Methods: A descriptive study design was employed with mainly qualitative data collection methods and analysis. The assessment made use of both qualitative and quantitative data, generated from primary sources through key informant interviews and from secondary sources through desk review methods. Results: DTCs were introduced in Ethiopia in the early 1980’s. The mandate of DTCs has been given to four different government organizations since this time. As a result, its implementation was lagging. Recently, the government and its partners have given attention to DTCs. More than 5847 professionals underwent DTC training from 2016 onwards. DTC establishment in health facilities (HFs) improved from 85% to 98% between 2015 and 2019 during baseline and endline assessments carried out by CHAI/CSP. Similarly, DTC functionality in HFs improved from 20% to 63%. The CHAI/CSP regular supervision data analysis revealed that DTC establishment improved from 83% to 100% of HFs, while its functionality improved from 5% to 72% between 2016 and 2019, respectively. A chi-square test of independence examining the relationship between facility and pharmacy head training on DTCs and functionality of DTC in the same facility revealed significant association between the two variables at p<0.0001. Conclusions: Providing consistent capacity building and availing strong monitoring and evaluation system improves functionality of DTCs. Moreover, national coordinating bodies for DTCs and similar structures at Regional Health Bureaus and woreda health offices should be established.

INTRODUCTION:The socioeconomic burden of diseases is increasing in Africa. For instance in 2011, 70 percent of the world's human immunodeficiency virus (HIV) population resided in sub-Sahara Africa. There are also growing rates of Antimicrobial Resistance (AMR), which necessitates newer more expensive antibiotics adding to costs. There is also a growing burden of non-communicable diseases (NCDs), three out of four patients with hypertension currently live in low and middle income countries (LMICs), with prevalence rates up to 30 to 45 percent among adults in Africa. Alongside this, up to 70 percent of total healthcare expenditure is spent on medicines in LMICs; much of this out-of-pocket. Consequently, there is an urgent need to strengthen collaborative research to improve medicine use.METHODS:Summary of groups working together in Africa including the Medicines Utilisation Research in Africa (MURIA) group.RESULTS:African Strategies for Health identifies and advocates best practices, as well as works with others to develop sustainable solutions. Pharmacology for Africa (PharfA) organises and promotes pharmacology on the African continent, including research in clinical pharmacology, alongside the International Union of Basic and Clinical Pharmacology (IUPHAR) sub-division. International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Africa co-ordinates activities from the different African country chapters. The South African Health Technology Assessment Society (SAHTAS) is a scientific and professional society for all those who produce, use, or encounter Health Technology Assessment (HTA) in Southern Africa, and the World Health Organization (WHO) International and Regional groups are improving antibiotic drug utilization capabilities in Africa. The MURIA group was established in 2015 (1). Ongoing collaborative research includes (i) initiatives to optimize antibiotic use; (ii) methods to enhance adherence to anti-infective prescribing guidance, (iii) approaches to improve adherence to HIV and NCDs; (iv) researching current anti-hypertensive utilization patterns and knowledge; (v) approaches to enhance Drugs and Therapeutic Committees (DTC) activities, and (vi) strengthening medicine utilization capabilities (2,3). These activities have already strengthened research ties across Africa.CONCLUSIONS:A number of groups are already working across Africa to enhance appropriate medicine use, and should continue. Ongoing MURIA activities include antibiotic point-prevalence studies, ongoing research into infectious diseases, NCDs and DTCs including adherence as well as the third workshop and symposium in Namibia in 2017.

Introduction: Novel approaches to treating patients (pts) with relapsed or refractory (R/R) classical Hodgkin lymphoma (cHL) have improved outcomes but some pts do not respond or, despite initial response, develop progressive disease and have limited treatment options. Camidanlumab tesirine (ADCT-301; Cami) is an antibody-drug conjugate composed of a human IgG1 anti-CD25 monoclonal antibody stochastically conjugated to a potent pyrrolobenzodiazepine (PBD) dimer warhead, which triggers cell death via formation of highly cytotoxic interstrand cross-links. Data from a Phase (Ph) 1 dose-escalation, dose-expansion trial demonstrated an overall response rate (ORR) in pts with cHL of 86.5% (48.6% complete response [CR] rate) at the 45 μg/kg dose. Cami had a generally acceptable safety profile at Ph 1 but there were 5/77 cases (6.5%) of Guillain-Barré syndrome (GBS)/polyradiculopathy (Preferred Terms: 4 GBS and 1 radiculopathy) (Collins et al, ICML June 18-22, 2019, Lugano, Switzerland, Abstract 055). Here, we present preliminary efficacy and safety results of a Ph 2 trial of single-agent Cami in pts with R/R cHL (NCT04052997). Methods: A single-arm, multi-center, open-label, Ph 2 trial is currently enrolling pts ≥16 yrs (US) and ≥18 yrs (outside US) with R/R cHL following ≥3 prior treatment lines (or ≥2 lines in pts ineligible for hematopoietic stem cell transplantation). Eligible pts had prior treatment with brentuximab vedotin and PD-1 blockade, measurable disease per 2014 Lugano Classification, and Eastern Cooperative Oncology Group performance status 0-2. The primary objective is to evaluate efficacy of single-agent Cami by ORR as determined by central review. Secondary objectives include further characterization of additional efficacy endpoints and safety. Pts receive 30-min IV infusions of Cami on Day 1 of each 3-week cycle at a dose of 45 μg/kg for 2 cycles, followed by 30 μg/kg for subsequent cycles for up to 1 yr or until discontinuation due to disease progression, unacceptable toxicity, or other reasons. Pts deriving clinical benefit at 1 yr may be able to continue treatment on a case-by-case basis. Treatment-emergent adverse events (TEAEs) were defined as AEs occurring/worsening from time of first dose to either 30 days post last dose or to start of new anticancer therapy/procedure, whichever occurs first. This analysis was conducted after meeting a protocol-specified criterion for pausing enrollment (≥2 cases of GBS or other relevant severe neurologic toxicity). Results: As of June 15, 2020, 47 pts with R/R cHL were enrolled and are included in this analysis. Median age was 36 (range 23-74) yrs and pts had received a median of 7 (range 3-20) lines of prior therapy, including transplant (Table 1). Pts received a median of 5 (range 2-10; mean 4.9 [SD 1.86]) cycles of Cami. ORR was 80.9% (38/47 pts), with 18 (38.3%) and 20 (42.6%) pts attaining CR and partial response, respectively; 6 pts (12.8%) had stable disease (Figure 1). TEAEs were experienced by all 47 pts; the most common (≥20% of pts) were fatigue (22, 46.8%); nausea, pyrexia, and maculopapular rash (18, 38.3% each); anemia and headache (12, 25.5% each); pruritus (11, 23.4%); arthralgia, constipation, diarrhea, hypophosphatemia, and rash (10, 21.3% each). TEAEs thought to be PBD-associated included skin reactions and nail disorders (36, 76.6%), liver function test abnormalities (14, 29.8%), and edema or effusion (7, 14.9%). There were 3 (6.4%) pts with GBS/polyradiculopathy (Preferred Terms: grade 4 subacute inflammatory demyelinating polyneuropathy, grade 2 radiculopathy, and grade 2 peripheral motor and sensory neuropathy updated to GBS after data cut-off date). In total, 27 (57.4%) pts had grade ≥3 TEAEs; the most common (≥5% of pts) were hypophosphatemia (6, 12.8%) and gamma-glutamyltransferase increased (3, 6.4%). Overall, 3 (6.4%) pts had TEAEs leading to dose reduction/delay and 6 (12.8%) pts had TEAEs leading to treatment discontinuation. Conclusions: Current data show that therapy with Cami has encouraging anti-tumor activity in heavily pretreated pts with R/R cHL. Safety was consistent with that reported at Ph 1, with no new safety concerns identified and similar incidence of GBS/polyradiculopathy. Following a positive risk-benefit assessment, the enrollment pause was lifted, and pts continue to be enrolled. Updated efficacy and safety results will be presented at the meeting. Funding: Study funded by ADC Therapeutics SA. Disclosures Herrera: Pharmacyclics: Research Funding; Bristol Myers Squibb: Consultancy, Other: Travel, Accomodations, Expenses, Research Funding; Merck: Consultancy, Research Funding; Gilead Sciences: Consultancy, Research Funding; Genentech, Inc./F. Hoffmann-La Roche Ltd: Consultancy, Research Funding; Seattle Genetics: Consultancy, Research Funding; Karyopharm: Consultancy; AstraZeneca: Research Funding; Immune Design: Research Funding. Carlo-Stella:Bristol-Myers Squibb, Merck Sharp & Dohme, Janssen Oncology, AstraZeneca: Honoraria; ADC Therapeutics and Rhizen Pharmaceuticals: Research Funding; Boehringer Ingelheim and Sanofi: Consultancy; Servier, Novartis, Genenta Science srl, ADC Therapeutics, F. Hoffmann-La Roche, Karyopharm, Jazz Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees. Collins:Taekda: Consultancy, Honoraria, Other: travel, accommodations, expenses, Speakers Bureau; Novartis: Consultancy, Honoraria, Speakers Bureau; Pfizer: Honoraria; Gilead: Consultancy, Honoraria, Speakers Bureau; ADC Therapeutics: Consultancy, Honoraria; BMS: Consultancy, Honoraria, Research Funding, Speakers Bureau; MSD: Consultancy, Honoraria, Research Funding; Celleron: Consultancy, Honoraria, Research Funding; Celgene: Research Funding; Amgen: Research Funding; BeiGene: Consultancy; Roche: Consultancy, Honoraria, Other: travel, accommodations, expenses , Speakers Bureau. Maddocks:Morphosys: Consultancy, Honoraria; BMS: Consultancy, Research Funding; Karyopharm: Consultancy; Seattle Genetics: Consultancy, Honoraria; ADC Therapeutics, AstraZeneca: Consultancy; Pharmacyclics: Consultancy, Honoraria; Celgene: Consultancy, Honoraria. Bartlett:Autolus: Research Funding; BMS/Celgene: Research Funding; Forty Seven: Research Funding; Immune Design: Research Funding; Seattle Genetics: Membership on an entity's Board of Directors or advisory committees, Research Funding; Janssen: Research Funding; Kite, a Gilead Company: Research Funding; Merck: Research Funding; Millennium: Research Funding; Pfizer: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Pharmacyclics: Research Funding; Seattle Genetics: Consultancy, Research Funding; Roche/Genentech: Consultancy, Research Funding; ADC Therapeutics: Consultancy; BTG: Consultancy; Acerta: Consultancy; Affimed Therapeutics: Research Funding. Savage:BeiGene: Other: Steering Committee; Roche (institutional): Research Funding; Merck, BMS, Seattle Genetics, Gilead, AstraZeneca, AbbVie: Honoraria; Merck, BMS, Seattle Genetics, Gilead, AstraZeneca, AbbVie, Servier: Consultancy. Caimi:ADCT, Kite Therapeutics, Genentech, Amgen, Verastem, TG Therapeutics, Bayer: Consultancy; ADC Therapeutics: Membership on an entity's Board of Directors or advisory committees; Genentech, ADC Therapeutics: Research Funding; Celgene: Speakers Bureau. Hess:ADC Therapeutics: Consultancy, Membership on an entity's Board of Directors or advisory committees; BMS, AstraZeneca: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Zinzani:ADC Therapeutics: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Sanofi: Consultancy, Membership on an entity's Board of Directors or advisory committees; Sandoz: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; EUSA Pharma: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Celltrion: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Verastem: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Janssen-Cilag: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Kirin Kyowa: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Merck: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Portola: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Janssen: Consultancy, Honoraria, Speakers Bureau; Servier: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Roche: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; TG Therapeutics, Inc.: Honoraria, Speakers Bureau; BMS: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Incyte: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Takeda: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Gilead: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; AbbVie: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; MSD: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Eusapharma: Consultancy, Speakers Bureau; Kyowa Kirin: Consultancy, Speakers Bureau; Immune Design: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Immune Design: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Cruz:ADC Therapeutics SA: Current Employment, Current equity holder in publicly-traded company. Wang:ADC Therapeutics America, inc: Current Employment, Current equity holder in publicly-traded company. Feingold:ADC Therapeutics: Current Employment, Current equity holder in publicly-traded company. Wuerthner:ADC Therapeutics SA: Current Employment, Current equity holder in publicly-traded company. Ansell:Takeda: Research Funding; Bristol Myers Squibb: Research Funding; Seattle Genetics: Research Funding; Regeneron: Research Funding; Trillium: Research Funding; ADC Therapeutics: Research Funding; Affimed: Research Funding; AI Therapeutics: Research Funding.

Australia�s policy on Quality Use of Medicines (QUM) aims to achieve appropriate use of medicines and improved health outcomes. Drug and Therapeutics Committees (DTCs) are educators, policy makers as well as financial gatekeepers in matters relating to medicine use. Increasingly, DTCs are also involved in risk management and clinical governance. As such, DTCs could be considered to be QUM advocates in the institutions in which they function. In a health care arena where there are escalating demands on high standards of clinical practice, quality assessment and improvement is essential in ensuring safe and effective patient care. Given the role DTCs play in safeguarding the interests of the stakeholders of the health care system, research into ways in which DTC performance could be enhanced is required. Although indicators specific to DTCs exist, the literature does not seem to provide straightforward answers to the question of what is currently being done in terms of quality assessment and quality improvement of DTCs. In the absence of such data, an opportunity for research is clearly identified. The first aim of this research project was to gain insight into the current activities undertaken by, and challenges facing Australian DTCs. Following this, the second aim was to explore ways in which DTC performance could be augmented. In addressing the first aim of this project, a national survey of Australian DTCs was conducted. These findings reinforce the evidence in the literature about the roles, structure and stakeholder expectations of DTCs. Our research also documents DTCs� quality improvement initiatives and barriers to DTC activities. It appears that there is little support available to Australian DTCs. Further, a case study was undertaken in order to gain an understanding of the depth and detail of DTC operations. An audit of a DTC in an Australian hospital was conducted. This study revealed that DTC decisions are being implemented in an ad hoc manner. In fact, there were no strategies (or action) planned to implement the majority of their decisions. This could have an impact on DTC performance. In view of this finding, qualitative methods were used to explore stakeholder opinions regarding the implementation of DTC decisions and policies. Stakeholders believed that strategies used to implement DTC policies should be targeted (to the audience as well as the type of decision/policy being implemented), timely, and delivered at the point of care. Face-to-face strategies were perceived to be more effective than printed materials, particularly when an influence on clinical practice was desired. Stakeholders also felt that the lack of resources was a significant barrier to DTC performance augmentation. This probably contributed to a lack of follow-up (or review) of implemented policies. According to stakeholders, other barriers to policy implementation include a lack of ownership of policies, low DTC profile, and an over-reliance on pharmacy to implement DTC decisions. Stakeholders felt one of the ways in which DTC performance could be improved was to prioritise DTC decisions for implementation. In pursuit of a method to prioritise DTC decisions, a survey was conducted. Stakeholders identified patient safety, cost, and the practice of evidence-based medicine as domains of important DTC decisions. The results also suggest that stakeholders recognise the need for the prioritisation of DTC decisions for implementation. Stakeholders implied that higher priority would be assigned to DTC decisions considered to be important. In a follow-up survey, stakeholders (including doctors, nurses, pharmacists, and DTC members) seemed to have agreement of the primary domains of DTC decisions. Higher levels of importance and higher priority were assigned to decisions involving the primary domains of patient safety and cost. However, level of importance and priority assignment were not consistently correlated. The work presented in this thesis suggests that there are ways to improve DTC performance. Although conducted primarily on hospital-based DTCs, it is anticipated that the lessons learnt could be applied to state-based, or even, Area Health-based DTCs. In conclusion, this research found that there was a range of views regarding �importance� and prioritisation for implementation. Social, organisational, as well as environmental factors may contribute to this. Future research should examine other possible factors contributing to the importance and priority of DTC decisions, so that DTC policy could be appropriately implemented into practice.

BACKGROUND: Hospital formularies are usually the gatekeepers for pharmaceutical drugs. Typical majority members of hospital formularies are physicians, although most of the time the formulary is chaired by a pharmacist. As German hospitals are struggling with a difficult economic environment the question arises: what kind of decision-making criteria are applied when pharmaceutical drugs should be added to the formulary list? Information regarding this topic is scarce due to the sensitive topic of decision-making. OBJECTIVES: Build a single decision-making framework which will be created to explain hospital drug funding decision-making and identify underlying mechanisms which explain processes and structures. The results can be used by hospitals to initiate knowledge sharing and provide a basis to analyse local formulary committee decision-making practice. Additionally, they can be used by the pharmaceutical industry to better adapt to the specific needs of the hospital decision-makers. METHODS: In this study, a mixed-methods approach has been used to confirm and further detail a preliminary hospital formulary decision-making framework derived from literature. An online survey was used to get insights on the structure of German hospital formularies and the relative importance of different decision-criteria. Additional semi-structured expert interviews were used to get in-depth information on the underlying mechanisms which influence decision-making on drug funding. RESULTS: Decisions for or against a pharmaceutical drug are influenced by a variety of perceived objective and specifically subjective criteria. Despite a consistency in a dominant, high impact role of pharmacists and lead physicians every hospital formulary member has different relative weighting of decision criteria. Drug funding decision-making in German hospital formularies is highly individual but usually starts with a quasi-rational preference influenced by a mixture of analytic and intuitive criteria. The decision to use more analytic or more intuitive criteria is influenced by a variety of factors. The two most important ones are uncertainty and power. The resulting individual preference is then challenged and adapted in a group decision-making process.

Professionals and researchers have developed a number of strategies aimed at improving the quality and safety of medication use. However, studies continue to demonstrate persistent problems. For instance, the first paper in this thesis reveals the prevalence of potentially harmful drug combinations among elderly people in Europe. The following four papers focus on two professional groups and how they have approached safety and quality issues related to medication use: 1) the Swedish drug and therapeutics committees (DTCs) and 2) pharmacist involved in pharmaceutical care, an international movement. Qualitative research approaches were applied.

Papers II and III focus on the DTCs: analyses indicate a development of the perception of the DTC role over time. The focus of the activities was broadened – from targeting prescribing physicians to incorporating decision-makers and patients. However, a clear patient-centered perspective was generally lacking. Moreover, the findings indicate a shift in focus from cost aspects of medication use to an increased focus on quality and safety aspects.

In the studies addressing pharmaceutical care (Papers IV and V), the findings propose that different classification systems for drug-related problems had different characteristics which reflected differences in goals in the pharmaceutical care process. It was also found that the concept of pharmaceutical care was understood in different ways and that the perceptions were based on at least two different understandings of health and illness. First, a patient-centered perspective characterized by a holistic understanding of health and illness, and, second, an “EBM perspective” primarily based on a biomedical understanding of health and illness.

This thesis has disclosed new aspects of how two groups of professionals perceive their work towards improved quality and safety of medication use. A patient-centered perspective among healthcare collectives is not obvious; therefore, efforts and comprehensive strategies supporting change are necessary. Strategies should focus on challenging the traditional thought patterns and care approaches among professionals and students.

Els estudis d'utilització de medicaments permeten identificar els patrons d'ús dels medicaments en diferents poblacions, ja siguin adults o nens. Un aspecte és l’anàlisi de l’ús de medicaments en condicions diferents a les autoritzades a la fitxa tècnica. Aquesta tesi és una anàlisi de diferents aspectes de l’ús de medicaments en condicions no aprovades després que es publiqués el Reial Decret 1015/2009 del 19 de juny pel qual es regula la disponibilitat de medicaments en diferents situacions especials (entre les quals hi ha l’ús en condicions no aprovades), i s’establís una normativa de l'Institut Català de la Salut (ICS), amb uns procediments estandarditzats de treball per revisar, avaluar i registrar l'ús de medicaments en les situacions especials. En la publicació Medicina Clínica 2014;143:327-9, es descriuen els medicaments utilitzats en condicions no aprovades i les indicacions en les que s'utilitzen a dos hospitals de tercer nivell durant els tres anys posteriors a la publicació del Reial Decret. Es van rebre 671 sol·licituds de 117 medicaments o combinacions per a ser emprats en aquestes condicions. Els subgrups més sol·licitats van ser antineoplàstics, immunosupressors i relaxants musculars, i els fàrmacs més freqüents van ser rituximab (170 sol·licituds), toxina botulínica (81), omalizumab (39) i anakinra (21). Les principals malalties van ser neoplàstiques, del sistema nerviós i òrgans dels sentits i de l’aparell digestiu. En la publicació European Journal of Clinical Pharmacology 2014;70:1385-93 s’analitzen les indicacions d'ús, els resultats clínics (resposta clínica i tolerabilitat de la medicació) i els resultats econòmics (cost total i per pacient) de l'ús dels medicaments en condicions no aprovades en cinc hospitals de l'ICS durant un any. S’hi van incloure de manera prospectiva 226 pacients, amb una edat mediana (RIQ) de 46 (33-62) anys; el 59% eren dones. Els pacients havien rebut una mediana de tres tractaments previs, i la principal raó per demanar l’ús no aprovat era la manca de resposta o resposta subòptima (72,1%). Es van donar 232 fàrmacs per a 102 indicacions diferents. Els fàrmacs més freqüents van ser rituximab (49; 21,1%), toxina botulínica (25; 10,7%) i omalizumab (14; 6,0%). En 117 casos (51,8%) el nivell d’evidència disponible per al seu ús era baix. Es va observar resposta parcial en 82 pacients (36,3%), completa en 71 (31,4%) i estabilització en 11 (4,9%). Va presentar efectes indesitjats 58 pacients (26,5%). El cost medià (RIQ) per pacient va ser de 2.943,07 € (541,9 – 5.872,54). En la publicació European Journal of Clinical Pharmacology 2013;69:1689-99 s’analitzen i es descriuen les indicacions d'ús, les evidències científiques disponibles i els resultats clínics a curt i llarg termini i econòmics, del fàrmac més utilitzat en condicions no aprovades que ha estat el rituximab. Es van analitzar 101 casos d’ús de rituximab en condicions no aprovades, amb una mediana d’edat (RIQ) dels pacients de 53 anys (37,5-68,0]; el 55,4% eren dones. Les indicacions principals eren malalties hematològiques (46%), malalties sistèmiques del teixit connectiu (27%) i malalties renals (20%). Les evidències eren principalment estudis de cohort individuals (53,5% dels casos) i sèries de casos (25,7%). La resposta observada a curt termini (mediana de 3 mesos [IQR 2-4]) va ser completa en el 38% dels casos i parcial en el 32,6%. Les més altes es van observar en el lupus eritematós sistèmic i la glomerulonefritis membranoproliferativa, i les respostes més baixes, en la neuromielitis òptica, i la púrpura trombocitopènica idiopàtica. En el 69,2% dels casos amb resposta a curt termini es va mantenir una certa resposta a llarg termini (mediana de 23 mesos [RIQ 12-30]). El cos per pacient (mediana va ser de 5.187,5€ (RIQ 5.187,5-7.781,3).
Drug utilization studies can be useful to identify patterns of drug use in different populations, whether adult or children. One aspect is the analysis of drug use in conditions other than those authorized in the technical specifications or labelling. This thesis is an analysis of various aspects of off-label drug use after the publication of a Royal Decree 1015/2009 of 19 June regulating the availability of medicines in various special situations (including off-label drug use) and after the establishment of a rule of the Catalan Institute of Health (ICS) with standardized work procedures to review, evaluate and register the use of drugs in special situations. The publication Medicina Clínica 2014;143:327-9 describes the drugs used in conditions not approved in two tertiary hospitals during the three years following the publication of Royal Decree. There were 671 applications for 117 single drugs or combinations to use in these conditions. The main groups were antineoplastic, immunosuppressive and muscle relaxants, and the most involved drugs were rituximab (170 applications), botulinum toxin (81), omalizumab (39) and anakinra (21). The main diseases were neoplastic diseases, those affecting nervous system and sense organs and digestive disorders. The publication European Journal of Clinical Pharmacology 2014;70:1385-93 describes the analysis of indications for use, clinical outcomes (clinical response and tolerability of the medication) and cost of off-label drug use in five hospitals of the ICS during one year. A total of 226 patients were included. The median [IQR] age of patients was 46 (33-62) years; 59% were women. Patients had received a median of three previous treatments, and a lack of response (or suboptimal) was the main reason for off-label use (72.1%). A total of 232 off-label medicines were administered for 102 different indications. The most frequent medicines were rituximab (49; 21.1%), botulinum toxin (25; 10.7%) and omalizumab (14; 6.0%). In 117 (51.8%) cases the level of clinical evidence for their use was low. A partial clinical response was observed in 82 patients (36.3%), complete response in 71 (31.4%) and stabilization in 11 (4.9%). A total of 58 (26.5%) patients had adverse effects, which in 11 (4.9%) were severe. The median (IQR) cost per patient was € 2,943.07 (541.9 – 5,872.54). The publication European Journal of Clinical Pharmacology 2013;69:1689-99 describes the indications for use, the available scientific evidence and clinical results in the short and long term, and the cost of off-label use of rituximab. A total of 101 cases of off-label rituximab use were analyzed (median [IQR] age 53 years [37.5-68.0]; 55.4% women). The requested indications were mainly haematological diseases (46%), systemic connective tissue disorders (27%) and kidney diseases (20%). Available evidence in these indications were mainly individual cohort studies (53.5% of cases), and case series (25.7%). Short-term outcome (median 3 months [IQR 2-4]) was a complete response in 38% of cases and partial response in 32.6%. The highest short-term responses were observed for systemic lupus erythematosus and membranous glomerulonephritis, and the lowest for neuromyelitis optica and idiopathic thrombocytopenic purpura. Some response was maintained in long-term follow-up (median 23 months [IQR 12-30]) in 69.2% of patients with short-term response. Median cost per patient was € 5,187.5 (IQR 5,187.5-7,781.3).

While the cost of health care rises in all public healthcare organizations, budgets for that care have remained the same or have decreased. This is most certainly true in correctional settings. Because pharmaceutical expenditures are a substantial percentage of a health care organization’s budget, medication utilization is closely scrutinized. Clinicians must consider the appropriateness, effectiveness, and safety of medications prescribed to incarcerated patients. The abundance of available drugs and the complex issues with respect to their safe and effective use make a sound program for maximizing rational drug use critical. This is a challenging task in jails and prisons that requires a reexamination of the treatments provided. This is not a process of arbitrarily limiting prescriber choices or their decision-making authority solely based on cost-saving incentives. Evidence-based, best practices that inform the development of, and adherence to, disease management guidelines and a preferred, restricted medication formulary enhances the quality, safety, and effectiveness of the care provided. This chapter details the process and procedures to develop, implement, and monitor prescription practice change by establishing an effective Pharmacy & Therapeutics Committee (P & TC). The chapter further addresses: the roles and responsibilities of a P & TC; P & TC decision-making processes; formulary development and modification; formulary process decision-making; medication therapy management guidelines; prescriber education; and data analytics to assist in monitoring outcomes, medication use, and prescriber adherence to P & TC policies.

This chapter focuses on the purpose of formularies and the potential ethical problems or issues that formularies raise. Pharmacists will increasingly find themselves on committees, such as Pharmacy and Therapeutics Committees, that set the standards for formularies used by health system pharmacies and insurers responsible for paying for the cost of pharmaceuticals. Some ethical principles that are relevant to decisions on setting limits on prescribing and dispensing practices include justice, beneficence, and nonmaleficence. Reducing costs overall, which may serve the principle of justice, may threaten the well-being or limit the autonomy of a few. Cases in this chapter deal with these issues as well as the ethical justification for overriding formulary decisions.

Medicines management 256Evaluating new drugs 258How to write a drug protocol 260Unlicensed use of medicines 262Drug and therapeutics committees 264Patient group directions (PGDs) 266Supplementary prescribing 268Independent prescribing 272Community (FP10) prescription use in hospitals 274Electronic prescribing 276...