Explore the vast range of titles available.

Dupilumab is a monoclonal antibody targeting IL-4Rα recently licensed for severe asthma (SA). A Named Patients Program (NPP) was created in Italy before its commercial availability for SA patients with no other available therapeutic options. We aimed to assess the real-world effectiveness of dupilumab in patients with SA and unmet needs. We performed a multicentre retrospective study, including SA patients admitted to the NPP treated with dupilumab for 12 months. Data on the number of exacerbations, Asthma Control Test (ACT), pre-bronchodilator FEV %, oral corticosteroids (OCSs) use, FeNO and eosinophils count in peripheral blood were recorded at baseline and after 3, 6, and 12 months. We included 18 SA patients (mean age 53.3±12.4 years, 66.7% female). Eleven (61.1%) were OCSs dependent. Five patients (27.8%) received previous anti-IgE and/or anti-IL-5 agents. A significant improvement in ACT score (from 15.7±5.1 to 18.8±4.8, p=0.023), OCSs intake [10 (5-25) mg/day to 0 (0-5) mg/day, p=0.0333] and FeNO [from 25 (20-80) ppb to 21 (10.9-55.3) ppb, p=0.0190] was already detected after 3 months of treatment. After 12 months, a statistically significant decrease in the number of exacerbations from 2 (0-3) to 0 (0-1) (p<0.0068) and increase in FEV % from 73.5±19.5% to 87.1±19.2% (p=0.0407) and ACT to a mean value of 22.4±1.7 (p<0.0001) and the interruption of OCSs in all the patients (p<0.0001) was observed. A transient increase in the eosinophil count was observed in five patients (above 1000 cells/μL in 2 cases) after 3 months, without any clinical effect. Dupilumab improved all the explored clinical outcomes after 12 months, and the transient hypereosinophilia did not modify treatment response. These real-world data confirm the results reported in randomized controlled trials and provide an important opportunity to characterize the clinical impact of the treatment in a non-trial setting. Further real-world studies with a larger cohort of patients are needed to confirm these findings.

Early access programs, (EAPs) are adopted by an increasing number of pharma companies due to several benefits offered by these programs. EAPs offer ethical, compliant, and controlled mechanisms of access to investigational drugs outside of the clinical trial space and before the commercial launch of the drug, to patients with life-threatening diseases having no treatment options available. In addition to the development of positive relationships with key opinion leaders (KOL), patients, advocacy groups and regulators, the data captured from the implementation of EAPs supports in the formulation of global commercialization strategies. This white paper outlines various circumstances to be considered for the implementation of EAPs named patient programs, the regulatory landscape, the benefits and challenges associated with implementing these programs and the key considerations for their successful implementation.

Background Intravenous (IV) artesunate is the treatment of choice for severe malaria. In Europe, however, no GMP-manufactured product is available and treatment data in European travellers are scarce. Fortunately, artesunate became available in the Netherlands and Belgium through a named patient programme. This is the largest case series of artesunate treated patients with severe malaria in Europe. Methods Hospitalized patients treated with IV artesunate between November 2007 and December 2010 in the Netherlands and Belgium were retrospectively evaluated. Patient characteristics, treatment and clinical outcome were recorded on a standardized form and mortality, parasite clearance times and the occurrence of adverse events were evaluated. Results Of the 68 treated patients, including 55 with severe malaria, two patients died (2/55 = 3.6%). The mean time to 50% parasite clearance (PCT50), 90% and 99% were 4.4 hours (3.9 - 5.2), 14.8 hours (13.0 - 17.2), and 29.5 hours (25.9 - 34.4) respectively. Artesunate was well tolerated. However, an unusual form of haemolytic anaemia was observed in seven patients. The relationship with artesunate remains uncertain. Conclusions Data from the named patient programme demonstrate that IV artesunate is effective and well-tolerated in European travellers lacking immunity. However, increased attention needs to be paid to the possible development of haemolytic anaemia 2-3 weeks after start of treatment. Treatment of IV artesunate should be limited to the period that IV treatment is required and should be followed by a full oral course of an appropriate anti-malarial drug.

Background The standard first- and second- line chemotherapy backbone regimens for metastatic colorectal cancer (mCRC) are 5-fluorouracil (5-FU)/capecitabine-based with addition of irinotecan or oxaliplatin. Until recently, evidence for optimal sequencing post second-line was sparse. Trifluridine/tipiracil (indicated for mCRC and gastric cancer after standard chemotherapies) was made available to UK patients via a named patient programme (NPP) before receiving marketing authorisation in Europe in 2016, allowing characterisation of UK treatment pathways, and evaluation of trifluridine/tipiracil in a UK non-trial population. Methods Data collected routinely for the NPP were analysed to describe the patient demographics, clinical characteristics and treatment pathways. Patients eligible for the programme were adults (≥18 years) with histologically or cytologically confirmed mCRC who had previously received chemotherapy treatment(s). Results Of the 250 eligible patients enrolled in the NPP, 194 patients received ≥1 dose of trifluridine/tipiracil and 56 patients did not receive trifluridine/tipiracil. The following results are reported first for patients who received trifluridine/tipiracil and second for those who did not receive trifluridine/tipiracil: median (IQR) age was 63.0 (54.0–69.0) and 62.0 (54.8–69.0) years; Eastern Cooperative Oncology Group performance status score was 0 for 28 and 14%, 1 for 65 and 70%, 2 for 7 and 16%. In terms of previous systemic treatments 47 and 43% had 2 prior lines of therapy. FOLFOX-, FOLFIRI- and CAPOX-based therapies were the most common first-line regimens in patients receiving trifluridine/tipiracil (37, 35 and 21%, respectively), and in patients not receiving trifluridine/tipiracil (41, 30 and 20%, respectively). Second-line treatment regimens in patients receiving and not receiving trifluridine/tipiracil were most commonly FOLFIRI-based (48 and 41%, respectively) and FOLFOX-based (19 and 21%, respectively). Patients received a median of 2 cycles of trifluridine/tipiracil with a median treatment duration of 1.8 (95% CI: 1.8–2.4) months. In patients who discontinued treatment due to disease progression, the median progression-free duration was 2.8 (95% CI: 2.4–2.9) months. Conclusions The results highlight the number of treatment pathways used to treat mCRC in routine UK clinical practice prior to the marketing authorisation and National Institute for Health and Care Excellence approval of trifluridine/tipiracil and highlight the lack of clinical guidelines for mCRC.

Background Intravenous (IV) artesunate is the treatment of choice for severe malaria. In Europe, this treatment is only available in a few countries via named patient programmes (NPPs). As a case study, the legal and organisational aspects and pharmacovigilance of these NPPs and possibilities for harmonisation within the EU were studied over time and space using IV artesunate (Malacef) in the Netherlands, Belgium and France. Methods The legal base and organisation of NPPs in the Netherlands, Belgium and France were studied. The diffusion and cumulative availability of IV artesunate and the pharmacovigilance components were compared among the three countries using distribution data from the period 2007 through 2012. Results Artesunate has quickly gained acceptance for treating severe malaria in the Netherlands, whereas both Belgium and France have introduced this treatment more hesitantly. This difference in acceptance is due to differences in the implementation of NPP legislation among the countries. France currently has a proactive system in which treatment requires the permission for each patient and an intensive follow-up protocol. On the other hand, Belgium and Dutch NPPs are more dependent on the investigators’ initiative and are therefore potentially faster and more flexible, facilitating the discovery of adverse effects that have not been reported by more formal comparative clinical trials. Conclusions NPPs provide a unique opportunity to study both the benefits and risks of unregistered products for treating rare diseases, provided that the patients are actively vigilated. Thus, we recommend that NPPs should be harmonised throughout Europe in order to ensure equal availability of treatment and therapeutic benefit to all Europeans without compromising patient safety.

Since 1972, many victims of endstage renal disease (ESRD) have received treatment under a unique Medicare entitlement. This book presents a comprehensive analysis of the federal ESRD program: who uses it, how well it functions, and what improvements are needed.The book includes recommendations on patient eligibility, reimbursement, quality assessment, medical ethics, and research needs.Kidney Failure and the Federal Government offers a wealth of information on these and other topics:The ESRD patient population.Dialysis and transplantation providers.Issues of patient access and availability of treatment.Ethical issues related to treatment initiation and termination.Payment policies and their relationship to quality of care.This book will have a major impact on the future of the ESRD program and will be of interest to health policymakers, nephrologists and other individual providers, treatment site administrators, and researchers.

In healthcare settings, the term ‘mentorship’ is normally used to describe the supervision of a pre-registration student by a qualified practitioner. Mentorship can be very formal or relatively informal. It can also be practised differently in particular locations, settings and healthcare professions.This clear, concise book transcends professional and geographical boundaries in order to focus on the essential characteristics of effective mentorship. It will therefore be useful to a very wide range of healthcare professionals who are involved in mentoring and assessing junior colleagues.The book examines learning theories, teaching and communication skills and assessment methods. It also contains helpful advice on dealing with overseas students and students with special needs. Activities encourage reflection, and quotations and tables enable readers to absorb the content and relate theory to practice.In this second edition, the text has been made even clearer and the authors have added further detail on learning theories such as social constructivism. Most importantly, they have added an Afterword written in the light of reports on the Mid Staffordshire NHS Foundation Trust Public Inquiry. This final section places new emphasis on the mentor’s role in helping to ensure that patients receive safe and effective care, which is provided with compassion as well as practical skill.

We know more about cancer prevention, detection, and treatment than ever before-yet not all segments of the U.S. population have benefited to the fullest extent possible from these advances. Some ethnic minorities experience more cancer than the majority population, and poor people-no matter what their ethnicity-often lack access to adequate cancer care. This book provides an authoritative view of cancer as it is experienced by ethnic minorities and the medically underserved. It offers conclusions and recommendations in these areas: Defining and understanding special populations, and improving the collection of cancer-related data. Setting appropriate priorities for and increasing the effectiveness of specific National Institutes of Health (NIH) research programs, to ensure that special populations are represented in clinical trials. Disseminating research results to health professionals serving these populations, with sensitivity to the issues of cancer survivorship. The book provides background data on the nation's struggle against cancer, activities and expenditures of the NIH, and other relevant topics.

In Nursing Civil Rights, Charissa J. Threat investigates the parallel battles against occupational segregation by African American women and white men in the U.S. Army. As Threat reveals, both groups viewed their circumstances with the Army Nurse Corps as a civil rights matter. Each conducted separate integration campaigns to end the discrimination they suffered. Yet their stories defy the narrative that civil rights struggles inevitably arced toward social justice. Threat tells how progressive elements in the campaigns did indeed break down barriers in both military and civilian nursing. At the same time, she follows conservative threads to portray how some of the women who succeeded as agents of change became defenders of exclusionary practices when men sought military nursing careers. The ironic result was a struggle that simultaneously confronted and reaffirmed the social hierarchies that nurtured discrimination.

The rhetoric of 'needs' has been used to legitimate all major turns in UK health policy since 1936. This study identifies the ethical, policy and technical issues arising from the concept of needs. In the first part a theory of needs is developed, which takes into account both the philosophical traditions and the practical problems arising in daily health care. In a second part, health systems throughout the world are described and compared, addressing ethical as well as economical questions. Its interdisciplinary approach will make The Need for Healthcare important reading not only for those interested in or employed in the health care sector but also for students of philosophy.