"Stick or Twist?" Negotiating Price and Data in an Era of Conditional Approval.

Background: Changes in the regulatory context enable faster approval of transformative medicines. They also lead to health technology assessment (HTA) agencies having to make decisions with less evidence. In response, HTA agencies have also initiated forms of conditional approval.

Principles for consistent value assessment and sustainable funding of orphan drugs in Europe.

The European Orphan Medicinal Products (OMP) Regulation has successfully encouraged research to develop treatments for rare diseases resulting in the authorisation of new OMPs in Europe. While decisions on OMP designation and marketing authorisation are made at the European Union level, reimbursement decisions are made at the national level. OMP value and affordability are high priority issues for policymakers and decisions regarding their pricing and funding are highly complex.

Ischaemic stroke and bleeding rates in 'real-world' atrial fibrillation patients.

Stroke prevention guidelines recommend oral anticoagulants (OAC) for atrial fibrillation (AF) patients at moderate/high risk of stroke, and antiplatelet or no therapy for those at low/moderate risk. Outcomes for AF patients receiving antiplatelet/no therapy in 'real-life' clinical practice were explored. This study compared clinical event rates (stroke/bleeding) for AF patients treated with OAC therapy, antiplatelets or no therapy in usual clinical practice to event rates in OAC-treated AF patients from optimally-monitored 'real-life' settings (anticoagulation clinics).

Characterization of the proportion of untreated and antiplatelet therapy treated patients with atrial fibrillation.

Despite the efficacy of oral anticoagulants for stroke prevention in atrial fibrillation (AF), evidence suggests that many patients with AF who should be treated with vitamin K antagonists (VKAs) are treated with antiplatelet therapy or remain untreated. The aims of this study were to determine the proportion of patients with AF in each treatment category in clinical practice and to ascertain whether treatment is appropriate for stroke risk. An extensive search of the biomedical research published since 1994 was performed.

Underuse of oral anticoagulants in atrial fibrillation: a systematic review.

Background: Atrial fibrillation is associated with substantial mortality and morbidity from stroke and thromboembolism. Despite an efficacious oral anticoagulation therapy (warfarin), atrial fibrillation patients at high risk for stroke are often under-treated. This systematic review compares current treatment practices for stroke prevention in atrial fibrillation with published guidelines.

A national survey of the management of atrial fibrillation with antithrombotic drugs in Italian primary care.

The aims of this study were to investigate trends in the incidence of diagnosed atrial fibrillation (AF), and to identify factors associated with the prescription of antithrombotics (ATs) and to identify the persistence of patients with oral anticoagulant (OAC) treatment in primary care. Data were obtained from 400 Italian primary care physicians providing information to the Health Search/Thales Database from 2001 to 2004. The age-standardised incidence of AF was: 3.

Reimbursement agency requirements for health related quality-of-life data: a case study.

A review was undertaken to identify relevant and appropriate heath-utility estimates for patients with atrial fibrillation who had stroke and to appraise them against the published requirements for several countries' Health Technology Assessment agencies: Australia (Pharmaceutical Benefits Advisory Committee), Canada (Common Drug Review), England and Wales (NICE), Germany (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen), Scotland (Scottish Medicines Consortium) and Sweden (Tandvårds-och läkemedelsförmånsverket). National agencies have created guidelines to support economic evaluations for their own countries but these guidelines differ. It may be more appropriate for agencies to be concerned primarily with the methodological quality of studies that report utilities rather than identifying local values.

Methods for integrating medication compliance and persistence in pharmacoeconomic evaluations.

Objectives: Suboptimal compliance and failure to persist with drug treatments are important determinants of therapeutic nonresponse and are of potential economic significance. The present article aims to describe the methodologies that may be appropriate for integrating noncompliance and nonpersistence in economic evaluations.

Methods: MEDLINE and NHS-EED were searched for economic evaluations published in the period between 1997 and 2005.

The association between compliance and persistence with bisphosphonate therapy and fracture risk: a review.

Background: Sub optimal levels of compliance and persistence with bisphosphonates are potentially compromising the reduction of post menopausal osteoporotic (PMO) fracture risk.

Methods: A structured literature search (1990-2006) was performed to identify primary research studies evaluating the relationship between compliance and persistence with bisphosphonates and post menopausal osteoporotic (PMO) fracture risk in clinical practice. Search criteria were: bisphosphonates; osteoporosis/osteopenia in postmenopausal women; all types of fractures; compliance and persistence.

Recombinant erythropoietin for chemotherapy-related anaemia: economic value and health-related quality-of-life assessment using direct utility elicitation and discrete choice experiment methods.

Objective: To assess both the health-related quality of life (HR-QOL) and the economic value of erythropoietin treatment in chemotherapy-related anaemia using direct utility elicitation and discrete choice experiment (DCE) methods from a societal perspective in the UK.

Methods: The time trade-off (TTO) method was employed to obtain utility values suitable for the calculation of QALYs for no, mild, moderate and severe anaemia. Health-state descriptions were developed using the Functional Assessment of Cancer Therapy - Anaemia (FACT-AN) subscale and the EQ-5D questionnaires, and were validated by clinical experts and patients.

The effect of dosing frequency on compliance and persistence with bisphosphonate therapy in postmenopausal women: a comparison of studies in the United States, the United Kingdom, and France.

Objectives: The aim of this study was to determine the effect of dose frequency on compliance and persistence with bisphosphonate therapy in postmenopausal women and to compare findings from 3 different health care systems.

Methods: Three independently performed retrospective cohort analyses were performed using observational data sources. In the United States, bisphosphonate-naive postmenopausal women were identified from a database providing information on health plan prescription drug claims; in the United Kingdom and France, bisphosphonate-naive postmenopausal women were identified from a database of medical records supplied by general practice physicians.

Assessing the total costs of blood delivery to hospital oncology and haematology patients.

Objective: To determine direct costs associated with a blood transfusion session in two hospital settings.

Research Design And Methods: The study was conducted in two United Kingdom hospital sites during April 2004. Transfusion sessions for patients receiving units of red blood cells within either haematology or oncology departments were followed using time and motion techniques to measure the direct costs.

The impact of dosing frequency on compliance and persistence with bisphosphonates among postmenopausal women in the UK: evidence from three databases.

Background: Bisphosphonates are currently among the most effective therapies for the treatment of osteoporosis and provide one of the mainstays of treatment in the UK. However studies in several countries have all reported sub-optimal compliance and persistence with treatment.

Objective: To examine the impact of dosing frequency on compliance and persistence with bisphosphonates in the UK.