ObjectiveTo review existing regulations and policies utilised by countries to enable patient access to orphan drugs.MethodsA review of the literature (1998 to 2014) was performed to identify relevant, peer-reviewed articles. Using content analysis, we synthesised regulations and policies for access to orphan drugs by type and by country.ResultsFifty seven articles and 35 countries were included in this review. Six broad categories of regulation and policy instruments were identified: national orphan drug policies, orphan drug designation, marketing authorization, incentives, marketing exclusivity, and pricing and reimbursement. The availability of orphan drugs depends on individual country’s legislation and regulations including national orphan drug policies, orphan drug designation, marketing authorization, marketing exclusivity and incentives such as tax credits to ensure research, development and marketing. The majority of countries (27/35) had in place orphan drug legislation. Access to orphan drugs depends on individual country’s pricing and reimbursement policies, which varied widely between countries. High prices and insufficient evidence often limit orphan drugs from meeting the traditional health technology assessment criteria, especially cost-effectiveness, which may influence access.ConclusionsOverall many countries have implemented a combination of legislations, regulations and policies for orphan drugs in the last two decades. While these may enable the availability and access to orphan drugs, there are critical differences between countries in terms of range and types of legislations, regulations and policies implemented. Importantly, China and India, two of the largest countries by population size, both lack national legislation for orphan medicines and rare diseases, which could have substantial negative impacts on their patient populations with rare diseases.

Nowadays, only a few data are available about left heart unloading in V-A ECMO support. Despite the well-known controversy, IABP remains widely used in combination with V-A ECMO. Percutaneous approaches utilizing unloading devices is becoming an increasingly used option. However, further studies are required to establish the optimal LV unloading method.

Patient-reported outcomes (PROs) are a distinctive method of evaluating patient response to health care or treatment. This systematic review aimed to analyse the impact of PROs in patients on direct oral anticoagulant (DOAC) treatment, prescribed for any indication (e.g. venous thromboembolism treatment or atrial fibrillation) using controlled trials (CT) and real-world observational studies (OS).Methods: A systematic search of articles was conducted according to PRISMA guidelines using databases, with the last update in November 2018. The Cochrane Collaboration tool for assessing bias in randomized CTs and the Newcastle-Ottawa Scale adapted for cross-sectional studies were used. Outcomes evaluated were related to health-related quality of life (HRQoL), satisfaction, adherence and compliance.Results: Twenty-one original studies (6 CT, 15 OS) were included. HRQoL was assessed by 6 (1 CT, 5 OS) studies and reported that HRQoL scores were similar in patients on DOACs and warfarin. Patients prescribed DOACs presented higher HRQoL scores which were attributed to lack of intense monitoring required compared with warfarin but this was not statistically significant. The majority of studies (5 CT, 9 OS) investigated patient-reported satisfaction, indicating greater satisfaction with DOACs with significantly lower burden and increased benefit scores for patients on DOACs. Patient-reported expectations, compliance and adherence were similar for patients on DOACs and warfarin. Conclusion:Patients appear to prefer treatment with DOACs vs warfarin. This is shown by the higher quality of life, satisfaction and adherence described in the studies. However, heterogeneity in the analysed studies does not allow firm conclusions.

2 Objective 3To review the effects of pharmaceutical care on hospitalizations, mortality and clinical outcomes in 4 patients. 5 Methods 6Systematic searches were conducted in MEDLINE, EMBASE and International Pharmaceutical Abstracts 7 (IPA) databases to identify studies that were published between 2004 and January 2017. Studies 8 included in this review were randomized controlled trials (RCTs) that spanned across both community and 9 hospital settings. Using strict inclusion/exclusion criteria studies were included if they reported level 1 or 2 10 outcomes in the hierarchy of outcome measure i.e. clinical and surrogate outcomes (e.g. blood pressure 11(BP) control, blood glucose level, cholesterol BMI). Each study was assessed for quality using the Jadad 12 scoring system. 13 Results 14Fifty-four RCTs were included in the present review. Forty-six of these studies ranked high quality 15 according to the Jadad scoring system. Studies were categorized into their general condition groups. 16Interventions in patients with diabetes, depression, respiratory disorders, cardiovascular disorders, 17 epilepsy, osteoporosis, and interventions in older adults were identified. In the majority of studies