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.
Cochrane Database of Systematic Reviews Data collection and analysisWe compared the intervention of discontinuing or reducing antihypertensive medication to usual treatment using mean di erences (MD) and 95% confidence intervals (95% CIs) for continuous variables and we used Peto odds ratios (ORs) and 95% CI for binary variables. Our primary outcomes included: mortality, myocardial infarction, development of adverse drug reactions or adverse drug withdrawal reactions. Secondary outcomes included: blood pressure, hospitalisation, stroke, success of withdrawing from antihypertensives, quality of life, and falls. Two authors independently, and in duplicate, conducted all stages of study selection, data extraction and quality assessment.
Economic evaluations of hospital and community pharmacy services are becoming increasingly commonplace to enable an understanding of which health care services provide value for money and to inform policy makers as to which services will be cost-effective in light of limited health care resources.
Background The Dundee classification of cellulitis severity, previously shown to predict disease outcomes, provides an opportunity to improve the management of patients with cellulitis. Methods We developed and implemented a pathway to guide the management of adults with cellulitis based on their Dundee severity class, and measured its effect on patient outcomes. We compared the outcomes in patients admitted to Auckland City Hospital (ACH) between July 2014 and July 2015 (the baseline cohort) with those in patients admitted between June 2017 and June 2018 (the intervention cohort). Results The median length of stay was shorter in the intervention cohort (0.7 days, IQR 0.1 to 3.0 days) than in the baseline cohort (1.8 days, IQR 0.1 to 4.4 days; P<0.001). The 30 day mortality rate declined from 1.8% (19/1092) in the baseline cohort to 0.7% (10/1362; P=0.02) in the intervention cohort. The 30 day cellulitis readmission rate increased from 6% in the baseline cohort to 11% (P<0.001) in the intervention cohort. Adherence to the ACH cellulitis antibiotic guideline improved from 38% to 48% (P<0.01) and was independently associated with reduced length of stay. Conclusions The implementation of the Auckland cellulitis pathway, readily generalizable to other settings, improved the outcomes in patients with cellulitis, and resulted in an annual saving of approximately 1,000 bed days.
This review systematically evaluated the current literature and identified key areas of difference in policy between Australia and NZ. Australia appears to cover and reimburse a greater number of medicines, while New Zealand achieves much lower prices for medicines than their Australian counterparts and has been more successful in controlling national pharmaceutical expenditure. Delays in patient access to new therapies in New Zealand have considerable implications for overall patient access to medicines; however, higher patient co-payments and relative pharmaceutical expenditure in Australia and its effect upon patient access to medicines must also be considered.
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