Ultra-orphan drugs are medicines used to treat exceptionally rare diseases that are chronically debilitating or life-threatening. Low patient numbers make it difficult for pharmaceutical companies to recoup research and development costs, and consequently these medicines are generally expensive on a per patient basis. European Union (EU) regulations promote the development of orphan drugs; but to contain costs, some EU healthcare systems assess the cost-effectiveness of therapies when deciding if they should be funded. As ultra-orphan drugs are invariably cost-ineffective, factors in addition to cost-effectiveness need to be considered if ultra-orphan drugs are to be provided by public health services. Health service funding of ultra-orphan drugs, which varies across the EU and within the UK, has led to geographical inequities in patients' access to treatment. In some instances, support for these drugs would appear to have been approved on the basis that diseases that are rare and severe are a special case. We explore whether ultra-orphan drugs merit special status by considering efficiency, effectiveness and equity criteria. Mechanisms are discussed for creating a policy that would reduce geographical inequalities in provision across Europe.

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ObjectiveTo examine the impact of a 5-week national lockdown on ambulance service demand during the COVID-19 pandemic in New Zealand.DesignA descriptive cross-sectional, observational study.SettingHigh-quality data from ambulance electronic clinical records, New Zealand.ParticipantsAmbulance records were obtained from 588 690 attendances during pre-lockdown (prior to 17 February 2020) and from 36 238 records during the lockdown period (23 March to 26 April 2020).Main outcome measuresAmbulance service utilisation during lockdown was compared with pre-lockdown: (a) descriptive analyses of ambulance events and proportions of event types for each period, (b) absolute rates of ambulance attendance (event types/week) for each period.ResultsDuring lockdown, ambulance patients were more likely to be attended at home and less likely to be aged between 16 and 25 years. There was a significant increase in the proportion of lower acuity patients (Status 3 and Status 4) attended (p<0.001) and a corresponding increase in patients not transported from scene (p<0.001). Road traffic crashes (p<0.001) and alcohol-related incidents (p<0.001) significantly decreased. There was a decrease in the absolute number of weekly ambulance attendances (ratio (95% CI), 0.89 (0.87 to 0.91), p<0.001), attendances to respiratory conditions (0.74 (0.61 to 0.86), p=0.01), and trauma (0.81 (0.77 to 0.85), p<0.001). However, there was a significant increase in ambulance attendances for mental health conditions (1.37 (1.22 to 1.51), p=0.005).ConclusionsDespite the relative absence of COVID-19 in the community during the 5-week nationwide lockdown, there were significant differences in ambulance utilisation during this period. The lockdown was associated with an increase in ambulance attendances for mental health conditions and is of concern. In considering future lockdowns, the potential implications on a population’s mental well-being will need to be seriously considered against the benefits of elimination of virus transmission.

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