Introduction Orphan diseases are low-prevalence conditions with chronically debilitating or life-threatening consequences. Their treatments are generally called orphan drugs (OD). Health technology assessment processes have traditionally considered cost-effectiveness analysis (CEA), when making reimbursement and pricing decisions for healthcare plans. Valuing OD with standard CEA raises important issues due to uncertain evidence, inability to meet cost-effectiveness thresholds for reimbursement and high budget impact, among others. Multi-criteria decision analysis (MCDA) allows to overcome these issues and improve technical and ethical quality of decisions regarding prioritization, coverage and reimbursement of OD. Areas covered A scoping review was conducted in order to characterize MCDA frameworks for assessing OD and implementation experiences. We reviewed electronic databases (Medline, Embase, Cochrane Library, EBSCO, CINAHL, EconLit, Web of Science, LILACS, Google Scholar) key journals (Orphanet Journal of Rare Diseases and Value in Health) and organization repositories. Expert opinion The theoretical framework for MCDA considers areas related to characteristics of orphan diseases and their technologies’ clinical and economic impact. Participation processes are critical in incorporating societal values in weighting different dimensions and constructing decision rules. Local implementation pilots considering different stakeholders are necessary in order to pinpoint specific barriers and opportunities.

One-dose universal varicella vaccination (UVV) was introduced in the Argentinian National Immunization Program in July 2015. This study examined the impact of one-dose UVV on varicella incidence and mortality in Argentina. Incidence and mortality data were obtained from official databases for pre-UVV (January 2008–June 2015) and post-UVV (July 2015–December 2019) periods. Time series analyses with autoregressive integrated moving average (ARIMA) modeling predicted varicella incidence and mortality in absence of UVV in the target (aged 1–4 years) and overall population. Predicted and observed values post-UVV were compared to estimate UVV impact. Mean annual incidence rates per 100,000 reduced from 1999 (pre-UVV) to 1122 (post-UVV) in the target population and from 178 to 154 in the overall population. Significant declines in incidence were observed, reaching reductions of 83.9% (95% prediction interval [PI]: 58.9, 90.0) and 69.1% (95% PI: 23.6, 80.7) in the target and overall populations, respectively, during peak months (September-November) post-UVV. Decreasing trends in mortality rate from 0.4 to 0.2 per 1,000,000 population were observed. Over the last four years, one-dose UVV has significantly reduced varicella burden of disease in Argentina. Continuous efforts to improve vaccination coverage rates and long-term follow-up are needed to better understand the benefits of the UVV program.

The DAPA-HF study has shown that dapagliflozin added to standard treatment reduced the risks of worsening of heart failure or cardiovascular death compared to placebo.To evaluate the cost- utility ...

To assess the burden of disease of chronic pain in Colombia.We estimated disability adjusted life years (DALYs) attributable to chronic pain using Global Burden of Disease (GBD) method. We identifi...

SGLT2 inhibitors or DPP-4 inhibitors are among the preferred options in patients with type 2 diabetes mellitus (T2DM) without established cardiovascular disease. To evaluate the incremental cost-effectiveness of dapagliflozin versus DPP-4 inhibitors as a complement to metformin in the treatment ofT2D, from the perspective of the Colombian health system. The Cardiff model was used to estimate the incremental cost-effectiveness ratio (ICER) of dapagliflozin plus metformin compared to DPP-4 inhibitors plus metformin in adults with T2DM who did not respond adequately to metformin monotherapy. We estimated the incidence of micro- and macrovascular complications from risk equations incorporating the effect of treatment. The time horizon for analysis was 5 years and a discount rate of 5% was applied, for both costs and outcomes. The costs were expressed in 2020 USD (1 USD = $3,693.36 COP). Dapagliflozin in association with metformin resulted in a higher number of quality-adjusted life years (QALYs) compared to the intervention. The ICER was US$1,964.80 per QALY gained. From the point of view of Colombian healthcare system, the combination of dapagliflozin with metformin is a cost-effective option compared to DPP-4 + metformin inhibitors in the treatment of T2D without established cardiovascular disease.