Current medical research and opinion
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To evaluate geographic variation in the prevalence of autosomal dominant polycystic kidney disease (ADPKD) in the US, including ADPKD at risk of rapid progression. ⋯ This estimate for ADPKD prevalence is consistent with previously reported national estimates, with regional variation suggesting that ADPKD might be under-diagnosed in rural areas with more limited access to care. More than one-third of ADPKD patients presented risk factors associated with rapid progression, highlighting the need for timely identification, as disease-modifying therapy may delay progression to end-stage renal disease.
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Limited real-world information exists on the characteristics or treatment patterns of patients with peripheral T-cell lymphoma (PTCL). We reported demographics, treatments and direct healthcare resource utilization (HRU) in a large cohort of US patients newly diagnosed with PTCL. ⋯ This retrospective analysis of patients with PTCL revealed high levels of comorbidity and HRU; novel interventions that improve patient outcomes and reduce the HRU burden of PTCL are needed.
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Favipiravir is a repurposed drug to treat coronavirus 2019 (COVID-19). Due to a lack of available real-world data, we assessed its effectiveness and safety in moderately to critically ill COVID-19 patients. ⋯ Favipiravir was associated with clinical benefits, including accelerated discharge rate and less progression to mechanical ventilation; however, no overall mortality benefits were seen across the severity spectrum.
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ABP 501 is a biosimilar to the anti-tumor necrosis factor-alfa monoclonal antibody adalimumab and despite its effectiveness and safety in the treatment of psoriasis was demonstrated in randomized clinical trials, no real-life data are available, in particular in patients undergoing non-medical switch from originator to biosimilar. ⋯ AB- 501 is an effective treatment for plaque-type psoriasis and psoriatic arthritis regardless if patients are originator-naïve or if they were switched from the reference product.
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Those invested in the outcome of specific decisions may seek to inform the decision-maker by generating relevant evidence. With multiple decision-makers, the evidence generator benefits from understanding whether their needs differ. This was explored using relevant stakeholders' preferences on quality indicators (QIs) of rare disease patient registries (RDRs), a common vehicle for generating evidence, to support the adoption of new medicines. ⋯ A multiple stakeholder approach for generating real-world evidence can be justified. The potential mismatch of 54.9% between pharma and non-pharma stakeholders indicates a critical misalignment between generator and consumer of its RWE. Pharmaceutical SHG's were highlighted for greater alignment with Patients and Payor (non-pharma) groups.