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Participant recruitment is challenging, especially in those indications in which there are competing clinical trials necessitating a comprehensive understanding of the disease research ‘landscape’ and targeted outreach and stakeholder collaboration. Read on to learn key lessons in rare disease drug development.
Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?
As the research and medical community learns more about population variances in disease outcomes, whether as a result of regional genetic profiles, socioeconomic factors, or environmental inputs, it’s become necessary to ensure that clinical research trials include broad swaths of global participants. billion in 2019.
Experts discuss the therapeutic landscape, treatment gaps, regulatory considerations and clinical trial strategies. Breast Cancer Multiomics: Unified Insights in Tumor Heterogeneity This on-demand webinar introduces a cutting-edge single-cell multiomics method designed for garnering detailed genetic insights into breast cancer.
Having presented the data from the 24-month phase 3 trial at the American Academy of Ophthalmology (AAO) annual meeting, Dr Wykoff set aside some time to go into further detail with pharmaphorum about how the results were received, and what glimmer of hope this might hold for patients with GA.
And even when Black people with AML have the same access to treatment as white patients, their survival is shorter — something genetic differences might explain. But even when patients of both races received the same treatments, Black patients’ outcomes were worse, clinical trial data showed.
Our findings could help to pick out those patients most likely to benefit from immunotherapy – and we’re keen to translate our work into clinical trials to test the benefit of different immunotherapeutic strategies to tackle this hard-to-treat form of pancreatic cancer.”.
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Data from the research were recently included in the company’s Investigational New Drug (IND) application to the FDA to initiate a Phase 2 clinical trial for COVID-19. Based on these studies, one of those drugs was licensed to Cantex Pharmaceuticals for the treatment of COVID-19 and other inflammatory lung diseases.
For example, antisense oligonucleotide (ASO)-based therapies have gained traction, with 100 Phase I clinical trials initiated and around 25 percent of these advancing to Phase II or Phase III trials in recent years. Furthermore, CRISPR/Cas9 presents a promising avenue for overcoming genetic diseases in the near future.
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