Publications by authors named "Mark Needles"
Background: Desirability of outcome ranking (DOOR) is an innovative approach to clinical trial design and analysis that uses an ordinal ranking system to incorporate the overall risks and benefits of a therapeutic intervention into a single measurement. Here we derived and evaluated a disease-specific DOOR endpoint for registrational trials for hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia (HABP/VABP).
Methods: Through comprehensive examination of data from nearly 4000 participants enrolled in six registrational trials for HABP/VABP submitted to the Food and Drug Administration (FDA) between 2005 and 2022, we derived and applied a HABP/VABP specific endpoint.
Article Synopsis
- - Allergic bronchopulmonary aspergillosis and invasive fungal diseases are serious health issues that lead to high rates of illness and death, but current inhaled antifungal treatments are not effective and can cause negative side effects.
- - The development of new inhaled antifungal therapies is complicated by issues like a lack of proper testing models, diverse patient backgrounds, and difficulty establishing clear clinical trial criteria and outcomes.
- - On September 25, 2020, the FDA held a workshop with experts to discuss ways to encourage the development of inhaled antifungal drugs, address the limitations of current research methods, and gather insights from patients and the industry.
Article Synopsis
- - The study focused on a new method called Desirability of Outcome Ranking (DOOR), which combines safety and efficacy measures in clinical trials for complicated intra-abdominal infections (cIAI) to assess overall patient outcomes.
- - Researchers developed a cIAI-specific DOOR endpoint using data from 9 Phase 3 trials, analyzing how well patients responded to study treatments compared to control groups in terms of health outcomes.
- - Key findings showed that patients with worse outcomes experienced more complications and surgeries, and the DOOR estimates indicated similar probabilities of better outcomes between treatment arms, demonstrating a structured way to evaluate clinical experiences in such trials.