Respiratory studies are complex, and for seamless execution of these studies, we must account for specific therapeutic knowledge, familiarity with different therapeutic guidelines, and a deep understanding of how various instruments work and how to interpret their readouts.
The symptomatology of respiratory diseases, in general, is often variable. The disease intensity can vary based on environmental factors, smoking history, genetic factors, nutrition, etc. Patient education about the disease helps circumvent the factors influencing the high variability of study outcome measures. Therefore, including such educated and well-trained patients in clinical studies is imperative.
Accelsiors primary specialty is Rare Pulmonary Disorders. Many challenges surround the execution of Clinical trials in Rare Pulmonary Disorders, like access to patients, an investigator(s) proficiency in the studied disease, the complexity of trial logistics, the availability of measuring instruments, the validation state, and the availability of patient-reported outcome measures in different languages, various regulatory constraints, the available budget for the study conduct, and many other factors.
Another critical challenge in rare disease clinical trials is demonstrating a clinically meaningful and statistically significant response to treatment. Selecting the most appropriate, sensitive, regulatory compliant, and sound efficacy endpoints for these trials is Accelsiors, ultimate specialty. The types of endpoints are dependent on many factors. Among others, they should align with the stage of development (e.g., proof of concept vs. confirmation of clinical efficacy), the patient characteristics and disease stage, and the treatment goal, whether curing, improving, or preventing the disease. For rare diseases in general, so for respiratory disorders, demonstration of clinical benefit, defined as how a patient feels, functions, or survives, is an essential endpoint and should be demonstrated for product registration in at least one adequate and well-controlled pivotal study. Change in a single or even in several biomarkers is usually not enough for product registration. In some cases, however, full regulatory approval may be obtained based on a validated surrogate endpoint, while accelerated or provisional regulatory approval can be obtained using a biomarker likely to predict clinical benefit.
Clinically meaningful and relevant endpoints require informative natural history studies and patient input.
Patient-focused drug development (PFDD) meetings.