From researchers to sponsors to patients to advocacy groups to clinicians, rare disease research is full of passionate, driven, and determined people. As the greatest asset to the field, all people involved must be utilized to their greatest potential for a study to be successful. That’s why significant attention must be paid to the operational challenges within...
Most trials for potential Alzheimer disease (AD) treatments fail—and the reason may be poor protocol design. After all, typical AD studies are double-blind placebo-controlled parallel group clinical trials with a dual outcome, including a cognitive measure and a global impression of aptitude for the activities of daily living—a trial design originally developed to study cholinesterase...
Phase I and II trials may have different overall goals (i.e., demonstrating safety vs. efficacy), but the two both struggle with a major challenge in oncology study design: finding the right dose. Luckily, decades of data and innovations have given researchers the tools necessary to plan a successful dose-finding trial. Read on for a look...
Sponsors of emerging hematologic therapies – including gene transfer and gene editing, adoptive cellular, and antibody-drug conjugates – face a unique set of challenges in conducting early-phase, dose-finding studies. Key considerations for developing early-phase trials that can more accurately define the recommended dose and identify adverse events for emerging therapies include: Using dose-finding trial designs...
This is the thirteenth installment of our look at the increasingly high placebo response that is plaguing clinical trials in analgesia and psychiatry. Read the rest of the posts in the series here. Until now, our Placebo Problem series has focused exclusively on the placebo response in adults. Today we turn to another population: kids....
Immuno-oncology is a unique approach to cancer treatment that leverages the body’s immune system to help fight cancer. In recent years, immune checkpoint inhibitors have changed the landscape of immunotherapy, and emerging therapies such as chimeric antigen receptor T-cells (CAR-T), dendritic cell vaccines and bi-specific T-cell engager (BiTE) antibodies are pushing the envelope even further....
While in many ways similar to pharmaceutical drug development, medical device development must work through some unique challenges, including: Variations in complexity — Devices may have multiple different constituent parts, including hardware, software, and medicinal components. Very different purposes — Almost all drugs are therapeutic, while devices may be therapeutic, diagnostic, monitoring, supportive, surgical, and more. Regulations regarding...
There is a great deal of variability when it comes to osteoarthritis (OA) clinical trial designs. From the type of drug being studied to the regulatory approach of the target market to the optimal route of administration, assessment selections must be carefully tailored to meet the unique needs of each individual trial. 1. Pick the...
For the estimated 30 million people worldwide who have Alzheimer’s disease, progress toward understanding and treating this most prevalent form of dementia is frustratingly slow. The few approved drugs address only the condition’s symptoms, though scores of drugs to prevent onset or alter the disease’s course are now under study. From high screen failure rates...
This is the next installment of our look at the increasingly high placebo response that is plaguing clinical trials in analgesia and psychiatry. Check out the other posts here. Over the past few weeks, we’ve discussed the psychological, neurobiological, and genetic mechanisms responsible for the placebo response. Today, we turn to the study designs used...