Failures of Phase III programs after successful Phase II programs is probably the worst outcome of a clinical development program, as it failed in the most costly way. Nevertheless, these failures occur not infrequently. In psychiatry, highly publicized Phase II success stories ended in discontinuations of development programs, such as the NK1-antagonist program in depression several years ago. More recently, other examples have emerged. Some skip the Phase II process altogether with designs, which are supposed to provide “pivotal” data for regulatory purposes in large Phase III-like studies, which are just labeled as Phase II. These failures do not come out of the blue. Sometimes it is important to go back to basics and consider the purpose of Phase II trials.
What is the purpose of a Phase II trial?
The purpose of Phase II trials, besides gaining insights into the safety of a compound, is broadly exploratory, i.e. to generate data, which help with the design of the pivotal Phase III program. In a therapeutic area, a reasonably performed Phase II study can provide insights into clinical and biological patient characteristics, which match the properties of the drug under study. With an increased interest in personalized medicine, these boundaries between patient populations have to be understood in order to be successful. This approach is in direct contradiction to the urge to generate a “pivotal” Phase II outcome. Continue reading
There should be no doubt that clinical trials in Alzheimer’s disease and dementia need to be faster and more cost-effective if new treatments are to reach patients.
Public interest in participation in clinical trials for dementia is growing as governments become more vocal about the need to find treatments. A number of well-known organizations have well-established processes for linking patients with clinical trials. Alzheimer’s Association’s Trialmatch in the US and the United Kingdom’s Clinical Research Network (UKCRN) Study Portfolio allow patients to search for trials that they would like to participate in. In the case of dementia however, the disease may hinder a person’s ability to search for trials, potentially limiting access to experimental treatments. As research focuses more on prevention of Alzheimer’s it is important to include people interested in research who have no symptoms of memory loss. Continue reading
Testing drives drug development. From laboratory tests on patient specimens comes almost all of the clinical data needed for a new drug application. How and where those specimens are collected, transported, stored, and analyzed impacts the quality and usefulness of the data they produce. In the past, most tests were processed by local, academic, and specialized testing laboratories and coordinated by each investigator. However, centralized testing is becoming an accelerated trend – one that uses advanced technology and global operations to concentrate oncology clinical trial tests in a single, central laboratory.
The core value of a central lab is consistency. When local laboratories perform testing, their results will be different and results vary over the course of the trial. Central laboratory testing, on the other hand, offers ‘combinable data.’ The end product is that a result from a central laboratory is similar regardless of the global location where it originated from and the lab location where it is tested. At all of Covance’s central laboratories — in Indianapolis, Geneva, Singapore, Shanghai, and Tokyo — we generate data from the same analytical method platform, SOPs, equipment, reagents, and standards, eliminating variables that affect tests results. Continue reading
Throughout my career at Aventis, Hoffmann-La Roche and Alcon (a Novartis company), we focused on the proof of concept (PoC) as the critical milestone when considering when to partner with or purchase a compound from a small biotech.
Your potential pharma partner is looking for answers to key development questions surrounding the mechanism of action and your compound, how your company has addressed those, the robustness of the results and the quality of the data. This drives their evaluation of the real stage of development and the valuation. Continue reading