Trial Design

FDA Commissioner Scott Gottlieb supports approaches that help biomedical research “become more agile and efficient” and reduce the cost of developing therapies, including decentralized trials and how real world data (RWD) and real world evidence (RWE) to support a range of drug development goals.

A recurring challenge in assessing and treating Post-Traumatic Stress Disorder or PTSD is the inherently complex, ill-defined, and sometimes downright puzzling nature of its symptoms.

For sponsors who are pursuing the goal of treating Alzheimer’s disease, understanding the obstacles inherent in Alzheimer’s clinical trials can help in planning for, and overcoming, these challenges.

Columnist Moe Alsumidae speaks to key Synteract executives on their acquisition of a dermatology CRO to augment its own expertise and grow the specific services necessary for this therapeutic specialty.

Here are four trends to watch as researchers continue to make headway in the development of biomarkers for cancer immunotherapy.

Until more sophisticated measures are created, a combination of investigator training and new imaging methods is the best way to create accurate, reproducible data across sites.

Due to a shifting landscape because of faster drug approvals, this article offers strategies for sponsors to design clinical trials.

In the search for more effective treatments and cures, there are challenges that sponsors and CROs must confront when conducting IMID clinical trials.

Drug developers are using surrogate endpoints to monitor the effectiveness of immunotherapies, taking into account the differences between immunotherapy and traditional treatments.

While block randomization has been shown to be a more frequently used form of trial randomization design, the use of dynamic randomization has been shown to have optimal treatment group balancing results.

Immunotherapy drugs have led to successes in the oncology space as the list of cancer survivors continues to grow. However, more work must be done to ensure that all patients who can benefit from these life-saving drugs receive them.

Progress and innovation within oncology has accelerated to a paradigm that includes immunotherapy and bio-genomics. Collaborative approaches in the future will continue to transform treatments in hopes of improving patient quality of life.

With significant technology advances in ECG recording through the years, the need to expand on traditional monitoring baselines-and include new variables such as time when designing clinical trial protocols-is important.

The EU-AIMS Longitudinal European Autism Project (LEAP) is combining genetic testing, neuroimaging, cognitive testing and clinical assessments to identify biological risk markers associated with subtypes of autism.

Poor understanding of the natural history of the proposed indication due to few observational studies studying disease progression, heterogeneous patient populations with variable phenotypes and clinical courses, geographic dispersion of patients and investigators, regulatory uncertainties, and lack of prior clinical studies to establish a template for study execution, can all prove challenging in creating a pharmaceutical development program for the treatment of a rare disease.

Alzheimer’s disease (AD) is and will remain one of the most unyielding public health crises of the 21st century. Aging global populations translate to a constantly growing prevalence of a disease with no cure and an immense cost of care. In the United States alone, recent estimates1 suggest that 28 million people will develop AD by midcentury. Approximately 25% of the annual Medicare budget will be spent treating the disease – a projected $330B in 2040 – with hundreds of billions more in indirect costs.

Background:

 Clinical trial recruitment and enrollment has presented its challenges in many ways.  For Quintiles’ Phase I Unit in Kansas, clinical trial recruitment was a matter of process and strategy that it improved with innovation and mobile health (mHealth) technology applications. This article and case study will delineate the challenges that Quintiles’ Phase I Unit underwent, and what it did to achieve transformative results in recruitment, study visit adherence, and operational efficiency.

The concept of the human challenge model (HCM) originated from early societal attempts to halt the spread of acute diseases within their own communities

Study shows that industry contributions to R&D go well beyond the applied area of clinical testing.

Successful conduct of hematological malignancy trials requires addressing several unique complexities.

How the practical application of these methods can help overcome the complex demands of cancer trials.

In the healthcare arena, the concept of patient-centeredness has expanded over the last 50 years, beginning as a term to describe patient engagement in self-health management and evolving to include various aspects of patient engagement in healthcare research.1

Cancer Research UK (CRUK) followed such advice when it decided to have its Center for Drug Development (CDD) adopt a risk-based monitoring (RBM) approach across its entire portfolio of clinical trials.

Sponsor companies face intense pressure to deliver higher levels of efficiency and drug development performance. A growing number of sponsors are now acting on the belief that improvements in protocol design feasibility hold the key to addressing and easing some of these pressures.

Over recent years global drug companies large or small have recognized that one root cause of the high failure rate in their clinical development lies in the selection of right therapeutic targets.

Case Study, Trigemina TI-001 for high frequency migraine