INSIGHTS ON CELL & GENE CLINICAL TRIALS

• Does RNAi Have A Future Beyond Liver Disease?

  On the heels of his company's RNA interference IP buying spree, Arrowhead Pharmaceuticals CEO Dr. Christopher Anzalone seeks to prove the merits of RNAi in diseases beyond the liver.

• How Technology Is Enabling Advancements In The Assessment Of Patient Safety

  Pharmacovigilance (PV) activities must be carefully planned from the outset of the drug development process. Read more about available process automation solutions available and if your company could benefit from working with external partners to manage PV activities.

• A Global Observational Registry Of Adult Patients With Homozygous Familial Hypercholesterolemia (HoFH)

  Clinical development service provider efficiently and strategically manages a global rare disease registry with rolling start-up, including mandated annual data collection and reporting.

• Developing TIL Therapies: Sample Collection And Processing

  While it has been advanced over many years, the process of developing TIL therapies still suffers from certain challenges. This article attempts to lay bare some of these challenges and discuss how novel technological solutions can help overcome them.

• Tumor-Infiltrating Lymphocytes: What, Why, And How

  As we explore immune mechanisms using technology, we find better ways to fight disease. Here, we focus on targeted, highly personalized cancer therapies called tumor-infiltrating lymphocytes.

• Addressing A Critical Unmet Need: Considerations For Designing And Conducting Neonatal Clinical Trials

  There is significant unmet need for approved treatments for neonatal-specific conditions. With improved understanding of the unique nuances of research in this young, vulnerable population, sponsors can increase their likelihood of developing and executing successful neonatal clinical trials.

• Real-World Data And Real-World Evidence: What Is Their Value As A Synthetic Control Arm?

  It is commonly accepted that a single-arm trial utilizing a synthetic control arm (SCA) can also be an adequate design for assessing a new treatment intervention. Single-arm trials demonstrate clinical benefit by showing the positive effects of a new therapy or treatment without the need to use placebo or standard of care as a control. Instead, RWD and RWE comparisons – leveraging a variety of sources – can serve as the comparator.

• Managing Pandemic-Related Protocol Deviations: Tips For Clinical Trial Conduct

  Conducting clinical trials of therapeutics and devices can be daunting, and COVID-19 has made this endeavor even more challenging. Here we share some tips for managing pandemic-related disruptions while collecting the information regulatory agencies will require regarding protocol deviations going forward.

• COVID-19 & Clinical Trials: Understanding The Long-Term Impact

  While most regulatory agencies have stated an intention to be flexible during these unprecedented times, it is critical that drug developers continue to monitor their risk diligently and develop contingency plans to adapt and respond to factors that affect their clinical development programs.

• How Ramping Up Communications Helped Overcome A Next-To-Impossible Recruitment Challenge

  We knew going in that it could be the perfect recruiting nightmare with extremely complex inclusion/exclusion criteria. By letting sites set their own goals and commit to them along with assigning project coordinators that stayed close to those sites the study is off to a good start.