Tag: product development 6 results

Real-World & Late Phase

What is Real-World Data and Evidence and How It Can Facilitate Clinical Development

The 21st Century Cures Act (Cures Act)1 signed into United States law in 2016 aims to accelerate medical product development and bring innovations to patients faster through real-world evidence (RWE) to support regulatory decision making. Incorporating real-world data (RWD) and real-world evidence (RWE) can help pharma and biotech companies optimize clinical trial designs and demonstrate…

Dermatology

Establishing Competitive Advantage in Dermatology Trials: Critical Study Design and Operational Considerations for a New Age

Skin diseases are the fourth leading cause of health afflictions, affecting nearly 900 million people worldwide.1,2 At the same time, there has been a remarkable increase in dermatology drug development activity in recent years. For conditions such as atopic dermatitis, psoriasis, and melanoma, there are hundreds of ongoing or planned clinical trials.3 In this new…

Rare Disease

How COVID-19 Is Making Operationalizing Gene Therapy Trials Even Tougher – And How We Handled It

Though gene therapy has been around for decades, it continues to pose extraordinary challenges in the areas of R&D, clinical development, and operation of clinical trials. Sponsors and CROs face shifting operational and regulatory demands amid rapid advances in the science of precision medicine. In our previous blog post, we discussed site selection, manufacturing, long-term…

Rare Disease

Site Selection, Manufacturing & Long-Term Follow-Up: Looming Challenges for a Rescue Phase 1/2 Gene Therapy Trial

Designing and conducting a gene therapy trial is a complex undertaking. Understanding, planning for, and overcoming the myriad challenges of operationalizing these studies will help you bring safe, breakthrough treatments to patients with unmet medical needs. In this blog post, we introduce a case study as a framework for exploring critical study design considerations of…

Rare Disease

How to Navigate Global Regulatory Frameworks & Expedited Programs for Gene Therapy Development in the U.S., EU, & Japan

As scientific knowledge, clinical experience, and acceptance of gene therapy products have evolved, so have the regulatory frameworks for ensuring the safety of these novel treatments. To date, there is no harmonized international standard for regulating gene therapy products; however, the U.S., EU, and Japan have established regulatory frameworks with subtle variations. Understanding how gene…