Clinical Research: Phase 1 - Phase 4

A Primer on Cancer Immunotherapy Part 2: The Cancer Immune Response & Mechanisms of Resistance

Immunotherapy has led to substantial advances in cancer therapy in recent years. Still, unpredictable response rates and immune-related adverse events have hampered the widespread use of immune checkpoint therapy to treat cancers. To tackle these challenges, sponsors are increasingly looking to combination therapies as a strategy for improving response and overcoming resistance. In our previous...

Clinical Research: Phase 1 - Phase 4

A Primer on Cancer Immunotherapy Part 1: Goals & Major Approaches

In recent years, immunotherapy has led to substantial advances in cancer therapy. In particular, the immune checkpoint inhibitors — PD-1/PD-L1 and CTLA-4 inhibitors — have revolutionized treatment for certain hematologic malignancies and solid tumors. The U.S. Food and Drug Administration (FDA) has approved six immunotherapies across 19 cancer types and two tissue-agnostic conditions. But widespread...

Clinical Research: Phase 1 - Phase 4

Introduction to CAR T-Cell Therapy Part 2: Additional Considerations & Future Opportunities

Beginning in 2017 with the approvals of tisagenlecleucel (Kymriah™) and axicabtagene ciloleucel (Yescarta™), chimeric antigen receptor (CAR) T-cell therapies have changed the treatment paradigm for patients with certain hematologic malignancies. Since those initial approvals, three other CAR T-cell therapies have been approved for various hematologic malignancies, but many challenges still limit the therapeutic efficacy of...

Clinical Research: Phase 1 - Phase 4

Introduction to CAR T-Cell Therapy Part 1: Background & Current Development Landscape

Chimeric antigen receptor (CAR) therapies use CAR T cells, a patient’s own immune cells that are programmed to recognize and kill cancer cells throughout the body. Beginning in 2017 with the approvals of tisagenlecleucel (Kymriah™) and axicabtagene ciloleucel (Yescarta™), CAR T-cell therapies have changed the treatment paradigm for patients with certain hematologic malignancies. Since those...

Consulting

New FDA Draft Guidance Provides Insights on Use of PROs in Oncology Trials

Incorporating patient-reported outcomes (PROs) into clinical trials can help sponsors better understand patients’ symptoms and how a therapy will affect their quality of life, and these insights can be particularly valuable in oncology research. However, there has been a lack of guidance to help sponsors ensure consistency and quality when incorporating PROs into cancer drug...

Premier Research Enhances Oncology Capabilities With Addition of Expert Dr. Ashley Herrick

Clinical Research: Phase 1 - Phase 4

Premier Insight 271: Enrolling Sickle Cell Patients Is Tough – So We Got Busy

Kick off a study of a drug to treat sickle cell disease and you’re facing a tough climb. Experimental drugs for sickle cell are few and far between, so there is little in the way of clinical trial infrastructure. The patient population is demographically challenging, too, made up largely of socioeconomically disadvantaged people who find...

Medical and Regulatory Affairs

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...

Medical and Regulatory Affairs

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...

Consulting

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...