Consulting

Clinical Research: Phase 1 - Phase 4

A Brief 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...

A Brief 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...

New FDA Draft Guidance Provides Insights on Use of PROs in Oncology Trials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

The Changing Landscape of Psoriasis Treatment

Over the past two decades, biologic therapies have revolutionized the treatment of psoriasis, with more than half of treated patients now able to achieve essentially complete clearing of their disease. In this white paper, we will explore the history of psoriasis treatment – including topical and oral therapies – and the evolution of biologics. Psoriasis...

Ashanthi DeSilva’s Story: A Look Back at the First Gene Therapy Trial – TEST

In 1976, pop culture icon John Travolta starred in the made-for-television movie The Boy in the Plastic Bubble about a young man with a compromised immune system. The premise of the film is that Travolta’s character has to decide between living in isolation behind a protective bubble or dying engaged with the physical world. Hollywood...

Rethinking Data Quality Best Practices in the Era of Decentralized Clinical Trials

Pandemic-related disruptions have accelerated much-needed change in clinical operations, but this change has been accompanied by questions about data collection and data quality. In a recent survey commissioned by Oracle Health Sciences, more than 75 percent of industry respondents indicated that limitations in patients’ ability to attend on-site visits sped up their adoption of decentralized...

Medical Device Development: Pathways to Clearance or Approval in the U.S. and Europe

Introduction Although medical device development is similar in many ways to drug development, devices are subject to different regulatory requirements and approval pathways than drugs. There are also key differences in the rules for the clinical investigations needed to support marketing approval, in part due to the durable nature of devices, which may be implanted...

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...

Choose the Right FDA Program to Expedite Your Rare Oncology Trial

Rare cancers account for 27 percent of all new cancer diagnoses in the U.S. and 22 percent of all new cancer diagnoses in the EU.1 With their poorly understood natural histories, phenotypic heterogeneity, and diverse clinical manifestations, rare cancers pose challenges to drug development and represent a significant unmet need in oncology. Faced with limited treatment...

Beware of Underestimating the Value of a Pre-NDA Meeting

Drug development is a resource-intensive endeavor. Seeking input from the U.S. Food and Drug Administration throughout the journey can help optimize those resources and maximize the likelihood of regulatory approval. When preparing to submit a new drug application (NDA), a pre-NDA meeting with the FDA can be a critical step in ensuring the submission of...