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Clinical Research: Phase 1 - Phase 4

Addressing a Critical Unmet Need: Considerations for Designing and Conducting Neonatal Clinical Trials

Introduction Due to the challenges associated with studying investigational medical products in neonates, healthcare professionals must often resort to administering drugs in neonatal intensive care units (NICUs) off-label. While numerous pediatric legislative initiatives have been passed worldwide, neonates are still largely treated with medicines that have not been specifically studied or approved in this young,...

The Role of Adaptive Trial Designs in Early Oncology Studies: Optimizing Flexibility and Agility

Targeted therapies and immunotherapies have changed not only cancer treatment paradigms, but also historical approaches to clinical trial design, especially in early development. Traditionally, early phase oncology trials have been focused on dose optimization, toxicity, and broad response signals. However, with the growth of personalized medicine, many early phase studies also place emphasis on identifying...

Med Ad News – Optimizing Early Oncology Studies Through Adaptive Trial Designs

Despite advances in technology and our understanding of the genetic and molecular underpinnings of cancer, making a meaningful impact on the survival and quality of life of patients with cancer remains a significant challenge. In fact, a recent review revealed that, among 59 cancer drugs approved by the U.S. Food and Drug Administration (FDA) based...

The State of Cell Therapies in Dermatology

Skin contains essential stem cell populations and other cell types that are critical for renewing and maintaining its structural integrity and function.1 Of the many types of skin stem cells that have been identified, epidermal stem cells — primarily keratinocyte stem cells — are recognized to play the key role in tissue repair and skin...

The State of Gene Therapy in Dermatology

When it comes to dermatologic conditions, gene therapy is still in its very early stages. However, we are seeing promising potential solutions for some rare genetic dermatology diseases, as well as ongoing research in more common skin conditions. The success of gene-based therapies relies on three critical elements:1 A well-defined disease geneA therapeutic geneAn efficient...

Understanding New FDA Guidance for Pediatric Oncology Studies: A Sponsor’s Guide to the FDA RACE for Children Act

Summary The Research to Accelerate Cures and Equity (RACE) for Children Act The Research to Accelerate Cures and Equity (RACE) for Children Act aims to improve and expand treatment options for pediatric cancer patients by mandating that all new adult oncology drugs also be tested in children when the molecular targets are relevant to a...

Snapshot: A New Guidance Document Program From the FDA

As a regulatory affairs professional advising various stakeholders on the interpretation and implementation of guidance documents from the FDA, I can safely say that the task is challenging in the best of cases. FDA guidance documents have a tendency to be vague and difficult to decode. As experts, we are always debating about what the...

Gene Therapy in Dermatology: Transfer Techniques and Delivery Systems

Gene therapy holds the potential of long-term efficacy or even a cure for diseases with an underlying genetic basis, offering hope to patients with currently incurable diseases. The use of gene therapy for dermatologic conditions is attractive for many reasons:1 As the largest organ of the human body, skin is easily accessible for gene deliveryThe...

UPDATE – Draft FDA Guidance Concerning Combination Products

As those working in the development of combination products likely know, the Food and Drug Administration (FDA) issued two new draft guidances in December 2019. The guidances – Requesting FDA Feedback on Combination Products and Bridging for Drug-Device and Biologic-Device Combination Products– were published in response to the 21st Century Cures Act and Prescription Drug...

INTERACT Meetings: Streamlining Early Clinical Development

In October 2018, the Food and Drug Administration (FDA) issued Standard Operating Policy and Procedure (SOPP) – SOPP 8214 Version 1.0: INTERACT Meetings with Sponsors for Drugs and Biological Products. This document details recommendations for the Center for Biologics Evaluation and Research (CBER)’s engagement with sponsors (such as industry and investigators) in an early development...