Bio

Debra Aub Webster, PhD, has over 20 years of experience in pharmaceutical research and the regulatory environment.  She received her undergraduate degree from Virginia Polytechnic Institute and State University, and her graduate degree in pharmacology and toxicology from the Virginia Commonwealth University’s Medical College of Virginia.  After leaving academia and bench research, Dr. Webster joined the United States Food and Drug Administration (FDA) as a reviewing toxicologist.  Dr. Webster was an inaugural member of the FDA’s Division of Anti-viral Drugs in the Center for Drug Evaluation and Research.  Here she was responsible for critical evaluation of the nonclinical pharmacology and toxicology sections of Investigational New Drug Applications (INDs) and New Drug Applications (NDAs).  She authored reviews, advisory opinions, and executive summaries, and was awarded a Medal of Appreciation from the Commissioner for her work.  Prior to leaving the FDA, she held a rotation as Assistant to the Director for Pharmacology/Toxicology.  Dr. Webster is the Director of Advanced Therapy Medicinal Product Development group of Cardinal Health Specialty Solutions. She is responsible for providing regulatory strategy consulting support across clinical, nonclinical, and CMC to lead product development from proof-of-concept through to approval. Dr. Webster has provided support and leadership for the development of regenerative medicines and advanced therapies, including CAR-T and other genetically engineered cells; stem cell therapies; gene therapies; genome editing therapies, oncolytic virus therapies; therapeutic cancer vaccines, recombinant human proteins, antibody-based therapies, biosimilars, and bioengineered tissue constructs.

Abstract

State of the Industry for Advanced Therapies

The revolution in cell therapy began with the use of autologous cord blood cells for hematopoietic and immunologic reconstitution in patients with disorders affecting the
hematopoietic system. Most recently, genetically modified cell therapies such as Yescarta® and Kymriah™ are proving the power that human cells have to treat disease in truly transformative ways. However, the translation of preclinical promise of efficacy to the reality of clinical efficacy is particularly challenging and expensive. These transformative therapies must be affordable and accessible in order to be truly transformative. The challenges faced in the development of these advanced therapies encompass all aspects of product development. Manufacturing is expensive and must exceed pace of both nonclinical and clinical development. Demonstration of proof-of-concept in traditional nonclinical studies is constrained by species specificity. Demonstration of clinical benefit may need to rely on biomarkers that need to be validated in patient populations that are limited. With the recent plethora of updated and final guidance documents issued by the FDA, perhaps the least challenging aspect of product
development will be regulatory submissions. Regardless of these challenges the landscape for regenerative medicines is expanding, with clinical trials encompassing indications in oncology to lymphatic diseases. While only a few advanced therapy products have received full FDA approval to date, there are thousands that are at various stages of development.