From The Editor | May 29, 2024

Pediatric Patient Access With Dr. Bambi Grilley


By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1


Cell & Gene: The Podcast features some of the CGT sector’s brightest leaders who share their expertise on the timeliest topics and trends. If you can’t tune in to every episode, I’ve got you covered. Here are highlights, quotes, and more from a recently aired episode.

The Episode:

The Current State of the Global Regulatory Landscape with ISCT's Dr. Bambi Grilley

Guest At-A-Glance

Dr. Bambi Grilley, Ph.D., is Professor of Pediatrics and the Director of Clinical Research and Early Product Development for the Center of Cell and Gene Therapy (CAGT) at Baylor College of Medicine and Chief Regulatory Officer for ISCT. She has worked in Clinical Research for over 30 years and has experience as an Investigational Drug Pharmacist, IRB administrator, IRB member and vice-Chair, Regulatory Affairs professional, and in the conduct of clinical trials. She has worked in oncology and, in addition to that, worked with cell and gene therapy protocols for over 20 years. She has provided in-depth regulatory guidance to investigators developing projects involving translational products and assisting in regulatory submissions in support of these projects. Additionally, she has taught and lectured on regulatory affairs and clinical research extensively both within BCM and internationally.

Dr. Grilley has overseen the regulatory submissions for over 2,000 clinical research studies, almost half of which are therapeutic. And, she has assisted in the submission of over 70 investigator-initiated INDs with over 200 studies conducted under them. She has prepared and implemented over 100 Standard Operating Procedures for clinical research and has established Quality Assurance and Quality Control programs for the protocols being conducted under her purview.

Organization At-A-Glance

The International Society for Cell & Gene Therapy (ISCT) was established in 1992 and is a global society of clinicians, regulators, researchers, technologists, and industry partners with a shared vision to translate cell and gene therapy into safe and effective therapies to improve patients' lives worldwide.

ISCT is the global leader focused on pre-clinical and translational aspects of developing cell and gene-based therapeutics, thereby advancing scientific research into innovative treatments for patients. ISCT offers a unique collaborative environment that addresses three key areas of translation: Academia, Regulatory, and Commercialization. Through strong relationships with global regulatory agencies, academic institutions, and industry partners, ISCT drives the advancement of research into a standard of care.

Comprised of over 3,000 cell and gene therapy experts across five geographic regions and representation from over 60 countries, ISCT members are part of a global community of peers, thought leaders, and organizations invested in cell and gene therapy translation.

Episode Highlights

The Global Regulatory Landscape  

During the episode, Dr. Grilley shared her take on the current state of CGT’s global regulatory landscape and her appreciation to Dr. Peter Marks and his team at FDA for their on-going relentless advocacy in moving the field forward. She stressed the need for regulatory framework in countries that currently lack such infrastructure. At ISCT 2024 in Vancouver, Dr. Grilley will take part in the Global Regulators Summit, which brings together leaders from multiple different countries to talk about different issues facing the cell and gene therapy space. The Global Regulators Summit had its inaugural meeting last year, and participants covered the proliferation of unlicensed stem cell products. This year’s Summit will focus on rare diseases.

Focus on Pediatrics

Dr. Grilley talked passionately about the sector’s need to focus more on overcoming barriers to treating pediatric patients. Barriers exist in part because pediatric patient populations are typically smaller than adult patient populations, resulting in limited interest from industry even though, regarding gene therapy, specifically, research is done in pediatric patients with many gene therapists at pediatric institutions. She argues that the on-going problem in all drug development is that the industry is focused on return on investment.

Top Quotes

7:53:  “I think the most important thing to remember if you’re in regulatory affairs in this field is just that the regulations themselves are unchanged. It’s the implementation of the regulations. It’s really in transition right now. For instance, in the U.S., Peter Marks has been a very vocal and an advocate for helping to push the field forward. And, and I think that any good regulator is there to put up the safety bars but not to be a block to research. And I think that Peter Marks and the FDA have really, really been very good at doing that.”

14:44: “There are some really big patient access issues. We talk about the cost of these products as they get approved. What does that really look like? The bluebird bio story from Europe where they ultimately had their product approved there. They couldn’t agree on the reimbursement code. The product came to the U.S., and the product is now available in the U.S. at $2.2 million per dose. What does that really mean? Well, that really means that for a European, the product’s not there. You can’t receive it even though it was approved. It means that the product’s not available in Africa where the majority of the patients actually are. And in the U.S., at a price point of $2.2 million, I would argue that we really must talk about how available it is for patients here.”

There’s More Where That Came From

This is just some of what Dr. Grilley shares during our conversation. Tune in to the full-length episode to hear her insight on the foreseeable future for the pediatric patient journey, her focus areas for her role as CRO of ISCT, and much more.