Cell and gene therapies hold the potential to transform medicine; however, if patients are to gain access to these therapies, we must increase public awareness and understanding of how these therapies can benefit them. According to a review of recent research studies that measured U.S. public opinion related to gene medicine,¹ the public is not yet aware of gene medicine; is unfamiliar with the terms being used to describe the topic; and, when faced with multiple therapeutic options, is worried about making informed decisions.

Cell & Gene Editorial Advisory Board member answers questions about his message to pharma executives, researchers, scientists, and doctors, what the industry should consider when developing new payment structures, and more.

The FDA’s RMAT and breakthrough designations have created a streamlined environment for product sponsors. To effectively leverage this regulatory policy environment, sponsors must conduct high-quality clinical trials that are often operationally complex. Clinical trial networks, capable of managing the array of regenerative medicine technologies, are well suited to manage this complexity.

Peter Marks, MD, PhD, Director Center for Biologics Evaluation and Research at U.S. Food and Drug Administration answered some of my questions about the current state of the FDA’s regulations around cell and gene therapies, its stance on international harmonization required to make cell and gene therapies more streamlined, the reasons why it is critical for companies to begin working with the FDA early in the development process, and more. Read on for Dr. Marks' insightful responses.

Current reimbursement models in general do not accommodate many of the unique factors that are common among gene and cell therapies, including smaller patient populations, shorter treatment windows, potentially curative efficacy, high up-front costs, lack of long-term efficacy and safety data, and fees associated with complex administration, dosing, and patient monitoring requirements.

Clinical trials are complex, but adding genomics to the equation has the potential to make them even more complicated. However, much of the genetic testing that is now being performed in the clinical space is done before a trial begins. That means companies need to do some thinking before adding a genetic test to the protocol.

Janet Lambert, joined the Alliance for Regenerative Medicine (ARM) as CEO just over a year ago. And what a year it’s been. Recently, I had the opportunity to talk to Lambert about her first year as CEO, what ARM is doing in cell and gene therapies from clinical and commercialization points of view, as well as its areas of short-term focus. Here’s what she had to say.

The FDA has been issuing guidance documents addressing gene therapy development issues for approximately 20 years — a remarkable dedication of resources to an area that did not have a licensed product until 2017. Of the six gene therapy-related draft guidances the agency issued last month, two represent the first of the “suite of disease-specific guidance documents on the development of specific gene therapy products” Commissioner Scott Gottlieb promised in Dec. 2017. 

Cell and gene therapies: Will their use become ubiquitous, changing the landscape forever as many have promised? Or will applications be confined to rare diseases as they have been thus far?

The FDA recently issued six draft guidance documents. This article discusses the guidance Human Gene Therapy for Hemophilia.

This is the fifth article in a six-part series summarizing each of the FDA's gene therapy draft guidance documents. It will discuss the draft guidance Human Gene Therapy for Retinal Disorders.

The FDA recently issued for public comment six draft guidance documents. This is the fourth article in a series and will discuss the guidance Human Gene Therapy for Rare Disease.

The FDA recently issued for public comment six draft guidance documents intended to serve as part of a modern, comprehensive framework for how CBER will help advance the field of gene therapy. This is the third article in a six-part series.

Despite recent progress in regenerative medicine, cell and gene therapy executives are still pioneering many new pathways as they shape science into never-before-realized medicinal applications. From innovation and growth come surmountable, yet challenging, issues which can hinder — even halt — groundbreaking progress in its tracks.

The draft guidance document provides recommendations regarding the testing for RCR during the manufacture of retroviral vector-based gene therapy products, and for follow-up monitoring of patients who have received retroviral vector-based gene therapy products.

The FDA recently issued for public comment six draft guidance documents intended to serve as part of a modern, comprehensive framework for how CBER will help advance the field of gene therapy. 

A range of factors — including small patient populations, complex manufacturing processes, and lack of specialized expertise — are positioned to both drive up costs and require new options for stakeholder engagement and risk sharing along the development pathway. New approaches in development are needed to support the next generation of novel drugs on the horizon.

Manufacturers, suppliers, and regulators have recognized that introducing new technologies also introduces a degree of unfamiliarity and uncertainty.

This article highlights the diverse interests and range of opinions about pricing innovative and often high-cost therapies, but also demonstrates that most players in the sector see an urgent need for new pricing models to accommodate cell and gene therapy products.