New annual report on cell and gene therapy market authorizations
ISCT, the International Society for Cell and Gene Therapy, the global professional society of clinicians, researchers, regulatory specialists, technologists and industry partners in the cell and gene therapy sector, announced on November 20, 2018 the first publication of its new annual report detailing global cell and gene therapies (CGT) with market authorization.
The CGT market authorization report is intended to provide a centralized resource and increased clarity for all of those involved in the CGT industry. This involves the scientific community, healthcare stakeholders and patient associations. The report will be a regularly updated document that will be republished yearly, and accessible through the ISCT website.
The report has been designed to react to the increase in momentum around a widening number of CGT authorizations, as well as a parallel increase of unproven approaches where cells are delivered as treatments without rigorous scientific and regulatory assessment and authorization.
The report has been prepared by select members of the ISCT Presidential Task Force (PTF) on the Use of Unproven and/or Unethical Cell & Gene Therapies (UCGT) with lead authors Natividad Cuende (Executive Director, Andalusian Initiative for Advanced Therapies, Seville, Spain) and Laertis Ikonomou (Assistant Professor of Medicine, Boston University School of Medicine, Boston, MA). The ISCT Presidential Task Force was formed in 2014 to support ISCT’s ongoing commitment to address the rise of commercially available unproven or insufficiently proven CGT treatments. These approaches are targeted at hopeful individuals seeking cures or health improvement for a variety of conditions. The PTF-UCGT characterizes unproven cellular interventions and promotes safe and effective practices worldwide.
The reliable, up-to-date resource will help patients to make informed decisions before receiving a CGT treatment so that they can avoid being exposed to unproven and unlicensed cell interventions not approved by a regulatory or medicine agency.
The report also provides an annual breakdown of CGTs that have received, as well in some cases, have withdrawn, market approval. This includes analysis on types of product and therapy as well as disease targeted. 44 unique products were identified. 84% were cell and tissue therapies, and 55% were autologous. More than one third target oncological or hematologic conditions.
In the US, there are over 16 cell, tissue and gene products with marketing authorization, 14 in Korea, eight in Europe, four in India and Japan, two in Canada, and one each in China and Australia. This contrasts with Russia, Middle East, Africa, Central and South America, which have no CGT market authorizations or data available.
“The number of market approved cell and gene therapy treatments continues to grow, with a high number of other therapies seeking approval. It is critical for all those involved in the sector, and most importantly those patients that may benefit from newly approved treatments, to have an annually updated resource detailing all approved cell and gene therapies,” said Massimo Dominici, Chair of the ISCT Presidential Task Force on Unproven and Unethical Cell & Gene Therapies. “This resource will give patients visibility on what cell and gene therapies have gone through proper approval processes, and help them alongside physicians to make more informed decisions about their treatment.”
In tandem with the efforts of professional societies like ISCT, it is imperative that the regulatory environment and guidance keep pace with the rapid advancements in cell and gene therapy. As such, this report also supports ISCT the FDA’s continued publication of numerous draft CGT guidance documents in 2018.