The evolution of medicines from small molecules to proteins has greatly advanced patient care, and up until now, our system of therapeutic delivery in the U.S. commercial system has been built with these therapeutics in mind. The next generation of cellular and gene therapies holds tremendous promise for patients, but only if the existing delivery system is revamped. There’s a real risk that most patients won’t benefit from these therapies as stakeholders struggle to find successful business models. In a recent article for Nature’s Gene Therapy, my industry colleague, Kris Elverum and I take a critical look at the U.S. system and outline what needs to change to make approved cell and gene therapies accessible to patients.


The U.S.’s first FDA-approved gene therapy, Novartis’s tisagenlecleucel, and technologies like CRISPR-Cas9 are poised to create a wave of new medicines, including as many as 39 gene therapy approvals by 2022. However, approval will not be enough to help patients. At least 95% of people receive medicines only through the commercial delivery system vs. clinical trials, with less than 5% of cancer patients participating in clinical trials. Cell and gene therapies have faced a variety of challenges: Sales of the recently approved CAR-T cell therapies have performed below expectations, the manufacturer of another personalized cell therapy (sipuleucel-t) declared bankruptcy, and hospitals and payers have struggled to provide access to these novel therapies. It’s clear that the delivery system designed for pills and biologics urgently needs to change to accommodate cell and gene therapies.


In the articlewe describe the challenges that healthcare stakeholders face along the following seven steps to deliver personalized, autologous, ex vivo cell and gene therapies: 1) patient selection; 2) care delivery approach; 3) patient protection and support; 4) risk and reimbursement; 5) raw material sourcing; 6) manufacturing and logistics; and 7) initial and ongoing care. This framework for cell and gene therapies highlights the systemic challenges, and hopefully will trigger proactive dialogue among stakeholders and a sense of urgency to detail and implement solutions to scale the entire delivery system. We must move beyond old systems to ensure that appropriate patients can benefit from the advances in cellular and genomics research. We hope that all stakeholders will join us in developing solutions for the next generation of medicine.