The number of companies targeting the development of new cell and gene therapies has risen dramatically in recent years.
On a global scale, clinical research programmes are now underway targeting almost 50 different indications for gene therapies alone, up from only ten a few years ago. Several clinical programmes have advanced to late-stage development with regulatory decisions pending or on the horizon.
Many of these drugs have the potential to bring both patients and clinicians historic and transformative advances in the treatment of many serious diseases, including the introduction of single-dose and potentially curative therapies to address major areas of unmet need.
With these potential benefits, cell and gene therapies also introduce some important and new considerations related to established reimbursement and payer strategies. It is likely that these considerations will affect a wide range of stakeholders, including manufacturers, payers, regulators, patients and clinicians as well as entire health systems.