The Institute for Clinical and Economic Review (ICER) recently conducted a study of value assessment methods considered for “cures” or “potential cures” and solicited input from international HTA bodies. The research will form the basis of a value framework that will be applied in the assessment of potential cures and other treatments that qualify as what ICER refers to as “single or short-term transformative therapies.”
As the cell and gene therapy landscape continues to mature, so too do the intricacies of commercialization. Inside the pages of this free collection of articles, you’ll find insightful data addressing the challenges critical for the sustainability of cell and gene therapy business models.
Delivering cell and gene therapies is an expensive and highly complex process, and there are a number of critical metrics that manufacturers should consider when selecting treatment sites. Subsequent activities — preparing sites to receive and initiate therapy, and managing site training and ongoing certification — can be equally, if not more, challenging.
The pharmaceutical industry’s success has become something of a double-edged sword, and our healthcare systems are burdened with the costs of lifestyle challenges. Everyday businesses and healthcare professionals must work diligently to improve our quality of life, but pricing and access will continue to be a battle globally that the industry must take note of.
In recent years, cell and gene therapies have been generating highly promising results in clinical studies, advancing them toward the market. However, the small number of products launched to date have not been proven commercial successes, with a number of advanced therapies being withdrawn from the EU market and limited sales of CAR-T therapies in the U.S.
Cell and gene therapy offers extended-term relief from disease states but comes at high cost with a complex reimbursement model. What proportion of intervention costs should be levied up front, and what proportion can be phased into the future (when the patient benefits accrue and are proven)? Separating the payment timeline from the treatment schedule is often referred to as outcome-based costing.
Innovation in gene therapy brings the potential for transforming patient care and obviating the need for chronic therapy through single-dose cures. Despite the potential long-term benefits of this new therapeutic modality, gene therapy companies face a number of underappreciated challenges.
Over the past four decades, and especially in recent years, progress in the development of cell and gene therapies has reached unprecedented levels. In gene therapy alone, between 1989 and 2015 there were more than 2,330 clinical research programs targeting almost 50 different indications.1,2 As more cell and gene therapies reach commercial stage, industry insiders expect research in the sector to continue to expand in the coming years. For patients, clinicians, and health systems, new cell and gene therapies bring the promise of historic and transformative advances in the treatment of many serious diseases.
Streamlining, connecting, and automating workflows to shape the future of cellular treatment delivery has the potential to transform how we treat and potentially cure once life-threatening diseases.
Emerging cellular therapies aim to manufacture cell populations for clinical indications, such as cancer, autoimmune, and cardiovascular diseases. As hospitals and translational facilities explore implementation of this technology they concurrently evaluate whether or not to manufacture the therapies onsite. Here is a guideline of how and when to get started.