The decades-long focus on more “personalized” medicines has paved the way for the recent and anticipated advances in gene and cell therapies and progress in many other areas. The first generation of gene and cell therapies has the potential to transform the way we treat some diseases and can bring new hope to patients with many rare and serious conditions.
Novel technologies to address areas left unaddressed by Kymriah and Yescarta have started streaming into the research arena. This article focuses on the barriers to widespread commercial adoption of the currently available CAR T cell therapies, and opportunities for developers of next-generation treatments.
Indeed, earlier this year at World Advanced Therapies & Regenerative Medicine (WATRM) Congress in London, it was predicted that manufacturing will be a major future differentiator between therapy companies. I spoke to Dr. Paul Lammers, MD, MSc, president, CEO, and Director of Triumvira Immunologics about the therapy pricing and manufacturing’s role in it.
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.