Cell and gene therapies hold the potential to transform medicine; however, if patients are to gain access to these therapies, we must increase public awareness and understanding of how these therapies can benefit them. According to a review of recent research studies that measured U.S. public opinion related to gene medicine,1 the public is not yet aware of gene medicine; is unfamiliar with the terms being used to describe the topic; and, when faced with multiple therapeutic options, is worried about making informed decisions.
Current reimbursement models in general do not accommodate many of the unique factors that are common among gene and cell therapies, including smaller patient populations, shorter treatment windows, potentially curative efficacy, high up-front costs, lack of long-term efficacy and safety data, and fees associated with complex administration, dosing, and patient monitoring requirements.
Manufacturing viral vector-based therapies such as vaccines and gene and cell therapies is complex, but a new manufacturing solution helps solve those challenges.
Cell and gene therapies: Will their use become ubiquitous, changing the landscape forever as many have promised? Or will applications be confined to rare diseases as they have been thus far?
Despite recent progress in regenerative medicine, cell and gene therapy executives are still pioneering many new pathways as they shape science into never-before-realized medicinal applications. From innovation and growth come surmountable, yet challenging, issues which can hinder — even halt — groundbreaking progress in its tracks.
A range of factors — including small patient populations, complex manufacturing processes, and lack of specialized expertise — are positioned to both drive up costs and require new options for stakeholder engagement and risk sharing along the development pathway. New approaches in development are needed to support the next generation of novel drugs on the horizon.
Single use technologies are expected to play a big role in the commercialization of cell and gene therapies because of their cost, speed, and sterility benefits. In some ways, will cell and gene therapy manufacturing be similar to biopharmaceutical manufacturing? What are the key differences and gaps in learning?
This article highlights the diverse interests and range of opinions about pricing innovative and often high-cost therapies, but also demonstrates that most players in the sector see an urgent need for new pricing models to accommodate cell and gene therapy products.
Part 2 of this two-part article reviews lessons from Spark Therapeutics’ pivotal program for Luxturna, a gene therapy approved for the treatment of patients with retinal dystrophy associated with confirmed biallelic mutation in the RPE65 gene, and summarize key considerations for the clinical development and commercialization of gene therapies.
Aside from the clinical opportunity that gene therapy products present; they do introduce tremendous complexity into both the manufacturing and commercial processes. Remember these three points before developing your gene therapy plan.