Despite the efficacious promise of these new modalities and their potential for commercial success, the next generation of therapies continue to face challenges that can limit their widespread consideration and commercialization.  This session will consider pre-commercialization concerns for design and delivery of new medicines and therapies, including gene therapy, cell therapy, CAR-T, and bispecific and RNA treatments.  Points of discussion will include:

The Current Pre-Commercialization Landscape:

• Examining the types of products that pharmaceutical, biotechnology and biopharmaceutical companies are seeking to develop now based on mRNA, CRISPR, and CAR-T
• Identifying impediments – through patent or regulatory restraints – which prevent these companies from pursuing the development of the desired product
• Identifying FDA hurdles that may not clear even if all patent and other IP hurdles are met
• Developing techniques for analyzing the value the product adds to the company’s portfolio, and methods for proving value

Regulatory Considerations:

• Understanding how the introduction of these new therapies will change the commercial landscape
• Examining the role of the Center for Medicare and Medicaid Services (CMS) in the approval process
• The connection between CMS approval and commercial viability via government payor systems and rebates
• Assessing the competition and analyzing potential therapeutic interchangeability considerations -if any