While factors including patient population, product value and efficacy benefit, and pricing play the major roles in successful commercialization of gene and cell therapies, it is essential for drug developers to recognize when and where decisions related to production can also have an impact. The application of technology is a critical consideration in planning related to production time, scalability, and product purity and safety, as well as in expansion of target indications. Without access to skilled expertise, many drug developers risk making decisions related to production that can limit or even jeopardize commercial potential.
Navigating JCA for Orphan Medicinal Products: From scope simulation to pragmatic evidence generation for optimal patient access
The EU’s JCA framework is intended to streamline clinical evaluations and create a more consistent foundation for health technology assessments (HTAs) across...