Kris Elverum & Maria Whitman; Gene Therapy (2019) Doi:10.1038/s41434-019-0074-7
This paper reviews the existing system to deliver cell and gene therapies and outlines the requirements to make them accessible to patients. Informed by interviews with experts, opportunities for improvement are identified along the patient and cell journeys, and a call to action is made for stakeholders to detail and implement change.
Healthcare professionals and patients: fight for progress. With all of the challenges and changes needed in our healthcare system, unfortunately action is not likely unless patients, caregivers, patient organizations, and healthcare professionals fight for progress. With many cell and gene therapies targeting rare diseases, coming together to push for these technologies is even more important to ensure that government, regulatory bodies, and payers have these issues on their priority agenda.
Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine