Developing novel policy proposals to support access to gene and cell therapies

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. Many gene therapies are designed to be effective after a single cycle of care (one-off transformational or potentially curative treatments), which brings many obvious administration and patient benefits. Given the way medicines are currently paid for, this often involves large one-time payments for these drugs, although the clinical benefits may not be observed for several months or even years. Given this, advanced therapies such as gene and cell therapies will present a number of challenges for healthcare systems. If left unaddressed, these challenges could result in the adoption of such therapies being restricted and ultimately limit patient access. Without new approaches in healthcare delivery, management, and funding models, treatment may be out of the reach of many patients and hinder the commercial success of these technologies.
On behalf of the American Chamber of Commerce to the European Union (AmCham EU), the Life Sciences Practice at CRA recently analyzed a range of emerging trends and challenges associated with access to the use of new integrated technologies, including gene and cell therapies. The goal of this analysis was to better understand the factors that could limit patient access to transformative technologies and promising new therapies and identify opportunities to improve healthcare policies to help expand access.