Personalized medicines such as gene and cell therapies, highly specific and tailored to the individual immune system and genetic make-up, offer promising opportunities for previously terminal conditions. Many of these can be considered to be curative therapies which accrue lifetime benefits after a single use. However, the long-term efficacy of cell and gene therapies is unproven and it remains unclear if they are actually curative.
As the number of curative therapies obtaining marketing authorization increases, challenges associated with affordability and uncertainty in clinical benefit will grow. In the short term, tailored approaches can help promote patient access to innovative products in a timely manner. In the long-run, legislative changes in both the US and Europe and improvements in infrastructure for collection of real-world evidence are needed.
The objective of this MTRG project is to identify the additional value and challenges posed by curative high-cost therapies for payers and healthcare systems in terms of clinical efficacy and effectiveness, financing, manufacturing, and real-world evidence. Challenges are explored from both a high-income country perspective and a low and middle-income country perspective.
In December 2020, LSE hosted a high-level virtual seminar on Financing Options for High-Cost Curative Therapies. The seminar hosted a panel of international experts including former payers and academic experts from France, Germany, Italy, Belgium, Spain, the UK and the US.