OBJECTIVES: Regenerative Medicines Advanced Therapy (RMATs) are cutting-edge technologies including gene, cell and tissue therapies. RMATs are usually associated with high prices and, therefore, raise affordability concerns. This analysis aims to forecast the expected budget impact of adoption of RMATs in the United States (US).

METHODS: A 10-year budget impact analysis was conducted comparing 2 scenarios. In scenario 1, 35 RMs were assumed to reach the market within the first year. In scenario 2, they were expected to reach the market sequentially: 5 RMs per year. Fifteen RMs targeted chronic diseases with high clinical unmet needs and 20 RMs targeted orphan diseases (ODs). RMs prices were 150,000$ and 450,000$ for RMs indicated to treat chronic diseases and ODs respectively (low end of current market prices). Based on the prevalence and incidence in US of each disease, the budget impact was calculated.

RESULTS: In scenario 1, a budget of $4.57 trillion in the first year is needed to pay for all RMATs, which decreased to $228 billion in the second year due to the expected curative effect of RMATs. In scenario 2, the budget fluctuated between $1.27 trillion and $635 billion per year for the first 7 years, depending on the disease epidemiology, and then the budget was stabilized around $229 billion for the following 3 years.

CONCLUSIONS: It is unlikely that payers and patients can afford to pay the whole RMATs costs upfront. The RMATs high prices has become a hot topic and it stimulated discussions on possible new mechanisms for paying the high prices of curative therapies and ensuring patient access to RMATs. A potential solution for affordability issue may be payment amortization, whereby the upfront cost is spread over an extended timeframe combined with outcomes-based payments. Further studies on the appropriateness of this model are needed.