Challenges impacting market access to CGTs and evaluation of the potential strategies

Abstract

Cell and gene therapies are advanced therapies which offer valuable potentially curative treatments with well-established clinical efficacy and safety. However, there are cases where these advanced therapies have setbacks in market access with some treatments being commercially roadblocked due to challenges. A scoping analysis of literature, stakeholder interviews, and survey data were used to identify and substantiate the critical challenges and potential strategies relating to market access of these therapies. The findings of the three methodologies were compared and combined to signify each challenge and strategy. There were some differences between the findings, but the main trend was that social and economic factors are responsible for the market access setbacks. Several strategies were assessed in their potential to address the market access issues especially in relation to combatting specific challenges. Outcome-based payment plans have been used often to improve market access; however, the high costs still outweigh this plan significantly. Various other strategies have been proposed to enhance market access for CGTs. These include improving manufacturing processes to reduce costs, utilizing conditional approvals to expedite access while gathering further clinical evidence, and encouraging use. The effectiveness of these strategies varies, and their successful implementation often depends on the significance of the specific challenges they act against. Strategies are intended for implementation and further enhancing to eventually minimize market access challenges and facilitate these groundbreaking treatments to the patients in need.