OBJECTIVES: Cell & gene therapies (CGTs) have heralded a new era of medicine, promising potentially curative treatments for patients with acute, chronic, and often fatal diseases. However, discrepancies between the high prices set by pharma and healthcare systems’ willingness-to-pay has restricted access and somewhat dampened the broadening potential of CGTs. In the current healthcare environment, the majority of emerging CGTs will struggle to establish a foothold, as currently it is simply not designed to accommodate this step-change in innovation. Through this research, we aimed to explore concerted motions that pharma and healthcare systems can action to unify and co-create win-win situations, opening access to more patients globally.

METHODS: A literature review was conducted to evaluate key drivers behind positive and negative CGT access decisions, from the perspectives of both healthcare system and pharma.

RESULTS: Our research highlighted a breadth of factors driving negative access such as pharma’s withdrawal from the HTA process, insufficient evidence generation or, at times, an apparent lack of communication between parties, leading to HTA misalignment. With these insights, we developed a framework with which pharma and healthcare systems can each meet their respective objectives. The framework details three key areas which pharma and healthcare systems must find mutual ground to streamline processes and extend access to those in need: the clinical evidence required to support CGTs, the value recognition of these innovative therapies, and the pricing models that could plausibly mitigate existing healthcare expenditure challenges.

CONCLUSIONS: Evolving evidence expectations, increasing collaboration between key stakeholders, and flexibility in adopting new pricing frameworks are vital steps to achieving access and unlocking the maximal value of CGTs. As CGTs broaden their applicability into more prevalent diseases, such as solid tumors and Alzheimer’s disease, the time is now to find solutions to access barriers that exist today for CGTs.