Pharma USA 2024

Mar 26, 2024 - Mar 27, 2024, Philadelphia

North America’s ultimate gathering for pharma changemakers

Cell and gene therapy: how to discover and communicate clinical worth

CGTs are often expensive, and the healthcare sector is increasingly working on a tight budget. To bolster CGT market access potential, pharma companies should take a holistic approach.



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Market access is more complex than ever. It requires a delicate balance of demonstrating value to payers and clinicians, ensuring the treatment meets patient needs, and that pharma companies are rewarded for innovation. As EMD Serono’s Nancy Ruscio-Bell described, the pharma sector’s challenge is to demonstrate a therapy’s value in a holistic way.  

The way the sector communicates with stakeholders is changing, which is particularly the case with CGTs. Historically, to bolster a drug’s uptake, sales teams go to physicians to inform them of a drug’s value. Now, all pharma stakeholders – patients, clinicians, regulatory bodies, payers, and policymakers – want customized information.  

All stakeholders should be targeted because, as an example, even if a therapy is approved by regulatory bodies, it might not be reimbursed unless payers agree with its clinical value. Indeed, tension exists about what different stakeholders deem as valuable. In oncology, for instance, payers might primarily look at a therapy’s potential to increase the likelihood of overall survival, but patients might prioritize better quality of life. The pharma company’s role is to engage all groups and find out areas where preferences of stakeholders overlap.  

Pharma companies should meet with patient advocacy groups and medical advisory boards at the same time. While there is value in meeting them separately, having these groups in the same room allows the company to find common ground more easily. Fortunately, the preferences of different stakeholders increasingly converge. For instance, regulatory bodies have been taking patient-reported outcomes and quality of life data in clinical trials into account.  

CGTs, despite their many clinical advantages, are generally expensive. For payers that are working within budgetary constraints, the cost is a significant downside. Tight budgets are further under pressure because of the COVID-19 pandemic and stretched healthcare resources. A way to address budgetary issues is for CGT companies to establish patient assistance programs, which can ease the pricing burden on payers as well as co-pays on patients.  

Many pharma companies have been experimenting with how best interact with payers, in terms of finding the balance between in-person and virtual interactions. The best approach depends on the type of information payers seek. Nevertheless, any virtual communications should complement existing personal interactions. As Bayer’s Reg Waldeck noted, talks should be in “surround sound.” 

Building relationships with patients and advocacy groups can help bolster access to CGTs. Increasingly, patients view themselves to be drivers in their own health journeys. Social listening can therefore be used to understand patient perception of therapies. In addition to revenues, patient satisfaction is an important indicator of a new therapy’s market success.  

The COVID-19 pandemic demonstrated to the public the value of innovative therapeutic approaches, which may have a positive impact on CGTs. The pandemic also showed how the pharma sector and regulators can work together to speed up development timelines. Yet questions remain as to which lessons will last. That said, the notion of getting a therapy to market sooner is gaining broad interest within the sector and it will likely influence many companies’ approaches in the coming years. 

Actionable Insights: 

  • Pharma companies should target all stakeholders: patients, clinicians, regulatory bodies, payers, and policymakers. While they might deem different things valuable, companies need find and capitalize on what priorities overlap 

  • Payer budget is further tightened by the COVID-19 pandemic and stretched healthcare resources: To boost uptake, pharma companies should establish patient assistance programs to ease financial burden on payers and patients 

  • Building relationships with patients is key to underscoring the therapy’s value: As patients are increasingly becoming drivers of their health journeys, they are also increasingly important in highlighting therapeutic worth 



Pharma USA 2024

Mar 26, 2024 - Mar 27, 2024, Philadelphia

North America’s ultimate gathering for pharma changemakers