The power of patient engagement with CGT innovation
By AmerisourceBergen
The Emily Whitehead Foundation is focused on supporting children and their families in fighting cancer through novel treatments, not limited to CAR-T therapies. The foundation’s Board Chair and Interim Executive Director, George Eastwood, joined Cencora’s two-day ThinkLive CGT Summit in May 2024 to share his experience with the foundation and the importance of patient engagement and patient support programs.
After many years in the cell and gene therapy space, collaborating with leading companies to solve the challenges involved in developing and manufacturing cell-based therapeutics, particularly in manufacturing, George was at a summit in Boston around 8 years ago where he heard Tom Whitehead share Emily’s story. Over the years, George became more and more involved with the foundation, helping guide it to a deeper and broader strategy to support patients along their entire journey.
“There are so many logistical and policy challenges that exist across the whole advanced therapy ecosystem and we’re working on supporting patients in that journey,” he said.
Key to this journey is integrating the patient voice into the value story for CGTs – and that’s not possible without knowing what those patients’ lives are like, George noted.
“I’ve seen parents and patients invite pharma companies into their living room to see what it’s like, and those stakeholders gain a drastically different view when they do that, rather than relying just on numbers and data in the development of drugs,” he said.
This is where organizations like the Emily Whitehead Foundation help raise awareness about the patient’s journey.
However, George noted that reaching more patients will require diagnosis and treatment earlier in a patient’s journey. He added that, in a recent research initiative to help guide patients, the foundation found that around 70% of patients reported that they received no support from pharma in their journey.
“The onus is on us, on patient groups and nonprofits and advocacy organizations to help build and connect those stories, long in advance of treatment,” he said.
While the pharmaceutical industry and services providers can play a role in building engagement, there can be mistrust from patients when information is shared from a manufacturer-sponsored site, even when that information is unbranded.
Another challenge George pointed out is that it can be a struggle to find vetted trials for CAR-T and other cell and gene therapies. This is made more challenging because of what George refers to as bad actors in the regenerative medicine space conducting trials that have uncertain FDA regulatory status.
One important initiative aimed at addressing this barrier is TrueTrials, which is focused on accelerating clinical trials in regenerative medicine by highlighting only clinical facilities providing cell-based treatments within FDA-authorized studiesii.
Another of the challenges CGTs often face is the negative press around the cost of these therapies. But, as Tom Whitehead has recounted, the cost to Medicare to care for Emily for 22 months was $2 million with no cure. The current cost of Kymriah is $473,000 for a one-and-done treatment and Emily has not spent a day in hospital for the past 12 years.
“CAR-Ts have a high price tag and it’s easy to pull that apart and critique it in the media, but we need to understand the total journey – starting with diagnosis, treatment and even past the cure date looking at mental health and support along the way, and truly looking at that whole picture when we're deciding value,” he said.
Building bridges and connections
To overcome some of these challenges, George pointed to the importance of meeting patients where they are and better understanding the journeys and struggles caregivers face.
There are also important roles for companies in the space that are not developing therapies – technology and services providers, contract development and manufacturing organizations (CDMOs) – to connect pharma and patients earlier.
“Companies like Cencora can help to build some of those bridges between pharma and advocacy groups to establish multi-stakeholder teams that can get patients the information they need, when they need it,” he said. For example, a CDMO could have a patient come on site and speak about their experience with a product that site is manufacturing, George noted. “There are interesting ways in which tool providers can integrate and help raise awareness for patients.”
Examples include improved data integration and interoperability between platforms to better serve patients and streamline processes for drug developers.
The emphasis for drug developers must be on incorporating the patient voice and thinking about the patient journey.
“You can never start too early or interact too much,” George said. “Ask questions and learn along the way, partner with the right organizations like the Emily Whitehead Foundation, even for connection to patients with rare journeys that are outliers. Also, can we build better solutions that are more integrated and more universal to deliver more medicines?”
“We are only 12 years from Emily being treated and six or seven years since the first approval,” George added. “There's lots of integration and understanding to happen. And we can only do that through working with the entire ecosystem, whether it's the patients and their journey, the clinical caregivers, the nurse navigators and the doctors that are in these institutions. How can we engage and learn more as we deliver more therapies?”
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The power of patient engagement: A trailblazing journey to elevate access
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