How HCPs and Patients Influence the CGT Approval Process

In a video interview with Pharma Commerce, Jenna Dale, direct of client relations at Cencora, shares how pharmaceutical manufacturers are able to handle supply chain disruptions. She explains that while these issues may arise due to political or environmental factors, they aren't new challenges for the industry. Manufacturers have faced similar hurdles before and are accustomed to managing risks that affect production. The key to overcoming these challenges, according to Dale, is having a robust continuity plan in place. Such plans help ensure that manufacturing remains uninterrupted, even when external factors like tariffs create obstacles. By identifying potential risks and preparing for them, manufacturers can continue the development and commercialization of therapies despite external pressures.

Dale also comments on key challenges preventing the United States from meeting the FDA’s forecast of 10 to 20 cell and gene therapy (CGT) approvals per year by 2025, the need for continued investment by the FDA in reviewers and staff to support clinical reviews, and much more.

A transcript of her conversation with PC can be found below.

PC: The involvement of HCPs and patient organizations is critical in understanding meaningful clinical outcomes. What role do these groups play in shaping the approval process, particularly for rare patient populations with significant unmet needs?

Dale: So I'll start from the provider perspective. I think, especially in rare disease, but really, this is applicable across cell and gene for different types of therapies than maybe what the agency may be used to seeing in terms of devices or traditional pharmaceuticals. That said, from a healthcare professional (HCP) perspective, allowing the opportunity to give their expert insights and clinical experience, not only to the agency, but to all the stakeholder communities, so that we know that we're developing therapies that ultimately are going to achieve some type of clinical significance, some type of clinical outcome that is meaningful from the provider's perspective, and of course, most importantly, from the patient's perspective.

To focus on where the HCP is coming from, being able to give their insight into how we can develop these therapies to achieve a meaningful clinical outcome, is really important. That helps us design trials and implement the trials operationally, more meaningfully as well, and then they can help really interpret and contextualize the data. Making this something that's clinically meaningful to the right audiences is really critical, and then the providers can also help advocate and educate the patients.

A lot of patients have a lot of questions about cell and gene therapies, and so being able to trust your provider and get more information through those medical channels is critically important for patients, not only thinking about participating in a trial, but ultimately enrolling in a clinical trial, or families, caregivers, parents, if it's a pediatric indication. Having that advocate and educational champion through the HCP lens is really important. And then, similarly, on the patient side of things, patients are the strongest voice in this entire community.

I think every manufacturer's goal is to develop a therapy that is going to help a patient, which involves listening to what matters to the patient. Do we have patient-centric clinical outcomes in our clinical trials? Are we developing this with the patient in mind that not only recruit patients into the clinical trials, but retains patients into the clinical trials if they have a better understanding up front of what that's going to take in terms of an investigational protocol? And then the patients can go be such a strong voice, both in terms of policy, but also in terms of the FDA’s review. If they have listening sessions or advisory committees where they want to hear directly from patient communities, engaging with the patient community and understanding from their perspective, really just opens the eyes of all of the different stakeholders involved, whose ultimate goal it is to help treat those patient populations more effectively.