Building patient trust and empowerment to improve the treatment experience

Cell and gene therapies have brought transformational advancement to the care of patients with rare diseases, including, in some cases, curative treatments. These therapies are, however, not well understood by patients and their caregivers, and there is also often uncertainty in the physician and payer communities.

During Cencora’s ThinkLive Cell and Gene Therapy Summit 2024 – “Beyond borders: Breaking barriers to enhance patient access in cell and gene therapy” – discussion turned to the patient journey and the importance of building trust with the patient community. Patrick Collins, Vice President, Corporate Relations, National Organization for Rare Disorders (NORD) shared insights from the patient perspective, followed by a panel discussion on the patient journey with George Eastwood, Board Chair / Interim Executive Director, Emily Whitehead Foundation; Matthew Lakelin, Ph.D., Head of Consultancy Services and Co-Founder, TrakCel; Vickie Simpson, Senior Manager, Operations, Cencora; and Patrick. The discussion was moderated by Dale Hanna, Director, Cell and Gene Therapy Solutions, Cencora.

Building patient trust

While a growing number of CGTs have received regulatory approval, long-term efficacy data is not yet available, which necessitates ongoing post-marketing surveillance . In his presentation, Patrick noted that to engage patients in follow-up will require time and effort to build trust.

The other side of the coin is the cost and whether payers are willing to accept that cost, he noted. That will likely become a bigger question as more therapies come to market, leading to more cost for payers – be they private insurers or state Medicaid.

“Are patients and providers willing to navigate all the hurdles of formulary placement or step therapy programs? And will payers be engaging in different types of reimbursement models such as value-based arrangements that will provide greater flexibility to reimburse these therapies?” he asked.

These are some of the unchartered waters that industry must navigate, which underscores the importance of building trust with patients and providing them with meaningful support, Dale said.

The treatment experience

Given how new CGTs are and how different the treatment regimen is, patients will need some guidance with the care journey.

Drawing on her nursing experience in the oncology space, Vickie said that patients and their caregivers have more knowledge of treatments such as chemotherapy since these have been around for many years.

“There’s less familiarity with cell and gene therapies and so there really needs to be a focus on education,” she said. “Education empowers, engages and motivates. And it’s critical to engage and empower the patient and caregiver because they’re in it together. Cell and gene therapies are very targeted treatments, so we likewise need to design and deliver very customized services. It’s important to identify and meet the individual needs of the patient and caregiver, where they are in the treatment journey.”

Educating individuals with rare diseases and trying to alleviate misconceptions about CGTs is key, Patrick said, for example the idea that these therapies are a panacea requiring one injection and the patient is cured.

“It is a process to be identified and to be accepted into the gene therapy protocol, and then there is the post treatment in terms of follow up and surveillance, so it’s not one shot and that's it,” he said. “That education can’t begin three months before the launch of a product. There needs to be 2, 3 or even 4 years of prepping that population that you potentially have this new curative model.”

Matt agreed that post-surveillance monitoring can be a challenge, because once a patient is well, where is the motivation for them to return for follow-up?

“It has to begin with education, and it needs to be spelled out early on in treatment,” he said. “There are other ways you can monitor patients, so you have to put those strategies in place, particularly if you are receiving long-term milestone payments for your therapies.”

In the rare space, while each patient journey is unique there are commonalities.

“A lot of what a caregiver goes through can be the same across rare diseases – the uncertainty, the lack of knowledge, the searching for information,” George said. “So, what commonalities can we find across modalities? And how can we meet patients where they are, especially when everybody wants a different level of information? How do you do that at scale? It's very difficult, but I think we can find ways to navigate across the ecosystem.”

Minding the gaps

One of the many challenges patients with rare disease face is simply getting to treatment centers.

“At NORD, we have a whole patient assistance department dedicated to assisting efforts such as travelling to and from a medical site,” Patrick said. “And I encourage companies that are involved in rare disease product development to have patient assistance programs that cover not only the therapy itself, but also the ancillaries of getting to and from a site, and other levels of patient support.”

“Patients need a whole level of support to be diagnosed, and also support to be able to have the initial visit at a medical institution,” he added. “There is a litany of activities that industry could be doing, that the patient organizations are doing, to support that whole ecosystem to allow the patient to be treated with a cell or gene therapy.”

Vickie agreed, adding that when patients are taken out of their familiar environment there are social and psychological barriers to address too.

“It’s about building services that help them to anticipate and plan for all of those needs,” she said.

While patient well-being must be addressed, from a more technical perspective, another challenge is data siloes, Matt said.

“You have to think about exchanging the information that's necessary for everyone to treat the patient as quickly as possible,” he said. “But you also need to recognize that just because you can exchange information between different systems and different groups, that doesn't mean you should. You need to be quite clear with your data models to protect the patient.”

Balancing the logistics

One of the fundamental differences between standard therapies and CGTs is very often these start off with the patient material.

“The actual manufacture of the drug product starts with a particular procedure, usually taking cells by apheresis,” Matt pointed out. “When you’re looking for sites to administer these therapies, you will need to find experienced apheresis centers.”

First there are the logistical capabilities of the centers themselves. They must be equipped to take viable cells from the patient into the manufacturing environment and back to the treatment center, and be accustomed to handling cryopreserved products, Matt said.

“You need to align the different parts of your supply chain carefully and make sure everyone is aware of the importance of their role,” he said. “The patient must be aware of this too, because there are very few manufacturing slots available for these products, so if a manufacturing slot becomes available, the patient needs to be at the right place at the right time to either donate starting material or potentially receive their drug product.”

Managing those logistics also requires educating physicians outside the major cell and gene therapy centers.

“All of us need to do more to address the unique hurdles within the cell and gene therapy ecosystem, and pursue tools and technology that will better enable access, patient support and delivery,” George said. “This is how the promise of cell and gene therapies will be fully realized for every patient who can benefit from them.”

Untangling the patient journey

As the patient journey continues to evolve, along with continued growth of CGTs, manufacturers and other key stakeholders will need to consider new ways of supporting and engaging with patients.

“We really have to understand and leverage technology to reach patients in the most effective manner and equip them with the tools, resources and the knowledge that they need to be their own best advocate,” Vickie said. “These are complex therapies and so really customizing services to the patient will be key, as well as empowering the patient to be their own best advocate and to take ownership of their care.”

The future direction is positive, Patrick said. “We’re seeing more and more companies engaging directly with patient organizations and a greater focus on patient-oriented drug development for rare diseases. On the medical side, NORD now has around 40 centers of excellence for rare diseases throughout the country that can serve as a network to educate other physicians as well. So, we’re heading in the right direction.”

Technology is key across the entire lifecycle, the panelists agreed.

“These advanced therapies can now potentially treat patients earlier in the disease cycle, which could mean more therapies and more centers administering these therapies, which in turn could make it easier to gain access to these treatments,” Matt said. “You want to standardize where you can, but, because these are very complex products, standardization is not going to be the panacea. So, instead, the goal should be simplification to make it as easy as possible, with all the different technologies at your disposal, to administer therapies and improve that patient journey.”

Dale Hanna concluded by highlighting: "By breaking down barriers and fostering collaboration across the healthcare ecosystem, we can truly empower patients and their caregivers to efficiently and effectively provide the individualized support they desperately need. The future of cell and gene therapy is not just about innovative treatments but about building a foundation of trust, education, and support that ensures every patient has the opportunity to thrive in a very complex ecosystem."