Cell and gene therapies and the tricky question of patient support

Cell and gene therapies represent the cutting edge of pharma technology, but how are patients experiencing this treatment revolution? Ben Hargreaves looks into the challenges facing pharma and patients to navigate the treatment journey, and what more can be done to help patients.

The emergence of cell and gene therapies has been one of the major success stories for the pharmaceutical industry over the last few years. Earlier this year, the area managed another breakthrough with the first FDA approval for a cell therapy to treat solid tumours. More such firsts are expected, with the pipeline of cell and gene therapies growing each year and currently over 4,000 such therapies in development, as of the first quarter of 2024. In line with this pipeline growth has been the spending by the industry, as investment rose to $5.9 billion in 2023, up 38% from 2022, according to IQVIA.

As the number of cell and gene therapies on the market increases, patients are increasingly gaining access to the treatments. This can be lifesaving for some and represent a significant boost in quality of life for others. However, perhaps because of the scale of the benefits provided to patients, the difficulties individuals face in order to have access to the treatment, and managing the logistics of receiving the treatments, are not as often covered. With the industry’s focus on patient centricity, understanding the patient’s perspective on treatments is more important than ever.

At the end of 2022, ZS Associates, a healthcare consulting and technology firm, conducted a study to understand patient reactions to treatment with cell and gene therapies, finding that more needed to be done to support patients receiving treatment. Sankalp Sethi, principal and leader of the cell and gene therapy vertical at ZS, spoke with pharmaphorum. One of the issues raised in the initial study had been that only 10% of patients found the pharma-led support to be valuable. Sethi stated that ”there is a gap between the need versus who actually ends up getting [support]. And there are a number of reasons that are creating that divide or that gap.”

Complicated patient journey

The number of reasons mentioned includes the complexity it takes to deliver these next-generation treatments to patients.

“Patients do require a lot of special attention, and there are a few different things here,” said Sethi “The treatment itself is more complicated from a logistics perspective, meaning, patients need to travel and be away from their families. They need different kinds of caregiver support in order to be able to go through the process to get these medicines delivered to them. This requires special attention, special education, special preparation for them to be ready to take these medicines.”

The logistical challenge may be one of the most daunting aspects for patients; for CAR-T therapies, this will mean travel for cell harvesting at a specific centre, which could involve a lengthy journey to arrive, and then spending time away from their home. This process is then extended as the product is manufactured and then eventually delivered to the patient, requiring further time in a medical facility and the associated travel.

On the subject of education, Sethi noted that this forms another complication for these types of treatments. Given the very recent rise of these therapies, there are still concerns from patients on potential side-effects and the long-term outlook once treated, though the research found that acceptance of the therapies increased after more information was provided.

A further challenge is ensuring that the information supplied to patients is readily understandable, as these types of treatments differ greatly from standard therapies, potentially requiring additional steps to ready the treatment and having a different mode of action, such as by replacing a disease-causing gene. In the ZS study, a patient explained that the scientific language of the information supplied was ‘intimidating’ and they therefore did not engage with it.

Individual treatment, individual support?

With the treatment area still in its infancy, it is no surprise that ZS found that patients were generally underwhelmed by the support they received when receiving treatment, if they received any at all. Of the patients consulted, 70% did not receive support from manufacturers.

When asked about this, Sethi stated that, in some cases, “there may be hesitancy or a lack of education and awareness from a provider perspective, in knowing what kind of services and supports are being offered. It's also that the programmes being offered aren't exactly what the patients specifically need. In other cases, it's eligibility from a patient perspective – the services may only be referred to certain kinds of patients with the right kind of insurance support.”

As a result, ZS found that 70% of patients reported ongoing distress, feelings of post-infusion abandonment (50%), and delays in treatment decisions (30%). Research that followed up with patients after treatment found that negative emotions, such as guilt and anxiety, were common.

To counter these negative experiences on patients’ journeys receiving cell and gene therapies, ZS proposed three actions: create greater awareness of cell and gene therapy patient solutions; design customisable programmes for evolving complex patient needs; and build solutions for the entire journey, not just treatment.

According to ZS, 80% of patients were unaware of pharma-led travel assistance to reach treatment centres, and this provides the reasoning behind the first action on the list. The second action advises pharma to tailor its materials to its patients in the same way that the treatments are often specific to them, which means paying attention to their degree of health literacy and ability to process new information. The last point relates to following the patient from even before treatment begins to well after it has finished to help alleviate the issues of ‘abandonment’ previously mentioned.

“There is that heterogeneity in what is today a small base of patients, but they're all individuals and different as human beings. So, the flexibility in terms of the format, in terms of the level of depth and type of information that's being shared – that must be there, to be able to make it applicable across your entire base of customers,” Sethi said of the information to be provided to patients.

This could equally be applied to the entire patient journey, where each patient will react differently at each stage of the process. Receiving these treatments provides therapeutic benefits that would previously have been impossible, but this does not help patients when the logistics are time-consuming or the fears over such new therapies are higher. However, in such a new area, there are going to be challenges in delivering the support and care successfully from the beginning. As more cell and gene therapies are approved and patient numbers increase, the ability to get patient support right will also grow.