Cell and Gene Therapies Test the Limits of Oncology Health Technology Assessment

Brazil’s National Cancer Institute (INCA) has started a public discussion on the role of clinical outcomes in oncology health technology assessment, especially cell therapies. It invited oncologist Alexandre Palladino to address questions of value, evidence, and patient access in cancer care.

Multi-stakeholder governance in cancer care

The talk formed part of INCA’s “ATS no INCA” project to share knowledge. Health technologist Rodrigo Sardá, from INCA’s Division of Health Technology Assessment (DATS), led the session. It covered current issues in oncology HTA, including multi-stakeholder governance and the evidence demands created by cell and gene therapies. Palladino noted that decisions on technology adoption involve more than clinical factors alone. Patients and families, advocacy groups, regulators, health managers, and drug makers each bring their own priorities. “Each defends its own interests alongside the collective interest, so that we can reach a better result at a lower, socially acceptable cost,” he said. He cited growing health expenditure, higher-priced technologies, and fast-track approvals that still require careful review. “In the end, it is the collective benefit that matters, and the bill is paid by all of us.”

Quality of life metrics in oncology assessment

Participants examined how measures centered on patients sit alongside standard oncology endpoints. Overall survival and progression-free survival continue to anchor the evidence base, yet Palladino said quality-of-life data and patient-reported outcomes now carry more weight. “Treatment may improve disease-related symptoms and, with that, quality of life and longer-term symptom control, even if the patient initially suffers some side effects,” he noted. Although often collected as a secondary endpoint, quality-of-life information helps support decisions on routine use.

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Cell therapies in oncology health technology assessment

The discussion moved to the evaluation of cell therapies such as CAR-T and newer gene therapies. “Evaluating cell therapies is a challenge we have to learn to do better, because it is new, high-cost, and aimed at a smaller niche of patients, potentially with great benefit, but for fewer people,” Palladino explained. He identified two practical difficulties. Standard endpoints can miss the full effect of these treatments, which sometimes produce deep and lasting responses; response rate and duration of response may therefore prove more useful. In addition, the real expense reaches beyond the product price. “Patients may experience important inflammatory responses and end up needing major hospital support, sometimes closed units or intensive care. When you assess the cost of incorporating a technology like this, you must account for the secondary costs tied to the entire health-system infrastructure.”

Real-world evidence and fast-track pathways

Both speakers noted the quick pace of oncology development tied to molecular biology and precision approaches. Single-arm trials, limited data at approval, and accelerated pathways now occur worldwide. “This happens worldwide; every health agency faces this challenge,” Palladino said. “It came with the growth of molecular biology knowledge and precision treatment, ever more specific patient niches, and we will have to adapt. There is no way around it.” Real-world data can help fill gaps left by tightly controlled trials, especially for therapies approved on smaller studies. Such data also support value-based contracts by confirming performance after launch and backing agreements that tie payment to delivered outcomes. “We can use real-world data in all these scenarios to optimise not only the results we bring, but also the cost of what we do.”

Palladino linked these points to patient navigation in an increasingly multi-professional cancer system. Navigation helps patients move through care without gaps and gain from every stage of treatment, “not only access to a new therapy, but even what we already have established.” He placed the broader task in the context of limited resources that affect health systems everywhere.