Patient Assistance Programs: Real-World Data Source?

Early last month, Kite Pharma, a subsidiary of Gilead Sciences, released results from an analysis combining data from clinical trials of the lymphoma drug Yescarta and patient data gathered from the support site Kite Konnect. The study concluded that the CAR T cell therapy Yescarta is more effective when used as a second-line treatment for relapsed/refractory large B cell lymphoma rather than as a third-line option.

Kite Pharma's utilization of data from a patient support program underscores the potential for these programs to provide real-world evidence about medical products. However, the prevalence of this practice is not well-documented. Several pharmaceutical companies, including Eli Lilly, Biogen, Kite Pharma, and GSK, declined to comment on their practices regarding the collection and use of patient outcome data from assistance programs when contacted by BioSpace.

Genevieve Kanter, an associate professor at the University of Southern California (USC) Price School of Public Policy, suggests that it is likely companies gather data from these patient programs. She notes that companies might use information related to adverse events, patient survival, and patient characteristics to inform the design and recruitment strategies for future trials.

Yescarta is an approved drug, and the Kite Konnect support site collects data on patients who receive the therapy as prescribed. Other sources of real-world evidence could include free drug programs for patients who cannot afford treatments and compassionate use programs for therapies not yet approved or withdrawn from the market.

In April, Amylyx announced the withdrawal of its amyotrophic lateral sclerosis (ALS) drug Relyvrio (AMX0035) following a failed Phase III trial. However, the company allowed existing patients to continue using the product if they chose to. Amylyx's Co-CEOs Josh Cohen and Justin Klee clarified that this decision was not for research purposes and that no data is being collected beyond mandatory adverse event reports to the FDA.

Erela Dana, a director of neurology at GlobalData, pointed out that this continuation could help gather real-world safety data for AMX0035, Amylyx's lead product. According to Dana, prolonged use in the real world could provide valuable safety information for both physicians and the company.

Amylyx is also exploring AMX0035 for conditions such as Wolfram syndrome, progressive supranuclear palsy, and Alzheimer’s disease. Dana believes that established safety profiles and physician familiarity are crucial for commercial success if approval for these additional indications is achieved. The extended use of AMX0035 for ALS may contribute to these goals.

Despite the potential benefits, patient assistance programs have limitations, as noted by Allison Bateman-House, an ethicist at New York University School of Medicine. Bateman-House, who co-founded NYU’s Working Group on Compassionate Use and Preapproval Access, emphasizes that while safety information from these programs can guide research design and development, it cannot replace clinical trials.

In clinical trials, numerous controls are put in place to ensure accurate results, whereas real-world data lacks these stringent measures. Therefore, real-world data is more suitable for assessing safety rather than efficacy. However, Bateman-House believes that real-world data can generate ideas for new trials.

Patience Asanga is a freelance science journalist based in Nigeria who specializes in writing about the environment, biotechnology, and life sciences.