Patient Assistance Programs A Source of Real-World Data – BioSpace

Patient Assistance Programs A Source of Real-World Data - BioSpace

The Potential of Patient Support Programs as a Real-World Evidence Source

Patient support programs have become an increasingly vital aspect of healthcare, providing assistance to patients as they navigate their treatment journey while also offering a way for drugmakers to gather real-world information about their products. Last month, Kite Pharma, a subsidiary of Gilead Sciences, reported that data gathered from their patient support site, Kite Konnect, showed that its lymphoma drug, Yescarta, was more effective as a second-line treatment compared to its use as a third-line treatment for refractory large B cell lymphoma. The company’s use of this data highlights the potential of these programs to offer a source of real-world evidence about a product.

The Use of Patient Assistance Programs

Many drugmakers, including Eli Lilly, Biogen, Kite Pharma, and GSK, are reluctant to discuss whether they collect patient outcome data as part of patient assistance programs and how this data is used. However, it is likely that companies do collect this information. According to Genevieve Kanter, an associate professor of public policy at the University of Southern California (USC) Price School of Public Policy, this data could be used to derive insights which inform “the design and recruitment of a different trial.”

Free drug programs that help patients access treatments they cannot afford and compassionate use programs that supply therapies not yet approved or which have been withdrawn from the market are also potential sources of such real-world evidence, experts say.

The Case of Amylyx’s ALS Drug, Relyvrio (AMX0035)

In April, Amylyx announced the withdrawal of its Amyotrophic Lateral Sclerosis (ALS) drug, Relyvrio (AMX0035), from the market. However, the company also announced that patients who were already using the product could continue to do so. Amylyx Co-CEOs Josh Cohen and Justin Klee reported in an email to BioSpace that this decision was not for research purposes, and the company is not collecting any data outside the statutory adverse event reports required by prescribing physicians to be submitted to the FDA.

The program to allow continued usage of Relyvrio comes as a benefit in testing the safety profile of AMX0035, Amylyx’s lead product. This benefit is due to the availability of more safety data for both physicians and the company from a longer duration of usage in the real world. The company aims to develop AMX0035 further in three other indications, including Wolfram syndrome, progressive supranuclear palsy, and Alzheimer’s disease. The drug’s continued usage for ALS could help further these aims by providing physician familiarity and an established safety profile for the product.

Limitations of Patient Assistance Programs as a Source of Real-World Evidence

While patient support programs offer benefits in terms of providing real-world information, these programs also have limitations. Allison Bateman-House, an ethicist at the New York University School of Medicine and co-founder of NYU’s Working Group on Compassionate Use and Preapproval Access, cautioned that patient support programs can’t substitute for clinical trials. Real-world data gathered from these sources is subject to biases and artificial barriers, which must be addressed in clinical trials.

However, this data can provide insights that inform trial design. Real-world data cannot be used for efficacy, but it can be a valuable source of information to generate ideas for another trial and provide safety data.


In conclusion, patient support programs offer immense potential in providing real-world evidence about a drug’s effectiveness, safety, and usefulness to patients. Companies see the value in gathering patient data from assistance programs, which are sources of real-world evidence. Nonetheless, it’s essential to recognize the limitations of this data and how it can be harnessed to inform trial design and provide safety data.

Originally Post From

Read more about this topic at
Real-World Evidence
Realizing the Promise of Real-World Evidence

“Powerball Winning Numbers Revealed: $20 Million Jackpot Up for Grabs!”

Fosun Pharma Advances Cancer Treatment Trial – TipRanks