In the life cycle of a new medicine, it’s a major milestone when regulatory agencies, such as the U.S. Food Administration (FDA), approve the product for use by patients following research that showed the product to be safe and effective.
A product approval is important for patients who are waiting for new and better treatments, the company that developed the medicine and for the scientists who did the research. But the scientific inquiry doesn’t stop there. After a medicine is approved, researchers keep gathering data, monitoring how patients are doing and watching out for safety concerns through adverse event reporting systems
CSL recently released data that assessed hemophilia patients receiving a gene therapy in their fourth year following a new treatment. Learn more in this news release.
The follow-up data can be collected in several ways, CSL’s Andres Brainsky said. Phase III clinical trials may have extension stages to continue monitoring patients who were in the research study. In addition, patient data may be collected in Phase IV clinical trials, which are clinical trials conducted with medicines after they have been approved by regulatory authorities. A patient registry can also be used to compile data collected by various clinical sites.
Research focuses on safety and efficacy
This latter phase of the research aims to uncover any safety issues and measure the medicine’s effectiveness over time, said Brainsky, who is CSL’s Vice President for R&D in Hematology. Regulatory agencies oftentimes play a role and may request that a pharmaceutical company gather a certain type of data over a defined amount of time, he said.
The way post-approval research is done can vary, in part, based on the number of people who have the condition or who are expected to start using the treatment once it’s approved. Rare diseases have smaller patient populations, so everything in rare disease research happens at a smaller scale, Brainsky said. The number of clinical trial participants is relatively small and the number of people who will be prescribed a rare disease medicine is also small. That makes post-approval surveillance especially important.
Rare disease patients do a service, not just for themselves, but for their entire patient community when they participate in a clinical trial and then continue to assist with follow-up studies, Brainsky said. That’s why CSL has been working, for a number of years, to reduce the burden on trial participants and be responsive to their needs.
Participating in research can require taking time off work, juggling family responsibilities, traveling to a clinical research site and sitting in waiting rooms. Understanding that, CSL tries to narrow the focus of its post-approval research to lighten the load and make it less likely participants will drop out, Brainsky said.
Clinical trials can only enroll a limited amount of patients, and even though the sample is considered representative of the overall disease population, the number of patients is too small to identify all potential safety concerns. Therefore, post-approval data collection makes it more likely that a safety concern will be identified, he said. People who continue to contribute their follow-up data help create a deeper well of information, beyond just lab report data points, Brainsky said. Their participation can provide information about how the treatment has impacted their quality of life, something that is measured using validated assessments.
“How is the medicine actually helping the patient? When we talk about quality of life, it's not just the patient saying, ‘yes, I feel better.’ There are very specific indicators and surveys that have been developed over time for specific diseases and patient populations,” Brainsky said.
If a product leads to a marked improvement in a person’s quality of life, patients stand to benefit in a broader sense and that data could influence governments and other payers to grant access to more patients, he said. It’s a way to show that a treatment provides a benefit, beyond simply correcting a medical problem, that other, similar treatments do not.
Learn more about how clinical trials work in the animation below.
