At the time of her first diagnosis, Deb was a computer company executive. Along with her husband (also a cancer survivor), she got involved with an oncology patient advocacy group. This was in the early 1990s when very little was known about effective cancer treatments, and she felt that this was important to change
A patient advocate contributes in a dramatic way to improve the experience of patients they don’t know through direct patient support, political advocacy, fundraising, and watchdog advocacy. A research advocate takes a long-term partnership approach. They focus on improving and speeding research results and ensure patient results and things that are important to the patient community, not just the scientific community, are a major focus.
Typically research has been all about science, product development, and regulatory compliance. No one has truly focused on the patient experience or the fact that the patient is the true end-user. This needs to change, and clinical research should set goals for finding better treatments, not just more, that give patients the improvements they’re looking for.
While they are different, they can be considered together so we can get to the answer we’re all looking for. Companies may be focused on getting label approval from regulatory agencies, rather than the usefulness of this product and how it can fit into the rest of a patient’s life. This is where the patient voice is crucial, because it helps us complete the picture and cover aspects that are actually important to the patient community.
Not recognizing the patient as the end-user can negatively impact the results patients get from a treatment. By involving patient advocates in the trial design process, sponsors can avoid or eliminate barriers that prevent individuals from enrolling in a clinical trial. Sponsors can also turn to adaptive trial design, which celebrated its 50th anniversary in 2019, as a potential way to improve the patient experience.
It does vary. In oncology, for example, cancer is very multi-faceted; it can be chronic, life-threatening, acute, and so on). The type of cancer or patient situation can make a big difference in terms of what will be effective.
Currently, the industry approach to “patient centricity” still views the patient as a target to get data from. To address this, we need to start thinking “what’s in it for patients?” instead of “what’s in it for me (as a sponsor)?” Focus on patient needs ensures that regulatory and product concerns will also naturally be addressed.
Things are definitely improving, and more people are understanding the difference in perspectives among trial stakeholders. However, the scientific and medical communities need to be more in line with the patient perspective of a “cure”. There also needs to be an increased recognition of the fact that clinical research is often an endurance test for patients, particularly when sponsors are looking to address retention or compliance issues.
A breast cancer study funded in part by the National Cancer Institute was the first trial that explored neoadjuvant therapy. Patient advocates were involved in the trial development process from the beginning. As a result, the study recruited patients quicker, required fewer amendments, cost less, and was completed faster. Bringing in patient advocates early was crucial to the trial’s success..
We’ve all heard the terms “real-world data” and “real-world evidence.” The concept is important and has a lot of promise, but we have to think about what’s in it for patients. A shift from real-world data (raw material), to real-world evidence (analytics) to real-world answers is key to addressing what patients, families, and doctors really need and want.