Clinical trials – those tightly controlled studies to determine if a medicine works – can’t happen without patients who are willing to participate. So it makes sense, given that they are experts in their disease, that patients are playing a greater role in how clinical trials are designed and delivered.
Positioning patients as partners in clinical trial design is the subject of a panel discussion I’m participating in on December 3 at Pharma & Patient USA, a virtual event. I’ll be one of of five panelists. Two of us represent the pharmaceutical industry; the other three panelists will bring the patient perspective: Dave Fuehrer of GRYT Health, a company that connects patients for peer-to-peer patient support; Susan Schaeffer of The Patients’ Academy for Research Advocacy; and Steven Taylor, President and CEO of the Sjögren's Foundation. Just the composition of this panel demonstrates how things are changing. Not long ago it would have been all industry with, hopefully, at least one patient. Things are evolving and it’s to everyone’s benefit.
This event brings together patients, patient advocacy groups, regulators and industry for a multi-dimensional discussion. I’m looking forward to hearing from the talented and passionate people who are doing this important collaborative work. And I’m especially honored to have the opportunity to listen to, and learn from, the patients we strive to serve.
Many decisions go into designing and setting up a clinical trial. The primary goal of any trial is to determine if the medicine is safe and effective. That requires designing the outcomes − the endpoints − carefully; this is critical in the process of expanding treatment options for patients. Just as important is the operational design, including where patients will go for treatment, whether any at-home care will be provided and how often they will have to come in for visits at the clinic trial site. If a trial’s requirements are too burdensome or don’t address what matters to patients, they might drop out or decline to sign up in the first place.
Acknowledging the obstacles, a recent journal article in Clinical Therapeutics attempted to “score” trials to reflect the burdens and demands on the patient. The researchers called it the “Patient Friction Coefficient” and recommended using the tool to design better trials that “reduce, and ultimately avoid, entirely inefficient practices” from the patient’s point of view.
For its part, CSL Behring has been working to involve patients more in the clinical trial process, and even earlier in the development timeline. For example, we asked sickle cell disease patients to help design an app to explain an upcoming clinical trial; we added a patient who has hereditary angioedema (HAE) as a member of the study team working on the design of a Phase 3 trial; and earlier this year, CSL invited patients who have the immune system disorder, Sjögren's Syndrome, to a roundtable discussion.
The goal of these and other engagements is to better understand the disease as the patient experiences it and incorporate that understanding into the design of the trial. Ideally, that involvement happens well before the company has a potential product available for a clinical trial such as it did with the Sjögren's roundtable.
Looking back to when I started in this industry, we have come a long way in acknowledging the patient as an educated expert in their condition. But we all need to keep going. I see such potential for growth and partnership. Whether you are a patient, an advocate, a regulator or a researcher, we all need to listen more and seek to understand one another’s perspectives and objectives. As one of my fellow panelists wisely said: “We all need to come with a humble heart.”
Pharma & Patient USA runs December 1 through December 4. Learn more about the patient groups appearing on the clinical trial design panel: