Deborah Collyar, President, Patient Advocates in Research (PAIR), stresses the importance of patients being at the front and center of clinical trials
Terms like “patient centric” are all the rage these days. It’s meaning, however, depends on who bandies them about, but the ways in which to effectively achieve patient centricity are in very short supply. Unfortunately, the needs of the patients are usually at the tail end of the research process rather than the beginning.
In truth, the system has focused on everything else first, before the patients. This is evident in every journal article and, frankly, in every conference – all you have to do is peruse the titles. Patients feel like afterthoughts (at best), and think system priorities create barriers to what they need and want most. Sponsors often spend a great deal more time, money and effort on endpoints, regulations, and publications than they do on learning true patient needs and issues. While the Merriam-Webster dictionary provides a generic definition for the term “centricity,” patients use a different mantra, “Nothing about us without us!”
If patient centricity is to mean anything, the research system must change terminology, mindsets, and approaches to drug and device development. Sounds like a tall order, I know, but bear with me!
I was asked to share some key points on this topic while speaking at the Arena International 8th Annual Outsourcing in Clinical Trials West Coast 2016 Conference. Our panel discussion, “Considering the growing trend of patient centricity to highlight the need for all sponsors to put patients in the centre,” included Yu Ping Yen, Vice President of Clinical Operations from Dance Biopharma, and me. We accepted the challenge to share key points and examples in 30 minutes (total), and then met later with attendees to field questions. Here is a copy of our presentation.
It is clear that we are now in a targeted medicine landscape. Targeted therapies demand that every aspect of a clinical trial also needs to be targeted, starting with patients. It is important to understand that people become patients, and they have already received conflicting messages along the medical path before being diagnosed with an illness, disease or condition. New molecular tests also complicate the issue, so learning how to find AND interest the specific patients you need becomes critical. It is also crucial to turn validated data into useful knowledge that actually brings affordable results to patients. Shortcuts that stop at “big data” can produce false information that can actually harm people instead of providing them with meaningful answers.
Back at the conference, the audience was asked what “patient” meant to them. Most nodded while seeing pictures of people in short gowns, waiting in clinics, or even as blood samples that were being analyzed by lab technicians. Clearly, the system has treated patients as disease data points or means to an end rather than key contributors who deserve respect. Instead, patients see themselves as whole people who are family members, and happen to have a disease or condition. These people are looking for answers, and they want to work with providers who can explain complicated concepts (and how it could impact) / (in context to) their daily lives.
We then ventured into different kinds of patient centricity. Yu Ping Yen provided information on diabetic care that was managed precisely with each patient during a clinical trial. Individualized plans were developed with patients after engaging discussions about lifestyle, diet, risk factors, comorbidities, and what type of support system existed for them. Discussions about modifiable risks and different types of insulin treatments were also explained to enhance the shared decision-making process.
The U.S. Food and Drug Administration (FDA) actions regarding patient centricity were also highlighted. The FDA actively seeks patient input to inform their pre-market and post-market decisions. This includes interaction at conferences, meetings, and grassroots efforts such as the Twitter #StripSafely campaign to ensure safe and accurate test strips for diabetes patients. The FDA has also had a Patient Representative Program for years that allows patient representatives to participate in pre-approval meetings with sponsors, and serve on Advisory Committees.
Other ways to incorporate patients into the development and implementation of clinical trials was presented. For instance, PAIR members have actively participated as partners and peers in clinical research since 1994. We bring reality to research by infusing patient needs and experiences into specific research dialogues, while helping to resolve barriers that hinder bringing relevant research results to patients.
Many examples of patient partnerships exist throughout the spectrum of clinical research, starting with feasibility of study designs and eligibility criteria. We also work in all phases of clinical trials and with key collaborators, including regulatory agents and IRBs, to help speed up the trial process and to minimize risk. In addition, we can develop successful recruitment plans and materials with sponsors and CROs that provide greater understanding and can impact specific communities and populations in mutually beneficial and ethical ways. Last, but certainly not least, we can create plain language study summaries that clearly explain trial results in meaningful ways to patients.
So, what does it take to build effective patient-centric clinical trials? The concepts are actually quite simple, although it takes renewed commitment to achieve them. Viewing patients (and their representatives) as valuable peers, and showing respect at each step is the key. This requires mutually agreed-upon ground rules that allow for an open exchange at each decision point, building jargon-free, reciprocal communication, and adjusting plans along the way. Sharing emerging research issues with your patient partners also ensures relevant input.
There are very few patient representatives that have clinical research expertise across the board. After all, we are no different than any other partner in the research system. Groups like PAIR know this, and can help fit the patient expertise needed for each stage of the clinical trial process.
Key points in a 30-minute conference slot may sound interesting. What we can do by bringing real patients into clinical trial design and operations, however, is create amazing advances that reflect the true meaning of patient-centered research. “Patient-centricity” means putting patient needs first, not last. We are all learning how to listen and learn together to build better products that meet patient needs for years to come.