By Lori Abrams, Patient Engagement Solutions, LLC (Bristol-Myers Squibb, retired)
Patient-centricity. Patient at the center. Voice of the patient. The pharmaceutical/biotech industry frequently uses these phrases when discussing the inclusion of patients and caregivers in clinical trial development. I used them when I was in the pharmaceutical industry, and they meant something to my R&D diversity and patient engagement team. We believed bringing patient and caregiver insights, experiences, and opinions into the drug development process was paramount. Without them, we wouldn’t understand their priorities and obstacles.
There are many ways to accomplish this, both long- and short-term. An example of a shorter-term engagement is inviting patient advocacy groups (PAGs) into Phase 2 planning meetings prior to development of a synopsis. This is an excellent opportunity to begin to build rapport with PAGs, but, more importantly, to ask questions about the patient journey and obstacles that may impact the trial. It is also a time to listen and ask the PAG what is important to patients and their families. Ultimately, this is the beginning of patient-centricity.
A long-term example of bringing patients, caregivers, and advocacy into the drug development process is to form a cohort of this group in the clinical trial team or drug program as early as possible. This cohort is taught the clinical trial process, the roles involved in the trial, and about the asset. They may meet in person or virtually. The group is involved in areas such as synopsis and protocol review, decisions for operationalizing the trials, creating recruitment materials, presenting at investigator meetings, and identification of new investigators. Ideally, this cohort stays together at least until regulatory submission.
Our team believed if we listened, we would improve the clinical trial process. After years managing clinical trials, we sometime forget there are others who know more than we do — the patients. One experience I will never forget occurred when I was in patient engagement on a Duchenne Muscular Dystrophy study. The team was discussing how significant it would be for the boys to be able to walk for a longer period. In fact, many believed it was the most important goal. However, I queried several parents and their sons, and all of them disagreed. To them, the most important aim was to be able to use their arms and hands for as long as possible, as this would continue their independence. They could eat on their own, use the computer, hold the telephone, and operate their wheelchairs.
We all learned a lesson from this. If patients and their loved ones shared their stories with our colleagues, it would make our day-to-day contributions more meaningful, while building empathy.
Conducting seminars with patients, caregivers, and advocacy groups empowers those living or working daily with the disease. It shows them the organization cares about them and their struggles. It is also important for colleagues, no matter their role, to learn about the company’s pipeline, not from a scientific perspective, but from a humanistic one. It is vital to collectively understand whom an organization is working for and to realize each role is important in its own way.
While validating our ideas and assumptions was part of the process, listening was more important. We often learned that while we were on the right track, the patient perspective helped us determine a clearer direction or develop more concrete questions. The number of changes to the draft and final trial executions validated this. It was understood from day one that not all recommendations from the cohort would be incorporated into the study. But we had an agreement that we would always explain the reason (e.g., scientific, legal, regulatory). Over time we realized we made more changes to the draft protocols and other documents than anticipated. I attribute that to two things: an educated cohort trained by our team and a clearer and better understanding by the clinical team stemming from the incorporation of the cohort.
Continually discovering methods of integrating patients into our processes made us a better company. Early on we were not sure how deep the cohort contributions to the clinical teams would be. It clearly evolved over time. As trust and confidence were built, some ideas came from the cohort and others from the clinical team. I believe there are opportunities still to be discovered.
The Advocacy/Patient Perspective
Today I stand on the outside looking in. As a consultant, I work with patients, advocacy groups, and the industry in patient engagement. The view is very different, but the task remains the same. Advocacy groups, patients, caregivers, and the industry want to partner. Patients and advocacy understand their insights and experiences can greatly impact the overall design and execution of a drug development program. And the industry realizes patient insights and participation are vital to success. However, they don’t always know how to make it happen.
Advocacy has perfected the grant proposal process, seeking money for walks, galas, and educational projects. However, they have not achieved a model for designing and bringing forth R&D clinical engagement plans to the industry. If they were to proactively approach the industry with strategies to partner with them to develop more patient-centric drug development programs, it would be a win-win.
Being on the outside, people are more open to share their concerns and feelings, especially patients and caregivers. They believe the future can be better with everyone rowing in unison toward a common goal. While intentions are good on both sides, there is still a gap. Let me explain.
Ask 10 people in the industry what “patient at the center” means to them, and you may receive 10 very diverse answers. Speak with a patient and be prepared to hear the hard truth. While there has been progress over the past seven to 10 years, from a patient’s perspective, the industry is not always listening, but, more importantly, not always asking. Patients convey that it is not unusual to be asked to participate in a transactional manner (reviewing a protocol or completing a survey). These are useful methods of collecting patient feedback. However, it’s often one-and-done and goes into the black hole of “feedback provided” without the patient seeing any impact.
Pharma would like to gain insights and develop partnerships and is often proactive in its approach. If it focuses on developing long-term relationships instead of showing up only when it needs help, PAGs would be more receptive. This is something I routinely hear from advocacy. In fact, there is a term for it: “helicoptering in and helicoptering out.” It means you only show up when you need something.
The industry has a unique opportunity to leverage knowledge and experience that was overlooked for decades. This does not mean inviting a group in to review a protocol. It is providing a tangible role for the patients and caregivers on your team. Each patient group has unique challenges that require up-front arrangements. This takes corporate support, precise organization, and careful planning; it takes real courage. But most of all it requires empathy, kindness, and the belief that it is the right thing to do, both scientifically and ethically.
Metrics: What Is The Outcome Of Your Relationships?
It took my team years to determine the best metrics to measure our success. We developed both qualitative and quantitative metrics for specific tasks. These included trial performance (endpoints, first patient last visit (FPLV), trial complexity), development portfolio decisions (development timelines, go/no go), and internal/external reach (number of new sites, number of new investigators). So, there are mechanisms to measure whether patient engagement in clinical trials is adding value to your development program.
Metrics are important to see if specific tasks have impact. But sometimes there are more important methods of measuring success:
Remember, today’s clinical trial partner is tomorrow’s post-approval patient. They are the experts who can shape a program over time, be a voice during the regulatory approval process, and provide insight on what other patients hope to find in a therapy.
About The Author:
Lori Abrams is the owner of Patient Engagement Solutions. She brings together advocacy and industry to identify when and how external stakeholders can impact the planning and execution of new drug development or clinical programs. Abrams previously was in R&D at Bristol-Myers Squibb for 20 years, the last five as director of diversity and patient engagement. This group integrated the voice of the patient and caregiver into the clinical trial process, developed relationships in the disease-based and minority-focused communities, and helped colleagues understand the challenges of those living with diseases. You can email her at firstname.lastname@example.org or connect with her on LinkedIn.