Practical Approaches to Improve Patient Centricity

Patients are at the heart of clinical research, but ensuring they remain the focus takes dedicated effort. A true focus on patient-centricity in clinical research will involve collaboration between all stakeholders in the drug development process: regulatory agencies, sponsors, sites, and payors, but most importantly, patients. Implementing more patient-centric tactics in drug development not only facilitates the successful completion of clinical trials, but can ensure the methods, measures, and intended outcomes are meaningful to patients. More and more in the drug development process, patients are being engaged as key stakeholders in the early design and development process. The FDA committed, following the 21^st Century Cures Act of 2016, to prioritize patient engagement in order to warrant the collection of outcomes that are meaningful to patients and examine how the disease affects both patients and caregivers. They issued the first two of four guidances on patient-focused drug development addressing key questions: 

• Guidance One (final): From whom do you get input and why?
• Guidance Two (draft): What do you ask and why?
• Guidance Three: How you do you decide what to measure in a trial? 
• Guidance Four: How do you incorporate a clinical outcome assessment or set of measures into a clinical study endpoint? 

The agency is shifting to engaging patients early in the conversation in order to build their feedback into design rather than just engaging patient advocacy groups further along in the development process or upon receipt of regulatory approval. Recognizing that patients are at the core of the work the FDA does, the agency has initiated multiple opportunities to include patients in the process from early development through to the approval of new products such as Patient-Focused Drug Development groups to obtain information from patients such as patient perspectives on current treatment approaches or clinical trial considerations, and collaborative forums or listening sessions to hear directly from patients about topics of most importance to patients and families such as disease burden or quality of life factors.

While the FDA is focused on outcomes and early patient engagement, sponsors have also begun implementing innovative strategies within study design that more actively engage patients. These strategies that use patient feedback in the planning and development of studies are intended to reduce patient burden, simplify the collection of data, and enhance the patient experience. Patient centricity is universally seen as important, but the implementation of new tactics and lasting change is slow. Recruitment and protocol adherence are two issues that plague the efficient completion of clinical trials; a shift in focus towards patients will address both issues acknowledging there is some investment early in the study design to make this happen.

Patient Centricity means different things to different people. The common theme and primary goal of having a patient-centric clinical trial is to shift the mindset by having patients be the forefront of treatment and be recognized as fundamental consumers of healthcare. They are the experts of their own disease. This will allow sponsors to look through the lens of the patient. It is critical that industry ensure patients are educated enough to become partners in the process. When patients are informed, they make better choices and have more control in making decisions relative to their treatment. Here are some ways industry can help patients be their partners:

• Industry experts can show the patient what companies do by walking them into a lab, a manufacturing suite, and a regulatory conversation. Manufacturers can identify the types of evidence that will best resonate with patients who are later evaluating their products. This can help nurture the relationship and is a practical and tangible way to let patients “behind the scenes.”
• Companies can ensure patients are educated and informed by showing them resources like patient advocacy organizations such as the Celiac Disease Foundation or Leukemia & Lymphoma Society. Having access to information on clinical trials will give patients the opportunity to learn and engage with industry stakeholders.
• Companies can engage select groups like caregivers, minorities and patients that live in geographically isolated areas. It is important to have patient opinion leaders as partners within the community to educate their peers and provide a diverse perspective. An example is Project LEAD®, an organization that helps breast cancer advocates become research advocates. It empowers patients with the skillsets they need to become actively engaged in real discussions with industry.
• Companies can have more meaningful communication and engagement with patients ensuring the communication is clear and fit for purpose. This goes from end to end, and it is key to begin early on throughout the product lifecycle. Relationship building is important as it allows trust between all parties. One way to do this is by designing or creating a storyboard for your clinical trial to tailor the patient’s experience and personalize it from beginning to end. Consider sharing data with patients by developing a lay summary while also then thanking patients to further build trust within the community.

New technologies have arisen focusing on advancing more efficient management of data while others have focused more on easing patient burden with the use of apps or wearables for at-home assessments, virtual or site less trials, telehealth, and at-home study visits. The challenge for sponsors is ensuring the technological advancements work efficiently together and do not create more of a burden on patients or sites. This is another area of feedback that sponsors are really chasing during early engagement with patients. With COVID-19, there is an impetus for industry to reimagine how trials are conducted. The use of telehealth options has been very successful in maintaining protocol compliance by allowing patients in some cases to be treated and evaluated in the comforts of their own home.

Ongoing engagement between patients and industry representatives is imperative to bring meaningful change to drug development. Shifting the mindset to see patients as partners will inevitably lead to trials becoming more patient-centric. For patients, that means clinical trial development that leads to improved patient experiences and satisfaction, greater accessibility to participate, decreased patient burden, and increased diversity among participants. For sponsors, that shift in focus towards patients is expected to help speed recruitment, engage hard to reach populations, reduce dropout rates, and increase adherence to study protocols which then speeds development, decreases costs, and increases data quality. All of these together lead to increased trust and strengthened partnerships among all stakeholders to better inform trial design.

Patients can provide invaluable insights based on their personal experiences, and sponsors have increasing opportunities to draw upon those experiences to bring products through higher-quality development. Just being a part of the conversation won’t be enough, it’s time for sponsors to take action to make this happen.