Historically, drug development has involved collaboration among life science companies, researchers, healthcare providers, and regulatory authorities. Clinical trials for potentially life-changing medicines were designed and conducted in a manner that was best suited to the study sponsors, and patients were merely “subjects” who enrolled in these trials and “adhered” to study protocols. Their input was not solicited, expected, or considered important. Indeed, the word “patient” itself implies a sense of passivity, derived from the Latin word “patiens,” meaning “to suffer or bear.” However, at the turn of the 21st century, a move toward democratization of the healthcare research process began—a push for research performed “with” or “by” the public rather than “for,” “about,” or something being done “to” them.
Today, patient-centered care has slowly but definitively taken center stage—but how did we get here?
As access to health-related information continues to increase, patients today are increasingly well-informed. Advances in technology, such as mobile applications and smart watches, allow patients ready access to real-time information related to their health. Armed with information and, arguably, the ability to research and form opinions about their healthcare, patients today are more engaged with their health and are empowered to actively participate across the continuum of drug development. Importantly, patients and caregivers, as the ones living with and closely observing a medical condition, can provide unique perspectives to help steer life science research and development in the direction of realizing patients’ unmet needs, ultimately leading to drug development that is more relevant and valuable.
Optimizing health outcomes starts with optimizing patient engagement
Life science companies are recognizing the importance of early and active patient involvement across the drug development lifecycle, from pre-clinical stages to post-market activities. Gathering perspectives and input from patients and caregivers at key stages can help infuse the “patient voice” in drug development and communication, making them feel seen and heard and creating a sense of ownership of the data. One way of achieving this is through the inclusion of patient-reported outcomes (PROs) in clinical trials. PROs are now not only commonplace but also mandated by some governments. For example, the UK has mandated PROs since 2009, and the US has started implementing PRO mandates in some surgical procedures as of FY 2023. PROs are a measure of a patient’s health as reported directly by the patient, without added or prior interpretation by a clinician or any other healthcare professional. PROs provide insight into patients’ quality of life and functional status and allow for a holistic view of treatment benefit.
Patient engagement can begin right from setting research priorities—this can help identify unmet patient needs and relevant patient populations and define patient-relevant outcomes. Empowering patients to define key elements of research has many benefits, for example,
- Patient involvement and input can help identify important research questions and patient-relevant protocols, eligibility criteria, and clinical endpoints during research design and planning.
- PROs can complement patient preferences in the design of later-stage trials, for example, by informing dose selection based on patient experience.
- Patient empowerment can generate tangible benefits for health equity and access by improving patient representation and increasing the diversity of the patient population in clinical trials.
- Patient engagement improves patient retention in clinical trials through the use of patient-friendly data collection methods and protocols, including digital innovations that allow for some trial activities to be conducted remotely in a patient’s preferred setting.
- Patient inclusion enhances the development and accuracy of patient information/education material and informed consent forms in terms of content as well as design, language, and readability.
During research conduct and operations, timely communication of protocol amendments and new safety information can help increase patient trust in the entire process. At this stage, patient input can also be solicited for reporting of study results. For example, many clinical trial participants have expressed interest in consulting on the dissemination of interim results and sharing of information within the patient community. Finally, post-approval, patients can be involved in post-study communications (including the development of plain-language summaries), contribute to real-world knowledge and insights, support access and reimbursement, and advise on patient education. Collectively, these measures can be useful in ensuring that patient priorities are addressed at every stage.
In summary, a structured collaboration between life science companies and patients, caregivers, and patient advocates can provide diverse perspectives and promote the exchange of ideas, which in turn can positively influence clinical research design, planning, conduct, and execution. Empowering patients and involving them as key stakeholders in the drug development lifecycle can help deliver impactful, long-lasting, and positive health outcomes to the ones the life science industry aims to serve—the patients. The future is well and truly here.