Patients today are more involved in their healthcare choices than ever before, and gaining access to accurate, relevant and easily understandable information can help empower them to make more informed decisions about their health. Providing patients with patient-reported data from clinical trials can shed light on the potential benefits and drawbacks experienced by participants, directly aiding their decision-making process.
Learning about the results of clinical trials can be a daunting prospect for patients. It’s here that information sheets or resource sheets, which provide information about clinical trials and are written to be understood by a lay audience, may be helpful. Developed through researchers and patients working together, a resource sheet can provide a means of sharing clinical trial information with patients in a way that is understandable, sensitive and empathetic, and which resonates with them, empowering their choices. The collaborative approach used to develop these patient resources ensures that clinical trial data is not just presented but explained and contextualized for the patient. By bridging the gap between research and patient needs, resource sheets can both inform and change healthcare decision-making and, ultimately, improve patient outcomes.
The rise of patient-focused drug development
In the US, patient-focused drug development (PFDD) gained traction in the 1980s when the Food and Drug Administration (FDA) responded to patient groups calling for increased involvement in drug development. Subsequent legislative and regulatory milestones, including the 1997 FDA Modernization Act and 2009 guidance on Patient-Reported Outcome (PRO) Measures, led to the launch of the PFDD program in 2012. This initiative sought to integrate patient perspectives throughout drug development stages, from identifying new drugs to assessing existing ones. More recently, in 2016, the 21st Century Cures Act advanced PFDD with FDA guidance on collecting and using patient experience data and the establishment of the Patient-Centered Outcomes Research Institute (PCORI) to compare treatment effectiveness and patient-reported outcomes (PRO).
Equally, in Europe, the European Medicines Agency (EMA) has also embraced a patient-centric approach to drug development, by working closely with patients and patient advocates, and most recently by highlighting the importance of the role of the patient as an active participant in clinical trials in their “Regulatory Science to 2025” strategic initiative.
All of these developments have not only increased the voice of the patient in the clinical trial space, but also have paved the way for the use and presentation of patient-reported clinical trial data in ways that patients prefer and help to promote patient understanding. As a result, researchers, clinical trial sponsors, patients and patient groups are now encouraged to work together to create informational materials that are clear, relevant, sensitive and understandable, meeting the needs of patients.
The power of resource sheets
Knowledge is power, as the saying goes, and providing patients with comprehensive information about clinical trials can alleviate worry, support dialog with family members and clinical care providers and empower informed decision-making. Providing individuals with information about the condition, its symptoms and treatment options, including benefits, potential side effects and what to expect during their treatment journey, in an easily understandable way supports clear communication between trial sponsors, clinicians and patients.
Historically, patients have provided valuable information to researchers and clinical trial sponsors about how drugs impact their conditions, and partnerships that increase patient engagement and information accessibility drive modern PFDD. By incorporating patient voices into clinical trials and providing accessible and relevant information back to patients, individuals may be better enabled to participate in their own healthcare decision-making. This engagement fosters greater understanding and has the potential to reduce fear or anxiety surrounding diseases.
Already, PFDD is gaining traction in fields such as oncology and rheumatology. By working with patients to determine which particular clinical outcomes are most prevalent and bothersome, researchers can drive the development of PROs such as symptom burden, quality of life, and the ability to function, all of which can in turn be presented to patients via resource sheets. In other clinical areas, for instance, in pediatrics where patients may be harder to communicate with or in populations that have neurodevelopmental or psychiatric conditions, the adoption of PROs and other clinical outcome assessments has been slower. However, it is still not only possible, but also valuable, to engage with these patients and their caregivers to understand what they are experiencing and what is important to them.
Working closely with patients often uncovers various patient perspectives, even within narrow areas such as specific oncology. When developing resource sheets, Patient Advocate Groups (PAGs) can be a valuable resource, partnering with the team to provide insights, determine key variables and criteria, work with patients to facilitate interviews and generally ensure that the tone, language and use of graphics are appropriate.
Building trust, addressing challenges
Creating resource sheets should be a collaborative process. Patients are key to providing real world personal experience, and as the scope of clinical trials expands to include more diverse patient populations, building trust in the relevance and value of the process is fundamental. But there are also challenges that should be recognized:
- Resources – Some of the biggest obstacles to developing resource sheets can be time and budget. Allocating sufficient resources to work closely with patients and fine-tune the methodology is necessary in order to develop resource sheets that truly reflect the patient experience, expectations, benefits and challenges and have true value for future patients.
- Transparency about intentions – There is a need to address any skepticism and mistrust relating to motives and legitimacy. This is particularly true for underserved or more vulnerable or hard-to-reach populations who may feel skeptical of healthcare resources. PAGs are helpful partners not only in identifying and recruiting patients but also in reinforcing the value and legitimacy of the work.
- Respect for the patient – It is also important to remember that patients are often navigating the health system during a life-changing and challenging time. Meeting patients where they are with full transparency, sensitivity, compassion and good intentions and engaging in meaningful dialogue is key.
Another obstacle can be clinician resistance to PROs. Although not always the case, some clinicians may be more accustomed to medical data from sources other than patients and may not always view them as reliable reporters of how they feel. However, there is an opportunity here to provide clinicians with additional resources to encourage more open discussions with patients about their care.
The value of empowered relationships
PFDD will continue to grow in importance, as regulatory bodies such as the FDA are heavily invested in this area and have implemented several initiatives to support PFDD. Researchers working collaboratively and iteratively with patients to integrate their voices into the clinical trial development process will also ensure the development of clear, relevant, sensitive and understandable data that meets patient needs, which is especially important as patients become more informed and demand more accurate and complete information.
Looking ahead, patient-reported clinical trial data will be leveraged and visualized in ways that help to meet patients’ preferences and promote understanding. Resource sheets could be accessible through various channels, including the patient portal, email, smart devices or physical mail. Digital information has the advantage of offering added flexibility for patients to find specific information more easily, such as potential side effects or expected quality of life. Access to this information can help patients make informed choices about managing their health by fostering meaningful conversations between patients, clinicians and family members about their medical care.
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