The landscape of clinical trials has evolved significantly over recent decades, moving from a purely scientific focus to a more patient-centric approach. This transformation acknowledges the crucial role patients play in providing data and insights that are essential for developing new drugs and treatments. However, the true value of patient collaboration in clinical trials extends beyond data collection: it encompasses improved study design, enhanced patient engagement, and ultimately, more meaningful research outcomes. Despite the obvious benefits, patient collaboration has not yet become standard practice in clinical trials.
The patient’s voice isn’t just important, it’s decisive
Who is more qualified than the patients themselves to guide us through their experience and quality of life?
While sounding obvious, patients are the cornerstone of clinical trials, providing critical data that are necessary to develop new drugs and treatments. Not only do patients provide unique biological samples, clinical measurements and imaging results that are fundamental for assessing the safety and efficacy of a drug, but they also offer invaluable direct feedback on their symptoms and experiences through electronic Patient-Reported Outcomes (ePROs). This feedback provides deeper insights into the impact of a drug on patients’ daily lives, going beyond clinical measurements to capture the “real” effects of treatment. ePROs help researchers understand the nuances of patient experiences, including any side effects and the overall quality of life during the treatment. This comprehensive data collection is crucial to accurately characterize a drug’s profile and determining its true benefit to patients.
Without patient involvement, it would be impossible to collect such comprehensive data, slowing progress in healthcare and medical research. Patients’ unique insights are critical for ensuring that drugs seeking marketing authorization are not only effective and safe but also beneficial and meaningful to those who will use them. These data allow researchers to fully understand a drug’s effects and potential benefits, tailoring treatments to better meet patient needs and improving overall healthcare outcomes.
As well as informed participation, strong engagement throughout the trial is essential
Patients must be motivated to participate, which requires a clear understanding of the trial process, duration, and the significance of their contributions. Patients not only need to be fully aware of the potential risks and benefits, the procedures involved, and their rights as participants, but their engagement will build trust and willingness to participate, leading to better enrollment and lower dropout rates.
As well as informed participation, through involving patient communities and advocates early in the trial design and feasibility assessment, researchers can ensure that the trial is truly patient-centric. This means considering patients’ needs, preferences, and experiences, to enhance the relevance and impact of the research. This also means allowing patients to play an active and crucial role in the clinical protocol decision-making process, such as influencing trial design and procedures, choosing endpoints that matter, and building relevant supportive material. A patient-centric approach fosters a sense of partnership and collaboration, ultimately leading to more successful trials and better healthcare outcomes for all.
A deeper exploration into how early collaboration with patients in protocol development can further enhance trial success is essential to fully understand the benefits of a patient-centric approach.
Early patient collaboration: Unveiling the true value in protocol design
Collaborating with patients’ representatives is important, but so is collaborating at the right time. Involving patients early in protocol development brings multiple advantages.
- More relevant protocol designs: Collaborating with patients early in the protocol development process leads to the creation of more relevant and patient-centered study designs. By incorporating patient input, researchers can ensure that the trial addresses issues that matter most to patients. This enhances the relevance of the research findings to everyday clinical practice, ultimately making the outcomes more impactful and useful to patients and healthcare providers.
- Studies are more feasible and practical: Early patient collaboration ensures that study designs are not only relevant but also feasible and practical. Patients can provide insights into potential logistical challenges and barriers to participation, such as the frequency of visits, travel distances, and the complexity of procedures. By addressing these issues upfront, researchers can develop protocols that are easier for patients to adhere to, thereby enhancing the overall feasibility of the study.
- More meaningful endpoints: Involving patients in the development of study protocols helps to identify and select endpoints that are truly meaningful to them. Patients can highlight outcomes that have a direct impact on their quality of life and daily functioning, ensuring that the study measures what matters most to them. This patient-centric approach to endpoint selection enhances the relevance and significance of the research findings.
- Selection of relevant Patient-Reported Outcomes (PROs): Patients play a crucial role in selecting the most relevant PRO measures. By incorporating their feedback, researchers can choose PROs that accurately reflect the patient experience and provide valuable insights into the efficacy and safety of the intervention from the patient’s perspective.
- Improved patient experience of clinical trials and digital solutions: Early patient involvement improves their overall experience of clinical trials, particularly when it comes to using digital solutions. Patients can provide feedback through participant feedback questionnaires and user tests, which can help researchers understand their perspectives and preferences regarding the use of digital solutions for protocol procedures. This feedback is invaluable for designing digital tools and platforms that are user-friendly and meet patients’ needs, and can be provided during or at the end of the study via an electronic Participant Feedback Questionnaire.
By making clinical trials more accommodating and less burdensome, patients will benefit from a better clinical trials experience, and this will have a real impact on patient engagement before and during the trial:
- Enhanced recruitment and retention: Patient-centric designs are more attractive to potential participants, leading to higher recruitment rates and improved retention. This reduces the overall duration of the study as fewer participants are likely to drop out.
- Reduced dropout rates: By addressing patient needs and preferences, dropout rates are minimized, which in turn reduces the number of patients needed to achieve the study’s goals. This leads to shorter timelines and lower costs.
- Increased patient compliance: Protocols that consider patient feedback tend to be more patient-friendly, resulting in higher compliance rates. This ensures the collection of more comprehensive and accurate data.
Involving patients early brings a lot of advantages, and has been pushed by regulatory agencies for several years.
Regulatory catalyst: Accelerating patient advocacy in clinical trials
Over the past two decades, regulatory have marked a turning point in patient centricity. By producing patient-centric guidelines, regulatory agencies have accelerated the shift towards clinical trials that are more aligned with patient needs, thereby improving the practicality of patient-focused trials. This has opened the door for industry stakeholders willing to collaborate with patients: now they have a framework!
Examples of key regulatory initiatives towards patient-centric approaches:
- The U.S. Food and Drug Administration (FDA) has been a major leader in integrating patient perspectives into drug development and regulatory decision-making. The FDA Patient-Focused Drug Development (PFDD) Initiative and Guidance Series, launched in 2012, aims to systematically gather patient perspectives on their conditions and available treatment options. This initiative includes a series of guidance documents that provide a framework for incorporating patient input throughout the drug development process. The guidance covers topics such as collecting comprehensive and representative patient input, developing and using patient-reported outcome (PRO) measures, and including patient input in benefit-risk assessments. One of these focuses on the Inclusion of PROs in Clinical Research. The FDA recognizes the value of Patient-Reported Outcomes (PROs) in clinical research, and issued guidance in 2009 on the use of PRO measures to support labeling claims emphasizing that PROs can provide critical information on the patient’s perspective. Additionally, the FDA’s guidance on patient-centered outcomes in oncology underscores the importance of incorporating patient feedback to better understand the impact of cancer treatments on patients lives
- The European Medicines Agency (EMA) has also taken significant steps toward patient-centricity. In 2006, the EMA published a reflection paper on the regulatory guidance for using health-related quality of life (HRQL) measures in evaluating medicinal products. This document highlights the importance of HRQL measures in understanding the broader impact of treatments on patients’ daily lives and overall well-being.
- EMA Initiatives with EURORDIS for Rare Disease Drug Development: The EMA has also initiated collaborations with patient organizations, such as EURORDIS, to advance drug development for rare diseases. These initiatives aim to ensure that the perspectives of patients with rare diseases are considered in the development and evaluation of new treatments. By working closely with patient advocacy groups, the EMA can better understand the unique challenges faced by these patients and ensure that their needs are addressed in clinical trials and regulatory processes.
This proactive engagement ensures that studies are designed with the patient’s perspective at the forefront, leading to more practical and impactful research outcomes. Ultimately, all these factors contribute to more efficient clinical trials. Studies are completed faster and more cost-effectively, with high-quality data that are truly reflective of patient experiences and outcomes.
The integration of patient-centric principles into clinical trials represents a significant advance in medical research. Regulatory bodies have taken positions and pharmaceutical companies—and some clinical trial organizations—have made strides in incorporating patient perspectives. However, to fully realize its value, patient-centricity should be globally recognized and systematically implemented. Stakeholders are poised to enhance patient collaboration and develop more effective clinical trials, ultimately leading to improved healthcare outcomes for all.
