In the US alone, an estimated 1.9 million new cases of cancer were diagnosed in 2022, positioning oncology as a key subject of clinical research. Throughout oncology trial development, it is important that stakeholders acknowledge that only patients can fully understand the impact of treatment on their lives. Regulators are now looking beyond clinical indications such as tumor size and delayed disease progression. When evaluating the risks and benefits of treatment, they want to know whether the side effects are tolerable for patients and how these treatments could be improved from the patient’s perspective. Marrying the scientific rigor of clinical research and the human experience captured by patient-reported outcomes (PROs) is essential to the successful development and evaluation of drugs and treatments.
Regulatory bodies, including the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), now expect PROs to be included in study protocols, objectives and even labeling claims. These insights help regulators, payers and providers understand treatments through the eyes of the patient, including how symptoms and adverse events impact study medication adherence and quality of life across patient populations.
However, stakeholders must bear in mind that oncology patients often have limited time and stamina for extensive surveys. Therefore, a patient-centric electronic clinical outcome assessment (eCOA) strategy will effectively capture the patient voice while minimizing patient burden. eCOAs provide patients with the flexibility to complete assessments on their own time from the comfort of their own homes, via a familiar device. This approach amplifies the patient voice across virtual and hybrid clinical trial models, equipping patients and researchers with a simple way to record and exchange data.
The FDA has identified 5 core aspects of the patient experience to consider throughout the development of an eCOA:
1. Disease-related symptoms
When collecting patient responses surrounding the severity of common symptoms for oncology-related drugs, the FDA recommends using numeric or verbal (none, mild, moderate, severe) rating scales. If the assessment is intended to quantify occurrence, a frequency scale (never, rarely, daily, etc.) may also be considered.
2. Symptomatic adverse events
To mitigate patient burden, sponsors should define a concise set of the most relevant symptomatic adverse events likely to occur and capture PRO data in both treatment and control arms.
3. An overall side effect impact summary
To properly evaluate tolerability, regulators need to understand the overall impact of side effects on a patient’s life. For instance, extreme fatigue preventing a patient from leaving their home is a valuable insight to capture.
4. Physical function
Regulators, payers and providers alike need the full picture when it comes to how treatment impacts patient mobility and other physical functions. The FDA recommends using a scale to measure levels of ability with clearly defined concepts.
5. Role function
Perhaps one of the most critical aspects of the patient experience to collect is the overall impact of a treatment on a patient’s ability to work and carry on with daily activities. The European Organisation for Research and Treatment of Cancer QLQ-C30 role function scale is one of the most commonly used assessments to capture this data.
While each of these categories enriches regulatory submissions, they must be carefully chosen with the patient burden in mind. In a perfect world, sponsors would create assessments that capture many or all of these insights in a single tool to make the most of each patient’s time.
In addition to these valuable assessment components outlined by the FDA, there are also several overall best practices for a patient-centric eCOA strategy:
1. Think outside the clinic
The pandemic propelled the clinical research industry to dramatically accelerate its approach to decentralization. While many COVID-19 restrictions have since been lifted, sponsors have continued to leverage technology solutions such as eCOAs to support virtual and hybrid clinical trials.
Whether a trial is conducted remotely or on-site, eCOA data collection eases both the patient and clinician burden. Patients can complete assessments electronically between visits or as part of a remote visit when they are too ill to travel to the site, while clinicians have the flexibility to use an eCOA to collect appropriate Clinician Reported Outcomes via telemedicine. The same eCOA technology can also be leveraged to capture Patient Reported Outcomes and Clinician Reported Outcomes during on-site visits.
2. Establish a ‘bring your own device’ (BYOD) approach
Another positive outcome of the pandemic was the widespread adoption of the BYOD approach in the clinical research space. This method allows patients to access all trial communications and alerts on their own devices, eliminating the time and cost of securing devices for each patient and ultimately accelerating study startup.
Research also indicates that patients are highly receptive to using their own personal devices for a clinical trial. Given that trial adherence depends on ease of use for participants, empowering patients to use devices that are familiar to them is a great way to maximize retention.
3. Prioritize ease of use
Another excellent way to increase trial retention is to cater the solution design itself to patient needs. For example, sick patients suffering from disease symptoms and treatment effects may require breaks while completing their eCOA assessments. Sponsors can proactively address this challenge by creating assessment windows that afford patients ample time for completion. Additionally, the eCOA solution design can be designed to accommodate breaks during and in between assessments as appropriate.
4. Integrate caregiver assistance options
For oncology patients especially, completing an assessment can quickly become too taxing for a variety of reasons, including disease progression, fatigue and treatment side effects. To alleviate this burden, sponsors can establish caregiver support options to assist the patient with inputting their data into the device. With some solutions, it is even possible to offer a caregiver log-in feature to ensure transparency, security and data attribution upon point of data entry.
5. Patients as research partners
In recent years, the clinical research industry has experienced a seismic shift toward patient-centricity. Patients are now viewed as collaborative partners in the research process and in their own care journeys. When studies are designed to prioritize both scientific rigor and the patient voice, sponsors maximize retention, enhance the patient experience and ultimately set the stage for the development of breakthrough oncology treatments.
About Melissa Mooney
Melissa Mooney has over 17 years of experience in the development of IVQIA eCOA solutions for use in clinical trials. Melissa’s area of expertise is eCOA solution design where she has supported clients and eCOA vendors in developing robust and usable eCOA software solutions that meet eCOA protocol requirements. She also brings a plethora of experience in eCOA requirement gathering, leading eCOA User Acceptance testing, eCOA data management, and BD support.