Putting Patients First: A Guide to Patient Centricity in Clinical Trials
What is the definition of patient centricity in clinical trials?
Patient centricity essentially entails bringing the patient voice into healthcare. It revolves around considering patients’ personal experiences and aiming to incorporate this perspective into the design of clinical trials to make their experience more fulfilling and comfortable.
In a patient-centric trial, the focus is not solely on testing the proposed treatment’s efficacy and safety and driving positive outcomes. Patient centricity means ensuring the trial and its treatment are also convenient for the patients and addressing their needs. The idea is to attain the trial’s research objectives while also focusing on creating a positive patient (and/or caregiver) experience.
How is patient centricity different from patient-centered care?
There tends to be some confusion between patient-centered healthcare and patient-centric healthcare. Patient-centered healthcare is focused on fulfilling patients’ needs as identified by the healthcare provider, researcher, medical professional, drug company, etc. In other words, it is a top-down approach. Patient-centric healthcare instead uses actual patient input to guide decision-making, in a bottom-up approach. In the case of clinical trials, the patient is conceptually placed at the center of the development of the research plan and the design of the study and protocol.
What you will find in this blog post
In this article, we will answer the following questions to guide an in-depth discussion about patient-centricity in clinical research:
- What does a patient-centric clinical trial look like?
- How is patient centricity incorporated into study design and conduct?
- What are some examples of patient-centricity in action?
- Why is patient-centricity in clinical trials important?
- What are the challenges involved in adopting a patient-centric approach in clinical trials?
What does a patient-centric clinical trial look like?
A patient-centric clinical trial will aim to build trust between the trial researcher or medical professional and the patients, making the process more comfortable for the patients. A patient-centric trial could involve all or some of the following aspects:
- Increased patient engagement (two-way communication, follow-up, interactive content, etc.)
- Use of clear, simple language to facilitate understanding
- Explaining the participation risk to benefit ratio to the patients in everyday terms
- Encouraging data transparency (providing patients with all the information they need and publishing all study results)
- Using informative online consent forms (eConsent)
- Employing telehealth and remote health endpoint collection (wearable devices, eCOA, etc. to minimize trial site visits)
An important factor in defining what a patient-centric trial would look like is knowing what patients actually want. According to a survey of 3,230 patients and healthcare industry stakeholders conducted by Boston Consulting Group and co-funded by AstraZeneca, patients expressed desire for the following characteristics of clinical trials, in descending order of importance:
- Provide affordable access to treatments and trials
- Reflect real world situations and deliver outcomes that matter to patients
- Information transparency
- Empower patients through services and data, and seek patient input
- Aim to achieve the best possible health outcomes
- Strive for medical innovation
- Easy-to-complete trials
- …for further detail, see the survey results
Adopting a patient-centric mindset
Conventionally, clinical trials have not been designed from a patient-centric mindset. Although patients have always been key members of the trial implementation, they were not usually considered essential stakeholders, especially not during the trial design phase. Communication and engagement with patients during the trial used to be minimal.
This old mindset is diminishing and giving way to a patient-centric mindset. The traditional trial approach is often plagued with challenges relating to low patient engagement, including poor enrollment and retention rates. Today, patient-centricity is almost becoming an essential requirement for successful clinical trials, as it leads to improved patient engagement as well as adherence to treatment, and thus improved trial results.
Adopting a patient-centric mindset means that patients are not seen just as research subjects, but as stakeholders involved in trial design and implementation.
What are some possible actions that can demonstrate to patients that you place importance on their lived experience?
Develop accessible information, support, and services in accessible and relatable language to make the patient feel seen, valued, and invested in the trial and its outcome. Listen to and take their concerns and feedback into consideration. Reduce patient inconvenience by considering the logistics of their participation, for example by minimizing long-distance travel to trial sites whenever possible. Empower your patients to input data themselves and take an active role in their trial participation, via wearable devices and technologies such as ePRO and eCOA.
Evaluating treatments beyond safety & efficacy
Treatment success, in the eyes of the patient, likely goes beyond its statistical or scientific efficacy. A treatment could be effective but require inconvenient dosage forms or schedules, unpleasant side effects, or could end up being extremely expensive when it hits the market. Under a patient-centric approach, these drawbacks would be taken into account in assessing the global success of the treatment. Adherence to the treatment plan can serve as an indirect indicator of participant satisfaction with the treatment.
Evaluation of treatments in a patient-centric manner thus necessitates communicating with the patient and understanding what makes a treatment “good” in their eyes and in the context of their unique personal situation. It may involve factors such as ease-of-use, convenience, adherence, cost to patient, ease of getting prescriptions filled, and the trade-off between benefits and side effects. In other words, the endpoints used for assessing the success of the new treatment may need to be defined or prioritized differently.
What does a patient-centric approach look like in clinical study design and conduct?
The design of a clinical trial is a basic factor in determining a patient’s experience, so in a patient-centric approach, forecasted patient experience should inform trial design. Here are some possible ways to incorporate patient factors in trial design:
Connect directly with patients to understand their condition and their needs
A good way to learn about how an illness or condition impacts the everyday lives of patients diagnosed with it is to speak with them directly. By talking to patients, researchers and sponsors can attain invaluable insights into how patients speak about their condition, where they go for support and information, and the types of treatments they may feel are lacking or wish were available. You may be able to arrange a patient focus group or reach out to specific groups on social media channels to reach patients directly. Consider their potential motivations for sharing their insights and experiences, and remember that the topic is likely to be sensitive.
If it isn’t feasible to reach out to patients directly, you can collaborate with a patient advocacy organization (PAO) or a nonprofit centered around the specific condition or patient rights. These organizations have direct connections with patients and may have plenty of data that could help you generate valuable insights into patients’ first-hand experiences. Working with these organizations will also enable you to start a dialogue around the areas where patient needs are not being fulfilled, and help you predict how patients might respond to your trial’s logistics.
Further, partnering with patient advocacy groups and establishing a presence could help you generate trust around the trial, setting you apart from other researchers and sponsors, and could even help you accrue more patients to your trials.
By understanding your patients’ symptoms and how their condition affects their quality of life, you can design protocols that maximize potential benefit and comfort for patients in consideration of their unique challenges and avoid trial designs that might prove to be particularly burdensome.
Incorporate components of a decentralized trial model
If the condition and drug you’re testing allow for it, consider using a decentralized trial (DCT) model – siteless, virtual, remote, or direct-to-patient, or a hybrid model incorporating some degree of decentralization. Decentralization, in this sense, refers to the move away from trials conducted from a single central site toward the use of local or mobile service providers, remote health endpoint and data collection, telehealth services, and drug distribution models that reduce the need for repeated travel to and from study sites and redistribute the workload for trial staff.
Decentralization of clinical trials gained momentum during the COVID-19 pandemic, as virtual models of connecting with patients became the go-to for clinicians and researchers to restrict direct contact and facilitate healthcare with stringent mobility restrictions in place.
Trials can be fully decentralized (i.e., fully remote, siteless, or direct-to-patient) or hybrid (with only the necessary minimum number of site visits) depending on the needs and possibilities of the specific trial (i.e., whether health endpoints or study procedures require specialized equipment or direct supervision). Such models offer many benefits, including:
- Reduced exposure to airborne and infectious diseases for trial participants who might be vulnerable to them
- Reduced travel burden (time and costs) for patients as certain study visits/check-ins can be performed remotely
- Lower recruitment barriers as virtual trials mean that patient’s geographical location is not an issue
- Higher-quality, accurate, real-time data input via wearable technologies
- Better patient engagement via the use of interactive technologies and increased participation in the study protocol
Utilize data from previous trials
Assessing data from previous trials regarding recruitment issues or delays, participant dropout and non-adherence/non-compliance, as well as patient feedback will help you understand issues stemming from protocol design and study logistics and avoid them in the future. Problematic aspects could include things such as unnecessarily or unintentionally restrictive eligibility criteria, non-representative study samples potentially caused by non-inclusive recruitment practices, burdensome study visits or treatment plans, or lack of participant engagement.
Be mindful of your patients’ health literacy
It was reported that only 12% of adults in the U.S. have proficient health literacy skills. This means that the majority of potential participants might have difficulty understanding medical terminology, even when relatively simplified. Communications, including clinical trial recruitment and outreach material, trial descriptions, and informed consent forms should be written in simple, easy-to-understand and direct language. This will facilitate comprehension and help patients feel engaged with the study. Beyond legibility, the terminology used in your communications should be appropriate, sensitive, and relatable in order to resonate with the target patient populations.
Lay out all information in a clear and transparent manner
It is important that potential participants understand exactly what the trial involves, who is eligible, and what the intention is. Not only will this empower participants to make an informed decision, it will save sponsors time wasted on screen failures due to interest by prospects who turned out to be ineligible or simply didn’t understand what the trial would have implicated for them.
Focusing on providing clear, concise, and transparent information will make it easier for a prospective patient to assess whether or not the trial fits what they’re looking for and will be manageable for their lifestyle. Details such as study visit timelines, site locations, trial duration, and potential out-of-pocket costs should be specified explicitly.
This does not apply only to recruitment and consenting, but also to reconsenting and any potential amendments to protocol during the trial. Any time patients are provided with written or verbal information, focus on using clear and digestible language. Ask questions to confirm they have understood clearly, and provide support channels for patients to clarify any doubts that may arise. Feeling confused about protocol and requirements is one reason for participant dropout.
Select appropriate trial sites and provide scheduling flexibility
Try to understand your patient demographic thoroughly and choose trial sites that best accommodate their location and schedules. Flexibility in scheduling will make lots of patients’ lives easier.
For instance, if your sample is concentrated in certain geographical regions, include average travel distance as a factor in selecting sites. If your study population mainly includes adults of working age, try to offer them the flexibility of weekend or evening study visits. Flexibility will also encourage participants to continue with the trial rather than dropping out due to inconvenience or frustration for not being able to make it to a study visit.
Work with multiple reliable lab partners
Work with local healthcare services such as laboratories or pharmacies for trial drug distribution, sample collection, or check-ups to further reduce travel time and costs and enhance accessibility. Collaborating with a direct-to-patient lab will enable you to ensure participants have convenient and safe access to the lab and diagnostic services.
Facilitate access to study sites
If your trial necessitates in-person study visits, assess the burden incurred for patients to travel to the site. Consider arranging transport for those who would need it the most (due to lack of transport or reduced mobility, for example), or offer reimbursement for all patients or those who would incur significant out-of-pocket expenses. Suggestions include partnering with a rideshare service to coordinate free site transfers or providing stipends to participants based on mileage, gas, or public transit costs.
Maintain two-way communication and support channels
To help participants remain engaged in the trial, offer support channels and encourage communication between patients and the study staff. Not only does this ensure that participants can receive clarification on any doubts (which could have otherwise led them to dropping out or becoming non-compliant), it helps them feel appreciated and also provides sponsors with extremely valuable feedback based on first-hand patient experiences.
Examples of patient-centricity in action
There are various companies, sponsors, and researchers already demonstrating practical applications of patient centricity in different aspects of clinical research. Here are some prime examples:
- Power is a platform that provides patients with direct and transparent access to clinical trials while simultaneously helping researchers and sponsors match and connect directly with these patients. The platform represents a patient-centric approach to the problem of trial accessibility and information transparency.
- AstraZeneca uses patient platforms and networks to capture patient information throughout their clinical studies, enabling its researchers to better understand their patients’ perspectives and fine-tune future studies. The company has more than 30,000 patients connected to its research projects.
In 2014 and 2015, AstraZeneca conducted two live clinical trial simulations for a systemic lupus erythematosus treatment to gather feedback about patient sentiment. The design enabled patients to provide prompt feedback and gave valuable feedback for improving patient recruitment, retention, and compliance for future trials. This clever example of gathering patient-centric insights into trial experience resulted in identifying the following principal patient desires: informative background material, knowledgeable staff, support from family and friends, personal results, shorter wait times, comfortable settings, and greater scheduling flexibility.
- A recent cutting-edge example of patient-centricity is Leal Health, an AI-powered platform that enables unbiased access to information about advanced and novel treatments for cancer patients. The platform helps patients find cancer clinical trials and treatments and educates them on all their possible options, while also collecting and using real-world data to assess patient diversity challenges and guide oncology research.
Why is patient centricity in clinical trials important?
The impact of patient centricity on clinical trial outcomes
Patient centricity enables patients to become part of the trial design, eliminating roadblocks to effective recruitment and retention. Moreover, when patients have all the information they need and the trial process is transparent, it encourages more people to enter the trial and stay with the program through to its completion.
Greater patient engagement and improved communication also result in enhanced data quality. Patients don’t feel like guinea pigs and develop a sense of ownership and involvement in the trial. They feel a sense of responsibility in seeing the trial through and inputting reliable and accurate data.
A bigger and higher-quality data pool results in considerably more reliable outcomes, enabling the trial to move forward and deliver life-changing treatment plans to market faster.
The impact of patient centricity on patients
The premise of patient centricity is that it enhances trial participants to be more engaged throughout the trial. A patient-centric approach also helps enhance patient safety by ensuring patients are made duly aware of the risks and benefits of participating in the trial. Their well-being is given center stage. Patients are also given the resources and education to report possible adverse effects quickly.
Moreover, patient-centricity reduces the disparities in healthcare faced by the less privileged by ensuring clinical trials are equitable, diverse, and inclusive. Patient-centric logic allows sponsors and recruiters to consider how different populations face different barriers to participation in clinical research. It instills the need to create trials representative of the global population rather than adopting a one-size-fits-all approach or defining overly restrictive eligibility criteria. This translates into improved accessibility for diverse groups, which then translates into study results that are more likely to be pertinent to the general population.
Patient centricity directly benefits trial participants by increasing access to trials that are less burdensome and more comfortable. It helps patients feel valued and encourages them to play an active role in their healthcare, with transparent information also acting as a way to educate participants about their conditions and health. In other words, patient centricity ultimately empowers patients.
The impact of patient centricity on researchers
Patient-centric trials prime researchers to better achieve the ultimate goal of their jobs - improving patient access to successful treatments for their conditions. It enhances researcher-patient communication, which builds trust and improves the chances of success. By aiming to simplify study design and operations for patients, it is simultaneously simplified for researchers as well. Fewer study visits means less scheduling, clear communication materials means fewer questions and explanations, and enhanced engagement, recruitment, and retention means fewer delays and less wasted resources.
Overcoming challenges in implementing a patient-centric approach to clinical trials
The transition from traditional trials to patient-centric trials involves new approaches, new considerations, and even new technologies and study designs, but the biggest hurdle is changing the existing culture.
Researchers and healthcare providers need to be educated on what patient centricity implies in terms of trial design and execution. It demands a shift from seeing patients as mere research subjects to active stakeholders to be considered throughout the entire trial. At the organizational level (pharma and biotech companies, CROs, etc.), seminars and professional development sessions can help all staff conceptualize the shift and understand why things are changing. Education is also important as it sets up individual staff to provide valuable feedback from their specific departments, under the context of the organizational objectives.
Another hurdle in the path of patient-centricity is the lack of content and resources designed to empower patients. Sponsors, CROs, and sites can assist by producing more (and more interactive) educational material for patients, before, during, and after their trials to encourage the paradigm shift. Even outside of any particular trial, these stakeholders can publish study outcomes, interim summaries (where possible), testimonies, or other developments to demonstrate transparency and garner interest in clinical research, or can create content such as blog posts or social media campaigns to simply share information and increase awareness of clinical research and the new directions it is taking to enhance patient experience. Negative perceptions about clinical research (many of them false) have been an important reason for people not being willing to participate in trials, even when they could potentially benefit; patient centricity is aiming to change that.
When investigators, sponsors and patients are educated about the new possibilities for patient-centric clinical trials, it is then important to connect these stakeholders. At Power, we have designed a patient-friendly platform to empower patients in finding treatments, which simultaneously provides significant benefit to trial sponsors by allowing them to connect with patients directly.
Patient centricity is about considering and incorporating first-hand patient experience into the clinical trial process, from trial design through all trial operations. It involves engaging with patients, understanding their pain points, and offering trials that check all the boxes. Patient-centric clinical trials aim to maximize patient satisfaction as the participants feel a sense of engagement with their own healthcare decisions as well as the trial’s research objectives. Ultimately, patient centricity seeks to address issues that have classically plagued the industry and cause significant delays and losses, in particular poor recruitment, retention, and engagement, thus supporting higher-quality data, more robust trial outcomes, and treatments that are not only effective and safe but also successful in the eyes of the patients themselves.