Achieving Patient Centricity in Clinical Trials: Uncovering the Mystery

Clinical Researcher—November 2018 (Volume 32, Issue 9)

RECRUITMENT & RETENTION

Rosamund Round, MSc; Kate Williams, MSc, PhD

 

Patient centricity has been deemed something of a buzz phrase within the pharmaceutical industry in recent years. Some see it as the key to commercial success, others as being at the heart of an ethical approach to business, and some discount it altogether.

Whatever your personal views, a recent report produced by The Economist Intelligence Unit and commissioned by PAREXEL{1} identified patient centricity as a key innovation within the healthcare industry. It showed that patient-centric trials took almost half the time to recruit participants, recruited double the number of patients, and the drug was 19% more likely to be launched.

Just as interestingly, only 5.2% of Phase II and III trials actually utilized this patient-centric approach. So, why the gap? Could it be the fact that the industry lacks a universal definition of patient centricity, making it challenging for people to pin down exactly what they should be focusing on? Or, is being patient-centric seen more as an ethos than a clear approach?

When exploring how various pharmaceutical companies define patient centricity, the common themes of putting patients’ needs at the center or heart of clinical research—and of wanting to enhance the patient experience—become evident. That seems like a great place to start, but how?

Our advice to achieving patient centricity in clinical trials is as follows:

Start with the Study Design

A truly patient-centric study should be designed with the patient at the forefront. A crucial step in this process is ensuring that the study is measuring outcomes that are important to patients.

Traditionally, measure selection was based solely on clinical opinion or the study goals, but in recent years there has been an increasing drive to involve patients in the process, underpinned by the publication of the U.S. Food and Drug Administration’s (FDA’s) Guidance for Industry on Patient-Reported Outcome Measures.{2} After all, it doesn’t matter how good a treatment is, if the study doesn’t capture outcomes that are important to patients then it won’t be able to demonstrate meaningful benefit to patients.

So how do we ensure that a trial is “measuring what matters”? Before even considering which measure to include in a study, we recommend starting with the concepts of interest (i.e., the symptoms or issues that are most important to patients, and that they would most like to see improved by a new treatment). There’s no better way to identify these concepts than to ask the patients themselves, through qualitative research, such as interviews or focus groups. These methods are employed by researchers in order to understand and learn about what’s important to patients in their own words.

Once the important concepts are identified, it’s then time to look for a measure(s) which assess these concepts. The FDA is particularly interested in direct measures of how a patient feels, functions, or survives.

Measures of how a patient feels or functions are often assessed using clinical outcome assessments (COAs), such as patient-reported outcomes (PROs). PROs are questionnaires for assessing concepts which are only known to patients, including symptoms, quality of life, treatment satisfaction, or the impact on their condition of their activities of daily living.

Although there are a wide range of PROs available, it is important to select one that has been developed and validated with input from patients. This is known as establishing “content validity,” and ensures that the measure is assessing an important concept and is therefore patient-centric.

Sometimes there is no existing COA available for measuring the concepts of interest to the study population, so a new COA may need to be developed. In this case, the same principles apply—start with the concepts of interest and obtain feedback from patients at each step of the way.

In addition to COA selection, obtaining patient input into other elements of the protocol design can help eliminate impractical elements from being included, which could otherwise deter a patient from enrolling in the study and staying for the duration. For example, visit length can often be a challenge. In such cases, exploratory endpoints sometimes add hours and unnecessarily increase the burden for patients and their family, and could be easily removed. Conversely, some time-consuming protocol elements may be required to maintain the scientific integrity of the study, in which case other services can be incorporated, such as providing transportation support and meal vouchers to reduce the patient burden.

In addition to patient feedback, running a mock trial visit can be beneficial to uncover potential issues prior to protocol finalization. Working through a day in the life of a study with patients, site staff, and the study team can help identify in real time where the experience could be enhanced from the patient perspective. Further, by incorporating direct feedback, the sponsor is less likely to require a costly protocol amendment later, whilst all participants are ensured a more positive study experience.

Enhance Patient Communication

It is our job to make it as easy as possible for the patient to learn about and participate in clinical trials. To achieve the former, it is important to remember the breadth of language, reading abilities, and health literacy across the patient population and countries in which a trial is to run.

Creating simple literacy and health literacy guidelines to help shape such communication is an important step, so that all stakeholders are working toward the same goal. Further, with illiteracy still experienced by many, providing such key information as the main details in the informed consent form in a visual format will help potential participants to make a truly informed decision about study participation.

Communicating with patients in a way that is familiar to them is also important, making patient apps and text messaging increasingly popular for many patient populations. Getting a reminder on your phone about an upcoming visit, or to provide encouragement for your efforts, can be an important part of study compliance and should not be underestimated.

Consider Deploying a Virtual Trial

Taking the trial to the patient can be viewed as the epitome of patient centricity, given that, if fully virtual, it eliminates the need for attendance at a physical study site. Such an approach not only positively impacts the practicality of a study by enabling patients to fit it around their daily lives more easily, it also reduces the geographical and financial barriers.

With only 70% of patients living within two hours of their nearest study site,{2} many patients are unable to access clinical trial opportunities under the traditional model, making virtual trials an attractive proposition. By not having to travel, pay for hospital parking, or miss significant days at work, financial barriers to participation are also minimized.

The fully virtual model does not, however, work for all study designs, particularly where MRI scans, lumbar punctures, or other complex and challenging tests and procedures are critical to study conduct. In such cases, a hybrid model can be deployed, which aims to minimize visit burden and take as much of the study as possible to the patient, with a few site visits remaining.

Patient transportation services can be utilized to ease the financial burden, wearables or sensors can be deployed to collect information at home rather than in the clinic, and telehealth visits can be arranged to enable patients to incorporate visits with their physicians virtually from their home, in addition to their onsite visits. There are many different variations on the model, dependant entirely on the specific needs of the patient population and the study design. However, making the patient the focus of study planning and conduct not only minimizes the trial burden, it can also enhance recruitment and retention to the benefit of all parties.

Ensuring Trial Ends as a Positive Experience

Patients can often feel frustrated and upset by a lack of acknowledgement of their efforts when a trial ends. Creating a closure plan for patients to address this can make all the difference. This can involve sending a thank you card and providing study results in layman’s terms so that they understand the outcome of the trial.

It is also important to look for ways to address continuity and transition of treatment beyond the trial during the trial planning phase. In doing so, we give patients the respect they deserve for the time and effort they have put into trial participation.

Conclusion

Patient centricity is an evolving space, and new ways of putting patients at the heart of the study continue to emerge as the pharmaceutical industry strives to simplify the patient journey. There’s more work to be done, but an ever-increasing focus on patient-centric matters from the FDA and European Medicines Agency is helping to accelerate change. So, where some still see patient centricity as a buzz phrase, there is without a doubt true change happening.

References

  1. The Economist Intelligence Unit. 2018. The innovation imperative: the future of drug development. https://druginnovation.eiu.com/
  2. Adams B. 2017. Sanofi launches new virtual trials offering with Science 37. fiercebiotech.com/cro/sanofi-launches-new-virtual-trials-offering-science-37

Rosamund Round leads PAREXEL’s Patient Innovation Center and is based in the United Kingdom.

Kate Williams is a Senior Research Manager with Acaster Lloyd Consulting Limited in London and former Senior Consultant for Clinical Outcomes Assessment at PAREXEL.