Developing Protocols For Patient-Centricity

Patient-centricity has become an increasingly important focus for clinical researchers in recent years. Creating a research experience that addresses patient-expressed needs and wants is an important part of recruiting and retaining sufficient numbers of qualified patients.

And it’s not enough to add a couple of features broadly considered to be what patients want, such as use of telehealth or home visits to reduce the number of site visits required for a study. The only sure way to ensure that sponsors can ensure their clinical trials are truly patient-centric is to design that focus into the protocol from the start, using a protocol optimization method such as Pro-ficiency’s Pro-Active Protocol solution.

In essence, patient-centricity marks a shift from the traditional disease-focused approach of conducting clinical trials to a philosophy that puts the well-being of patients enrolled in clinical studies first and treats them as “informed collaborators whose participation is crucial for the success of the research.”

Patient centricity has been at the forefront of the collective industry mind for the last several years. For instance, in 2016, the Drug Information Assn. (DIA) reported that a survey of pharma and biotech companies conducted in partnership with Tufts University showed that 65% of those firms had invested in patient-centric initiatives as part of their drug development programs.

By 2019, a SCOPE conference seemed to indicate widespread dedication to patient-centric trials, with representatives from various sponsors, CROs and other companies discussing how to incorporate more patient-centric aspects into research. 
The FDA has also highlighted the importance of patient-centricity, as well, developing a series of four patient-focused drug development guidance documents to address various ways of collecting and using patient experience data to support clinical research:  

  • Patient-Focused Drug Development: Collecting Comprehensive and Representative Input, a final guidance issued in June 2020;
  • Patient-Focused Drug Development: Methods to Identify What Is Important to Patients, a final guidance issued in February 2022;
  • Patient-Focused Drug Development: Selecting, Developing or Modifying Fit-for-Purpose Clinical Outcomes Assessments, a draft guidance issued in June 2022; and
  • Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision-Making, a yet-to-be-issued draft guidance.


Why is patient-centricity important?

One of the driving forces behind the move to more patient-centric studies is concern over patient enrollment and retention. About 80% of clinical trials struggle with enrollment, and some studies estimate that up to 30% of patients drop out before a clinical trial concludes, according to a March 2022 Antidote blog

“When product-centric trials neglect patient needs, dropout rates are high and the integrity of the data becomes compromised,” the MD Group agreed in a 2020 blog. Conversely, patient-centric approaches to clinical trial design can result in higher patient recruitment and retention rates.

And speaking at the 2019 SCOPE conference, Beth Zaharoff, senior director for patient-focused engagement and partnership at TESARO noted at the conference that enhanced convenience for the patient and focus on unmet needs expressed by the target patient population can help boost enrollment and ensure that patients remain in the trial for its entirety.  Additionally, patient centricity can improve trial speed and efficiency by helping to ensure the protocol is simple to administer for the patient, she said.
What constitutes a patient-centric approach to research? Schedule flexibility and use of decentralized features are factors that are often touted as means of making research participation more attractive to patients. Other options are also popular among sponsors, such as these steps recommended by the MD Group in its 2020 blog:

  • Make information accessible;
  • Involve patient associations;
  • Ensure that patients feel valued;
  • Reduce inconvenience wherever possible; and
  • Empower patients through technology.

But the answer doesn’t lie in randomly selecting features with the label “patient-centric” and attaching them to all clinical trials. Speaking at the SCOPE conference, Melanie Goodman, Pfizer director of patient recruitment programs, emphasized that patient-centricity must permeate every aspect of a clinical trial, from the protocol to the site, to be effective.

In other words, patient-centricity must be built into the protocol from inception. And that means that protocols must be optimized with an eye toward real-life patient needs and preferences. Pro-ficiency’s proprietary optimization methodology, Pro-Active Protocol, launched last month, can help achieve this goal by applying simulation to visually represent the operational flow of a clinical study. The service aims to shed light on protocol inconsistencies and gaps so sponsors and researchers can adjust and revise before a study begins.

A critical part of the service is gaining patient insight—along with opinions from study teams and regulatory agencies—about how easy or difficult it would be for target patients to participate in a study.

The only way to make sure that the protocol is written in a way that is feasible for the target patient population is to get those patients involved in the early stages of study design. This can be done via workshops that involve researchers, patient advocates and the patients themselves, for instance.

Logistical issues that don’t even register with sponsors during protocol design can have a large impact on a study’s ability to recruit and retain participants. For example, patients with long-term or serious conditions that cause fatigue may lack energy to travel at certain times. And patients whose condition causes dexterity problems may struggle to operate some devices or open certain types of drug containers.

The Pro-Active Protocol solution can “help to visualize and animate the patient journey through the study,” Beth Harper, Pro-ficiency Chief Learning Officer, explained. “This helps to set expectations for how involved the study participant will be and to help potential patients understand their commitment prior to participation; they will see how long the study will be and how involved from a procedure and time perspective.”

Having this information available makes it easier for sponsors to get feedback from patients on how likely they would be to participate in a clinical trial, including potential barriers to participation and opportunities to further simplify the study design and schedule of events.

Pro-Active Protocol can serve as an invaluable resource, whether used as a stand-alone resource or integrated with Pro-ficiency’s existing simulation-based protocol modules to boost site and study team understanding of the protocol requirements upon start-up of the clinical trial.


Site feasibility also supported

But ensuring that the protocol is truly designed with the target patient group in mind is just one of two critical areas that loom large for protocol optimization efforts. The second, and equally important, key area is ensuring that the protocol is clear and understandable for the research sites that will be implementing it.

“The most patient-centric thing sponsors can do is to make the protocol easy, or easier, to implement,” Harper said. “The easier it is to implement, the less change of errors (deviations), both on the part of the sites and the patients.”

Aidan Gannon, Advarra director of client services and innovation, agreed in a 2021 blog that there is a close relationship between patient-centricity and site-centricity, as well.

“One could argue patient centricity is not possible without site centricity, as sites deliver the patient experience in clinical trials,” he wrote. “The best intentions of any sponsor or contract research organization (CRO) make little difference if sites fail to engage with participants.”

Sponsors should listen to site experiences and suggestions for improvements, as these often have very practical implications to how well a clinical trial is conducted, he suggested. Sponsors should also offer process improvements and other support to help address any challenges that sites report. 

These types of improvements can help sites run clinical trials more smoothly, efficiently and accurately. When not struggling with study-specific challenges, sites can turn more time, attention and resources to ensuring a patient-centric experience.

For example, Advarra noted one large pharma sponsor conducted a Phase 3 breast cancer study involving a short timeline and small patient population in 16 countries. The company provided custom training, engagement options and guided visits, among other things, which reduced screen failures by 50% and protocol deviations by 21%.

The better sites understand the protocol, the more confidence they will have in explaining it to patients, Harper said. In this way, clarity for sites ties back into patient needs, by providing sites with the intimate knowledge about how a protocol will really work so that their staff can communicate that information clearly to patients.

With this growing focus on improved patient-centricity, it is critical that sponsors provide protocols that address real-world patient needs, constraints and priorities. And they must make sure that the research sites they select to carry out their studies both understand and are able to implement the protocols in the most patient-focused way possible.

But many sponsors lack the tools in-house to develop the necessary insight to implement these types of changes to their protocol development process. This is where resources like Pro-Active Protocol come in. By pairing industry expertise with effective simulation techniques, this product can help sponsors ensure deep understanding of patient needs that can be incorporated into their protocols and communicated with crystal clarity to sites. The end result: well-written protocols that account for real-world patient experiences and are easy to implement correctly.


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