Calling Out ‘Check-the-Box’ Patient Engagement

Ken Getz, MBA, Executive Director and Research Professor, Tufts Center for the Study of Drug Development, Tufts University School of Medicine

Listening to conversations and attending presentations among clinical research professionals, one would be hard pressed to question the drug development community’s commitment to supporting the patient engagement movement. But beneath the surface there are many signs that we are not delivering on that commitment.

A 2023 Tufts Center for the Study of Drug Development (Tufts CSDD) survey among several hundred sponsor companies found that only 27% are routinely soliciting patient input into protocol design decisions.

Despite high and rising study participation burden in our clinical trials, less than 10% of study volunteers reports receiving convenience-enhancing support (e.g., transportation assistance and the use of virtual and remote solutions), according to a recent study of 4,558 participants just completed by the Center for Information and Study on Clinical Research Participation (CISCRP). In that same study, CISCRP found that only 35% of clinical trial volunteers reports receiving a formal verbal or written “Thank You” for their participation and less than 30% reports receiving any information about the results of their clinical trial.

Without exception, stakeholders throughout the research enterprise are well-intentioned in their desire to treat patients as partners in clinical research. But many factors are contributing to a high level of reluctance and—equally, if not, more concerning—to poorly executed initiatives.

These factors include internal resistance to modifying legacy practices and processes; insufficient funding and staffing to adequately support initiatives; lack of clarity from regulatory and health authorities; uncertainty about the expected impact and return on investment; and too little time devoted to planning and implementation.

This article is part of a series that is intended to call out the many ways that patient engagement is falling short.

It is my hope that insights from these articles will inform and help strengthen our collective commitment to this essential movement.

Few ... and hard-to-find plain language trial results

A major tenet of patient engagement is providing accessible, reliable, complete, and accurate information on clinical trial results. Doing so ensures that patients and their families are informed about their care options, conveys appreciation for the gift of volunteer participation, and instills trust in the patient-professional partnership.

A little more than a decade ago, sponsor companies began providing clinical trial results information to their patients. That information was only available in scientific and technical language. At the present time, an estimated one-in-five (20%) sponsor companies are providing plain language trial results summaries. That proportion is gradually rising largely in response to the EU Clinical Trial Regulation 536/2014, which went into effect in January 2022. The regulation requires sponsors to publish a plain language results summary on the clinical trial information system (CTIS) of any clinical research study conducted in an EU member state within a specified timeframe. Although the FDA has yet to issue a similar regulation, most drug development sponsors are complying with the EU regulation as the majority of Phase II and III clinical trials are conducted globally and include EU member state locations.

Once the clinical trial results summaries have been prepared, many sponsors publish them on government and private websites. Sadly—and embarrassingly—these listings cannot be easily accessed. A 2020 study found, for example, that clinical trial results listed on the EUDRACT system could not be found using a commercially available search engine (e.g., Google) unless a specific unique sponsor-company identifier or a specific EUDRACT number was included in the search term.

Limited usefulness

A recent CISCRP study has uncovered an even more alarming issue: the published listings omit even rudimentary information that patients and their families need to understand around an investigational treatment and its risks and benefits.

A brief background about this study: in late 2022 and into 2023, CISCRP randomly selected 50 clinical trial results listings published on ClincialTrials.gov. Next, the CISCRP team manually evaluated each of these listings using a framework introduced in an earlier (2021) study to ensure that the results summaries were presented in plain language (i.e., at an 11-13 year-old reading level, with defined technical terms, and in a format and design that was easy to read) and that they communicated basic, useful, and meaningful information for patients, their families, and care providers. Basic information includes:

• Study title
• The rationale for and purpose of the trial
• Eligibility criteria
• Description of the study design and participant subgroups
• Results of the clinical trial (i.e., primary and secondary endpoints, adverse events, and adverse reactions to the study drug)
• Information on where to go to learn more about the clinical trial

The CISCRP team found that all 50 clinical trial results summaries provided basic background information, though rarely in plain language. Study title, location, point of contact, and medical condition were presented in all the trial results summaries evaluated. Less than two-thirds (60%) of the results provided an explanation of how the study drug might treat the medical condition and in all cases the explanation was only provided in technical language.

Very few summaries provided complete information about the study design and only a small percentage provided any information about the results of the study. Virtually none of this information was in plain language. All of the clinical trials that CISCRP evaluated provided a partial description of the study design, but only 16% discussed whether the primary endpoint had been met and only 13% discussed whether key secondary endpoints had been demonstrated. In no instance were these study outcomes presented in plain language. And although 60% of the published clinical trial results provided information on adverse and serious adverse events observed, in no instance was this information provided in plain language. None of the trial results provided information about adverse reactions to the study drug.

More harm than good

The low level of adoption and the poor execution of plain language trial results summaries reflect badly on the entire clinical research professional community. Ultimately, failure to deliver on our commitment compromises the study volunteer experience, harms efforts to engage patients as partners in the research process, and elevates public and patient frustration and mistrust.

We can and must do more to ensure the return of accessible and useful clinical trial results summaries to all patients who participate in our studies and to those who seek promising new treatment options.

Public and patient demand for greater transparency and disclosure is high and rising, as is their hunger for useful information about their medical care. It is critical that research sponsors take steps to address shortcomings in their published clinical trial results summaries. Providing rudimentary essential information in plain simple language must be the starting point, including discussion about whether primary and key secondary outcomes were met and whether adverse reactions to the investigational drug were observed.

Although the majority of sponsor companies have yet to even begin returning clinical trial results summaries to their patients, a few sponsors are already considering automating the preparation of clinical trial results summaries, including the use of artificial intelligence/machine learning to shorten timelines and reduce costs. Given the poor and incomplete current state of clinical trial results summary content, we must be thoughtful and avoid moving hastily in this direction.

There will be ample opportunity in the future to optimize the process for returning plain language clinical trial result summaries to patients; most sponsors need to first begin the process and to commit to doing it well.

Ken Getz, MBA, Executive Director and Research Professor, Tufts Center for the Study of Drug Development, Tufts University School of Medicine