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Patient input into study design – participation considerations. The last, in a series about the science of patient input.

Previously, we have discussed how patient preference can be taken into account as either an input to designing a clinical study, or as an endpoint measuring the effectiveness of a device in terms of patients’ satisfaction with the result. Since medical device companies develop products with the intent to fulfill unmet needs or to improve outcomes delivered by existing products, it seems reasonable that patient opinion may have a role to play in determining what kinds of devices get worked on and what kind of results would be acceptable. The healthcare industry is built on serving patients, but the details about the specific health conditions, prognoses, treatment availability, cost, and patients’ social cultures all factor in to their preferences.

The majority of clinical trials run over time and budget, often due to challenges with recruitment and retention of patients. When designing medical device trials to optimize patient participation, what better guide to use than patients themselves? Patient input can be used to design trials that are more attractive to patients by minimizing barriers to enrollment and reducing the burdens of participation. Those two key areas can lead to trials being completed faster, with better patient compliance and better quality data. These outcomes lead in turn to significant cost savings and potentially reduced time to market. In a recent survey of patients performed by the Medical Device Innovation Consortium (MDIC), patients who had been invited but chose not to participate in a clinical trial listed their main concerns as being the risks of the study procedure or experimental product, the number of required visits, and the chance of receiving a sham/placebo instead of treatment. Similarly, those who were considering participation in a clinical trial responded that a need to travel farther to visit the trial site, an experimental treatment that contained extra risks, and a chance of receiving a sham/placebo would make them less likely to participate in the trial.

On the other hand, patients reported being strongly motivated to participate in clinical trials that offered access to new treatments and technologies, the chance for a benefit to their personal health, the chance to contribute to the body of knowledge for their medical condition, periodic updates on the progress of their clinical trial, or a summary of the trial results once it is finished. The complete results of the MDIC patient survey can be found on its website: https://mdic.org/program/science-of-patient-input/

Most of these considerations are self-evident and are addressed in study design, where possible, to increase likelihood of patients choosing to participate. For example, a literature-derived performance goal might be appropriate to take the place of a placebo arm, so patients would not be concerned about being randomized to the placebo group. A similar approach is to use unbalanced sample size, with the treatment group being larger with a commensurate reduction in the likelihood of patients not receiving the experimental treatment. Other considerations are not so obvious, but can still be incorporated into trial planning fairly easily. For example, periodic trial progress updates written for laypersons could be shared with the trial sites to distribute to participants. Such an update could also emphasize the importance of patients completing their treatment/follow-up/data collection obligations. However, the content of these updates would need to be well-considered, to avoid introducing bias (e.g., preliminary trial results typically should not be shared with participants).

By taking the time to learn about patients’ concerns and motivations up front, clinical trials can be tailored to mitigate the challenges that would deter patients from enrolling in the trial and to emphasize the motivating factors. Patients will more likely choose to participate in such a trial and be less likely to drop out before completing the trial requirements. This can lead to trials that finish more quickly, with more complete data, and/or with fewer total patients enrolled (to meet the required sample size) which can ultimately save time and resources.

If you have any questions about how to design more efficient, cost-effective clinical trials, please call us at 855.463.1633 or visit our website medinstitute.com.