Expert Insights

Expert Insights

Part I: Key Challenges in Dermatology Clinical Trials – Assessing Qualitative Efficacy Endpoints

Part I: Key Challenges in Dermatology Clinical Trials – Assessing Qualitative Efficacy Endpoints
14 March 2019

Measuring clinical trial efficacy end point data is critical to establish statistically significant outcomes in dermatology clinical trials. However, it’s not always easy. 

Often, dermatologic conditions, such as acne, atopic dermatitis, and psoriasis, can present variable and subjective symptoms, making consistent endpoint assessments all the more difficult to achieve. With the qualitative nature of many dermatological indications, endpoint data frequently comes from visual descriptions of observations from an investigator’s trained eye versus mostly quantitative results, as in other therapeutic indications (e.g., A1C in diabetes).

Early and frequent training of investigators is key. Clinicians must thoroughly understand how to consistently assess clinical trial end points. The investigator meeting is one opportunity to build knowledge and consensus on these assessments. Face-to-face expert training can be made available here and throughout the study, as well as via videos, PowerPoint presentations, and other tools to ensure rater consistency.

Whether Investigator’s Global Assessment (IGA) scale, Psoriasis and Severity Index (PASI), or others are being used, investigators should be thoroughly versed in the appropriate rating scales for the respective indication. Additionally, advances in technology, photographic imaging, and mobile assessments can make it easier to track endpoints.

In some instances, patient reported outcomes such as pruritus (itch) may be the primary endpoint assessment for the investigational drug (treating the itch and not necessarily the underlying disease). Thus, the scales for patients to report severity of itch in this case must be validated and can be reproduced.

In addition to these measures of efficacy, assessing patient reported outcomes for quality of life (QOL) on social, functional, and emotional dimensions is becoming more heavily weighted in dermatology clinical trials, with the FDA expressing the desire for sponsors to include patient reported outcomes in study designs. Even in diseases like acne, which are chronic yet not life threatening, there can be huge quality of life implications.

As it’s also subjective, assessing QOL responses can be challenging to interpret. It can be difficult to collect consistent, measurable data. For instance, this study on poor reporting of quality of life outcomes in dermatology clinical trials suggests that from the 67 studies obtained, 95% did not calculate the number of patients necessary to obtain statistical power; another 95% did not disclose before/after results for scores and dimensions; 87% did not fully explain questionnaires; 75% did not address absent data.

Ensuring rater consistency also becomes all the more important at the Phase III level, when the drug needs to be showing distinctive efficacy results in narrow ranges to advance to pivotal NDA studies.

While assessing qualitative efficacy endpoints can be a challenge in dermatological clinical trials, putting a plan in place that leverages the right strategies can help to ensure rater consistency.

With extensive experience across psoriasis, atopic dermatitis, acne, alopecia, skin cancer, and numerous other indications, as well as skincare products, Synteract can bring your dermatology clinical trial to life. Contact us to learn how!

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