Researchers Emphasize Importance of Patient Perspective in Dermatology Drug Development

As the entire health care system continues pivot toward a more patient-centered focus, researchers have published a new paper on how the patient voice is being incorporated into dermatology drug development. The findings were published in Dermatologic Clinics.

In recent years, patient-reported outcomes (PROs) have been incorporated as secondary end points in symptomatic dermatologic conditions, such as psoriasis. The researchers underscored the importance of the patient voice in the field, as some aspects of treatment can only be assessed through the patient.

“In dermatology, capturing the patient voice isparticularly relevant because the patient’s perspective is critical in evaluating treatment outcomes in symptomatic dermatologic conditions, as well as aesthetic procedures,” explained the researchers. “One way to capture the patient voice in drug development is by gathering patient and other stakeholder input on what symptoms and impacts matter most to patients with respect to their disease or condition and can also change with treatment and, subsequently, using appropriate clinical outcome assessments to capture the outcomes of interest.”

Dermatologic indications that incorporate PRO data include atopic dermatitis, which includes patient-reported itching intensity, measured using a numeric rating scale. Within plaque psoriasis, patient-reported symptoms and itching severity are captured with signs and symptoms measures and a numeric rating scale, respectively.

Other examples include interdigital tinea pedis, tinea cruris, and tinea corporis, which include patient-reported pruritus symptoms measured by a pruritis assessment. Submental fat includes patient-reported submental convexity, measured with a submental fat rating scale and the visual and emotional impact of the condition is measured by patients with a 6-item survey.

As PROs continue to be incorporated in dermatology drug development, the researchers highlighted several challenges within certain indications, including those for pediatric patients and/or rare conditions. Challenges outlined by the researchers include:

• Fit-for-purpose instruments for rare diseases and pediatric populations do not exist for many conditions
• Rare diseases may have heterogeneous presentation, symptoms, and impacts
• Instruments may not be sensitive for tracking children as they develop
• Measuring vocabulary, numeracy, and comprehension may be difficult among younger patients
• Self-reporting may be limited in younger patients

The researchers outlined several ways to potentially overcome these barriers, including leveraging digital health technology through the use of sensors to measure sleep disruption, scratching, and other behaviors.

“As stakeholders work to overcome these challenges, we encourage them to plan for early interactions with FDA to obtain feedback about their COA [clinical outcome assessment] measurement strategy and collaborationwithin the precompetitive setting (eg, through consortia),” wrote the researchers. “Collaboration with FDA outside drug development, such as through the COA Qualification Program, may be another option. The COA Qualification Program manages the qualification process for COAs intended to address unmet public health needs.”

Outside of capturing patient perspectives on symptoms, the researchers underscored the potential around measuring quality of life effects, including physical and emotional functioning. For example, conditions that involve wounds or blisters, such as epidermolysis bullosa, and conditions that lead to emotional or psychological effects, including those that cause social embarrassment or anxiety.

Reference

Daniels S, Marcus K, Bent R, Papadopoulos E. Measuring what matters to patients in dermatology drug development: a regulatory perspective. Dermatol Clin. Published online June 1, 2022. doi:10.1016/j.det.2022.03.002