Expert Insights

Expert Insights

8 Things to Know About Rare Dermatologic Trials for Children

8 Things to Know About Rare Dermatologic Trials for Children
14 May 2021

The EU has identified more than 1,000 rare skin diseases — many of them genetic in origin and manifesting in childhood.

When addressing pediatric rare diseases, prompt treatment is paramount. In some cases, if skin cells die in childhood, patients may not ever recover lost function. However, conducting pediatric clinical trials for dermatologic conditions isn’t a quick-and-easy process. Consider the following as you plan your next pediatric dermatology study:

  1. Carefully consider clinically meaningful outcomes: With little to no robust longitudinal disease models, sponsors may have a difficult time gathering the information needed to understand natural history. In addition, the subjective nature of dermatology creates variability in interpretation. Make sure your investigators are thoroughly and consistently trained.
  2. Standardizing assessment: Standardizing global assessments for dermatological indications is complex but important. When conditions are rare, there is even less room for error.
  3. Standardize dosing: Variability in dosing impacts drug supply. For topical treatments, factor in body surface area when determining ways to standardize dosing.
  4. Ensure rater consistency when measuring outcomes: To ensure consistency in assessments, establish ways to account for lesion size reduction and other factors that are as precise as possible. Consistency across participating investigators is key to producing the most accurate data.
  5. Quality of life is key: Quality of life is hard to measure, but it’s an important component of treatment, especially for children and their families. Make quality of life measurements an important element of your clinical trial.
  6. Consider technology and innovation: With the rise of gamification for questionnaires, rewards for survey participation, and the addition of wearables, technology can add to the patient centricity of pediatric clinical trials. When developing wearables, consider the skin condition you’re researching to make sure it doesn’t hurt the skin.
  7. Perform genetic testing: Numerous investigational treatments show optimal benefit when initiated pre-symptomatically. Genetic testing helps improve trial participation and generates outcomes that impact quality of life throughout childhood and beyond.
  8. Take a multidisciplinary approach: Develop an approach including a care team that can address diverse clinical presentations or side effects. Your team of experts may include an ophthalmologist, respiratory specialist, general practitioner, and/or an immunologist, depending on the condition or disease.

To explore these considerations in depth, download our guide: Eight Must-Know Considerations About Rare, Dermatologic Trials for Children.

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