By Jasmina Jankicevic, M.D., M.S., CCRP, Vice President, Medical Affairs, Dermatology and Medical Aesthetics, Premier Research
Skin diseases are the fourth leading cause of health afflictions, affecting nearly 900 million people worldwide. It’s not surprising, then, that there has recently been a noticeable increase in dermatology drug development. There are currently thousands of dermatology clinical trials planned or already launched, including a variety of programs investigating innovative, targeted therapies.
Much of the development progress in dermatology, however, has been made in a small subset of diseases, such as melanoma, psoriasis, and atopic dermatitis. There is still a significant unmet need for safe and effective treatments for the vast majority of the more than 3,000 known skin disorders.
For investigative dermatology therapies, the likelihood of regulatory approval and successful market adoption increases when clinical trials are created with — and for — the patient. Such studies are designed to incorporate all relevant criteria for participation, including a well-defined patient population and key endpoints, and optimized to decrease the burden of participation on the patient in the investigation and to increase access to a diverse population of qualified participants.
Designing a patient-focused trial
In recent years, the U.S. Food and Drug Administration and European Medicines Agency, among others, have placed increasing emphasis on collecting and submitting patient experience data and other pertinent information from caregivers as part of the product development and regulatory decision-making process. For instance, the FDA is developing a series of four guidance documents to address how stakeholders collect and analyze these data and incorporate them into clinical trial endpoints.
Reducing the burden of study participation is critical in patient-focused study design. Through inviting patients and their caregivers to review draft protocols through patient advisory boards, focus groups, and targeted surveys, sponsors can gather feedback on the acceptability of a trial and the reasonableness of proposed procedures, assessments, and endpoints. Patient input is particularly valuable in rare dermatology conditions where knowledge of the natural history of the disease may be lacking and standard endpoints have not been developed.
Reducing start-up and enrollment timelines
Having an established network of qualified investigators and sites with experience in the indication of interest can help minimize additional paperwork, streamline study start-up, and lead to higher quality of the data.
Given the large number of dermatology clinical trials in progress, the intense competition for eligible participants is a threat to enrollment timelines. Patient-focused study designs are an opportunity for sponsors to differentiate their trials. For example, optimizing the number and length of site visits and the type and number of invasive procedures will boost enrollment. Moreover, increasing the use of technology through mobile health (mHealth) technologies including remote electronic consent, televisits, wearables, and apps not only cuts the burden on both patients and sites, but also makes studies more attractive for potential participants.
Involving patients and their families in the process of raising trial awareness and developing patient-facing marketing or study-related documents helps ensure that all messaging is on point and easy to understand. Establishing partnerships with patient organizations and patient advocacy groups further amplifies awareness, enhancing study visibility, recruitment, and enrollment.
Ensuring data quality
Securing patient safety and high data quality starts with rigorous protocol training and testing for investigators and site personnel. Many of the endpoints used in dermatology clinical trials rely on globally recognized visual evaluations of the skin to produce an accurate, reliable, and reproducible approach to assessments, which is crucial for study success. Developing and implementing robust, assessment tool-specific rater training programs and ongoing communication helps ensure both accuracy and consistency.
The training of investigators and their teams on study protocols and procedures minimizes variance in processes and assessment within and across sites. If placebo is part of study design, considerations regarding placebo effect are important for both clinician and patient-reported outcomes.
Dermatology clinical research is evolving rapidly in response to increased development activity, pandemic-related adaptations, and a regulatory framework that favors clinical trials that incorporate the patient perspective and minimize the burden of participation. When a study is both meaningful and manageable to patients and their families — not to mention investigators and site teams — it is more likely to succeed. Partnering with an experienced CRO that understands how to optimize patient-focused clinical development can help sponsors differentiate their clinical trials, increasing study visibility while reducing start-up and enrollment timelines and increasing efficiency.