Medical aesthetics is one of the most rapidly growing therapeutic fields as research continues to demonstrate its numerous benefits to personal health, including:

• Improved Self-Esteem: Medical aesthetic therapies can help improve skin appearance and boost self-esteem, leading to increased confidence and a higher quality of life.

• Improved Physical Health: Some medical aesthetic treatments, such as laser therapy and chemical peels, can improve the health of the skin by promoting cell renewal and reducing inflammation.

• Reduced Stress and Anxiety: Many skin issues, such as acne, wrinkles, and sun damage, can cause stress and anxiety. Medical aesthetic products can reduce the appearance of these skin concerns leading to a greater sense of well-being.

In this blog post, we will discuss the advantages and challenges of integrating electronic PROs in medical aesthetics clinical trials.

What are patient-reported outcomes (PROs)?

As many of the primary efficacy outcomes of medical aesthetic therapies are subjective (e.g., self-esteem improvement, stress and anxiety reduction, perception of appearance, severity of deficit, etc.), patient-reported outcomes (PRO) (or participant-reported outcomes in the context of clinical research), which refers to the self-reported data collected from research participants about their condition, treatment, and experience, play an important part in clinical trials.

From validated quality of life and self-esteem questionnaires to diaries, PROs are used to support the regulatory approval of investigational aesthetic products. In recent years, electronic PROs have emerged as new and innovative methods for collecting these types of data, replacing the paper diaries that research participants had to complete are return to the clinic at their scheduled visit.

What are the most common PROs used in medical aesthetics clinical trials?

Some of the most common PROs used in medical aesthetics clinical trials include:

• Wrinkle Severity Rating Scale (WSRS): The WSRS is a commonly used PRO that assesses the severity of wrinkles in the face, neck, and hands.

• FACE-Q: The FACE-Q is a multi-modular PRO instrument that includes more than 40 independently functioning scales and checklists. The FACE-Q scales measure outcomes that matter to participants, including facial appearance, quality of life, and adverse effects.

• Patient and Observer Scar Assessment Scale (POSAS): The POSAS is a PRO that measures the appearance, symptoms, and impact of scars on patients’ quality of life.

• Dermatology Life Quality Index (DLQI): The DLQI is a PRO that measures the impact of skin diseases on a patient’s quality of life.

• Participant Satisfaction Questionnaire (PSQ): The PSQ is a PRO that measures patient satisfaction with cosmetic procedures, such as dermal fillers and laser treatments.

• Visual Analog Scale (VAS): The VAS is a PRO that measures pain, itching, and other symptoms on a scale of 0 to 10.

• Facial Appearance Satisfaction Scale (FASS): The FASS is a PRO that measures patient satisfaction with their facial appearance after cosmetic procedures.

• Participant Global Assessment (PGA): The PGA is a PRO that assesses the overall improvement or change in a patient’s condition.

These measures help clinical investigators evaluate the effectiveness and research participants satisfaction with the investigative aesthetic product, whether it is a cosmetic/cosmeceutical, drug, or medical device.

What are the advantages of ePROs compared to paper-based PROs?

Electronic Participant-Reported Outcome Measures (ePROs) offer several advantages compared to traditional paper-based PROs, such as:

• Increased accuracy and reliability: ePROs can use algorithms to ensure the accuracy and completeness of data, reducing the risk of human error and missing data.

• Real-time data collection: ePROs can be administered at any time and from any location. This is particularly important for participants who may have mobility issues or live in remote areas. ePROs allow for real-time data collection, which is more accurate and reliable compared to traditional paper-based methods where there is a risk of introducing recall bias.

• Improved participant engagement: ePROs can be designed to be user-friendly and interactive, potentially increasing research participant engagement and motivation to participate. By using digital tools, research participants are more likely to complete the assessments and provide valuable feedback, which can help improve the overall quality of the trial.

• Reduced administrative burden: ePROs can automate data entry and analysis, reducing the administrative burden on research staff and improving efficiency.

• Increased data security: ePROs can use secure electronic systems to protect research participant privacy and confidentiality, reducing the risk of lost data, data breaches, and other unauthorized access to sensitive information.

• Cost savings: ePROs can save time and reduce costs associated with data entry, printing, and shipping of paper-based PROs.

Compared to traditional paper-based PROs, ePROs offer several advantages including increased accuracy, real-time data collection, improved research participant engagement, reduced administrative burden, increased data security, and cost savings.

What are the challenges of integrating ePROs into medical aesthetics clinical trials?

It is important to consider that ePROs also present new challenges that must be addressed to ensure the successful integration of ePROs into medical aesthetics clinical trials (e.g., technical support, internet access, access to appropriate devices such as smartphones or tablets).

Technical Challenges

Electronic PRO relies on technology that can be prone to technical difficulties such as software malfunctions and network disruptions. Technical issues, such as software compatibility and lost data, can impact the accuracy and completeness of clinical trial data.

Therefore, it is important to not only use technologies with solid infrastructures, proven track records and responsive trouble-shooting processes, but also to properly validate and test the technologies prior to their deployment.

Data Quality

While ePROs can increase the accuracy and reliability of clinical trial data compared to traditional paper-based PROs, the quality of ePROs data is dependent on several factors, including the design of the questionnaire, the research participant’s ability to use the technology, and the level of training provided to both the investigational teams and research participants.

Site-specific training on ePROs is an essential component of the investigational site initiation process as well as on-demand support from clinical project managers.

Research participant enrolment difficulties

As with any PRO, research participants may feel that the burden of reporting symptoms/outcomes through ePROs is too high. Participants may feel that the time commitment required to complete the ePROs is too much, especially if they have other responsibilities or obligations; a problem that is common to any PROs used in clinical trials. Furthermore, not all research participants may have access to digital devices compatible with the proposed ePRO technologies or be comfortable using them.

Indeed, despite the benefits of electronic PROs, some research participants may be hesitant to use the technology or may not fully understand how to complete the questionnaire. Research participants may also be skeptical about the reliability of ePROs and may question the accuracy of the information they are reporting. This can result in a limited research participant population, which can either impact the generalizability of the trial’s results or delay research participant enrolment timelines.

As overcoming research participant resistance and promoting the benefits of electronic PROs can be challenging, it is essential that the CRO provides investigational sites with the tools and support necessary to face these potential objections.

Data Analysis

The large amounts of data generated by ePROs can pose a challenge for trial teams in terms of data analysis and interpretation. Ensuring that the data are analyzed and interpreted accurately by the CRO’s data management team is critical.

Privacy and Security Concerns

While they provide added security compares to paper-based PROs, electronic PROs do involve collecting sensitive personal and medical information which raises concerns about privacy and security.

It is essential to ensure that the digital tools used in a trial comply with data protection regulations such as HIPAA, PIPEDA, and GDPR, as well as applicable state and provincial privacy legislation. It is also fundamental that research participant data are protected from unauthorized access. Robust cybersecurity measures are necessary to protect the data.

Regulatory Compliance

Beyond privacy legislations, ePROs are also subject to various regulations and guidelines and it can be challenging to ensure compliance. For example, the FDA has elaborated specific requirements for the acceptance of ePROs in clinical trials.

What are FDA requirements for including ePROs in a clinical trial?

The US Food and Drug Administration (FDA) has established guidelines[1] for the use of electronic Participant-Reported Outcome Measures (ePROs) in clinical trials. The FDA requirements for including ePROs in clinical trials are similar to those for traditional PROs and include the following:

• Validity: The ePRO must be valid, meaning it accurately measures what it is intended to measure. This includes a review of the development and testing of the ePRO and its ability to produce consistent results.

• Reliability: The ePRO must be reliable, meaning it produces consistent results when used with the same research participants over time. This includes a review of the stability and consistency of the ePRO results.

• Sensitivity: The ePRO must be sensitive to change, meaning it can detect changes in the research participant’s health status over time. This includes a review of the ePRO’s ability to detect changes in the research participant’s quality of life, health status, and or sign/symptom severity. This is a fundamental component to minimize risks to research participants, a requirement under the ethical principle of beneficence.

• Relevance: The ePRO must be relevant, meaning it is appropriate for the research participant population being studied and the goals of the clinical trial. This includes a review of the ePRO’s ability to accurately capture information about the research participants’ health status and quality of life, as previously discussed above.

• Statistical Analysis: The data collected using the ePRO must be analyzed in a way that is statistically meaningful and appropriate. This includes the use of appropriate statistical methods to analyze the ePRO data and the presentation of results in the clinical study report and scientific publications in a way that is easily understandable.

• Data Security: The ePRO must be secure and protect research participant privacy. This includes the use of secure servers and encryption to protect the data and the implementation of strict data access controls to prevent unauthorized access to the data.

• Electronic Recordkeeping: The ePRO data must be kept in a format that complies with FDA regulations for electronic records and electronic signatures (21 CFR Part 11). This includes the use of electronic signatures, secure storage, and electronic backup procedures to ensure the integrity of the data[2].

Overall, the FDA requires that ePROs used in clinical trials meet certain standards for validity, reliability, sensitivity, relevance, statistical analysis, data security, and recordkeeping.

The FDA also requires that the results of ePROs be reported in a clear and concise manner and that the information gathered using ePROs be used to support the approval of new medical aesthetic treatments.

How to successfully implement ePROs in medical aesthetics clinical trials

Integrating electronic PROs in clinical trials requires careful planning and attention to detail to overcome the above-mentioned challenges and to ensure that trials are conducted effectively and efficiently.

When research participants are asked to use their own devices to complete ePROs, several strategies can be employed to ensure a successful and seamless experience. These strategies include:

• Informed consent: To ensure that research participants make an informed decision about using ePROs to collect their health information the informed consent process should explain the type of information that will be collected by the ePRO and how that information will be used by the research team. The informed consent process should also specify who may have access to data collected through the ePRO during or after the clinical trial (e.g., sponsor, CRO, investigator, research participant, ePRO service provider, other third parties) and for how long.

• Device compatibility: Ensuring that the ePRO platform is compatible with the most commonly used devices, such as smartphones and tablets, to minimize technical difficulties.

• Accessibility: Ensuring that the ePRO platform is accessible to participants with disabilities, such as those with visual or hearing impairments, using accessibility features, such as screen readers and magnifiers.

• User-friendly design: Designing the ePRO platform to be user-friendly, with clear instructions, and an intuitive interface to minimize research participant confusion and frustration.

• Technical support: Providing both investigational sites and research participants with easy access to technical support, such as a help desk or online resources, in the event that technical difficulties are encountered.

• Data security: Ensuring that participant data are securely stored and protected from unauthorized access, and that the participant data are de-identified to protect privacy.

• Compensation: Offering participants a financial compensation for their time completing the ePROs shows respect and may ensure research participant compliance. Other non-financial incentives, like access to additional resources, may increase research participants motivation and engagement in the study.

• Communication: Regularly communicating with investigative sites and research participants to keep them informed about the study and to provide updates on the status of their data may also contribute to research participants’ motivation and engagement in the study.

Improve the quality of your clinical trials with ePROs

Electronic PROs can significantly improve the quality of medical aesthetics clinical trials. However, it is important to address the associated challenges such as technical difficulties, research participant engagement, privacy/security concerns, and regulatory compliance.

By addressing these challenges, electronic PRO can help improve research participant-reported outcomes measurements in clinical trials and bring safer and more efficient medical aesthetics products on the market.

Strategies such as device compatibility, accessibility, user-friendly design, technical support, data security, acceptable compensation and incentives, and communication can help ensure a successful and seamless experience. These strategies can help increase participant engagement and satisfaction while ensuring the integrity and validity of the study data.

Ethica CRO is a medical aesthetics and dermatology CRO that employs ePRO as part of our suite of real-time data management technology. Contact us today to learn more about our multidisciplinary team with extensive scientific expertise and a wealth of clinical management experience for managing your medical aesthetics clinical trials.

[1] Food and Drug Administration, “Digital Health Technologies for Remote Data Acquisition in Clinical Investigations”, Draft Guidance, December 2021, online: https://www.fda.gov/media/155022/download ; See also Food and Drug Administration, “Patient-Reported Outcome Measures:  Use in Medical Product Development to Support Labeling Claims”, Final Guidance, December 2009, online: https://www.fda.gov/media/77832/download .

[2] Food and Drug Administration, “Electronic Systems, Electronic Records, and Electronic Signatures in Clinical Investigations Questions and Answers”, Draft Guidance, March 2023, online: https://www.fda.gov/media/166215/download