On 25th January, we hosted the 13th webinar in the “Keeping it real” series.
How to design a digital real-world study? Real-World Evidence Generation Approaches in rare disease and oncology.
Mark Larkin PhD, Founder and CEO, and Dr Catherine Bottomley, Chief Scientific Officer, discussed Real-World Evidence Generation Approaches in rare disease and oncology.
Watch this webinar that includes a Q&A session on demand at your convenience
BlogDesigning a digital patient-reported real-world study
While there is no one-size-fits-all solution, there are many considerations to make in the early stages of development, which can optimize study conduct and outcomes. This blog covers just a few methodological approaches for robust digital real-world study design. Digital real-world studies are becoming increasingly prevalent in the biopharmaceutical industry – but what is the best approach to their design?
1. Develop a strong informed consent document
As with all research involving patients and carers, potential participants must be given the opportunity to voluntarily provide informed consent to contribute. In digital real-world research, it is beneficial for the study data collection app or website to have consent integrated within the platform, in order to streamline the process. Various considerations should then be made in document creation.
- Use a patient-friendly template.
- Think about the user interface, and how the information appears to participants on a variety of screens.
- Consider opt-in clauses, such as consent-to-contact for additional research or for data linkage.
Implementing informed consent opt-in clauses for Vitaccess-led studies has allowed us to gain up to 100% consent-to-contact about future research. This can be hugely valuable in developing patient research cohorts and maximizing the potential of study data.
2. Consider country-specific ethics requirements
Ethical approval is the foundation for patient protection, as well as for publication of research. Ethics boards will review the protocol, informed consent, and recruitment materials to ensure that research is ethically sound and being carried out in the best interests of participants. Key considerations in gaining ethical approval include:
- Determining which review boards need to be contacted to gain approval depending on the study design, dataset, and population –e., international and/or local review boards.
- Researching between-country differences in requirements – for instance, local study representatives may be needed.
- Planning approval timings, i.e., gaining initial approval before commencing recruitment or making development commitments, and preparing for re-approvals if modifications are made to study documents.
More than this, local ethics review boards can vary massively in the length of time required to receive a decision – from weeks to several months! Having gained ethical approval for real-world research from local and international boards globally, we recognize the importance of planning carefully to ensure that study timelines can be adhered to and endpoints met.
3. Apply expertise in clinical outcome assessment migration
When it comes to digital real-world studies, the process of migrating paper-based clinical outcome assessments (COAs) (e.g., patient-reported outcome [PRO] instruments) to electronic versions is an important aspect to take into account – for example:
- Are electronic versions of the instruments already available?
- What are the best practice guidelines for migrating from paper to electronic?
- Are there implications for specific user groups?
- Do you require evidence for measurement equivalence/comparability?
Sometimes, the migration process is simple, as with the example of a straightforward item and Likert scale (right).
In other cases, migration may require more thinking through. Below is an example of where the process can fall outside of the “classic” design, involving scales and split stem questions.
Our in-house experts in COA migration are well-acquainted with the range of instruments available, as well as the corresponding approaches to effective electronic migration.
4. Recognize the power of rewards programs
Implementing rewards programs can be an effective means of recognizing participants’ time and contribution to the study, as well as increasing patient engagement. Vitaccess led an international, 18-month rare disease study involving adult and pediatric (with caregiver proxies) patients. To thank participants for their time, direct monetary rewards were provided in the form of e-vouchers for the completion of study surveys.
As there were a number of participants who did or did not receive rewards, the survey completion rates for both groups could be compared. As can be seen in the table below, completion rates were notably higher – with no attrition – for participants who received rewards.
The list of considerations to make when designing a digital real-world study is extensive – here, we have simply scratched the surface. To learn more about our capabilities, get in touch at info@vitaccess.com.
By Mark Larkin, Catherine Bottomley and Fatemeh Amini
