Contact Us
Looking to explore real-world study solutions – get in touch
Frequently Asked Questions
How can we make best use of real-world data?
There are a multitude of uses for real-world data, most typically addressing gaps in, or providing data that are complementary to, a clinical trial data package. Real-world data can support a wide range of regulatory and market access needs, such as mapping disease burden and progression; complementing clinical trial data with patient- or caregiver-reported outcomes; strengthening a product’s evidence base during an early access or post-launch period with broader, more diverse, or long-term data. Data from observational studies can give stakeholders confidence that decisions are based on evidence grounded in real-world care.
Ultimately, the suitability of real-world data is a question best answered on a case-by-case basis. So if you are wondering about its potential for your evidence-generation needs, please get in touch.
How useful is it to work with key opinion leaders and patient advocacy groups when conducting real-world research?
It is invaluable to work closely with clinicians and patient advocacy groups with experience in the target condition, as it helps to ensure we are asking the right questions, in the right way. Including patient advocacy groups in study design and communication also helps to build participant trust in the study, giving them reassurance that their data are being used in a meaningful and patient-friendly way.
Why do you think patients are motivated to participate in real-world studies?
There are multiple reasons, which vary depending on the individual. We have had feedback that some participants want to give something back, or want to be part of new, innovative research to improve future management of their condition. Some find completing study activities therapeutic, or find features in the study that are useful to them (e.g., a symptom diary), or wish to keep up-to-date with the latest research information. If a study is not designed in a patient-friendly way, all these motivational factors can be lost, with a lack of engagement potentially leading to poor retention and completion rates.
At what stage of product development should RWE generation begin?
RWE generation should ideally be started early and continue throughout the product development and marketing lifecycle, informing and complementing other research efforts. If you are going to invest in a disease registry, invest early: set-up costs will be the same, and it is cost-effective to keep a registry running; plus there is often significant value in capturing longitudinal data. To allow for a seamless transition when the product is approved, safety reporting can then be added, transforming a pre-approval disease study into a post-approval/phase IV study.
What type of data can be gathered from a rare disease registry?
A broad range of data can be collected from a registry – here are a few examples:
- Treatment patterns
- Treatment preference/satisfaction
- Quality of life
- Diversity
- Demographics
- Healthcare-resource use
- Patient experience
- Physical function
- Productivity losses
- Burden of disease
- Adverse events.
How do you ensure RWE is published?
There are rich publication opportunities for RWE. We frequently build publication plans in parallel with our studies, and have been very successful in helping our study sponsors to publish. In our experience, successful publication of RWE is dependent on selecting an appropriate target journal or conference, with a scope that encompasses such research, and to ensure that the study methodology is robust and transparently reported.
