Cecilia Jimenez-Moreno
Associate Director, Kielo Research
Incorporating patient voice into rare disease drug development can drive more meaningful progress, explains Cecilia Jimenez-Moreno, Associate Director at Kielo Research.
Can you provide an introduction to what your research is about?
Kielo Research is a boutique consultancy specialising in integrating patient voice into drug development. As a highly focused team, we apply unique methodologies to ensure patient insights shape research and decision-making. Our clients include pharmaceutical and biotech companies seeking to enhance clinical research with real-world patient experiences.
How do you capture the patient voice in drug development?
Incorporating patient voice in drug development is not a new concept, but it has evolved and improved over the years. We use a range of methodologies to collate feedback, from direct patient interviews to large-scale surveys involving those actually living with the condition. We employ structured approaches, including focus groups and patient preference studies, to ensure that patient perspectives are collected in a scientifically meaningful way. These methods support decision-making in contexts where objective evidence is required.
It is increasingly common for industry
partners to collaborate with patient experts
and advocates to ensure there is a
patient-centric approach throughout.
Why is the patient voice crucial in rare disease research?
This is particularly important due to several factors: lack of available medications; limited awareness of the real impacts on those living with rare diseases; insufficient understanding of the diseases themselves; and sparse evidence regarding their natural history and progression. These elements contribute to high unmet needs that must be further explored from the perspective of the individuals affected.
How is the industry engaging with rare disease patients in drug development?
The integration of the patient voice in drug development has evolved due to increased awareness of its importance in decision-making. The industry now frequently utilises patient perspectives to inform aspects of clinical trial design, endpoint selection, awareness and dissemination and reimbursement and regulatory submissions, using structured methods to enhance understanding of diseases and identify treatment preferences. It is increasingly common for industry partners to collaborate with patient experts and advocates to ensure there is a patient-centric approach throughout.
What advice do you have for the rare disease community?
With over a decade of experience working with rare diseases, I want to encourage patients, advocates and patient organisations to feel more confident in taking the initiative to push for studies that capture patient experiences and preferences regarding treatments. They can be the driving force that leads the industry to gather this information and include it in their drug development strategies. This creates a win-win situation; we can learn from each other and ultimately improve the lives of those with rare diseases.
