Generating robust evidence of the value of your product in real world
Once the efficacy and safety of a new health care intervention has been proven in clinical trials, the key question for decision-makers is what benefits this intervention will effectively provide to patients in real-world conditions. How is clinical improvement perceived and valued by patients? The patients receiving the drug in real practice may have different characteristics from those included in clinical trials, compliance may be lower, other treatment options may be available etc. Does clinical improvement effectively occur under these conditions? These are some of the questions addressed by Outcomes Research.
We can help you determine the value of your drug or device in real world via:
- Analyses of large administrative databases or electronic medical records databases
- Systematic literature reviews and meta-analysis
- Epidemiological and observational studies
- Patient surveys
- Development and validation of patient-reported outcome measures
- Qualitative research (patient interviews, focus groups)
Contact us to find out more on how our expertise in Outcomes Research can determine the best value of your product.
Evaluation of the burden associated with cytomegalovirus disease among transplant recipients using the PMSI database
At an early stage of the development of a vaccine to prevent cytomegalovirus (CMV) disease following transplantation, new evidence about the burden of disease was required. Previous studies showed that CMV disease was a cause of transplant failure, graft-versus-host disease (GVHD, following stem cell transplantation) and mortality. However, there was uncertainty around the magnitude of the association with CMV disease, in particular for late-onset CMV disease. There was great interest in late-onset disease, because its incidence increased as a result of wider utilisation of prophylaxis among patients with solid organ transplant. We conducted a study on the burden of CMV disease using data from the French hospitalization databases (PMSI). Our dataset covered 5 years and included 20,473 patients with solid organ transplantation and 18,809 patients with stem cell transplantation. Patients with CMV disease in different time windows were compared to matched patients without CMV disease, in order to determine relative risks of transplant failure, GVHD, and mortality, as well as excess hospitalisation costs. Despite limitations associated with the utilisation of administrative data, our results were generally consistent with those of previous studies. Our study had the advantages of providing results specific to late-onset disease, to cover a longer timeframe than most previous studies, and being based on real-world data. It showed in particular a strong association between CMV disease and mortality, and suggested that there was scope to reduce the burden of disease despite existing pre-emptive and prophylactic strategies, and therefore scope for vaccination.
Online survey on health status, quality of life and productivity among women with recurrent vulvovaginal candidiasis (RVVC)
Our client was a small biotech developing a vaccine to prevent recurrences of yeast infections among patients with recurrent vulvovaginal candidiasis (RVVC). Very little data existed about the burden of RVVC, and such information was needed to determine the value of the vaccine for potential investors. Creativ-Ceutical conducted a survey among over 600 RVVC patients in the US and the five largest European countries. Patients were recruited through online panels, which made it possible to collect the data over a short time. We showed that quality of life is strongly diminished and that RVVC has a significant impact on patient’s daily activities and productivity. Since the data were collected from an online panel, several tests were performed to assess the internal and external validity of the data. We found in particular that the correlations between different outcome measures were consistent with correlations found in previous studies, which suggested satisfactory validity. This survey was recently published and used to inform potential investors about the value of developing a vaccine for women suffering from RVVC.
Aballéa S, Guelfucci F, Wagner J, Khemiri A, Dietz JP, Sobel J, Toumi M. Subjective health status and health-related quality of life among women with Recurrent Vulvovaginal Candidosis (RVVC) in Europe and the USA. Health Qual Life Outcomes. 2013 Oct 11;11(1):169. http://www.hqlo.com/content/11/1/169