1. Study identification
EU PAS Register NumberEUPAS10691
Official titleDisease progression and resource utilization in treated relapse/refractory multiple myeloma in Spain
Study title acronymPREMIERE
Study typeObservational study
Brief description of the studyThis is a retrospective chart review study to explore progression-free survival (PFS), overall survival (OS), health resource utilisation (HRU) and their associated costs in a sample of patients treated for a first episode of RRMM in a real-world setting in Spain. While data on treatments and resources will be collected from patients’ clinical notes, costs will be extracted from a local cost databases. Such information will be used to populate a cost-effectiveness model for Spain using real world data.
Was this study requested by a regulator?No
Is the study required by a Risk Management Plan (RMP)?
Not applicable
Regulatory procedure number (RMP Category 1 and 2 studies only)
Other study registration identification numbers and URLs as applicable
2. Research centres and Investigator details
Coordinating study entity
Centre to which the investigator belongsOXON
Department/Research group
Organisation/affiliationOxon Epidemiology Ltd.
Details of (Primary) lead investigator
Title Dr
Last name Qizilbash MBChB MRCP(UK) BSc MSc DPhil(Oxon.)
First name Nawab
Is this study being carried out with the collaboration of a research network?
No
Other centres where this study is being conducted
Multiple centres
In total how many centres are involved in this Study?27
Countries in which this study is being conducted
National study
Spain
3. Study timelines: initial administrative steps, progress reports and final report
PlannedActual
Date when funding contract was signed30/10/2014
Start date of data collection07/09/201511/09/2015
Start date of data analysis01/09/201514/12/2015
Date of interim report, if expected30/12/2015
Date of final study report31/10/201623/12/2016
4. Sources of funding
Please provide estimates of the percentage of funding by source for this study
Names(s)Approximate % funding
Pharmaceutical companiesBMS, Bristol Myers Squibb100
Charities
Government body
Research councils
EU funding scheme
5. Contact details for enquiries
Scientific Enquiries
Title Dr
Last name Qizilbash MBChB MRCP(UK) BSc MSc DPhil(Oxon.)
First name Nawab
Address line 1The Euston Office
Address line 21 Euston Square
Address line 340 Melton Street
CityLondon
PostcodeNW1 2FD
CountryUnited Kingdom
Phone number (incl. country code)442035744617
Alternative phone number34-629846059
Fax number (incl. country code)442033977497
Public Enquiries
Title Dr
Last name Qizilbash MBChB MRCP(UK) BSc MSc DPhil(Oxon.)
First name Nawab
Address line 1The Euston Office
Address line 21 Euston Square
Address line 340 Melton Street
CityLondon
PostcodeNW1 2FD
CountryUnited Kingdom
Phone number (incl. country code)442035744617
Alternative phone number34-629846059
Fax number (incl. country code)442033977497
6. Study drug(s) information
Not applicable (disease/epidemiology study)
7. Medical conditions to be studied
Medical condition(s)Yes
Plasma cell myeloma
8. Population under study
Age
Adults (18 - 44 years)
Adults (45 - 64 years)
Adults (65 - 74 years)
Adults (75 years and over)
Sex
Male
Female
9. Number of subjects
Estimated total number of subjects375
10. Source of data
Is this study being carried out with an established data source?Yes
Data sources not registered with ENCePP
eSalud, Spain
Sources of data
Patient´s clinical records
11. Scope of the study
What is the scope of the study?
Drug utilisation study
treatment patterns and resource utilisation
Primary scope : treatment patterns and resource utilisation
12. Main objective(s)
What is the main objective of the study?
The primary objective is to estimate progression free survival (PFS) in a cohort of patients in Spain treated for a first episode of relapse/refractory multiple myeloma (RRMM).
Are there primary outcomes?Yes
Progression-free survival (PFS)
Are there secondary outcomes?Yes
Overall survival (OS), socio-demographic, clinical and treatment characteristics, healthcare resource utilisation and associated costs, and reasons for change in Line of Therapy (LOT)
13. Study design
What is the design of the study?
Retrospective chart review
14. Follow-up of patients
Will patients be followed up?Yes
Please describe duration of follow up
3 years
15. Data analysis plan
Please provide a brief summary of the analysis method
PFS and OS will be described using Kaplan-Meier plots with estimation of the median PFS and 95% CIs. The number of events and the number of patients at risk by 1 month periods will be reported. If the median level (50%) is not reached, cumulative incidence by 3 month periods will be provided. Analyses will be performed overall and stratified by age, presence of previous treatment with immunomodulatory drugs and prior LOT (1st; ≥ 2).
Patient characteristics at the index date will be summarized. Descriptive statistics will be used to describe treatment characteristics by LOT during follow-up. Summary descriptive statistics will report HRU and costs during the follow-up in the pre-/post-progression states. The overall proportion of RRMM patients who undergo a change in LOT during follow-up, proportions by LOT and reasons for change will be presented. To identify patient- and center-level characteristics associated with PFS Cox regression models will be used.
16. ENCePP seal
Are you requesting the ENCePP seal for this study?
No
17. Full protocol
Not submitted
18. Study Results
Not submitted
Please list the 5 most relevant publications using data from your study
ReferenceLink to web-publication
None
19. Other relevant documents
Conflict(s) of interest of
investigator(s)Not submitted
Composition of Steering Group and
ObserversNot submitted
Other documentsNot
submitted
Signed Code of
Conduct Checklist
Not submitted
Signed Code of Conduct Declaration
Not submitted
Signed Checklist for Study
Protocols
Not submitted