Status: Finalised First registered on: 21/04/2017
Last updated on: 24/11/2022
1. Study identification
EU PAS Register NumberEUPAS18568
Official titleBudget impact analysis of discontinuing Tyrosin Kinase Inhibitors in patients with chronic myeloid leukemia achieving a complete molecular response by using probabilistic Markov approach
Study title acronymECOSTIM
Study typeObservational study
Brief description of the studyChronic myeloid leukemia (CML) is an hematopoietic stem cell disorder in which a t(9;22) (q34;q11) reciprocal chromosomal translocation gives rise to Philadelphia chromosome (Ph) and generates the BCR-ABL1 fusion gene encoding a constitutively activated tyrosine kinase protein. Over the past decade, a broad array of drugs designed to selectively inhibit protein tyrosine kinases [i.e., tyrosine kinase inhibitors, (TKI)] have emerged as novel therapies. These treatments induce durable responses and prolong survival allowing CML patients to have a near-normal life expectancy. Two important issues must be then considered in the future: 1-the quality of life and ethical aspects of the lifetime treatment during lifetime, 2- the economic impact of treating patients during lifetime. One of the best ways to consider these two points is to ask the question about stopping TKI in good responder patients. Previous studies showed promising results concerning patients who remained in complete molecular remission (CMR, i.e. undetectable residual disease on quantitative RT-PCR), for at least two years after imatinib was withdrawn. All molecular relapsing patients were sensitive when imatinib was re-challenged. Around 40% of these patients remain in a prolonged treatment-free remission (TFR) after treatment cessation. Considering the cost of imatinib and the number of months without treatment based on these studies, the savings in France would be 9 million €. However, since only 40 % of patients are in treatment free remission, a study, assessing the real budget impact for the healthcare system of stopping TKI in the eligible population seems necessary as no published study has ever addressed this question in France. The French National Health Insurance database (SNIIR-AM) is well suited to conduct this study since it provides exhaustive information about total costs induced by CML patients in France in both strategies (continuing or stopping TKI treatment).
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 belongsBordeaux PharmacoEpi
Department/Research grouppharmacoepidemiology
Organisation/affiliationUniversity of Bordeaux
Website/Homepagewww.pharmacoepi.eu
Details of (Primary) lead investigator
Title Professor
Last name MOORE
First name Nicholas
Is this study being carried out with the collaboration of a research network?
Yes
Dr Antoine Bénard (Methodologist) - Bordeaux University Hospital – CHU de Bordeaux Clinical Trial Unit (Unité de soutien Méthodologique à la recherche Clinique et épidémiologique, USMR)
Nathalie Hayes (Health economist) - Bordeaux University Hospital – CHU de Bordeaux Clinical Research Department (Direction de la recherche Clinique et de l’innovation, DRCI)
Pr François-Xavier Mahon (Hematologist) - Bergonie Institut, Bordeaux
Other centres where this study is being conducted
Not applicable (single centre)

Countries in which this study is being conducted
National study

France
3. Study timelines: initial administrative steps, progress reports and final report
PlannedActual
Date when funding contract was signed29/12/2016
Start date of data collection31/12/201722/03/2018
Start date of data analysis31/03/201815/06/2018
Date of interim report, if expected
Date of final study report28/02/201908/04/2019
4. Sources of funding
Please provide estimates of the percentage of funding by source for this study
Names(s)Approximate % funding
Pharmaceutical companies
Charities
Government bodyDirection Générale de l’Offre de Soins (DGOS)100
Research councils
EU funding scheme
5. Contact details for enquiries
Scientific Enquiries
Title Dr
Last name BOSCO-LEVY
First name Pauline
Address line 1146 rue Léo Saignat
Address line 2 
Address line 3 
CityBordeaux 
Postcode33000 
CountryFrance
Phone number (incl. country code)33557579209 
Alternative phone number 
Fax number (incl. country code) 
Email address pauline.bosco-levy@u-bordeaux.fr
Public Enquiries
Title Dr 
Last name BOSCO-LEVY 
First name Pauline 
Address line 1146 rue Léo Saignat 
Address line 2 
Address line 3 
CityBordeaux 
Postcode33000 
CountryFrance 
Phone number (incl. country code)33557579209 
Alternative phone number 
Fax number (incl. country code) 
Email address pauline.bosco-levy@u-bordeaux.fr 
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