Budget impact analysis of discontinuing Tyrosin Kinase Inhibitors in patients with chronic myeloid leukemia achieving a complete molecular response by using probabilistic Markov approach (ECOSTIM)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/49932
• EU PAS number:
  EUPAS18568
• Study ID:
  49932
• Official title and acronym:
  Budget impact analysis of discontinuing Tyrosin Kinase Inhibitors in patients with chronic myeloid leukemia achieving a complete molecular response by using probabilistic Markov approach (ECOSTIM)
• DARWIN EU® study:
  No
• Study countries:
  France
• Study description:
  Chronic 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remission (CMR, i.e. undetectable residual disease on quantitative RT-PCR), for at least two years after imatinib was withdrawn.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).
• Study status:
  Finalised

Research institution and networks

Institutions

Bordeaux PharmacoEpi, University of Bordeaux

France

Contact details

Pauline BOSCO-LEVY

Study contact

[e-mail]

Nicholas MOORE

Primary lead investigator

Study timelines

Date when funding contract was signed

Actual: 29/12/2016

Data collection

Planned: 31/12/2017

Actual: 22/03/2018

Start date of data analysis

Planned: 31/03/2018

Actual: 15/06/2018

Date of final study report

Planned: 28/02/2019

Actual: 08/04/2019

Sources of funding

Other

More details on funding

Direction Générale de l’Offre de Soins (DGOS)

Study protocol

Initial protocol

ECOSTIM-v2.1_clean version 20170322

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable