Economic Evaluation of Innovative Molecular Analyses in Onco-haematology

Study Overview
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Brief Summary
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To evaluate the impact of innovative molecular diagnostics on the clinical management of patients with haematological malignancies via updated Appropriate-Prescribing-Guides including Next-Generation Sequencing (NGS) panels, facilitated therapeutic orientation, and optimised use of costly novel therapeutics and risk-adapted treatment. A micro-costing approach will be used to develop flat fee tarifs for NGS analyses.

Detailed Description
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The 12 somatic genetic cancer tests that have received temporary authorisation in France form the basis of this study. These tests are not yet in the national biology reimbursement nomenclature but are supported by the ministry of health in a temporary list Le référentiel des actes innovants hors nomenclature de biologie et d'anatomocytopathologie (RIHN). The PRME RuBIH2 will focus on 5 clinical situations in onco-haematology: Myelodysplasia (MDS) Acute lymphocytic leukemia (T) (ALL) Lymphoproliferative disorders (LPD) Acute myeloblastic leukemia (AML) Myeloproliferative disorders (MPD) The project is organised in 4 complementary work packages (WP): WP1 Cost evaluation, WP2 Prescription Guidelines, WP3 Clinical Validation and WP4 Budget Impact and Organisation. WP1 will provide costing information on molecular tests and will build on previous studies conducted in France. WP2 will update existing prescription guidelines based on evidence from the literature and evidence from the WP3. These prescription guidelines will in turn be valued and provide recommendations for a flat fee bundle for pre-specified clinical situations. WP3 will provide evidence on the clinical impact of molecular diagnosis (in particular NGS) in the 5 pre-specified conditions. Changes in patient management will be measured using a prospective questionnaire for an estimated 3960 molecular tests. The impact of the test on the patient clinical pathway will be analysed. The impact of molecular tests on patient outcome will not be measured. WP4 will use information from WP1 and WP2 to estimate the budget impact and to provide scenario analyses on the territorial organisation of molecular biology platforms. Based on the estimation of the national activity of molecular onco-haematology platforms the annual functioning budget required to implement molecular diagnosis in France will be estimated.

Official Title
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Economic Evaluation of Innovative Molecular Analyses in Onco-haematology (PRME-K 2016)

Conditions
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Haematological Malignancy

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with haematological malignancies referred for molecular diagnosis workup. RuBIH2 will focus on 5 clinical situations in onco-haematology: Myelodysplasia (MDS) Acute lymphocytic leukemia (T) (ALL) Lymphoproliferative disorders (LPD) Acute myeloblastic leukemia (AML) Myeloproliferative disorders (MPD) Exclusion Criteria: Other haematological diseases not included in the list above.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Next Generation Sequencing (NGS) tests that have a clinical impact for the patient for five hematological malignancies. |  | 2 years | 
| Percentage of Next Generation Sequencing (NGS) that are from the oncologists internal to the platform versus external centres. |  | 2 years | 
| Average time in days between the Next Generation Sequencing (NGS) prescription being issued and the results being rendered to the clinician. |  | 2 years | 
| Percentage of prescriptions for diagnostics, prognostic, theranostics or treatment response |  | 2 years | 
| Percentage of the genetic targets that are analysed for research purposes versus immediate clinical utility for the patient. |  | 2 years | 
| Percentage of patients prescribed the Next Generation Sequencing (NGS) at the diagnostic stage or before second (or higher) line treatment. |  | 2 years |