Economic Evaluation of Innovative Molecular Analyses in Onco-haematology

• Conditions: Haematological Malignancy

• Registration Number: NCT03750994
• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

Brief Summary

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

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:

1. Myelodysplasia (MDS)

2. Acute lymphocytic leukemia (T) (ALL)

3. Lymphoproliferative disorders (LPD)

4. Acute myeloblastic leukemia (AML)

5. 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.

Study Timeline

• Study First Submitted: 2017-08-11
• Study Start: 2018-10-18
• Status Verified: 2018-11
• Study First Submitted QC: 2018-11-21
• Last Update Submitted: 2018-11-21
• Study First Posted: 2018-11-23
• Last Update Posted: 2018-11-23
• Primary Completion: 2020-10
• Study Completion: 2020-10

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 3960

Inclusion Criteria

• Patients with haematological malignancies referred for molecular diagnosis workup. RuBIH2 will focus on 5 clinical situations in onco-haematology:

  1. Myelodysplasia (MDS)
  2. Acute lymphocytic leukemia (T) (ALL)
  3. Lymphoproliferative disorders (LPD)
  4. Acute myeloblastic leukemia (AML)
  5. Myeloproliferative disorders (MPD)

Exclusion Criteria

• Other haematological diseases not included in the list above.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (26)

Centre hospitalier régional universitaire de Lille; 🇫🇷 Lille, Hauts De France, France

CHU Angers; 🇫🇷 Angers, France

Hôpital Avicenne AP-HP; 🇫🇷 Bobigny, France

CHU de Bordeaux; 🇫🇷 Bordeaux, France

CHRU Brest; 🇫🇷 Brest, France

Hôpital Henri Mondor AP-HP; 🇫🇷 Créteil, France

CHU Estaing; 🇫🇷 Clermont Ferrand, France

CHRU Dijon Bourgogne; 🇫🇷 Dijon, France

CHU Limoges; 🇫🇷 Limoges, France

CHU Lyon Sud Pierre Bénite; 🇫🇷 Lyon, France

CHU Hôtel Dieu; 🇫🇷 Nantes, France

CHU Nice; 🇫🇷 Nice, France

Hôpital Pitié-Salpêtrière AP-HP; 🇫🇷 Paris, France

Hôpital Robert Debré; 🇫🇷 Paris, France

Hôpital St Louis AP-HP; 🇫🇷 Paris, France

Hôpital Cochin AP-HP; 🇫🇷 Paris, France

Hôpital Necker AP-HP; 🇫🇷 Paris, France

Hôpital Saint Antoine AP-HP; 🇫🇷 Paris, France

CHU Robert Debré Reims; 🇫🇷 Reims, France

CHU Pontchaillou; 🇫🇷 Rennes, France

Centre Henri-Becquerel; 🇫🇷 Rouen, France

Centre hospitalier universitaire de Saint-Étienne; 🇫🇷 Saint-Étienne, France

Hôpitaux Universitaires Strasbourg; 🇫🇷 Strasbourg, France

CHU Toulouse; 🇫🇷 Toulouse, France

Institut Gustave Roussy; 🇫🇷 Villejuif, France

CHU Montpellier; 🇫🇷 Montpellier, France