Economic Evaluation of Innovative Molecular Analyses in Onco-haematology

Haematological Malignancy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03750994
• Other study ID #: RuBIH2
• Status: Recruiting
• Start date: October 18, 2018
• Est. completion date: October 2020

Study information

• Verified date: November 2018
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: Claude Preudhomme, Professor
• Phone: + 33 3 20 44 47 82
• Email: claude.preudhomme[@]chru-lille.fr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 3960
• Est. completion date: October 2020
• Est. primary completion date: October 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Haematological Malignancy
• Hematologic Neoplasms

Locations

Country	Name	City	State
France	CHU Angers	Angers
France	Hôpital Avicenne AP-HP	Bobigny
France	CHU de Bordeaux	Bordeaux
France	CHRU Brest	Brest
France	CHU Estaing	Clermont Ferrand
France	Hôpital Henri Mondor AP-HP	Créteil
France	CHRU Dijon Bourgogne	Dijon
France	Centre hospitalier régional universitaire de Lille	Lille	Hauts De France
France	CHU Limoges	Limoges
France	CHU Lyon Sud Pierre Bénite	Lyon
France	CHU Montpellier	Montpellier
France	CHU Hôtel Dieu	Nantes
France	CHU Nice	Nice
France	Hôpital Cochin AP-HP	Paris
France	Hôpital Necker AP-HP	Paris
France	Hôpital Pitié-Salpêtrière AP-HP	Paris
France	Hôpital Robert Debré	Paris
France	Hôpital Saint Antoine AP-HP	Paris
France	Hôpital St Louis 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

Sponsors (2)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris	Ministry of Health, France

Country where clinical trial is conducted

France, 

Outcome

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