Clinical trial NCT03750994: Economic Evaluation of Innovative Molecular Analyses in Onco-haematology
Official title: Economic Evaluation of Innovative Molecular Analyses in Onco-haematology (PRME-K 2016)
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Haematological Malignancy
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
Collaborators: name: Ministry of Health, France
First posted: 2018-11-23
Enrollment: 3960
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients referred for molecular diagnosis at one of the certified molecular onco-haematology platforms in France.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Percentage of Next Generation Sequencing (NGS) tests that have a clinical impact for the patient for five hematological malignancies.
timeFrame: 2 years
measure: Percentage of Next Generation Sequencing (NGS) that are from the oncologists internal to the platform versus external centres.
timeFrame: 2 years
measure: Average time in days between the Next Generation Sequencing (NGS) prescription being issued and the results being rendered to the clinician.
timeFrame: 2 years
measure: Percentage of prescriptions for diagnostics, prognostic, theranostics or treatment response
timeFrame: 2 years
measure: Percentage of the genetic targets that are analysed for research purposes versus immediate clinical utility for the patient.
timeFrame: 2 years
measure: Percentage of patients prescribed the Next Generation Sequencing (NGS) at the diagnostic stage or before second (or higher) line treatment.
timeFrame: 2 years
Secondary outcomes:
Other outcomes:
Locations:
city: Lille
country: France
facility: Centre hospitalier régional universitaire de Lille
state: Hauts De France
city: Angers
country: France
facility: CHU Angers
city: Bobigny
country: France
facility: Hôpital Avicenne AP-HP
city: Bordeaux
country: France
facility: CHU de Bordeaux
city: Brest
country: France
facility: CHRU Brest
city: Clermont Ferrand
country: France
facility: CHU Estaing
city: Créteil
country: France
facility: Hôpital Henri Mondor AP-HP
city: Dijon
country: France
facility: CHRU Dijon Bourgogne
city: Limoges
country: France
facility: CHU Limoges
city: Lyon
country: France
facility: CHU Lyon Sud Pierre Bénite
city: Montpellier
country: France
facility: CHU Montpellier
city: Nantes
country: France
facility: CHU Hôtel Dieu
city: Nice
country: France
facility: CHU Nice
city: Paris
country: France
facility: Hôpital Pitié-Salpêtrière AP-HP
city: Paris
country: France
facility: Hôpital Robert Debré
city: Paris
country: France
facility: Hôpital St Louis AP-HP
city: Paris
country: France
facility: Hôpital Cochin AP-HP
city: Paris
country: France
facility: Hôpital Necker AP-HP
city: Paris
country: France
facility: Hôpital Saint Antoine AP-HP
city: Reims
country: France
facility: CHU Robert Debré Reims
city: Rennes
country: France
facility: CHU Pontchaillou
city: Rouen
country: France
facility: Centre Henri-Becquerel
city: Saint-Étienne
country: France
facility: Centre hospitalier universitaire de Saint-Étienne
city: Strasbourg
country: France
facility: Hôpitaux Universitaires Strasbourg
city: Toulouse
country: France
facility: CHU Toulouse
city: Villejuif
country: France
facility: Institut Gustave Roussy
Study officials:
affiliation: DRCI AP-HP
name: Claude Preudhomme, Professor
role: PRINCIPAL_INVESTIGATOR
References: